Document:

EX-10.23

 Exhibit 10.23 

CONFIDENTIAL TREATMENT REQUESTED 

LICENSE AGREEMENT 
 This LICENSE AGREEMENT
(“Agreement”) is entered into as of December 2, 2013 (“Effective Date”) by and between ReGenX Biosciences, LLC, a limited liability company organized under the laws of the State of Delaware, with offices at 750
17th Street, NW, Suite 1100, Washington, DC 20006 USA (“Licensor”), and Lysogene Société par Actions Simplifiée, a simplified joint stock company organized under the laws of France, with offices at 52 rue de la
Boetie, 75008 - Paris, France (“Licensee”). Licensor and Licensee are hereinafter referred to individually as a “Party” and collectively as the “Parties.” 

WHEREAS, Licensor has rights under certain Licensed Patents (as defined herein) pertaining to adeno-associated virus serotype rh10; and 

WHEREAS, Licensee desires to obtain an exclusive license under the Licensed Patents under the terms set forth herein; 

NOW, THEREFORE, in consideration of the promises and covenants contained in this Agreement, and intending to be legally bound, the Parties hereby agree as
follows: 
 ARTICLE 1: DEFINITIONS 
 1.1
“AAVrh10” means (a) the recombinant adeno-associated virus serotype rh10 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype rh10 vector
that are covered by the claims of the Licensed Patents. 
 1.2 “Affiliate” means any legal entity directly or indirectly controlling,
controlled by, or under common control with another entity. For purposes of this Agreement, “control” means the direct or indirect ownership of more than 50% of the outstanding voting securities of a legal entity, or the right to receive
more than 50% of the profits or earnings of a legal entity, or the right to control the policy decisions of a legal entity. 
 1.3 “Calendar
Quarter” means each three-month period or any portion thereof, beginning on January 1, April 1, July 1, and October 1. 

1.4 “Confidential Information” means and includes all technical information, inventions, developments, discoveries, software, know-how,
methods, techniques, formulae, animate and inanimate materials, data, processes, finances, business operations or affairs, and other proprietary ideas, whether or not patentable or copyrightable, of either Party that are (a) marked or otherwise
identified as confidential or proprietary at the time of disclosure in writing; or (b) if disclosed orally, visually, or in another non-written form, identified as confidential at the time of disclosure and summarized in reasonable detail in
writing as to its general content within 30 days after original disclosure. The Parties acknowledge that (i) the terms and conditions of this Agreement and (ii) the records and reports referred to in Section 3.6 will be deemed the
Confidential Information of both Parties, regardless of whether, such information is marked or identified as confidential. In addition, information provided to Licensee pursuant to the provisions of Section 7.1 will be deemed the Confidential
Information of Licensor, regardless of whether such information is marked or identified as confidential. Notwithstanding the foregoing, 

  
 ****CERTAIN
INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL
TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
Confidential Information will not include the following, in each case, to the extent evidenced by competent written proof of the Receiving Party: 

1.4.1 information that was already known to the Receiving Party, other than under an obligation of confidentiality, at the time of disclosure
by the Disclosing Party; 
 1.4.2 information that was generally available to the public or otherwise part of the public domain at the time
of its disclosure to the Receiving Party; 
 1.4.3 information that became generally available to the public or otherwise part of the public
domain after its disclosure, other than through any act or omission of the Receiving Party in breach of this Agreement; 
 1.4.4 information
that is independently discovered or developed by the Receiving Party without the use of Confidential Information of the Disclosing Party; or 

1.4.5 information that was disclosed to the Receiving Party, other than under an obligation of confidentiality, by a Third Party who had no
obligation to the Disclosing Party not to disclose such information to others. 
 1.5 “Disclosing Party” has the meaning set forth in
Section 5.1. 
 1.6 “Domain Antibody” ****. 

1.7 “FDA” means the United States Food and Drug Administration, or a successor agency in the United States with responsibilities comparable
to those of the United States Food and Drug Administration. 
 1.8 “Field” means the treatment of the Sanfilippo syndrome Type A (MPSIII
Type A) in humans by in vivo gene therapy using AAVrh10. 
 1.9 “GSK Agreement” means that certain License Agreement entered into
between Licensor and SmithKline Beecham Corporation, effective on March 6, 2009, as amended by that certain Amendment to License Agreement dated April 15, 2009, and as amended from time to time. 

1.10 “Licensed Patents” means (a) all United States patents and patent applications listed in Exhibit A, and (b) any re-examination
certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications. 
 1.11 “Licensed
Product” means (a) any AAVrh10 product that is made, made for, used, sold, offered for sale, or imported by Licensee, its Affiliates and any of its or their Sublicensees, the manufacture, use, sale, offer for sale, or import of which
-product, in the absence of the license grafted pursuant to this Agreement, would infringe or is covered by at least one Valid Claim in the country of manufacture, use, sale, offer for sale, or imp*, including products manufactured by a process that
would infringe or is covered by at least one Valid Claim in the country of manufacture, use, sale, offer for sale, or import; or (b) any service with respect to the 

  
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REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
administration of AAVrh10 product to patients that, in the absence of the licenses granted pursuant to this Agreement, would infringe or is covered by at least one Valid Claim in the country of
sale. 
 1.12 “NDA” means a New Drug Application filed with the FDA as described in 21 C.F.R. § 314, a Biological License
Application (BLA) pursuant to 21 C.F.R. § 601.2, or any equivalent or any corresponding application for regulatory approval in any country or regulatory jurisdiction other than the United States. 

1.13 “Net Sales” means the gross receipts from sales or other disposition of a Licensed Product (including fees for services within the
definition of “Licensed Product”) by Licensee and/or its Affiliates and/or any Sublicensees to Third Parties less the following deductions that are directly attributable to a sale, specifically and separately identified on an
invoice or other documentation and actually borne by Licensee, its Affiliates, or any Sublicensees: ****. In the event consideration other than cash is paid to Licensee, its Affiliates, or any Sublicensees, for purposes of determining Net Sales, the
Parties shall use the cash consideration that Licensee, its Affiliates, or any ‘Sublicensees would realize from an unrelated buyer in an arm’s length sale of an identical item sold in the same quantity and at the time and place of the
transaction, as determined jointly by Licensor and Licensee based on transactions of a similar type and standard industry practice, if any. 
 1.14
“Penn Agreement” means that certain License Agreement entered into between Licensor and The Trustees of the University of Pennsylvania, effective on February 24, 2009, as amended by that letter agreement dated March 6,
2009, and as amended from time to time. 
 1.15 “Phase 3 Clinical Trial” means a pivotal clinical trial in humans performed to gain
evidence with statistical significance of the efficacy of a product in a target population, and to obtain expanded evidence of safety for such product that is needed to evaluate the overall benefit-risk relationship of such product, to form the
basis for approval of an NDA and to provide an adequate basis for physician labeling, as described in 21 C.F.R. § 312.21(c) or the corresponding regulation in jurisdictions other than the United States. 

1.16 “Prosecute” means preparation, filing, and prosecuting patent applications and maintaining patents, including any reexaminations,
reissues, oppositions, and interferences. 
 1.17 “Receiving Party” has the meaning set forth in Section 5.1. 

1.18 “Retained Rights” has the meaning set forth in Section 2.2. 

  
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REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

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 1.19 “Sublicensee” means any Third Party or Affiliate to whom Licensee grants a sublicense
of some or all of the rights granted to Licensee under this Agreement as permitted by this Agreement. 
 1.20 “Third Party” means any
person or entity other than a Party to this Agreement or Affiliates of a Party to this Agreement. 
 1.21 “Valid Claim” means a claim of an
issued and unexpired patent (including any patent claim the term of which is extended by any extension, supplementary protection certificate, patent term restoration, or the like) included within the Licensed Patents or a claim of a pending patent
application included within the Licensed Patents, which has not lapsed, been abandoned, been held revoked, or been deemed unenforceable or invalid by a non-appealable decision or an appealable decision from which no appeal was taken within the time
allowed for such appeal of a court or other governmental agency of competent jurisdiction. 
 ARTICLE 2: LICENSE GRANT 

2.1 License Grant. Subject to the terms and conditions of this Agreement, including the Retained Rights, Licensor hereby grants to Licensee an
exclusive (even as to Licensor), sublicensable (as provided in Section 2.4 only), non-transferable (except as provided in Section 10.2), royalty-bearing, worldwide license, under the Licensed Patents to make, have made, use, import, sell,
and offer for sale Licensed Products solely in the Field, including, for the avoidance of doubt, the right to conduct research and development. 
 2.2
Retained Rights. Except for the rights and licenses specified in Section 2.1, no license or 1 other rights are granted to Licensee under any intellectual property of Licensor, whether by implication, estoppel, or otherwise, whether any
such intellectual property dominates or is dominated by the Licensed Patents. Notwithstanding anything to the contrary in this Agreement, Licensor may use and permit others to use the Licensed Patents for any research, development, commercial, or
other purposes outside of the Field. Without limiting the foregoing, and notwithstanding anything in this Agreement to the contrary, Licensee acknowledges and agrees to the following rights retained by Licensor and its direct and indirect licensors
(individually and collectively, the “Retained Rights”), whether inside or outside the Field: 
 2.2.1 The rights and
licenses granted in Section 2.1 shall not include any right (and Licensor and its direct and indirect licensors retain the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents to make, have made, use, sell,
offer to sell, and import Domain Antibodies that are expressed by an adeno-associated vector, including AAVrh10. 
 2.2.2 Licensor and its
direct and indirect licensors retain the following rights with respect to the Licensed Patents: 
  

	 	(a)	A non-exclusive, sublicensable right under the Licensed Patents to make, have made, use, sell, offer to sell, and import products that deliver RNA interference and antisense drugs using an adeno-associated vector,
including AAVrh10; and 

  
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	 	(b)	A non-exclusive right for Licensor’s direct and indirect licensors (which right is sublicensable by such licensors) to use the Licensed Patents for non-commercial research purposes and to use the Licensed Patents
for such licensors’ discovery research efforts with non-profit organizations and collaborators. 

 2.2.3 The rights and
licenses granted in Section 2.1 shall not include any right (and Licensor retains the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents: 

 

	 	(a)	to conduct commercial reagent and services businesses, which includes the right to make, have made, use, sell, offer to sell, and import research reagents, including any viral vector construct; provided that, for
clarity, such rights retained by Licensor shall not include the right to conduct clinical trials in humans in the Field; or 

  

	 	(b)	to use the Licensed Patents to provide services to any Third Parties; provided that Licensee’s license under Section 2.1 does include the right to provide the service of the administration of Licensed Products
to patients. 

 2.2.4 Licensor retains the fully sublicensable right under the Licensed Patents to grant non-exclusive
research and development licenses to Affiliates and Third Parties; provided that such development rights granted by Licensor shall not include the right to conduct clinical trials in humans in the Field or any rights to sell products in the Field.

 2.2.5 The University of Pennsylvania may use and permit other non-profit organizations or other non-commercial entities to use the
Licensed Patents for educational, research, and other non-commercial purposes. 
 2.3 Government Rights. Licensee acknowledges that the United States
government retains certain rights in intellectual property funded in whole or part under any contract, grant, or similar agreement with a federal agency. The license grant hereunder is expressly subject to all applicable United States government
rights, including any applicable requirement that products resulting from such intellectual property sold in the United States must be substantially manufactured in the United States. 

2.4 Sublicensing. 
 2.4.1 The license
granted pursuant to Section 2.1 is sublicensable by Licensee to any Affiliates or Third Parties; provided that any such sublicense must comply with the provisions of this Section 2.4 (including Section 2.4.2). 

2.4.2 The right to sublicense granted to Licensee under this Agreement is subject to the following conditions: 

 

	 	(a)	Licensee may grant sublicenses **** but only pursuant to a written sublicense agreement with the Sublicensee. Licensor must 

  
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receive written notice as soon as practicable following execution of any such sublicenses. 

  

	 	(b)	In each sublicense agreement, the Sublicensee must be required to comply with the terms and conditions of this Agreement to the same extent as Licensee has agreed and must acknowledge that Licensor is an express third
party beneficiary of such terms and conditions under such sublicense agreement. 

  

	 	(c)	The official language of any sublicense agreement shall be English. 

  

	 	(d)	Within **** after entering into a sublicense, Licensor must receive a copy of the sublicense written in the English language for Licensor’s records and to share with Licensor’s direct and indirect licensors.
The copy of the sublicense may be redacted to exclude confidential information of the applicable Sublicensee, but such copy shall not be redacted to the extent that it impairs Licensor’s (or any of its direct or indirect licensors’)
ability to ensure compliance with this Agreement; provided that, if any of Licensor’s direct or indirect licensors require a complete, unredacted copy of the sublicense, Licensee shall provide such complete, unredacted copy. 

 

	 	(e)	Licensee’s execution of a sublicense agreement will not relieve Licensee of any of its obligations under this Agreement. Licensee is and shall remain **** to Licensor for all of Licensee’s duties and
obligations contained in ‘this Agreement and for any act or omission of an Affiliate or Sublicensee that would be a breach of this Agreement if performed or omitted by Licensee, and Licensee will be deemed to be in breach of this Agreement as a
result of such act or omission. 

 2.5 Improvements. 

2.5.1 Licensee hereby grants to Licensor a non-exclusive, worldwide, royalty-free, transferable, sublicensable, irrevocable, perpetual
license: 
  

	 	(a)	to use any Licensed Back Improvements (and any intellectual property rights with respect thereto) consummate in scope to the Retained Rights, and 

 

	 	(b)	to practice the Licensed Back Improvements (and any intellectual property rights with respect thereto) in connection with AAVrh10, including the right to research, develop, make, have made, use, offer for sale, and sell
products and services; provided that, during the term of this Agreement, Licensor shall have no right, under the license in this Section 2.5.1(b), to practice the Licensed Back Improvements in the Field. 

  
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REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

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 2.5.2 For purposes of this Agreement, “Licensed Back Improvements” means any
patentable modifications or improvements developed by Licensee, any Affiliates, or any Sublicensees to any vector that is the subject of a claim within the Licensed Patents. 

2.5.3 Licensee agrees to provide prompt notice to Licensor upon the filing of any patent application covering any Licensed Back Improvement,
together with a reasonably detailed description of or access to such Licensed Back Improvement to permit the practice of any such invention or improvement by Licensor or its direct or indirect licensors or licensees as provided for in
Section 2.5.1 above. 
 ARTICLE 3: CONSIDERATION 

3.1 Initial Fee. In consideration of the license granted to Licensee under Section 2.1, Licensee shall pay Licensor an initial fee of $500,000, of
which ****will be paid upon the Effective Date and ****will be paid upon the **** of the ****provided that such **** portion of the initial fee will be immediately payable upon any termination of this Agreement prior to the **** of the Effective
Date. 
 3.2 Annual Maintenance Fee. In consideration of the license granted to Licensee under Section 2.1, Licensee shall pay Licensor on-going annual maintenance fees of **** on each anniversary of the Effective Date. 
 3.3 Milestone Fees. In
consideration of the license granted to Licensee under Section 2.1, Licensee shall pay Licensor the following milestone payments: 
  

					
	 	 	 Milestone
	  	Milestone Payment
	 1.
	 	First treatment in Phase 3 Clinical Trial (i.e., first patient, first dose)	  	****
	 2.
	 	NDA submission in the United States	  	****
	 3.
	 	NDA submission in the European Union	  	****
	 4.
	 	NDA approval in the United States	  	****
	 5.
	 	NDA approval in the European Union	  	****
	 6.
	 	First rolling 12-month period during which aggregate Net Sales are greater than $50.0 million	  	****

 For clarity, the milestone payments set forth in this Section 3.3 are payable **** with respect to the
first Licensed Product that achieves the milestone event, ****. To the extent that the development milestone in this Section 3.3 (i.e., first treatment in Phase 3 Clinical Trial) has not been paid at the time of achievement of either NDA
submission milestone, then, upon the achievement of either of such NDA submission milestones, the preceding unpaid development milestone payment shall be made in addition to the payment corresponding to the NDA submission milestone that has been
achieved. 

  
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REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

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 3.4 Royalties. In further consideration of the license granted to Licensee under Section 2.1,
Licensee shall pay to Licensor the following royalties based upon Net Sales of Licensed Products: 
  

			
	 Cumulative Annual Net Sales of all Licensed Products Worldwide
	  	Royalty Percentage
	 Portion of Net Sales less than $300 million
	  	****
	 Portion of Net Sales between (and including) $300 million through (and including) $600 million
	  	****
	 Portion of Net Sales greater than $600 million
	  	****

 By way of example, if the cumulative annual Net Sales of all Licensed Product worldwide equal $700,000,000 in a calendar year,
the royalty rate on the first $299,999,999 of such Net Sales will be ****, the royalty rate on the next $300,000,002 of such Net Sales will be ****, and the royalty rate on the remaining $99,999,999 of such Net Sales will be ****. 

3.4.1 Third Party Royalties Stacking Provision. If Licensee must obtain a license from a Third Party to avoid infringement of such
Third Party’s rights in order to manufacture, use, or commercialize a given Licensed Product and if the royalties required to be paid to such Third Party for such license, together with those royalties payable to Licensor, in the aggregate,
exceed **** of Net Sales for any Licensed Product, then the royalty owed to Licensor for that Licensed Product will be reduced by an amount calculated as follows: 

STACKING ROYALTY CALCULATIONS 

R = (C * (A / (A+B))) 
 Where 

R = reduction of Licensor royalty, 

A = unreduced Licensor royalty, 
 B
= sum of all Third Party royalties, 
 C = increment of projected total royalty above **** 

Example Calculation: 
 assume: 

 

	 	i)	all Third Party royalties = **** 

  

	 	ii)	unreduced Licensor royalty =**** 

  

	 	iii)	projected total royalty = **** 

 R = (****–****) * (**** / (**** + ****)) 

R = **** * **** 
 R = **** 

  
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 Licensor Stacked Royalty = **** – **** = **** 

Notwithstanding the foregoing, Licensee will pay to Licensor no less than **** of the royalties that Licensee would otherwise pay to Licensor
with respect to Net Sales of Licensee if there were no royalties due to Third Parties. 
 3.4.2 Royalty Payment Period.
Licensee’s obligation hereunder for payment of a royalty under this Section 3.4 on the Net Sales of Licensed Products in a given country will end on a country-by-country basis when all Valid Claims in that country claiming the Licensed
Product have expired, lapsed, been abandoned, or been invalidated. 
 3.5 Sublicense Fees. 

3.5.1 In further consideration of the license granted to Licensee under Section 2.1, Licensee will pay Licensor a percentage of any
sublicense fees (including upfront payments and milestone payments) received by Licensee for the Licensed Patents from any Sublicensee or from any person or entity granted any option to obtain a sublicense. The applicable percentage due to Licensor
for each sublicense (or option) shall be as follows: 
  

			
	 Event
	  	Sublicense Fee Rate
	 If sublicensed (or optioned) on or before the third anniversary of the Effective Date
	  	****
	 If sublicensed (or optioned) after the third anniversary of the Effective Date
	  	****

 For the avoidance of doubt, with respect to an option to obtain a sublicense, if a sublicense is later granted as a result of
the exercise of such option, the sublicense fees applicable to such sublicense will be determined by reference to the date the original option was granted, not the date the actual sublicense was granted. 

3.5.2 With respect to the obligations under this Section 3.5, Licensee shall not be required to submit any amounts received from a Third
Party for the following: 
  

	 	(a)	Reimbursement for research, development, and/or manufacturing activities performed by Licensee corresponding directly to the development of Licensed Products pursuant to a specific agreement; 

 

	 	(b)	Consideration received for the purchase of an equity interest in Licensee at fair market value or in the form of loans at commercially reasonable rates of interest; and 

 

	 	(c)	Any and all amounts paid to Licensee by a Sublicensee as royalties on sales of Licensed Product sold by the Sublicensee under a sublicense agreement. 

3.5.3 To the extent Licensee receives payment from a Third Party relating to one or more of the milestone events set forth in the table in
Section 3.3, then the amount of the payment 

  
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made to Licensor under such Section 3.3 with respect to such milestone event shall not be deemed sublicense fees under this Section 3.5; instead, the amounts due under this
Section 3.5 shall be calculated by applying the applicable sublicense fee rate set forth in Section 3.5.1 above to the sublicense fees received by Licensee from such Third Party after deducting the amount of the payment under
Section 3.3. By way of example, 
  

			
	Assume:	 	(a) Sublicensee was granted a sublicense prior to the first anniversary of the Effective Date,
		
		 	(b) Sublicensee achieves an NDA submission in the United States, and
		
		 	(c) Sublicensee pays Licensee **** upon the achievement of such event.

 Then, Licensee would owe Licensor ****, calculated as follows: 

(i) **** pursuant to Section 3.3 (based on milestone event #2), and 

(ii) **** pursuant to this Section 3.5, which is calculated as follows: 

 

	
	(x) the sublicense fee rate of **** multiplied by
	
	(y) **** (which is determined by subtracting the **** milestone payment under Section 3.3 from the **** payment received from Sublicensee).

  

			
	Alternatively:	 	(A) If Sublicensee paid Licensee nothing upon the achievement of an NDA submission in the United States, then Licensee would owe Licensor (1) **** pursuant to Section 3.3 (based on milestone event #2) and (2) no
amounts under this Section 3.5; and
		
		 	(B) If Sublicensee paid Licensee **** upon the achievement of an NDA submission in the United States, then Licensee would owe Licensor (1) **** pursuant to Section 3.3 (based on milestone event #2) and (2) no
amounts under this Section 3.5.

 3.5.4 If Licensee receives sublicense fees from Sublicensees or from any person or entity granted any option
to obtain a sublicense under this Agreement in the form of non-cash consideration, then, at Licensor’s option, Licensee shall pay Licensor payments as required by this Section 3.5 (a) in the form of the non-cash consideration received
by Licensee or (b) a cash payment determined based on the fair market value of such non-cash consideration. 
 3.6 Reports and Records. 

3.6.1 Licensee must deliver to Licensor within **** after the end’ of each Calendar Quarter after the first commercial sale of a Licensed
Product a report setting forth the calculation of the royalties due to Licensor for such Calendar Quarter, including: 

  
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	 	(a)	Number of Licensed Products included within Net Sales, listed by country; 

  

	 	(b)	Gross consideration for Net Sales of Licensed Product, including all amounts invoiced, billed, or received; 

  

	 	(c)	Qualifying costs to be excluded from the gross consideration, as described in Section 1.13, listed by category of cost; 

  

	 	(d)	Net Sales of Licensed Products listed by country; 

  

	 	(e)	A detailed accounting of any royalty reductions applied pursuant to Section 3.4.1; 

  

	 	(f)	Royalties owed to Licensor, listed by category; and 

  

	 	(g)	The computations for any applicable currency conversions. 

 3.6.2 Licensee shall pay the
royalties due under Section 3.4 within **** following the last day of the Calendar Quarter in which the royalties accrue. Licensee shall send the royalty payments along with the report described in Section 3.6.1. 

3.6.3 Within **** after the occurrence of a milestone event described in Section 3.3, Licensee must deliver to Licensor a report
describing the milestone event that occurred, together with a payment of the applicable amount due to Licensor pursuant to Section 3.3. In addition, within **** after the receipt of sublicense fees from any Sublicensee as described in
Section 3.5, Licensee must deliver to Licensor a report describing the fees received, together with a payment of the applicable amount due to Licensor pursuant to Section 3.5. 

3.6.4 All financial reports under this Section 3.6 will be certified by the chief financial officer of Licensee. 

3.6.5 Licensee shall maintain and require its Affiliates and all Sublicensees to maintain, complete and accurate books and records which
enable the royalties, fees, and payments payable under this Agreement to be verified. The records must be maintained for **** after the submission of each report under Article 3. Upon reasonable prior written notice to Licensee, Licensee and its
Affiliates and all Sublicensees will provide Licensor and/or its direct or indirect licensors (and their respective accountants) with access to all of the relevant books, records, and related background information required to conduct a review or
audit of the royalties, fees, and payments payable to Licensor under this Agreement to be verified. Access will be made available: (a) during normal business hours; (b) in a manner reasonably designed to facilitate the auditing
party’s review or audit without unreasonable disruption to Licensee’s business; and (c) no more than once each calendar year during the term of this Agreement and for a period of **** thereafter. Licensee will promptly pay to Licensor
the amount of any underpayment determined by the review or audit, plus accrued interest. Licensor will promptly reimburse to License the amount of any overpayment determined by the review or audit, but no accrued interest will apply. If the review
or audit determines that Licensee has underpaid any payment 

  
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by **** or more, then Licensee will also promptly pay the costs and expenses of Licensor and or its direct or indirect licensors and accountants in connection with the review or audit. 

3.7 Currency, Interest. 
 3.7.1 All
dollar amounts referred to in this Agreement are expressed in United States dollars. All payments to Licensor under this Agreement must be made in United States dollars. 

3.7.2 If Licensee receives payment in a currency other than United States dollars for which a royalty or fee or other payment is owed under
this Agreement, then (a) the payment will be converted into United States dollars at the conversion rate for the foreign currency as published in the eastern edition of the Wall Street Journal, N.Y. edition, as of the last business day of the
Calendar Quarter in which the payment was received by Licensee; and (b) the conversion computation will be documented by Licensee in the applicable report delivered to Licensor under Section 3.6. 

3.7.3 All amounts that are not paid by Licensee when due will accrue interest from the date due until paid at a rate equal to 1.5% per
month (or the maximum allowed by law, if less). 
 3.8 Taxes and Withholding. 

3.8.1 All payments hereunder will be made free and clear of, and without deduction or deferment in respect of, and Licensee shall pay and be
responsible for, and shall hold Licensor harmless from and against, any taxes, duties, levies, fees, or charges, including sales, use, transfer, excise, import, and value added taxes (including any interest, penalties, or additional amounts imposed
with respect thereto) but excluding withholding taxes to the extent provided in Section 3.8.2. At the request of Licensee, Licensor will give Licensee such reasonable assistance, which will include the provision of documentation as may be
‘required by the relevant tax authority, to enable Licensee to pay and report and, as applicable, claim exemption from or reduction of, such tax, duty, levy, fee, or charge. 

3.8.2 If any payment made by Licensee hereunder becomes subject to withholding taxes with respect to Licensor’s gross or net income under
the laws of any jurisdiction, Licensee will deduct and withhold the amount of such taxes for the account of Licensor to the extent required by law and will pay the amounts of such taxes to the proper governmental authority in a timely manner and
promptly transmit to Licensor appropriate proof of payment of such withholding taxes. At the request of Licensor, Licensee will give Licensor such reasonable assistance, which will include the provision of appropriate certificates of such deductions
made together with other supporting documentation as may be required by the relevant tax authority, to enable Licensor to claim exemption from or reduction of, or otherwise obtain repayment of, such withholding taxes, and will upon request provide
such additional documentation from time to time as is reasonably required to confirm the payment of withholding tax. 
 ARTICLE 4:
DILIGENCE 
 4.1 Diligence Obligations. Licensee will use commercially reasonable efforts to develop, commercialize, market, promote, and sell
Licensed Products in the Field. Commercially reasonable efforts means efforts equivalent to those utilized by **** 

  
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****. Without limiting the foregoing, Licensee will achieve first treatment in a Phase 3 Clinical Trial (i.e., first patient, first dose) by no later than ****. Licensee will notify Licensor in
writing as soon as Licensee believes in good faith that Licensee will not be able to achieve the foregoing milestone by the relevant deadline date, and, upon the payment to Licensor of **** within ****of the original deadline date, the deadline date
for such milestone set will be extended for ****from the original deadline date; provided that Licensee will only be ****. 
 ****. 

4.2 Reporting. Within **** after the Effective Date and within **** of each December 1 thereafter, Licensee shall provide Licensor with written
progress reports, setting forth in such detail as Licensor may reasonably request, the progress of the development, evaluation, testing, and commercialization of each Licensed Product. Licensee will also notify Licensor within **** of the first
commercial sale by Licensee, its Affiliates, or any Sublicensees of each Licensed Product. Such a report (“Development Progress Report”), setting forth the current stage of development of Licensed Products, shall include: 

4.2.1 Date of Development Progress Report and time covered by such report; 

4.2.2 Major activities and accomplishments completed by Licensee, its Affiliates, and any Sublicensees relating directly to the Licensed
Product since the last Development Progress Report; 
 4.2.3 Significant research and development projects relating directly to the Licensed
Product currently being performed by Licensee, its Affiliates, and any Sublicensees and projected dates of completion; 
 4.2.4 A
development plan covering the next two years at least, which will include future development activities to be undertaken by Licensee, its Affiliates, or any Sublicensees during the next reporting period relating directly to the Licensed Product,
Licensee’s strategy to bring the Licensed Product to commercialization, and projected timeline for completing the necessary tasks to accomplish the goals of the strategy; 

4.2.5 Projected total development remaining before product launch of each Licensed Product; and 

  
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 4.2.6 Summary of significant development efforts using the Licensed Patents being performed
by Third Parties, including the nature of the relationship between Licensee and such Third Parties. 
 4.3 Confidential Information. The Parties
agree that Development Progress Reports shall be deemed Licensee’s Confidential Information; provided that Licensor may share a copy of such reports with its direct and indirect licensors. 

4.4 Improvements. Simultaneously with the Development Progress Report, Licensee shall deliver a detailed description of any Licensed Back Improvements,
if not previously provided pursuant to Section 2.5.3. 
 ARTICLE 5: CONFIDENTIALITY 

5.1 Treatment of Confidential Information. Each Party, as a receiving party (a “Receiving Party”), agrees that it will (a) treat
Confidential Information of the other Party (the “Disclosing Party”) as strictly confidential; (b) not disclose such Confidential Information to Third Parties without the prior written consent of the Disclosing Party, except as may be
permitted in this Agreement; provided that any disclosure permitted hereunder be under confidentiality agreements with provisions at least as stringent as those contained in this Agreement; and (c) not use such Confidential Information for
purposes other than those authorized expressly in this Agreement. The Receiving Party agrees to ensure that its employees who have access to Confidential Information are obligated in writing to abide by confidentiality obligations at least as
stringent as those contained under this Agreement. 
 5.2 Public Announcements. The Parties agree they will release a joint press release in the form
attached hereto as Exhibit B. Any other press releases by either Party with respect to the other Party or any other public disclosures concerning the existence of or terms of this Agreement shall be subject to review and approval by the other Party.
Once the joint press release or any other written statement is approved for disclosure by both Parties, either Party may make subsequent public disclosure of the contents of such statement without the further approval of the other Party. 

5.3 Authorized Disclosure. Notwithstanding the provisions of Section 5.1 or 5.2, either Party may disclose Confidential Information or make such a
disclosure of the existence of and/or terms of this Agreement to any ****; provided that, in each case, such recipient of Confidential Information is obligated to keep such information confidential on terms no less stringent than those set forth in
this Agreement. Furthermore, Licensee agrees that Licensor may share a copy of this Agreement, reports and notices provided by Licensee to Licensor pursuant to the terms of this Agreement, and copies of sublicense agreements provided to Licensor
hereunder with any of Licensor’s direct and indirect licensors of the Licensed Patents. In the event l that the Receiving Party receives service of legal process that purports to compel disclosure of the Disclosing Party’s Confidential
Information 1 or becomes obligated by law to disclose the Confidential Information of the Disclosing Party or the existence of or terms of this Agreement to any governmental authority, the Receiving Party shall promptly notify the 

  
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Disclosing Party, so that the Disclosing Party may seek an appropriate protective order or other remedy with respect to narrowing the scope of such requirement and/or waive compliance by the
Receiving Party with the provisions of this Agreement. The Receiving Party will provide the Disclosing Party with reasonable assistance in obtaining such protective order or other remedy. If, in the absence of such protective order or other remedy,
the Receiving Party is nonetheless required by law to disclose the existence of or terms of this Agreement or other Confidential Information of the Disclosing Party, the Receiving Party may disclose such Confidential Information without liability
hereunder; provided that the Receiving Party shall furnish only such portion of the Confidential Information that is legally required to be disclosed and only to the extent required by law. 

5.4 Term of Confidentiality. The obligations of this Article 5 shall continue for a period of **** following the expiration or termination of this
Agreement. 
 ARTICLE 6: TERM AND TERMINATION 

6.1 Term of Agreement. This Agreement, unless sooner terminated as provided in this Agreement, expires upon the expiration, lapse, abandonment, or
invalidation of the last Valid Claim to expire, lapse, or become abandoned or unenforceable in all countries of the world. 
 6.2 Licensee’s Right
to Terminate. Licensee may, upon 90 days’ prior written notice to Licensor, terminate this Agreement for any reason, with or without cause; provided that, if such termination notice is sent prior to the first anniversary of the Effective
Date, such termination notice shall be accompanied by Licensee’s payment of $400,000 in satisfaction of the remainder of the initial fee under Section 3.1. 

6.3 Termination for Breach. 
 6.3.1
Licensor may terminate this Agreement, if Licensee is late in paying to Licensor royalties, fees, or any other monies due under this Agreement, and Licensee does not pay Licensor in full within 15 days upon written demand from Licensor, which
termination shall be effective immediately upon the expiration of such 15-day cure period. 
 6.3.2 Either Party may terminate this
Agreement, if the other Party materially breaches this Agreement and does not cure such material breach within 30 days after written notice of the breach, which termination shall be effective immediately upon the expiration of such 30-day cure
period. 
 6.4 Termination for Insolvency. 

6.4.1 Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, if Licensee or any of its Affiliates
experiences any Trigger Event. 
 6.4.2 Licensee shall include in each sublicense agreement entered into with a Sublicensee a right of
Licensee to terminate such sublicense agreement if such Sublicensee experiences any Trigger Event; and Licensee shall terminate the sublicense agreement, effective immediately upon written notice to the Sublicensee, if the Sublicensee experiences
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licensor a right of termination under the Penn Agreement, then, upon receipt of such notice, Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, if any
Sublicensee experiences any Trigger Event. 
 6.4.3 For purposes of this Section 6.4, “Trigger Event” means any of the
following: (a) if Licensee, any Affiliate, or any Sublicensee, as applicable, (i) becomes insolvent, becomes bankrupt, or generally fails to pay its debts as such debts become due, (ii) is adjudicated insolvent or bankrupt,
(iii) admits in writing its inability to pay its debts, (iv) suffers the appointment of a custodian, receiver, or trustee for it or its property and, if appointed without its consent, is not discharged within 30 days, (v) makes an
assignment for the benefit of creditors, or (vi) suffers proceedings being instituted against it under any law related to bankruptcy, insolvency, liquidation, or the reorganization, readjustment, or release of debtors and, if contested by it,
not dismissed or stayed within ten days; (b) the institution or commencement by Licensee, any Affiliate, or any Sublicensee, as applicable, of any proceeding under any law related to bankruptcy, insolvency, liquidation, or the reorganization,
readjustment, or release of debtors; (c) the entering of any order for relief relating to any of the proceedings described in Section 6.4.3(a) or (b) above; (d) the calling by Licensee, any Affiliate, or any Sublicensee, as
applicable, of a meeting of its creditors with a view to arranging a composition or adjustment of its debts; or (e) the act or failure to act by Licensee, any Affiliate, or any Sublicensee, as applicable, indicating its consent to, approval of,
or acquiescence in any of the proceedings described in Section 6.4.3(b) through (d) above. 
 6.5 Patent Challenge. 

6.5.1 Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, upon the commencement by Licensee or any
of its Affiliates of a Patent Challenge. 
 6.5.2 Licensee shall include in each sublicense agreement entered into with a Sublicensee a
right of Licensee to terminate such sublicense agreement if such Sublicensee commences a Patent Challenge; and Licensee shall terminate the sublicense agreement, effective immediately upon written notice to the Sublicensee, if the Sublicensee
commences a Patent Challenge. In addition, if the Sublicensee’s commencement of a Patent Challenge gives Licensor’s licensor a right of termination under the Penn Agreement, then, upon receipt of such notice, Licensor may terminate this
Agreement, effective immediately upon written notice to Licensee, if any Sublicensee commences a Patent Challenge. 
 6.5.3 For purposes of
this Section 6.5, “Patent Challenge” means any action against Licensor, the University of Pennsylvania, or any direct or indirect licensor of Licensor, including an action for declaratory judgment, to declare or render invalid or
unenforceable the Licensed Patents, or any claim thereof 
 6.6 Effects of Termination. The effect of termination by Licensee pursuant to
Section 6.2, by either Party, as applicable, under Section 6.3, or by Licensor pursuant to Section 6.4 or 6.5 shall be as follows: 

  
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 6.6.1 The licenses granted by Licensor hereunder shall terminate, and Licensee and its
Affiliates shall cease to make, have made, use, import, sell, and offer for sale all Licensed Products and shall cease to otherwise practice the Licensed Patents; provided that Licensee, its Affiliates, and Sublicensees shall have the right to
continue to sell their existing inventories of Licensed Products for a period not to exceed **** after the effective date of such termination; 

6.6.2 All sublicenses granted to Third Parties to the extent of the rights licensed to Licensee hereunder and sublicensed to the Sublicensee
shall be assigned to Licensee; provided that (i) prior to such assignment, Licensee shall advise Licensor whether such Sublicensee is then in full compliance with all terms and conditions of its sublicense and continues to perform thereunder,
and, if such Sublicensee is not in full compliance or is not continuing to perform, Licensor may elect not to have such sublicense assigned; and (ii) following such assignment, Licensor shall not be liable to such Sublicensee with respect to
any obligations of Licensee to the Sublicensee that are not consistent with, or not required by, Licensor’s obligations to Licensee under this Agreement; 

6.6.3 If termination is by Licensee pursuant to Section 6.2 or by Licensor pursuant to Section 6.3, 6.4, or 6.5, Licensee shall
grant, and hereby grants to Licensor a non-exclusive, perpetual, irrevocable, worldwide, royalty-free, transferable, sublicensable license under any patentable modifications or improvements (and any intellectual property rights with respect thereto)
developed by Licensee, any Affiliates, or any Sublicensees to any vector that is the subject of a claim within any of the Licensed Patents, for use by Licensor for the research, development, and commercialization of products in any therapeutic
indication; 
 6.6.4 Licensee shall pay all monies then-owed to Licensor under this Agreement, and, if applicable, Licensor shall pay all
monies then-owed to Licensee under this Agreement; 
 6.6.5 The Parties acknowledge and agree that, if the GSK Agreement is terminated as
described in Section 6.5 of the GSK Agreement, then, as provided in Section 6.5.2 thereof, Licensor will assign this Agreement to the licensor of the GSK Agreement to the extent this Agreement is related solely to the rights and products
licensed to Licensor under the GSK Agreement; and 
 6.6.6 Each Receiving Party shall, at the other Party’s request, return all
Confidential Information of the Disclosing Party. Notwithstanding the foregoing, one copy may be kept by either Party for a record of that Party’s obligations. 

6.7 Survival. Licensee’s obligation to pay all monies due and owed to Licensor under this Agreement, and Licensor’s obligation to pay all
monies due and owed to Licensee under this Agreement, in each case, which have matured as of the effective date of termination or expiration shall survive the termination or expiration of this Agreement. In addition, the provisions of
Section 2.2, (Retained Rights), 2.3 (Government Rights), 2.5 (Improvements), 3.1 (Initial Fee), Article 3 (Consideration) (with respect to any final reports or to the extent any amounts are due but unpaid), Section 3.6 (Reports and
Records), Article 5 (Confidentiality), Article 6 (Term and Termination), Section 8.3 (Disclaimer of Warranties, Damages), Section 8.4 (Indemnification), Section 8.5 (Insurance), Article 9 (Use of Name), and Article 10 (Additional 

  
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Provisions) shall survive such termination or expiration of this Agreement in accordance with their respective terms. 

ARTICLE 7: PATENT MAINTENANCE; PATENT INFRINGEMENT 

7.1 Prosecution of Licensed Patents. As between Licensor and Licensee, the Parties agree as follows: 

7.1.1 Licensor shall have the sole right, but not the obligation, to Prosecute patent applications and issued patents within Licensed Patents,
in Licensor’s sole discretion. Subject to Section 7.1.3, Licensor shall provide Licensee with a reasonable opportunity to review and provide comments in connection with the Prosecution of the Licensed Patents; and Licensor shall keep
Licensee reasonably informed as to all material developments with respect to such Licensed Patents and shall supply to Licensee copies of material communications received and filed in connection with the Prosecution of such Licensed Patents. 

7.1.2 Nothing in this Agreement obligates Licensor to continue to Prosecute any patent applications or issued patents, and Licensee
acknowledges that Licensor shall have no obligation to undertake any inter-party proceedings, such as oppositions or interferences, or to undertake any re-examination or re-issue proceedings, in either case, with respect to the Licensed Patents.

 7.1.3 Licensee acknowledges that the University of Pennsylvania controls Prosecution of the Licensed Patents, with Licensor having
certain rights to review. Licensee acknowledges and agrees that (a) the rights and obligations under this Section 7.1 are subject to the rights of Licensor’s direct and indirect licensors with respect to the Licensed Patents, and
(b) Licensor’s obligations under this Agreement only apply to the extent of Licensor’s rights with respect to participation in Prosecuting the Licensed Patents under its agreements with its direct and indirect licensors. 

7.2 Infringement Actions Against Third Parties. 

7.2.1 Licensee is responsible for notifying Licensor promptly of any infringement of Licensed Patents (other than Retained Rights) that may
come to Licensee’s attention. Licensee and Licensor shall consult one another in a timely manner concerning any appropriate response to the infringement. 

7.2.2 As between Licensor and Licensee, Licensor shall have the first right, but not the obligation, to prosecute any such infringement ****.
In any action to enforce any of the Licensed Patents, Licensee, at the request and expense of Licensor, shall cooperate to the fullest extent reasonably possible, including in the event that, if Licensor is unable to initiate or prosecute such
action solely in its own name, Licensee shall join such action voluntarily and shall execute all documents necessary to initiate litigation to prosecute, maintain, and settle such action. 

7.2.3 If Licensor elects not to pursue any infringement of a Licensed Patent, then, to the extent that a Licensed Product is covered by an))
such License Patent and such Licensed Patent is being infringed by another product in the Field (such infringement, the “Competitive Infringement”), Licensee shall have the second right, but not the obligation, to prosecute such 

  
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Competitive Infringement with respect to such other product in the Field, at Licensee’s own expense. In any such action to enforce any of the Licensed Patents, Licensor, at the request and
expense of Licensee, shall cooperate to the fullest extent reasonably possible, including in the event that, if Licensee is unable to initiate or prosecute such action solely in its own name, Licensor shall join such action voluntarily and shall
execute all documents necessary to initiate litigation to prosecute and maintain such action. In prosecuting any such Competitive Infringement, Licensee (a) shall not take any actions that would be detrimental to the Licensed Patents and
Licensor’s rights with respect thereto outside the Field and (b) shall not settle any such Competitive Infringement without the prior consent of Licensor. 

7.2.4 Any recovery of damages by Licensor for any infringement other than a Competitive Infringement shall be ****. Any recovery of damages by
the Party undertaking enforcement or defense of a suit for Competitive Infringement shall be applied, as between Licensor and Licensee but subject to the obligations to Licensor’s direct and indirect licensors, first to reimburse each such
Party for costs and expenses (including reasonable attorneys’ fees and costs) incurred by such Party in connection with such suit, and the balance remaining, if any, from any such recovery shall be ****. 

7.2.5 Licensee acknowledges and agrees that (a) the rights and obligations under this Section 7.2 are subject to the rights of
Licensor’s direct and indirect licensors of the Licensed Patents (including any consent or approval rights or rights to control or participate in any enforcement actions); and (b) Licensor’s obligations under this Agreement only apply
to the extent that Licensor has any rights with respect to enforcing the Licensed Patents under its agreements with its direct and indirect licensors. Furthermore, Licensee acknowledges the following: 

7.2.5.1 All monies recovered upon the final judgment or settlement of any action with respect to Competitive Infringement will
also need to be allocated to Licensor’s direct and indirect licensors (a) to reimburse the costs and expenses (including reasonable attorneys’ fees and costs) of such licensors, (b) to take into account the royalties payable to
such licensors; and (c) to take into account the relative extent of such licensors’ financial participation in such action, if applicable. 

7.2.5.2 Licensor’s direct and indirect licensors retain the continuing right to intervene at their own expense and join
Licensor or Licensee in any claim or suit for infringement of the Licensed Patents. 
 7.2.5.3 In any infringement prosecuted
by Licensors’ direct and indirect licensors, all financial recoveries will be ****. 
 7.2.5.4 In any infringement
prosecuted by Licensor’s direct and indirect licensors, Licensee agrees, at the request and expense of such licensors, to cooperate to the fullest extent reasonably possible, to the same extent as though Licensor were prosecuting such suit (as
provided in this Section 7.2, including Section 7.2.2). 
 7.2.5.5 The written consent of Licensor’s direct
and indirect licensors will be required (a) for any decision that would have a materially adverse effect on the validity, 

  
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scope of patent claims, or enforceability of the Patent Rights and (b) for any settlement or compromise of any infringement suit that would impose any obligations or restrictions on any of
its direct or indirect licensors, or grants any rights to the Licensed Patents other than rights that Licensee has the right to grant under this Agreement. 

7.3 Defense of Infringement Claims. In the event Licensee or Licensor becomes aware that Licensee’s or any of its Affiliates’ or any
Sublicensees’ practice of the Licensed Patents is the subject of a claim for patent infringement by a Third Party, that Party shall promptly notify the other, and the Parties shall consider the claim and the most appropriate action to take.
Licensee shall cause each of its Affiliates and each Sublicensee to notify Licensee promptly in the event such entity becomes aware that its practice of the Licensed Patents is the subject of a claim of patent infringements by another. To the extent
Licensor takes any action, Licensor (or its direct or indirect licensors) shall have the right to require Licensee’s reasonable cooperation in any such suit, upon written notice to Licensee; and Licensee shall have the obligation to participate
upon Licensor’s request, in which event, Licensor shall bear the cost of Licensee’s participation. Without Licensor’s prior written permission, Licensee must not settle or compromise any such suit in a manner that imposes any material
obligations or restrictions on Licensor or any of its direct or indirect licensors or grants any rights to the Licensed Patents other than rights that Licensee has the right to grant under this Agreement. 

ARTICLE 8: WARRANTIES; INDEMNIFICATION 
  

	8.1	Warranty by Licensor. Licensor represents and warrants to Licensee as of the Effective Date: 

8.1.1 Licensor is duly organized, validly existing, and in good standing under the laws of the jurisdiction of its formation; 

8.1.2 Licensor has taken all necessary action on its part to authorize the execution of this Agreement and the performance of all of its
obligations under this Agreement and the persons executing this Agreement are authorized to execute it; 
 8.1.3 Licensor has the right,
power, and authority to enter into this Agreement and to grant to Licensee the rights specified in this Agreement; 
 8.1.4 This Agreement
when executed shall become the legal, valid and binding obligation of it, enforceable against it, in accordance with its terms; 
 8.1.5
There are no actions, suits, proceedings, or arbitrations pending or, to Licensor’s knowledge, threatened against Licensor relating to the Licensed Patents that would impact activities under this Agreement; 

8.1.6 To Licensor’s knowledge, (a) the Licensed Patents are solely owned by the University of Pennsylvania, and (b) no Third
Party (other than Licensor’s direct and indirect licensors) has any right, interest? or claim in or to such Licensed Patents in the Field that are inconsistent with those granted td Licensee in the Field under this Agreement; 

  
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 8.1.7 To Licensor’s knowledge, no Third Party is infringing any of the Licensed Patents
in a manner that is inconsistent with the scope of rights granted to Licensee in the Field under this Agreement; 
 8.1.8 Licensor has not
received any written notice from any Third Party patentee alleging infringement of such Third Party’s patents by the practice of the Licensed Patents in the Field; 

8.1.9 To Licensor’s knowledge, the Penn Agreement and GSK Agreement are in full force and effect and Licensor is not in breach of any
provisions thereof; and 
 8.1.10 To Licensor’s knowledge, Licensor does not Control as of the Effective Date any patent or patent
application (other than the Licensed Patents) that has a claim expressly reciting a composition of matter of AAVrh10 (as defined in Section 1.1(a)). For the purpose of this Section 8.1.10, “Control” means the possession by
Licensor (whether by ownership or license, other than pursuant to this Agreement) of the ability to grant to Licensee access, a license, or a sublicense (as applicable) to the applicable patent or patent application on the terms and conditions set
forth herein without violating the terms of any agreement or other arrangement with any Third Party. 
  

	8.2	Warranty by Licensee. Licensee represents and warrants to Licensor as of the Effective Date that: 

8.2.1 Licensee is duly organized, validly existing, and in good standing under the laws of the jurisdiction of its formation; 

8.2.2 Licensee has taken all necessary action on its part to authorize the execution of this Agreement and the performance of all of its
obligations under this Agreement and the persons executing this Agreement are authorized to execute it; 
 8.2.3 Licensee has the right,
power, and authority to enter into this Agreement and to grant the rights granted by it hereunder; 
 8.2.4 This Agreement when executed
shall become the legal, valid and binding obligation of it, enforceable against it, in accordance with its terms; 
 8.2.5 Licensee has the
ability and the resources, including financial resources, necessary to carry out its obligations under this Agreement; and 
 8.2.6 There
are no actions, suits, proceedings, or arbitrations pending or, to the Licensee’s knowledge, threatened against Licensee that would impact activities under this Agreement. 

8.3 Disclaimer of Warranties, Damages. EXCEPT AS SET FORTH IN SECTION 8.1, THE LICENSED PATENTS, LICENSED PRODUCTS, AND ALL RIGHTS LICENSED UNDER THIS
AGREEMENT ARE PROVIDED ON AN “AS IS” BASIS, AND LICENSOR MAKES NO REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, WITH RESPECT THERETO. BY WAY OF EXAMPLE BUT NOT OF LIMITATION, LICENSOR MAKES NO

  
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REPRESENTATIONS OR WARRANTIES, AND HEREBY DISCLAIMS ALL EXPRESS AND IMPLIED REPRESENTATIONS AND WARRANTIES, (i) OF COMMERCIAL UTILITY, ACCURACY, COMPLETENESS, PERFORMANCE, MERCHANTABILITY,
FITNESS FOR A PARTICULAR PURPOSE, VALIDITY OR ENFORCEABILITY OF THE LICENSED PATENTS, AND PROFITABILITY; OR (ii) THAT THE USE OF THE LICENSED PATENTS OR LICENSED PRODUCTS WILL NOT INFRINGE ANY PATENT, COPYRIGHT, TRADEMARK, OR OTHER PROPRIETARY
RIGHTS OF THIRD PARTIES. EXCEPT AS SET FORTH HEREIN, NONE OF LICENSOR OR ANY OF LICENSOR’S DIRECT OR INDIRECT LICENSORS SHALL BE LIABLE TO LICENSEE, LICENSEE’S SUCCESSORS OR ASSIGNS, ANY SUBLICENSEES, OR ANY THIRD PARTY WITH RESPECT TO:
(a) ANY CLAIM ARISING FROM USE OF THE LICENSED PATENTS, LICENSED PRODUCTS, AND ANY OR ALL RIGHTS LICENSED UNDER THIS AGREEMENT OR FROM THE DEVELOPMENT, TESTING, MANUFACTURE, USE, OR SALE OF LICENSED PRODUCTS; OR (b) ANY CLAIM FOR LOSS OF
PROFITS, LOSS OR INTERRUPTION OF BUSINESS, OR FOR INDIRECT, SPECIAL, INCIDENTAL, PUNITIVE, OR CONSEQUENTIAL DAMAGES OF ANY KIND, INCLUDING ANY ARISING FROM OR RELATING TO ANY BREACH OF THIS AGREEMENT OR THE EXERCISE OF RIGHTS HEREUNDER, REGARDLESS
OF ANY NOTICE OF SUCH DAMAGES. NOTHING IN THIS SECTION 8.3 IS INTENDED TO LIMIT OR RESTRICT THE INDEMNIFICATION RIGHTS OR OBLIGATIONS OF EITHER PARTY UNDER SECTION 8.4 OR TO LIMIT A PARTY’S LIABILITY FOR BREACHES OF ITS OBLIGATION REGARDING
CONFIDENTIALITY UNDER Article 5. 
 8.4 Indemnification. 

8.4.1 By Licensee. Licensee shall defend, indemnify, and hold harmless Licensor, its direct and indirect licensors of the Licensed
Patents, and their respective shareholders, members, officers, trustees, faculty, students, contractors, agents, and employees (individually, a “Licensor Indemnified Party” and, collectively, the “Licensor Indemnified Parties”)
from and against any and all Third Party liability, loss, damage, action, claim, fee, cost, or expense (including attorneys’ fees) (individually, a “Third Party Liability” and, collectively, the “Third Party Liabilities”)
suffered or incurred by the Licensor Indemnified Parties from claims of such Third Parties that result from or arise out of: ****; provided, however, that Licensee shall not be liable for claims based on any breach by Licensor of the
representations, warranties, or obligations of this Agreement or the gross negligence or intentional misconduct of any of the Licensor Indemnified Parties. Without limiting the foregoing, Licensee must defend, indemnify, and hold harmless the
Licensor Indemnified Parties from and against any Third Party Liabilities resulting from: 
  

	 	(a)	any **** or other claim of any kind related to the **** by a Third Party of a **** by Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors; 

  
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	 	(b)	any claim by a Third Party that the ****; and 

  

	 	(c)	**** conducted by or on behalf of Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors relating to the Licensed Patents or Licensed Products, including any claim by or on behalf of a ****.

 8.4.2 By Licensor. Licensor shall defend, indemnify, and hold harmless Licensee, its shareholders, members,
officers, contractors, agents, and employees (individually, a “Licensee Indemnified Party” and, collectively, the “Licensee Indemnified Parties”) from and against any and all Third Party Liabilities suffered or incurred by the
Licensee Indemnified Parties from claims of such Third Parties that result from or arise out of: **** provided, however, that Licensor shall not be liable for claims based on any breach by Licensee of the representations, warranties, or obligations
of this Agreement or the gross negligence or intentional misconduct of any of the Licensee Indemnified Parties. 
 8.4.3 Indemnification
Procedure. Each Party, as an indemnifying party (an “Indemnifying Party”), shall not be permitted to settle or compromise any claim or action giving rise to Third Party Liabilities in a manner that imposes any restrictions or
obligations on any indemnified party (an “Indemnified Party”) without the Indemnified Party’s prior written consent or, if Licensee is the Indemnifying Party, that imposes any restrictions Or obligations on Licensor’s direct or
indirect licensors or grants any rights to the Licensed Patents or Licensed Products other than those Licensee has the right to grant under this Agreement without Licensor’s prior written consent. The Indemnifying Party shall be permitted to
control any litigation or potential litigation involving the defense of any claim subject to indemnification pursuant to this Section 8.4, including the selection of counsel, with the reasonable approval of the Indemnified Party. If an
Indemnifying Party fails or declines to assume the defense of any such claim or action within **** after notice thereof, the Indemnified Party may assume the defense of such claim or action at the cost and risk of the Indemnifying Party, and any
Third Party Liabilities related thereto shall be conclusively deemed a Third Party Liability of the Indemnifying Party. The indemnification rights of a Indemnified Party contained in this Agreement are in addition to all other rights that such
Indemnified Party may have at law or in equity or otherwise. The Indemnifying Party will pay directly all Third Party Liabilities incurred for defense or negotiation of any claim hereunder or will reimburse the Indemnified Party for all documented
Third Party Liabilities incident to the defense or negotiation of any such claim within **** after the Indemnifying Party’s receipt of invoices for such fees, expenses, and charges. 

8.5 Insurance. Licensee will procure and maintain insurance policies for the following coverages with respect to product liability, personal injury,
bodily injury, and property damage arising out of Licensee’s (and its Affiliates’ and any Sublicensees’) performance under this Agreement: (a) during the term of this Agreement, comprehensive general liability, including 

  
 23 

 ****CERTAIN INFORMATION HAS BEEN
OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN
REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
broad form and contractual liability, in a minimum amount of **** combined single limit per occurrence (or claim) and in the aggregate annually; (b) prior to the commencement of clinical
trials involving Licensed Products and thereafter for a period of not less than **** (or such longer period as Licensee is required by applicable law to continue to monitor the participants in the clinical trial), clinical trials coverage in amounts
that are reasonable and customary in the U.S. pharmaceutical industry, subject always to a minimum limit of **** combined single limit per occurrence (or claim) and in the aggregate annually; and (c) from prior to the first commercial sale of a
Licensed Product until **** after the last sale of a Licensed Product, product liability coverage, in amounts that are reasonable and customary in the U.S. pharmaceutical industry, subject always to a minimum limit of **** combined single limit per
occurrence (or claim) and in the aggregate annually. Licensor may review periodically the adequacy of the minimum amounts of insurance for each coverage required by this Section 8.5, and Licensor reserves the right to require Licensee to adjust
the limits accordingly. The required minimum amounts of insurance do not constitute a limitation on Licensee’s liability or indemnification obligations to the Licensor Indemnified Parties under this Agreement. The policies of insurance required
by this Section 8.5 will be issued by an insurance carrier with an A.M. best rating of **** or better and will name Licensor as an additional insured with respect to Licensee’s performance (and its Affiliates’ and any
Sublicensees’) under this Agreement. Licensee will provide Licensor with insurance certificates evidencing the required coverage within **** after the Effective Date and the commencement of each policy period and any renewal periods. Each
certificate will provide that the insurance carrier will notify Licensor in writing at least **** prior to the cancellation or material change in coverage. Licensee will cause all Sublicensees to comply with the terms of this Section 8.5 to the
same extent as Licensee. 
 ARTICLE 9: USE OF NAME 

Licensee, its Affiliates, any Sublicensees, and all of its and their employee’s and agents must not use Licensor’s, the University of
Pennsylvania’s, or SmithKline Beecham Corporation’s name, seal, logo, trademark, or service mark (or any adaptation thereof) or the name, seal, logo, trademark, or service mark (or any adaptation thereof) of any of such entities’
representative, school, organization, employee, or student in any way without the prior written consent of Licensor or such entity, as applicable; provided, however that Licensee may acknowledge the existence and general nature of this Agreement.

 ARTICLE 10: ADDITIONAL PROVISIONS 

10.1 Relationship. Nothing in this Agreement shall be deemed to establish a relationship of principal and agent between Licensee and Licensor, nor any
of their agents or employees for any purpose whatsoever, nor shall this Agreement be construed as creating any other form of legal association or arrangement which would impose liability upon one Party for the act or failure to act of the other
Party. 
 10.2 Assignment. The rights and obligations of Licensee and Licensor hereunder shall inure to the benefit of, land shall be binding upon,
their respective permitted successors and assigns. Licensee may not assign this Agreement or any of its rights or obligations under this Agreement without the prior written consent of Licensor; provided, however, that Licensee may assign this 

  
 24 

 ****CERTAIN INFORMATION HAS BEEN
OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN
REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
Agreement, without Licensor’s prior written consent, pursuant to a merger or sale of all or substantially all of the assets to which the Agreement relates; provided that, as part of any
permitted assignment, (a) Licensee provides Licensor with notice of such assignment at least five business days prior to the effectiveness of such assignment, and (b) Licensee requires any such assignee to agree in writing to be legally
bound by this Agreement to the same extent as Licensee and provides Licensor with a copy of such assignee undertaking. Licensor may assign this Agreement and its rights and obligations without the consent of Licensee. No assignment shall relieve the
assigning Party of responsibility for the performance of any accrued obligations which it has prior to such assignment. Any attempted assignment by Licensee in violation of this Section 10.2 shall be null and void and of no legal effect. 

10.3 Waiver. A waiver by either Party of a breach of any provision of this Agreement will not constitute a waiver of any subsequent breach of that
provision or a waiver of any breach of any other provision of this Agreement. 
 10.4 Notices. Notices, payments, statements, reports, and other
communications under this Agreement shall be in writing and shall be deemed to have been received as of the date received if sent by public courier (e.g., Federal Express), by Express Mail, receipt requested, or by facsimile (with a copy of such
facsimile also sent by one of the other methods of delivery) and addressed as follows: 
  

			
	If for Licensor:	  	with a copy to:
		
	 ReGenX Biosciences, LLC

750 17th Street, NW
 Suite
1100
 Washington, DC 20006

USA
 Attn: Chief Executive
Officer
 Telephone: 202-785-7438

Facsimile: 202-785-7439
	  	 ReGenX Biosciences, LLC

750 17th Street, NW
 Suite
1100
 Washington, DC 20006

USA
 Attn: General
Counsel
 Telephone: 202-785-7438

Facsimile: 202-785-7439

		
	If for Licensee:	  	with a copy to:
		
	 Lysogene Société par Actions Simplifiée

52 rue de la Boetie, 75008

Paris, France
 Attn: Chief
Executive Officer
 Telephone: +33 1 56 88 52 87

Facsimile: + 33 1 56 88 52 81
	  	 Colombus Audit & Expertise

52 rue de la Boetie, 75008

Paris, France
 Attn:
President
 Telephone: + 33 1 56 88 52 90

Facsimile: + 33 1 56 88 52 81

 Either Party may change its official address upon written notice to the other Party. 

10.5 Applicable Law. This Agreement shall be construed and governed in accordance with the laws of the State of New York, without giving effect to
conflict pf law provisions that may require the application of the laws of another jurisdiction. Subject to Section 10.6, the Parties hereby submit to the exclusive jurisdiction of and venue in the courts located in the State of New York with
respect to any and all disputes concerning the subject of this Agreement. 

  
 25 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 10.6 Dispute Resolution. In the event of any controversy or claim arising out of or relating to this
Agreement, the Parties shall first attempt to resolve such controversy or claim through good faith negotiations for a period of not less than **** following notification of such controversy or claim to the other Party. If such controversy or claim
cannot be resolved by means of such negotiations during such period, then such controversy or claim shall be resolved by binding arbitration administered by the American Arbitration Association (“AAA”) in accordance with the Commercial
Arbitration Rules of the AAA in effect on the date of commencement of the arbitration, subject to the provisions of this Section 10.6. The arbitration shall be conducted as follows: 

10.6.1 The arbitration shall be conducted by three arbitrators, each of whom by training, education, or experience has knowledge of the
research, development, and commercialization of biological therapeutic products in the United States. The arbitration shall be conducted in English and held in New York, New York. 

10.6.2 In its demand for arbitration, the Party initiating the arbitration shall provide a statement setting forth the nature of the dispute,
the names and addresses of all other parties, an estimate of the amount involved (if any), the remedy sought, otherwise specifying the issue to be resolved, and appointing one neutral arbitrator. In an answering statement to be filed by the
responding Party within **** after confirmation of the notice of filing of the demand is sent by the AAA, the responding Party shall appoint one neutral arbitrator. Within **** from the date on which the responding Party appoints its neutral
arbitrator, the first two arbitrators shall appoint a chairperson. 
 10.6.3 If a Party fails to make the appointment of an arbitrator as
provided in Section 10.6.2, the 1 AAA shall make the appointment. If the appointed arbitrators fail to appoint a chairperson within the time specified in Section 10.6.2 and there is no agreed extension of time, the AAA shall appoint the
chairperson. 
 10.6.4 The arbitrators will render their award in writing and, unless all Parties agree otherwise, will include an
explanation in reasonable detail of the reasons for their award. Judgment upon the award rendered by the arbitrators may be entered in any court having jurisdiction thereof, including in the courts described in Section 10.5. The arbitrators
will have the authority to grant injunctive relief and other specific performance; provided that the arbitrators will have no authority to award damages in contravention of this Agreement, and each Party irrevocably waives any claim to such damages
in contravention of this Agreement. The arbitrators will, in rendering their decision, apply the substantive law of the State of New York, without giving effect to conflict of law provisions that may require the application of the laws of another
jurisdiction. The decision and award rendered by the arbitrators will be final and non-appealable (except for an alleged act of corruption or fraud on the part of the arbitrator). 

10.6.5 The Parties shall use their reasonable efforts to conduct all dispute resolution procedures under this Agreement as expeditiously,
efficiently, a d cost-effectively as possible. 
 10.6.6 All expenses and fees of the arbitrators and expenses for hearing facilities and
other expenses of the arbitration will be borne equally by the Parties unless the Parties agree otherwise or unless the arbitrators in the award assess such expenses against one of the Parties or 

  
 26 

 ****CERTAIN INFORMATION HAS BEEN
OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN
REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
allocate such expenses other than equally between the Parties. Each of the Parties will bear its own counsel fees and the expenses of its witnesses except to the extent otherwise provided in this
Agreement or by applicable law. 
 10.6.7 Compliance with this Section 10.6 is a condition precedent to seeking relief in any court or
tribunal in respect of a dispute, but nothing in this Section 10.6 will prevent a Party from seeking equitable or other interlocutory relief in the courts of appropriate jurisdiction, pending the arbitrators’ determination of the merits of
the controversy, if applicable to protect the confidential information, property, or other rights of that Party or to otherwise prevent irreparable harm that may be caused by the other Party’s actual or threatened breach of this Agreement. 

10.7 No Discrimination. Licensee, its Affiliates, and any Sublicensees, in their respective activities under this Agreement, shall not discriminate
against any employee or applicant for employment because of race, color, sex, sexual, or affectional preference, age, religion, national, or ethnic origin, handicap, or because he or she is a disabled veteran or a veteran (including a veteran of the
Vietnam Era). 
 10.8 Compliance with Law. Licensee (and its Affiliates’ and any Sublicensees’) must comply with all prevailing laws,
rules, and regulations that apply to its activities or obligations under this Agreement. Without limiting the foregoing, it is understood that this Agreement may be subject to United States laws and regulations controlling the export of technical
data, computer software, laboratory prototypes, and other commodities, articles, and information, including the Arms Export Control Act as amended in the Export Administration Act of 1979 and that Licensee’s obligations are contingent upon
compliance with applicable United States export laws and regulations. The transfer of certain technical data and commodities may require a license from the cognizant agency of the United States Government and/or written assurances by Licensee that
Licensee shall not export data or commodities to certain foreign countries without prior approval of such agency. Licensor neither represents that a license is not required nor that, if required, it will issue. 

10.9 Entire Agreement. This Agreement embodies the entire understanding between the Parties relating to the subject matter hereof and supersedes all
prior understandings and agreements, whether written or oral, including that certain Mutual Non-Disclosure Agreement between the Parties dated January 1, 2012. All “Confidential Information” disclosed by the Parties pursuant to such
Mutual Non-Disclosure Agreement shall be deemed “Confidential Information” under this Agreement (unless and until it falls within one of the exclusions set forth in Section 1.4). This Agreement may not be varied except by a written
document signed by duly authorized representatives of both Parties. 
 10.10 Marking. Licensee, its Affiliates, and any Sublicensees shall mark any
Licensed Product (or their containers or labels) made, sold, or otherwise distributed by it or them with any notice of patent rights necessary or desirable under applicable law enable the Licensed Patents to be enforced to their full extent in any
country where Licensed Products are made, used, sold, offered for sale, or imported. 

  
 27 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 10.11 Severability and Reformation. If any provision of this Agreement is held to be invalid or
unenforceable by a court of competent jurisdiction, then such invalid or unenforceable provision will be automatically revised to be a valid or enforceable provision that comes as close as permitted by law to the Parties’ original intent;
provided that, if the Parties cannot agree upon such valid or enforceable provision, the remaining provisions of this Agreement will remain in full force and effect, unless the invalid or unenforceable provisions are of such essential importance to
this Agreement that it is to be reasonably assumed that the Parties would not have entered into this Agreement without the invalid or unenforceable provisions. 

10.12 Further Assurances. Each Party hereto agrees to execute, acknowledge, and deliver such further instruments, and to do all other acts, as may be
necessary or appropriate in order to carry out the purposes and intent of this Agreement. 
 10.13 Interpretation; Construction. The captions to the
several Articles and Sections of this Agreement are included only for convenience of reference and shall not in any way affect the construction of, or be taken into consideration in interpreting, this Agreement. In this Agreement, unless the context
requires otherwise, (a) the word “including” shall be deemed to be followed by the phrase “without limitation” or like expression; (b) references to the singular shall include the plural and vice versa;
(c) references to masculine, feminine, and neuter pronouns and expressions shall be interchangeable; (d) the words “herein” or “hereunder” relate to this Agreement; (e) “or” is disjunctive but not
necessarily exclusive; (f) the word “will” shall be construed to have the same meaning and effect as the word “shall”; (g) all references to “dollars” or “$” herein shall mean U.S. Dollars;
(h) unless otherwise provided, all reference to Sections and exhibits in this Agreement are to Sections and exhibits of and in this Agreement; and (i) whenever this Agreement refers to a number of days, such number shall refer to calendar
days unless business days are specified. Business days shall mean a day on which banking institutions in Washington, D.C. are open for business. Each Party represents that it has been represented by legal counsel in connection with this Agreement
and acknowledges that it has participated in the drafting hereof. In interpreting and applying the terms and provisions of this Agreement, the Parties agree that no presumption will apply against the Party which drafted such terms and provisions.

 10.14 Cumulative Rights and Remedies. The rights and remedies provided in this Agreement and all other rights and remedies available to either
Party at law or in equity are, to the extent permitted by law, cumulative and not exclusive of any other right or remedy now or hereafter available at law or in equity. Neither asserting a right nor employing a remedy shall preclude the concurrent
assertion of any other right or employment of any other remedy, nor shall the failure to assert any right or remedy constitute a waiver of that right or remedy. 

10.15 Counterparts. This Agreement may be executed in one or more counterparts, each of which will be deemed an original, but all of which together
will constitute one and the same instrument. 
 10.16 Recordation of License. During the term of this Agreement, if Licensee determines that it Is
necessary to record a confirmation of the license granted under this Agreement in a country where a Licensed Patent is filed, which recording is necessary to comply with law or to make such license effective against Third Parties, Licensee may
notify Licensor in writing of such 

  
 28 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
requirement, including an explanation thereof and a draft document to be recorded. Licensor will discuss such requirement in good faith with Licensee, together with the form of document to be
filed, which the Parties intend will be a summary or acknowledgement of the license granted under this Agreement. If the Parties agree to a recording, Licensee may make the applicable recording in the form agreed to by Licensor, at Licensee’s
sole expense. Licensee agrees that it will not make any recordings or filings with respect to this Agreement without the prior written consent of Licensor both as to the requirement of the recording and the form of the recording to be made. Licensor
agrees that Licensee may make a recording relating to this Agreement in France, subject to the Parties agreeing on the form of document to be recorded. Following expiration or termination of this Agreement for any reason, Licensor may record, at
Licensee’s expense, any documentation needed to reflect such expiration or termination, and Licensee agrees to provide Licensor reasonable assistance thereto, including by executing any necessary acknowledgements. 

[REMAINDER OF PAGE INTENTIONALLY LEFT BLANK] 

  
 29 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 IN WITNESS WHEREOF, the Parties, intending to be legally bound, have caused this License
Agreement to be executed by their duly authorized representatives. 
  

									
	REGENX BIOSCIENCES, LLC	 		 	LYSOGENE SOCIÉTÉ PAR ACTIONS SIMPLIFIÉE
					
	By	 	 /s/ Kenneth Mills
	 		 	By:	 	 /s/ Karen Arach

	Name:	 	 Kenneth Mills
	 		 	Name:	 	 Karen Arach

	Title:	 	 President and CEO
	 		 	Title:	 	 President and CEO

 CONFIDENTIAL TREATMENT REQUESTED 

 
 Exhibit A 

Licensed Patents 
  

									
	 Appin #
	  	 Title
	  	 Inventors
	  	 Nos
	  	 Docket

	****	  	****	  	****	  	****	  	****

  

															
	 Docket
	  	 Country
	  	 Appin No
	  	 Filing Date
	  	 Patent
Number
	  	 Issue Date
	  	 Pubn
Number
	  	 Pub Date

	****	  	****	  	****	  	****	  	****	  	****	  		  	
	****	  	****	  	****	  	****	  		  		  		  	
	****	  	****	  	****	  	****	  		  		  		  	
	****	  	****	  	****	  	****	  		  		  	****	  	****
	****	  	****	  	****	  	****	  		  		  		  	
	****	  	****	  	****	  	****	  		  		  		  	
	****	  	****	  	****	  	****	  	****	  	****	  		  	
	****	  	****	  	****	  	****	  	****	  	****	  		  	
	****	  	****	  	****	  	****	  		  		  		  	

  
 ****CERTAIN
INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL
TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 Exhibit B 

Press Release 

 CONFIDENTIAL TREATMENT REQUESTED 

 

			
	

	  	 DRAFT-NOT FOR DISTRIBUTION
  

 REGENX BIOSCIENCES AND LYSOGENE ENTER INTO EXCLUSIVE LICENSE 

AGREEMENT FOR DEVELOPMENT OF TREATMENTS FOR SERIOUS, RARE 

LYSOSOMAL STORAGE DISORDER USING NAV® rAAVrh10 VECTORS 

WASHINGTON, DC and PARIS, FRANCE [December     , 2013] — REGENX Biosciences, LLC (REGENX) and LYSOGENE SAS (LYSOGENE) announce
that they have entered into an agreement enabling the development and commercialization of products to treat mucopolysaccharidosis type IIIA (MPS IIIA or Sanfilippo syndrome Type A) using NAV rAAVrh10. 

Under the terms of the Agreement, REGENX granted LYSOGENE an exclusive worldwide license, with rights to sublicense, to REGENX’s NAV
rAAVrh10 vectors for treatment of MPS IIIA in humans. In return for these rights, REGENX receives payments in the form of an up-front payment, certain milestone fees and royalties on net sales of products incorporating NAV rAAVrh10.

 “We believe this exclusive license agreement will enable LYSOGENE to advance the development of its NAV based treatment for
patients with MPS IIIA,” said Ken Mills, President and CEO of REGENX. “As a leader in gene therapy, we are pleased to be formally collaborating with the Lysogene team that, by the successful completion of a recent Phase I/II trial,
demonstrates outstanding expertise, resources, and commitment to patients. Providing partners with access to our NAV technology further advances REGENX’s mission to enable the development of successful new AAV therapeutics.”

 “Lysogene is a leading clinical stage gene therapy company committed to the development of breakthrough therapies in rare diseases. The company
successfully completed a phase VII study (NCT01474343/EudraCT 2010-019962-10) using the NAV rAAVrh10 technology in Sanfilippo syndrome. We are very 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
pleased to enter into this agreement with REGENX, which we believe offers us the best path to expeditiously advance the clinical development of our lead product towards the market and to patients
in extraordinary high demand”, said Karen Aiach, Founder, President and CEO of LYSOGENE. 
 “Sanfillipo disease is a complete unmet medical
need and our clinical study using NAV rAAVrh10 indicates that gene therapy may become an outstanding treatment option”, said Olivier Danos PhD, Co-founder and Senior Scientific Advisor to LYSOGENE. 

About MPS III A (Sanfilippo syndrome Type A) 

Sanfilippo syndrome is a lethal, rare, autosomal recessive condition characterized by rapid neurodegeneration, severe and invasive behavioural
disorders, and mild peripheral symptoms. Patients generally do not live above their second decade. There is currently no treatment. Sanfilippo syndrome is caused by mutations in a gene that encodes N-sulfoglucosamine sulfohydrolase
(sulfamidase) which is needed to break down glycoaminoglycans - used in a number of biological functions. It affects approximately 1 in 100,000 births, and is still largely underdiagnosed. 

About REGENX Biosciences 
 REGENX Biosciences is
the leading AAV gene therapy company that is developing a new class of personalized therapies, based on its proprietary NAV vector technology platform, for a range of severe diseases with serious unmet needs. NAV vector technology
includes novel AAV vectors such as rAAV7, rAAV8, rAAV9, and rAAVrh10. Our treatments in development include programs for hypercholesterolemia, mucopolysaccharidoses, and retinitis pigmentosa. REGENX leadership in AAV gene therapy and corresponding
intellectual property has enabled it to establish collaborations with leading global partners including Chatham Therapeutics, Fondazione Telethon, and Audentes Therapeutics. In addition, together with Fidelity Biosciences, REGENX has formed
Dimension Therapeutics a company focused on the development and commercialization of AAV gene therapies for rare diseases. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 For more information regarding REGENX, please visit www.regenxbio.com. 

About LYSOGENE 
 LYSOGENE is a clinical stage
biotechnology company committed to the development of innovative therapies for patients affected with rare disorders and high unmet medical needs. LYSOGENE’s team translated its rAAVrh10 lead product for Sanfilippo from bench to bedside in an
unprecedented fashion over the last years. Its lead product is for Sanfilippo syndrome, a neurodegenerative lysosomal storage disorder considered to be a perfect model for gene therapy. LYSOGENE is currently expanding its pipeline to additional
diseases with high unmet medical needs. 
 For more information about LYSOGENE, please visit www.lvsogene.com. 

### 
 Contacts: 

REGENX Biosciences 
 Vit Vasista, 202-785-7438 

vvasista@regenxbio.com 
 LYSOGENE 

Karen AIACH 
 karen.aiach@lysogene.comEX-10.24

 Exhibit 10.24 

CONFIDENTIAL TREATMENT REQUESTED 

LICENSE AGREEMENT 
 This LICENSE AGREEMENT
(“Agreement”) is entered into as of May 28, 2014 (“Effective Date”) by and between ReGenX Biosciences, LLC, a limited liability company organized under the laws of the State of Delaware, with offices at 750
17th Street, NW, Suite 1100, Washington, DC 20006 (“Licensor”), and Voyager Therapeutics, Inc., a corporation organized under the laws of the State of Delaware, with offices at 75 Sidney Street, Cambridge, MA 02139
(“Licensee”). Licensor and Licensee are hereinafter referred to individually as a “Party” and collectively as the “Parties.” 

WHEREAS, Licensor has rights under certain patents pertaining to various recombinant adeno-associated virus vectors; 

WHEREAS, Licensee desires to obtain from Licensor, and Licensor is willing to grant to Licensee, (a) a non-exclusive research license to conduct certain
research to identify and select Specified Vectors for specified indications and (b) an option to obtain a non-exclusive license to research, develop, and commercialize Licensed Products for specified indications under the terms set forth
herein; 
 NOW, THEREFORE, in consideration of the promises and covenants contained in this Agreement, and intending to be legally bound, the Parties hereby
agree as follows: 
 ARTICLE 1: DEFINITIONS 

1.1 “AAVrh10” means (a) the recombinant adeno-associated virus serotype rh10 vector with the specified sequence set forth in GenBank
**** and (b) any recombinant adeno-associated virus derivatives of such serotype rh10 vector that are covered by the claims of the Licensed Research Patents. 

1.2 “AAV Materials” means recombinant adeno-associated virus serotype vectors, and any materials that are made or used for the sole purpose
of making recombinant adeno-associated virus serotype vectors, in each case, which, in the absence of the license granted pursuant to Section 2.1, would infringe or is covered by at least one Valid Claim of the Licensed Research Patents in the
country of manufacture or use. 
 1.3 “Affiliate” means any legal entity directly or indirectly, during the term of this Agreement,
controlling, controlled by, or under common control with another entity. For purposes of this Agreement, “control” means the direct or indirect ownership of more than 50% of the outstanding voting securities of a legal entity, or the right
to receive more than 50% of the profits or earnings of a legal entity, or the right to control the policy decisions of a legal entity. For clarity, an entity may be or become an Affiliate of an entity and may cease to be an Affiliate of an entity,
in each case, during the term of this Agreement. Notwithstanding the foregoing, any person or entity that would otherwise qualify as an Affiliate of Licensee hereunder by this definition will not be deemed to be, and will not be treated as, an
Affiliate of Licensee if (i) the primary business of such person or entity is investing in securities, debt, or other investment vehicles; provided that a person or entity that satisfies the criteria under this clause (i) who, directly or
indirectly, during the term of this Agreement, controls Licensee will be deemed an Affiliate under Sections 6.6 and 8.4.1 during the period of time in which such person or entity 

  
 1 

 

	****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE
COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
controls Licensee; or (ii) such person or entity is a portfolio company of a person or entity that satisfies the criteria under clause (i). Licensee represents and warrants that, as of the
Effective Date, Third Rock Ventures and its related funds satisfy the criteria under clause (i) of the preceding sentence; and, as such, the Parties agree that, for so long as the foregoing representation and warranty remains true, Third Rock
Ventures and its related funds will be excluded from classification as Affiliates of Licensee under this Agreement to the extent provided in the immediately prior sentence. 

1.4 “Calendar Quarter” means each three-month period or any portion thereof, beginning on
January 1, April 1, July 1, and October 1. 
 1.5 “Commercial Field” means the treatment or prevention of a
Disease Indication (if and when a Commercial Option is exercised for such Disease Indication by Licensee under Section 2.3) in human beings by in vivo gene therapy with the applicable Specified Vector selected for the applicable Disease
Indication. 
 1.6 “Commercial Option” has the meaning set forth in Section 2.3. 

1.7 “Confidential Information” means and includes all technical information, inventions, developments, discoveries, software, know-how,
methods, techniques, formulae, animate and inanimate materials, data, processes, finances, business operations or affairs, and other proprietary ideas, whether or not patentable or copyrightable, of either Party that are (a) marked or otherwise
identified as confidential or proprietary at the time of disclosure in writing; or (b) if disclosed orally, visually, or in another non-written form, identified as confidential at the time of disclosure and summarized in reasonable detail in
writing as to its general content within 30 days after original disclosure. The Parties acknowledge that (i) the terms and conditions of this Agreement and (ii) the records and reports referred to in Section 3.7 will be deemed the
Confidential Information of both Parties, regardless of whether such information is marked or identified as confidential. Notwithstanding the foregoing, Confidential Information will not include the following, in each case, to the extent evidenced
by competent written proof of the Receiving Party: 
 1.7.1 information that was already known to the Receiving Party, other than under an
obligation of confidentiality, at the time of disclosure by the Disclosing Party; 
 1.7.2 information that was generally available to the
public or otherwise part of the public domain at the time of its disclosure to the Receiving Party; 
 1.7.3 information that became
generally available to the public or otherwise part of the public domain after its disclosure, other than through any act or omission of the Receiving Party in breach of this Agreement; 

1.7.4 information that is independently discovered or developed by the Receiving Party without the use of Confidential Information of the
Disclosing Party; or 
 1.7.5 information that was disclosed to the Receiving Party, other than under an obligation of confidentiality, by a
Third Party who had no obligation to the Disclosing Party not to disclose such information to others. 

  
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 1.8 “Disclosing Party” has the meaning set forth in Section 5.1. 

1.9 “Disease Indication(s)” means one or more of the following indications: (a) Friedreich’s Ataxia that is treated or prevented by
administration of the applicable recombinant adeno-associated virus serotype vector directly to the central nervous system (brain and spinal cord) (“Friedreich’s Ataxia (CNS)”), (b) Friedreich’s Ataxia that is treated
or prevented by administration of the applicable recombinant adeno-associated virus serotype vector by any route except administration directly to the central nervous system (brain and spinal cord) (“Friedreich’s Ataxia
(Systemic)”), (c) Huntington’s Disease, and (d) Amyotrophic Lateral Sclerosis. 
 1.10 “Domain Antibody” ****. 

1.11 “FDA” means the United States Food and Drug Administration, or a successor agency in the United States with responsibilities comparable
to those of the United States Food and Drug Administration. 
 1.12 “GSK Agreement” means that certain License Agreement entered into
between Licensor and SmithKline Beecham Corporation, effective on March 6, 2009, as amended by that certain Amendment to License Agreement dated April 15, 2009, and as amended from time to time. 

1.13 “Licensed Commercial Patents” means, on a Specified Vector-by-Specified Vector basis, to the extent they cover such Specified Vector,
(a) all United States patents and patent applications listed in Exhibit D (or on Exhibit A, until such time as this Agreement is amended to add Exhibit D in accordance with Section 2.3.3), including patents arising or
issuing from such patent applications; and (b) any re-examination certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications; provided that “Licensed Commercial Patents”
will not include any claim of a patent or patent application covering any Manufacturing Technology. 
 1.14 “Licensed Patents” means the
Licensed Commercial Patents or Licensed Research Patents, as applicable. 
 1.15 “Licensed Product” means (a) any product using the
applicable Specified Vector capsid protein that is made, made for, used, sold, offered for sale, or imported by Licensee, its Affiliates, and any of its or their Sublicensees, the manufacture, use, sale, offer for sale, or import of which product,
in the absence of the license granted pursuant to this Agreement, would infringe or is covered by at least one Valid Claim of the Licensed Commercial Patents in the country of manufacture, use, sale, offer for sale, or import; or (b) any
service sold by Licensee, its Affiliates, and any of its or their Sublicensees with respect to the administration of any product using the applicable Specified Vector capsid protein to patients that, in the absence of the licenses granted pursuant
to this Agreement, would infringe or is covered by at least one Valid Claim of the Licensed Commercial Patents in the country of sale. 
 1.16
“Licensed Research Patents” means (a) all United States patents and patent applications listed in Exhibit A, including patents arising or issuing from such patent applications; and (b) any

  
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re-examination certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications; provided that “Licensed Research Patents” will
not include any claim of a patent or patent application covering any Manufacturing Technology. 
 1.17 “Manufacturing Technology” means any
and all patents, patent applications, know-how, and all intellectual property rights associated therewith that are owned or controlled by Licensor, and including all tangible embodiments thereof, that are necessary or useful for the manufacture of
adeno-associated viruses, adeno-associated virus vectors, research or commercial reagents related thereto, Licensed Products, or other products, including manufacturing processes, technical information relating to the methods of manufacture,
protocols, standard operating procedures, batch records, assays, formulations, quality control data, specifications, scale up, any and all improvements, modifications, and changes thereto, and any and all activities associated with such manufacture.
Any and all chemistry, manufacturing, and controls (CMC), drug master files (DMFs), or similar materials provided to regulatory authorities and the information contained therein are deemed Manufacturing Technology. 

1.18 “NDA” means a New Drug Application filed with the FDA as described in 21 C.F.R. § 314, a Biological License Application (BLA)
pursuant to 21 C.F.R. § 601.2, or any equivalent or any corresponding application for regulatory approval in any country or regulatory jurisdiction other than the United States. 

1.19 “Net Sales” means the gross receipts from sales or other disposition of a Licensed Product (including fees for services within the
definition of “Licensed Product”) by Licensee and/or its Affiliates and/or any Sublicensees to Third Parties less the following deductions that are directly attributable to a sale, specifically and separately identified on an invoice or
other documentation and actually borne by Licensee, its Affiliates, or any Sublicensees: ****. In the event consideration other than cash is paid to Licensee, its Affiliates, or any Sublicensees, for purposes of determining Net Sales, the Parties
shall use the cash consideration that Licensee, its Affiliates, or any Sublicensees would realize from an unrelated buyer in an arm’s length sale of an identical item sold in the same quantity and at the time and place of the transaction, as
determined jointly by Licensor and Licensee based on transactions of a similar type and standard industry practice, if any. 
 1.20 “Penn
Agreement” means that certain License Agreement entered into between Licensor and The Trustees of the University of Pennsylvania, effective on February 24, 2009, as amended by that letter agreement dated March 6, 2009, and as
amended from time to time. 

  
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 1.21 “Phase 3 Clinical Trial” means a pivotal clinical trial in humans performed to gain
evidence with statistical significance of the efficacy of a product in a target population, and to obtain expanded evidence of safety for such product that is needed to evaluate the overall benefit-risk relationship of such product, to form the
basis for approval of an NDA and to provide an adequate basis for physician labeling, as described in 21 C.F.R. § 312.21(c) or the corresponding regulation in jurisdictions other than the United States. 

1.22 “Prosecute” means preparation, filing, and prosecuting patent applications and maintaining patents, including any reexaminations,
reissues, oppositions, inter partes review, and interferences. 
 1.23 “Receiving Party” has the meaning set forth in Section 5.1.

 1.24 “ReGenX Licensors” means SmithKline Beecham Corporation (or any successor thereto under the GSK Agreement) and The Trustees of the
University of Pennsylvania (or any successor thereto under the Penn Agreement). 
 1.25 “Research Field” means Licensee’s internal
research and pre-clinical development for the treatment or prevention of any of the Disease Indications in humans by in vivo gene therapy using AAV Materials. Notwithstanding the foregoing, “Research Field” specifically excludes the
use of AAVrh10 for the treatment or prevention of Friedreich’s Ataxia (Systemic). Furthermore, “Research Field” specifically excludes (without limitation) (a) all human clinical trial use, diagnostic use, therapeutic use, and
prophylactic use, and (b) any commercial uses. 
 1.26 “Research Term” means, on a Disease Indication-by-Disease Indication basis, a
period beginning with the Effective Date and ending on the earlier of (a) the Grant Date, if any, with respect to the applicable Disease Indication and (b) the 18-month anniversary of the Effective Date, or if the Research Term is extended
pursuant to Section 2.2, the 30-month anniversary of the Effective Date. 
 1.27 “Retained Rights” has the meaning set forth in
Section 2.4. 
 1.28 “Secondary Disease Indications” collectively mean (a) Friedreich’s Ataxia (Systemic),
(b) Huntington’s Disease, and (c) Amyotrophic Lateral Sclerosis. 
 1.29 “Specified Vector” means the recombinant
adeno-associated virus serotype vector with a specified sequence set forth in GenBank that is selected by Licensee pursuant to Section 2.3 and which is specified on Exhibit C (to be attached hereto as of the applicable Grant Date as
provided in Section 2.3). 
 1.30 “Sublicensee” means (i) any Third Party or Affiliate to whom Licensee grants a sublicense of
some or all of the rights granted to Licensee under this Agreement as permitted by this Agreement; and (ii) any other Third Party or Affiliate to whom a sublicensee described in clause (i) has granted a further sublicense as permitted by
this Agreement. 
 1.31 “Third Party” means any person or entity other than a Party to this Agreement or Affiliates of a Party to this
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 1.32 “Third Party Collaborator” means a Third Party with whom Licensee has entered into a
collaboration for a particular Disease Indication under which (a) research and development activities will be performed on a shared basis during the Research Term for the purpose of Licensee and such Third Party determining which Specified
Vector would be selected if the Commercial Option for such Disease Indication were exercised, and (b) the Third Party will be granted commercial rights upon exercise of a Commercial Option for such Disease Indication. For the avoidance of
doubt, a Third Party Collaborator will not include a Third Party who is granted the right to conduct research and development activities independent of Licensee or unrelated to the exercise of a Commercial Option. 

1.33 “Valid Claim” means (a) a claim of an issued and unexpired patent (including any patent claim the term of which is extended by any
extension, supplementary protection certificate, patent term restoration, or the like) included within the Licensed Patents or (b) a claim of a pending patent application included within the Licensed Patents that has not been pending for more
than 15 years from the earliest filing date to which such claim or the applicable patent application is entitled to claim priority, in each case under clauses (a) and (b) which has not lapsed, been abandoned, been held revoked, or been
deemed unenforceable or invalid by a non-appealable decision or an appealable decision from which no appeal was taken within the time allowed for such appeal of a court or other governmental agency of competent jurisdiction. 

ARTICLE 2: LICENSE GRANTS 
 2.1
Research License Grant. Subject to the terms and conditions of this Agreement, including the Retained Rights, during the Research Term, Licensor hereby grants to Licensee a non-exclusive, sublicensable (as provided in Section 2.6 only),
non-transferable (except as provided in Section 10.2), worldwide license under the Licensed Research Patents to make, have made, and use any and all AAV Materials in the Research Field (including, for the avoidance of doubt, the right to
conduct research and pre-clinical development) solely for purposes of identifying and selecting Specified Vector(s) for use in the Commercial Field upon exercise of a Commercial Option. For the avoidance of doubt, the foregoing license in this
Section 2.1 does not include the right to sell, offer for sale, or import any AAV Materials. 
 2.2 Research License Extension Option. Licensee
may extend the Research Term with respect to any or all of the Disease Indications with respect to which the Commercial Option has not been exercised pursuant to Section 2.3 prior to the **** of the Effective Date by providing written notice to
Licensor of such extension and simultaneously paying Licensor a fee of ****, which notice and payment must be received by Licensor at least **** prior to the **** of the Effective Date. If Licensee does not extend the Research Term under this
Section 2.2, the Research Term with respect to any or all of the Disease Indications with respect to which the Commercial Option has not been exercised pursuant to Section 2.3 or otherwise terminated by Licensee pursuant to
Section 6.3 prior to the **** of the Effective Date will expire on the **** of the Effective Date. 
 2.3 Commercial License Option. Subject to
the terms and conditions of this Agreement, Licensor hereby grants to Licensee the option, exercisable at Licensee’s sole discretion, to obtain a non-exclusive worldwide license with respect to each of the Disease Indications and a single

  
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Specified Vector for such Disease Indication (each such right with respect to a particular Disease Indication, a “Commercial Option”) in accordance with the following provisions:

 2.3.1 Method of Exercise. To exercise the Commercial Option for a particular Disease Indication, Licensee must provide written
notice to Licensor prior to the end of the applicable Research Term, which written notice must specify the Disease Indication(s) and Specified Vector (as further described in Section 2.3.2) with respect to which Licensee desires to exercise its
Commercial Option. For each of the Secondary Disease Indications, such written notice must be accompanied by a wire transfer of the commercial option fee set forth in Section 3.2 for such Secondary Disease Indication. 

2.3.2 Specified Vector. For purposes of selecting a Specified Vector for use with a Disease Indication, the Specified Vector must be a
recombinant adeno-associated virus serotype vector with a specified sequence. Licensee’s notice of the specified sequence will provide Licensor with a published source that refers to the sequence (which may include a reference to the Licensed
Research Patents), if there is a public source. The sequence of the Specified Vector will be provided to Licensor in a written format setting forth the entire DNA sequence and amino acid sequence in Vector NTI format (from Life Technologies) (or
such other format, as the Parties agree) that will enable Licensor to analyze the sequence through the Vector NTI electronic sequence editing program. Licensee may not select AAVrh10 as the Specified Vector for the treatment or prevention of
Friedreich’s Ataxia (Systemic). Upon Licensor’s receipt of the notice and, if applicable, fee described in Section 2.3.1, this Agreement will be amended to add a new Exhibit C (or amend a then-existing Exhibit C)
prepared by Licensor setting forth the Specified Vector for each Disease Indication with respect to which a Commercial Option is exercised. 

2.3.3 Licensed Commercial Patents. Within **** after Licensor’s receipt of the notice and, if applicable, fee described in
Section 2.3.1, Licensor will prepare a new Exhibit D setting forth the applicable Licensed Commercial Patents that apply to the Specified Vector and applicable Disease Indication, which Licensed Commercial Patents will be taken solely
from the Licensed Research Patents. Upon Licensee’s acceptance of the new Exhibit D (which acceptance will not be unreasonably withheld, conditioned, or delayed), this Agreement will be amended to add such new exhibit. If different
Specified Vectors are specified for use in connection with different Disease Indications, then Licensor may create a separate exhibit (labeled Exhibit D-1 through D-4, as necessary) for each Specified Vector. Until this Agreement is
amended to include the new Exhibit D, Exhibit A will continue to form the basis for determining the scope of the applicable Licensed Commercial Patents. 

2.3.4 License Grant Upon Exercise. If Licensee exercises the Commercial Option for a particular Disease Indication, effective upon both
(a) Licensor’s receipt of the notice and (b) in the case of a Secondary Disease Indication, the fee described in Section 2.3.1 for such Secondary Disease Indication (the date on which the notice and the fee (if applicable) are
received shall be deemed to be the “Grant Date” for such Disease Indication), subject to the terms and conditions of this Agreement, including the Retained Rights, Licensor shall grant, and hereby grants, to Licensee a
non-exclusive, sublicensable (as provided in Section 2.6 only), non-transferable (except as provided in Section 10.2), royalty-bearing, worldwide license under the applicable Licensed Commercial Patents to make, have made, use, import,
sell, and offer for sale Licensed 

  
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Products using the Specified Vector solely in the Commercial Field for such Disease Indication, including, for the avoidance of doubt, the right to conduct research and development. 

2.3.5 Disease Indications. For the avoidance of doubt, the foregoing license granted pursuant to Section 2.3.4 will be deemed
granted on the Grant Date on a Disease Indication-by-Disease Indication basis, solely with respect to the Commercial Field associated with the Disease Indication for which the Commercial Option was exercised under this Section 2.3 and solely
with respect to Licensed Products using the Specified Vector selected for the particular Disease Indication. The Parties acknowledge that there may be different Grant Dates for each Disease Indication, depending on when and if Licensee exercises the
Commercial Option for a particular Disease Indication. As set forth above, Licensee, at its sole discretion, may exercise the Commercial Option with respect to any or all of the four Disease Indications. If Licensee exercises the Commercial Option
with respect to only some of the Disease Indications but not all, the Commercial Option will terminate with respect to any unexercised Disease Indications and the license granted under Section 2.1 will also terminate, in each case, at the end
of the Research Term, and Licensee will have no further rights under this Agreement with respect to such unexercised Disease Indications. 
 2.4 Retained
Rights. Except for the rights and licenses specified in Sections 2.1 and, if applicable, 2.3.4, no license or other rights are granted to Licensee under any intellectual property of Licensor, whether by implication, estoppel, or otherwise and
whether such intellectual property is subordinate, dominant, or otherwise useful for the practice of the Licensed Patents. Notwithstanding anything to the contrary in this Agreement, Licensor may use and permit others to use the Licensed Patents for
any research, development, commercial, or other purposes inside or outside of the Commercial Field or the Research Field. Without limiting the foregoing, and notwithstanding anything in this Agreement to the contrary, Licensee acknowledges and
agrees to the following rights retained by Licensor and the ReGenX Licensors (individually and collectively, the “Retained Rights”), whether inside or outside the Commercial Field or Research Field: 

2.4.1 The rights and licenses granted in Sections 2.1 and, if applicable, 2.3.4 shall not include any right (and Licensor and the ReGenX
Licensors retain the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents to make, have made, use, sell, offer to sell, and import Domain Antibodies that are expressed by an adeno-associated vector, including any
Specified Vector. 
 2.4.2 Licensor and the ReGenX Licensors retain the following rights with respect to the Licensed Patents: 

 

	 	(a)	A non-exclusive, sublicensable right under the Licensed Patents to make, have made, use, sell, offer to sell, and import products that deliver RNA interference and antisense drugs using an adeno-associated vector,
including any Specified Vector; and 

  

	 	(b)	 A non-exclusive right for the ReGenX Licensors (which right is sublicensable by such licensors) to use the Licensed Patents for non-commercial
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licensors’ discovery research efforts with non-profit organizations and the ReGenX Licensors’ collaborators. 

2.4.3 The rights and licenses granted in Sections 2.1 and, if applicable, 2.3.4 shall not include any right (and Licensor retains the
exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents: 
  

	 	(a)	to conduct commercial reagent and services businesses, which includes the right to make, have made, use, sell, offer to sell, and import research reagents, including any viral vector construct; provided that for
clarity, such exclusive rights retained by Licensor shall not include the right to conduct clinical trials in humans in the Commercial Field, though Licensor retains the non-exclusive right to do so; or 

 

	 	(b)	to use the Licensed Patents to provide services to any Third Parties; provided that Licensee’s license under Section 2.3.4, if applicable, does include the right to provide the services of the administration
of Licensed Products to patients. 

 2.4.4 Licensor retains the fully sublicensable right under the Licensed Patents to grant
non-exclusive research and development licenses to Affiliates and Third Parties. 
 2.4.5 The Trustees of the University of Pennsylvania may
use and permit other non-profit organizations or other non-commercial entities to use the Licensed Patents for educational and research purposes. 
 2.5
Government Rights. Licensee acknowledges that the United States government retains certain rights in intellectual property funded in whole or part under any contract, grant, or similar agreement with a federal agency. The license grants
hereunder are expressly subject to all applicable United States government rights, including any applicable requirement that products that result from such intellectual property and are sold in the United States must be substantially manufactured in
the United States. 
 2.6 Sublicensing. 

2.6.1 The research license granted pursuant to Section 2.1 is sublicensable by Licensee (a) to Affiliates of Licensee and (b) to
one Third Party Collaborator with respect to each Disease Indication; any other sublicenses to Third Party Collaborators or Third Parties of the research license granted pursuant to Section 2.1 requires Licensor’s prior written consent,
which consent may not be unreasonably withheld, conditioned, or delayed. The license granted, if applicable, pursuant to Section 2.3.4 is sublicensable by Licensee to any Affiliates or Third Parties. Any sublicense of the rights under this
Section 2.6, whether to an Affiliate or Third Party and whether relating to a sublicense of rights under Section 2.1 or 2.3.4, must comply with the provisions of this Section 2.6 (including Section 2.6.2). 

2.6.2 The right to sublicense granted to Licensee under this Agreement is subject to the following conditions: 

  
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	 	(a)	Licensee may grant a sublicense to an Affiliate of Licensee; provided that (i) such sublicense must comply with the terms of this Section 2.6.2 (except to the extent such terms are limited to Third Party
Sublicensees), including being granted pursuant to a written agreement and requiring the Sublicensee to comply with the applicable terms and conditions of this Agreement; (ii) Licensee must provide Licensor with written notice of any such
sublicense within **** after entering into a sublicense, which notice will identify the Affiliate, the applicable Disease Indication, and the scope of the rights sublicensed; (iii) such sublicense must only remain in effect for as long as such
sublicensee remains an Affiliate of Licensee; and (iv) without limiting Section 2.6.2(f) below, Licensee will be responsible for any and all obligations of any such Affiliate as if such Affiliate were “Licensee” hereunder. If
either of the ReGenX Licensors requires additional information, including a copy of the sublicense agreement, Licensee shall provide such information, including such copy, to Licensor. 

 

	 	(b)	Licensee may only grant sublicenses pursuant to a written sublicense agreement with the Sublicensee. Licensee may grant a direct Sublicensee (as defined in Section 1.30(i) only) of the rights under
Section 2.3.4 the right to grant further sublicenses ****. For the avoidance of doubt, any further sublicenses granted by any Sublicensees must comply with the provisions of this Section 2.6 (including Section 2.6.2) to the same
extent that Licensee would have to comply if Licensee were granting a sublicense directly to a Third Party (including the obligation of requiring the Sublicensee to comply with the applicable terms and conditions of this Agreement and providing
Licensor with a copy of the sublicense). For clarity, Licensee is entitled to grant to a Sublicensee a sublicense with respect to any or all of the Disease Indications. 

 

	 	(c)	In each sublicense agreement, (i) the Sublicensee must be required to comply with the terms and conditions of this Agreement to the same extent as Licensee has agreed, except to the extent that such terms and
conditions do not relate to the specific rights granted to the Sublicensee pursuant to this Agreement (e.g., obligations related to a Disease Indication that has not been sublicensed); and (ii) if such Sublicensee is a Third Party, such
Sublicensee must acknowledge that Licensor is an express third party beneficiary of such terms and conditions under such sublicense agreement. 

  

	 	(d)	The official language of any sublicense agreement shall be English. 

  
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	 	(e)	Within **** after entering into a sublicense with a Third Party Sublicensee, Licensor must receive a copy of the sublicense written in the English language for Licensor’s records and to share with the ReGenX
Licensors. The copy of the sublicense may be redacted to exclude confidential information of Licensee or the applicable Sublicensee, but such copy shall not be redacted to the extent that it impairs Licensor’s (or the ReGenX Licensors’)
ability to ensure compliance with this Agreement; provided that, if either of the ReGenX Licensors requires a complete, unredacted copy of the sublicense, Licensee shall provide such complete, unredacted copy. 

 

	 	(f)	Licensee’s execution of a sublicense agreement will not relieve Licensee of any of its obligations under this Agreement. Licensee is and shall remain **** to Licensor for all of Licensee’s duties and
obligations contained in this Agreement and for any act or omission of an Affiliate or Sublicensee that would be a breach of this Agreement if performed or omitted by Licensee, and Licensee will be deemed to be in breach of this Agreement as a
result of such act or omission. 

 2.7 Improvements. 

2.7.1 Licensee hereby grants to Licensor a non-exclusive, worldwide, royalty-free, transferable, sublicensable, irrevocable, perpetual license
to use any Licensed Back Improvements (and any intellectual property rights with respect thereto) consummate in scope to the Retained Rights. 

2.7.2 Licensee hereby grants to Licensor a non-exclusive, worldwide, royalty-free, transferable, sublicensable, irrevocable, perpetual license
to use and practice any Licensed Back Improvements (and any intellectual property rights with respect thereto) for any and all purposes, including the right to research, develop, make, have made, use, offer for sale, and sell products and services;
provided that Licensor shall have no right, under the license in this Section 2.7.2, to use or practice the Licensed Back Improvements, on a Disease Indication-by-Disease Indication basis, (i) inside the Research Field during the Research
Term for such Disease Indication or (ii) if the Commercial Option for such Disease Indication is exercised, inside the Commercial Field during the term of this Agreement for such Disease Indication. 

2.7.3 For purposes of this Agreement, but subject to Sections 2.7.5 and 2.7.6, “Licensed Back Improvements” means
(a) with respect to Section 2.7.1, any patentable modifications or improvements developed, during the term of this Agreement, by Licensee, any Affiliates, or any Sublicensees to any vector that is the subject of a claim within the Licensed
Patents, and (b) with respect to Section 2.7.2, any patentable modifications or improvements developed, during the term of this Agreement, by Licensee, any Affiliates, or any Sublicensees to any vector that is the subject of a claim as of
the Effective Date within the Licensed Patents. 
 2.7.4 Licensee agrees to provide prompt notice to Licensor upon the filing of any patent
application covering any Licensed Back Improvement, together with a reasonably detailed 

  
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description of or access to such Licensed Back Improvement to permit the practice of any such Licensed Back Improvement in accordance with the rights granted hereunder. 

2.7.5 With respect to any patentable modifications or improvements developed by any Third Party Sublicensee, the definition of “Licensed
Back Improvement” under Section 2.7.3 will only include patentable modifications or improvements that are (a) developed by such Third Party Sublicensee during the term of the applicable sublicense granted to such Third Party
Sublicensee; and (b) developed by such Third Party Sublicensee (i) to any vector if developed during the Research Term for the particular Disease Indication(s) sublicensed to such Third Party Sublicensee or (ii) to the Specified
Vector(s) for the particular Disease Indication(s) sublicensed to such Third Party Sublicensee if developed following the Grant Date for such Disease Indication(s). 

2.7.6 Notwithstanding Section 2.7.3, if Licensee undergoes a Change of Control pursuant to which a Third Party acquirer becomes an
Affiliate of Licensee hereunder, patentable modifications and improvements that were developed by such acquirer and such acquirer’s Affiliates (excluding Licensee and Licensee’s Affiliates prior to such Change of Control) prior to such
Change of Control will not become “Licensed Back Improvements” hereunder solely because of such Change of Control transaction, but thereafter the provisions of Section 2.7.3 will apply to patentable modifications or improvements of
such acquirer and its Affiliates (if also Affiliates of Licensee) developed after such Change of Control. 
 ARTICLE 3: CONSIDERATION

 3.1 Initial Fee. In consideration of the rights and licenses granted to Licensee under this Agreement, Licensee shall pay Licensor an initial
fee of $500,000 within **** after the Effective Date. 
 3.2 Commercial Option Fee. If Licensee elects to exercise the Commercial Option granted to
Licensee under Section 2.3 with respect to any Secondary Disease Indication, Licensee shall pay Licensor a fee of **** for the first Secondary Disease Indication and **** for each of the second and third Secondary Disease Indications. For
clarity, no such fee will be required with respect to Friedreich’s Ataxia (CNS). 
 3.3 Annual Maintenance Fee. In consideration of the rights
and licenses granted to Licensee under this Agreement, Licensee shall pay Licensor on-going annual maintenance fees on each anniversary of the Effective Date. Licensor will invoice Licensee for the amount of such maintenance fee, and the invoiced
amount will be due and payable by Licensee on the later of (i) 30 days after receipt of the invoice and (ii) the applicable anniversary of the Effective Date. The annual maintenance fees will equal (a) on each anniversary prior to
Licensee exercising the Commercial Option with respect to any Disease Indication, ****, and (b) on each anniversary after Licensee has exercised the Commercial Option with respect to any Disease Indication, **** for each Disease Indication with
respect to which the Commercial Option has been exercised as of such anniversary, up to a maximum under this clause (b) of **** for all four Disease Indications. If the royalty obligation with respect to any Disease Indication has expired or
such Disease Indication has otherwise been terminated, the amount due pursuant to this 

  
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Section 3.3 will be decreased by **** for each Disease Indication with respect to which the royalty obligation has expired or such Disease Indication has otherwise been terminated. 

3.4 Milestone Fees. If Licensee exercises the Commercial Option granted to Licensee under Section 2.3 with respect to any Disease Indication, in
consideration of the rights and licenses granted to Licensee under this Agreement, Licensee shall pay Licensor the following milestone payments on a per-Disease Indication basis for the first Licensed Product for such Disease Indication to achieve
such milestone event: 
  

					
	 Milestone
	  	Milestone Payment	 
	 1. First treatment of human subject in a clinical trial (i.e., first patient, first dose)
	  	 	****	  
	 2. First treatment in Phase 3 Clinical Trial (i.e., first patient, first dose)
	  	 	****	  
	 3. NDA submission in the United States
	  	 	****	  
	 4. NDA submission in the European Union or the rest of the world (excluding the United States)
	  	 	****	  
	 5. NDA approval in the United States
	  	 	****	  
	 6. NDA approval in the European Union or the rest of the world (excluding the United States)
	  	 	****	  
	 Total (per Disease Indication):
	  	$	5,000,000	  
		  	  
	  
	 

 For clarity, the milestone payments set forth in this Section 3.4 are payable **** with respect to each
Disease Indication within the Commercial Field with respect to the first Licensed Product for such Disease Indication that achieves the milestone event, ****. To the extent that either of the two development milestones in this Section 3.4
(i.e., first treatment of human subject in a clinical trial or first treatment in Phase 3 Clinical Trial) has not been paid at the time of achievement of either NDA submission milestone, then, upon the achievement of either of such NDA
submission milestones, the preceding unpaid development milestone payments shall be made in addition to the payment corresponding to the NDA submission milestone that has been achieved. 

3.5 Royalties. If Licensee exercises the Commercial Option granted to Licensee under Section 2.3 with respect to any Disease Indication, in
consideration of the rights and licenses granted to Licensee under this Agreement, Licensee shall pay to Licensor the following royalties based upon the annual Net Sales worldwide of all Licensed Products for all Disease Indications in the
Commercial Field in a given calendar year, subject to the reductions in royalty rates set forth in Section 3.5.1: 

  
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COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

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	 Cumulative Annual Net Sales of all Licensed

Products for all Disease Indications in the

Commercial Field Worldwide
	  	Royalty Percentage
	 Portion of Net Sales less than $300,000,000
	  	****
	 Portion of Net Sales between (and including) $300,000,000 through (and including) $600,000,0000
	  	****
	  
	 Portion of Net Sales greater than $600,000,000
	  	****

 3.5.1 Third Party Royalties Stacking Provision. If Licensee must obtain a license from a Third Party to
avoid infringement of such Third Party’s rights in order to manufacture, use, or commercialize a given Licensed Product and if the royalties required to be paid to such Third Party for such license, together with those royalties payable to
Licensor, in the aggregate, exceed **** of Net Sales for any Licensed Product, then the royalty owed to Licensor for that Licensed Product will be reduced by an amount calculated as follows: 

STACKING ROYALTY CALCULATIONS 

R = (C * (A / (A+B))) 
 Where 

R = Reduction of Licensor royalty, 

A = Unreduced Licensor royalty, 
 B
= sum of all Third Party royalties, 
 C = increment of projected total royalty above ****. 

Example Calculation: 
  

											
		 	        assume:        	 	i)	 	all Third Party royalties = ****	  	
		 		 	ii)	 	    unreduced Licensor royalty = ****	  	
		 		 	iii)	 	    projected total royalty = ****	  	
			
		 	            R = (**** - ****) * (**** / (****+****))	  	
		 	            R = (**** * ****)	  	
		 	            R = ****	  	
		 	 Licensor Stacked Royalty = **** — **** = ****%
	  	

 Notwithstanding the foregoing, Licensee will pay to Licensor no less than **** of the royalties that Licensee
would otherwise pay to Licensor with respect to Net Sales of Licensee if there were no royalties due to Third Parties. 
 3.5.2 Royalty
Payment Period. Licensee’s obligation hereunder for payment of a royalty under this Section 3.5 on the Net Sales of Licensed Products in a given country will expire on a Licensed Product-by-Licensed Product and country-by-country basis
when the Licensed Product ceases to infringe or be covered by a Valid Claim within the Licensed Commercial Patents in that country. 

  
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COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 3.5.3 No Multiple Royalties. If the manufacture, use, sale, offer for sale, or import
of any Licensed Product infringes or is covered by more than one of the Licensed Commercial Patents, multiple royalties shall not be due. 
 3.6
Sublicense Fees. 
 3.6.1 In further consideration of the rights and licenses granted to Licensee under this Agreement, Licensee will
pay Licensor **** of any sublicense fees (including upfront payments and milestone payments) received by Licensee or its Affiliates for the Licensed Commercial Patents from any Third Party Sublicensee or from any Third Party granted any option to
obtain a sublicense. 
 3.6.2 With respect to the obligations under this Section 3.6, Licensee shall not be required to submit any
amounts received from a Third Party for the following: 
  

	 	(a)	Reimbursement for research, development, and/or manufacturing activities performed by Licensee or its Affiliates corresponding directly to the development of Licensed Products pursuant to a specific agreement;

  

	 	(b)	Consideration received for the purchase of an equity interest in Licensee or its Affiliates at fair market value or in the form of loans at commercially reasonable rates of interest; and 

 

	 	(c)	Any and all amounts paid to Licensee or its Affiliates by a Third Party Sublicensee as royalties on sales of Licensed Product sold by such Sublicensee under a sublicense agreement. 

3.6.3 If Licensee or its Affiliate receives sublicense fees from Third Party Sublicensees or from any Third Party granted any option to obtain
a sublicense under this Agreement in the form of non-cash consideration, then, at Licensor’s option, Licensee shall pay Licensor payments as required by this Section 3.6 (a) in the form of the non-cash consideration received by
Licensee or its Affiliates or (b) a cash payment determined based on the fair market value of such non-cash consideration. If Licensee or its Affiliate enters into any sublicense with a Third Party Sublicensee that is not an arm’s length
transaction, fees due under this Section 3.6 will be calculated based on the fair market value of such transaction, at the time of the transaction, assuming an arm’s length transaction made in the ordinary course of business, as determined
jointly by Licensor and Licensee based on transactions of a similar type and standard industry practice, if any. 
 3.6.4 To the extent
Licensee receives payment from a Third Party relating to one or more of the milestone events set forth in the table in Section 3.4, then the amount of the payment made to Licensor under such Section 3.4 with respect to such milestone event
shall not be deemed sublicense fees under this Section 3.6; instead, the amounts due under this Section 3.6 shall be calculated by applying the sublicense fee rate set forth in Section 3.6.1 above to the sublicense fees received by
Licensee from such Third Party after deducting the amount of the payment under Section 3.4. 

  
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COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
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 3.6.5 If a sublicense or option is part of a transaction in which Licensee or its Affiliates
also licenses, sublicenses, or grants rights to technology, patent rights, or other intellectual property rights other than Licensed Patents, that portion of the consideration received by Licensee or its Affiliates and subject to this
Section 3.6 shall be equitably apportioned between the Licensed Patents and those other rights, and such apportionment shall be reasonable and in accordance with customary standards in the industry. Licensee shall promptly deliver to Licensor a
written report setting forth such apportionment and shall describe in reasonable detail the rationale for such allocation, together with a copy of all underlying documents necessary to determinate the basis and accuracy of such allocation. If
Licensor disagrees with the determination made by Licensee, Licensor shall so notify Licensee within **** of receipt of Licensee’s report, and the Parties shall meet to discuss and resolve such disagreement in good faith. If the Parties are
unable to agree as to such apportionment within ****, then the matter shall be submitted in accordance with the dispute resolution process set forth in Section 10.6. 

3.7 Reports and Records. 
 3.7.1 Licensee
must deliver to Licensor within **** after the end of each Calendar Quarter after the first commercial sale of a Licensed Product a report setting forth the calculation of the royalties due to Licensor for such Calendar Quarter, including: 

 

	 	(a)	Number of Licensed Products included within Net Sales, listed by country; 

  

	 	(b)	Gross consideration for Net Sales of Licensed Product, including all amounts invoiced, billed, or received; 

  

	 	(c)	Qualifying costs to be excluded from the gross consideration, as described in Section 1.19, listed by category of cost; 

  

	 	(d)	Net Sales of Licensed Products listed by country; 

  

	 	(e)	A detailed accounting of any royalty reductions applied pursuant to Section 3.5.1; 

  

	 	(f)	Royalties owed to Licensor, listed by category; and 

  

	 	(g)	The computations for any applicable currency conversions. 

 3.7.2 Licensee shall pay the
royalties due under Section 3.5 within **** following the last day of the Calendar Quarter in which the royalties accrue. Licensee shall send the royalty payments along with the report described in Section 3.7.1. 

3.7.3 Within **** after the occurrence of a milestone event described in Section 3.4, Licensee must deliver to Licensor a report
describing the milestone event that occurred, together with a payment of the applicable amount due to Licensor pursuant to Section 3.4. 

  
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PORTIONS. 

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 3.7.4 Within **** after the receipt of any fees from any Third Party as described in
Section 3.6, Licensee must deliver to Licensor a report describing the fees received, together with a payment of the applicable amount due to Licensor pursuant to Section 3.6. 

3.7.5 All financial reports under this Section 3.7 will be certified by the chief financial officer of Licensee. 

3.7.6 Licensee shall maintain and require its Affiliates and all Sublicensees to maintain, complete and accurate books and records which
enable the royalties, fees, and payments payable under this Agreement to be verified. The records must be maintained for **** after the submission of each report under Article 3. Upon reasonable prior written notice to Licensee, Licensee and its
Affiliates and all Sublicensees will provide Licensor and/or the ReGenX Licensors (and their respective accountants) with access to all of the relevant books, records, and related background information required by this Section 3.7.6 to conduct
a review or audit of the royalties, fees, and payments payable to Licensor under this Agreement to be verified. Access will be made available: (a) during normal business hours; (b) in a manner reasonably designed to facilitate the auditing
party’s review or audit without unreasonable disruption to Licensee’s business; and (c) no more than once each calendar year during the term of this Agreement and for a period of **** thereafter. Licensee will promptly pay to Licensor
the amount of any underpayment determined by the review or audit, plus accrued interest. If the review or audit determines that Licensee has underpaid any payment by **** or more, then Licensee will also promptly pay the costs and expenses of
Licensor and the ReGenX Licensors and their respective accountants in connection with the review or audit. 
 3.8 Currency, Interest. 

3.8.1 All dollar amounts referred to in this Agreement are expressed in United States dollars. All payments to Licensor under this Agreement
must be made in United States dollars. 
 3.8.2 If Licensee receives payment in a currency other than United States dollars for which a
royalty or fee or other payment is owed under this Agreement, then (a) the payment will be converted into United States dollars at the conversion rate for the foreign currency as published in the eastern edition of the Wall Street
Journal, N.Y. edition, as of the last business day of the Calendar Quarter in which the payment was received by Licensee; and (b) the conversion computation will be documented by Licensee in the applicable report delivered to Licensor under
Section 3.7. 
 3.8.3 All amounts that are not paid by Licensee when due will accrue interest from the date due until paid at a rate
equal to 1.5% per month (or the maximum allowed by law, if less). 
 3.9 Taxes and Withholding. 

3.9.1 All payments hereunder will be made free and clear of, and without deduction or deferment in respect of, and Licensee shall pay and be
responsible for, and shall hold Licensor harmless from and against, any taxes, duties, levies, fees, or charges, including sales, use, transfer, excise, import, and value added taxes (including any interest, penalties, or additional amounts imposed
with respect thereto) but excluding withholding taxes to the extent provided in Section 3.9.2. At the request of Licensee, Licensor will give Licensee such reasonable 

  
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 assistance, which will include the provision of documentation as may be required by the relevant tax
authority, to enable Licensee to pay and report and, as applicable, claim exemption from or reduction of, such tax, duty, levy, fee, or charge. 

3.9.2 If any payment made by Licensee hereunder becomes subject to withholding taxes with respect to Licensor’s gross or net income under
the laws of any jurisdiction, Licensee will deduct and withhold the amount of such taxes for the account of Licensor to the extent required by law and will pay the amounts of such taxes to the proper governmental authority in a timely manner and
promptly transmit to Licensor appropriate proof of payment of such withholding taxes. At the request of Licensor, Licensee will give Licensor such reasonable assistance, which will include the provision of appropriate certificates of such deductions
made together with other supporting documentation as may be required by the relevant tax authority, to enable Licensor to claim exemption from or reduction of, or otherwise obtain repayment of, such withholding taxes, and will upon request provide
such additional documentation from time to time as is reasonably required to confirm the payment of withholding tax. 
 ARTICLE 4:
DILIGENCE 
 4.1 Diligence Obligations. If Licensee elects to exercise the Commercial Option granted to Licensee under
Section 2.3 with respect to any Disease Indication, Licensee will use commercially reasonable efforts to develop, commercialize, market, promote, and sell at least one Licensed Product for each Disease Indication in the Commercial Field.
Commercially reasonable efforts means efforts equivalent to those utilized by ****. 
 4.2 Reporting. Within **** after the Grant Date and within
**** of each December 1 thereafter, Licensee shall provide Licensor with written progress reports, setting forth in such detail as Licensor may reasonably request, the progress of the development, evaluation, testing, and commercialization of
each Licensed Product. Licensee will also notify Licensor within **** of the first commercial sale by Licensee, its Affiliates, or any Sublicensees of each Licensed Product. Such a report (“Development Progress Report”), setting
forth the current stage of development of Licensed Products, shall include: 
 4.2.1 Date of Development Progress Report and time covered by
such report; 
 4.2.2 Major activities and accomplishments completed by Licensee, its Affiliates, and any Sublicensees relating directly to
the Licensed Product since the last Development Progress Report; 
 4.2.3 Significant research and development projects relating directly to
the Licensed Product currently being performed by Licensee, its Affiliates, and any Sublicensees and projected dates of completion; 

  
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COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 4.2.4 A development plan covering the next two years at least, which will include future
development activities to be undertaken by Licensee, its Affiliates, or any Sublicensees during the next reporting period relating directly to the Licensed Product, Licensee’s strategy to bring the Licensed Product to commercialization, and
projected timeline for completing the necessary tasks to accomplish the goals of the strategy; 
 4.2.5 Projected total development
remaining before product launch of each Licensed Product; and 
 4.2.6 Summary of significant development efforts using the Licensed Patents
being performed by Third Parties, including the nature of the relationship between Licensee and such Third Parties. 
 4.3 Confidential Information.
The Parties agree that Development Progress Reports shall be deemed Licensee’s Confidential Information; provided that Licensor may share a copy of such reports with the ReGenX Licensors. 

4.4 Improvements. Simultaneously with the Development Progress Report, Licensee shall deliver a detailed description of any Licensed Back Improvements,
if not previously provided pursuant to Section 2.7.4. 
 ARTICLE 5: CONFIDENTIALITY 

5.1 Treatment of Confidential Information. Each Party, as a receiving party (a “Receiving Party”), agrees that it will (a) treat
Confidential Information of the other Party (the “Disclosing Party”) as strictly confidential; (b) not disclose such Confidential Information to Third Parties without the prior written consent of the Disclosing Party, except as
may be permitted in this Agreement; provided that any disclosure permitted hereunder be under confidentiality agreements with provisions substantially similar to those contained in this Agreement; and (c) not use such Confidential Information
for purposes other than those authorized expressly in this Agreement. The Receiving Party agrees to ensure that its employees who have access to Confidential Information are obligated in writing to abide by confidentiality obligations substantially
similar to those contained under this Agreement. 
 5.2 Public Announcements. 

5.2.1 The Parties agree they will release a joint press release in the form attached hereto as Exhibit B. Except as provided in
Section 5.2.2, any other press releases by either Party with respect to the other Party or any other public disclosures concerning the existence of or terms of this Agreement shall be subject to review and approval by the other Party. Once the
joint press release or any other written statement is approved for disclosure by both Parties, either Party may make subsequent public disclosure of the contents of such statement without the further approval of the other Party. 

5.2.2 Notwithstanding Section 5.2.1, Licensor has the right to publish (through press releases, scientific journals, or otherwise) and
refer to any clinical, regulatory, or research results related to Licensee’s Licensed Product or Specified Vector program that have been publicly 

  
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disclosed by Licensee, including referring to Licensee by name as a licensee of Licensor, which publication or referral by Licensor shall not require the prior consent of Licensee. 

5.3 Authorized Disclosure. Notwithstanding the provisions of Section 5.1 or 5.2, either Party may disclose Confidential Information or make such a
disclosure of the existence of and/or terms of this Agreement to any ****; provided that, in each case, such recipient of Confidential Information is obligated to keep such information confidential on terms substantially similar to those set forth
in this Agreement. Furthermore, Licensee agrees that Licensor may share a copy of this Agreement, reports and notices provided by Licensee to Licensor pursuant to the terms of this Agreement, and copies of sublicense agreements provided to Licensor
hereunder with the ReGenX Licensors. In the event that the Receiving Party receives service of legal process that purports to compel disclosure of the Disclosing Party’s Confidential Information or becomes obligated by law to disclose the
Confidential Information of the Disclosing Party or the existence of or terms of this Agreement to any governmental authority, the Receiving Party shall promptly notify the Disclosing Party, so that the Disclosing Party may seek an appropriate
protective order or other remedy with respect to narrowing the scope of such requirement and/or waive compliance by the Receiving Party with the provisions of this Agreement. The Receiving Party will provide the Disclosing Party, at the Disclosing
Party’s expense, with reasonable assistance in obtaining such protective order or other remedy. If, in the absence of such protective order or other remedy, the Receiving Party is nonetheless required by law to disclose the existence of or
terms of this Agreement or other Confidential Information of the Disclosing Party, the Receiving Party may disclose such Confidential Information without liability hereunder; provided that the Receiving Party shall furnish only such portion of the
Confidential Information that is legally required to be disclosed and only to the extent required by law. 
 5.4 Term of Confidentiality. The
obligations of this Article 5 shall continue for a period of **** following the expiration or termination of this Agreement. 
 ARTICLE 6:
TERM AND TERMINATION 
 6.1 Term of Agreement. This Agreement, unless sooner terminated as provided in this Agreement, expires upon the
expiration, lapse, abandonment, or invalidation of the last Valid Claim of the Licensed Commercial Patents to expire, lapse, or become abandoned or unenforceable in all the countries of the world. 

6.2 Termination for Failure to Exercise Option. This Agreement will terminate automatically at the end of the Research Term if Licensee does not
exercise the Commercial Option with respect to any Disease Indication in accordance with Section 2.3. If Licensee does not exercise the Commercial Option with respect to all Disease Indications, this Agreement will terminate with respect to all
unexercised Disease Indications at the end of the Research Term. 
 6.3 Licensee’s Right to Terminate. Licensee may, upon 90 days’ prior
written notice to Licensor, terminate this Agreement for any reason, with or without cause. In exercising such termination right, Licensee may terminate the Agreement in its entirety or, if desired, Licensee

  
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may specify in the written notice that this Agreement is terminating only with respect to one or more of the Disease Indications within the Research Field or Commercial Field, as applicable. 

6.4 Termination for Breach. 
 6.4.1
Licensor may terminate this Agreement, if Licensee is late in paying to Licensor royalties, fees, or any other monies due under this Agreement, and Licensee does not pay Licensor in full within 15 days upon written demand from Licensor, which
termination shall be effective immediately upon the expiration of such 15-day cure period. 
 6.4.2 Either Party may terminate this
Agreement, if the other Party materially breaches this Agreement and does not cure such material breach within 30 days after written notice of the breach, which termination shall be effective immediately upon the expiration of such 30-day cure
period. 
 6.5 Termination for Insolvency. 

6.5.1 Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, if Licensee, any of its Affiliates, or any
Sublicensees experiences any Trigger Event. 
 6.5.2 For purposes of this Section 6.5, “Trigger Event” means any of
the following: (a) if Licensee, any Affiliate, or any Sublicensee, as applicable, (i) becomes insolvent, becomes bankrupt, or generally fails to pay its debts as such debts become due, (ii) is adjudicated insolvent or bankrupt,
(iii) admits in writing its inability to pay its debts, (iv) suffers the appointment of a custodian, receiver, or trustee for it or its property and, if appointed without its consent, is not discharged within 30 days, (v) makes an
assignment for the benefit of creditors, or (vi) suffers proceedings being instituted against it under any law related to bankruptcy, insolvency, liquidation, or the reorganization, readjustment, or release of debtors and, if contested by it,
not dismissed or stayed within ten days; (b) the institution or commencement by Licensee, any Affiliate, or any Sublicensee, as applicable, of any proceeding under any law related to bankruptcy, insolvency, liquidation, or the reorganization,
readjustment, or release of debtors; (c) the entering of any order for relief relating to any of the proceedings described in Section 6.5.2(a) or (b) above; (d) the calling by Licensee, any Affiliate, or any Sublicensee, as
applicable, of a meeting of its creditors with a view to arranging a composition or adjustment of its debts; or (e) the act or failure to act by Licensee, any Affiliate, or any Sublicensee, as applicable, indicating its consent to, approval of,
or acquiescence in any of the proceedings described in Section 6.5.2(b) through (d) above. 
 6.6 Patent Challenge. 

6.6.1 Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, upon the commencement by Licensee or any of
its Affiliates of a Patent Challenge. Licensee shall include in each sublicense agreement entered into with a Sublicensee a right of Licensee to terminate such sublicense agreement if such Sublicensee commences a Patent Challenge; and Licensee shall
terminate the sublicense agreement, effective immediately upon written notice to the Sublicensee, if the Sublicensee commences a Patent Challenge. In addition, if the Sublicensee’s commencement of a Patent Challenge gives The Trustees of the

  
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University of Pennsylvania (or any successor thereto under the Penn Agreement) a right of termination under the Penn Agreement, then, upon receipt of notice from the Trustees of the University of
Pennsylvania, Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, if any Sublicensee commences a Patent Challenge. If Licensor obtains actual knowledge of a Patent Challenge commenced by a Sublicensee,
Licensor shall use commercially reasonable efforts to provide Licensee with written notice of such Patent Challenge; provided that Licensor’s failure to provide such notice will not affect Licensee’s obligations hereunder. 

6.6.2 For purposes of this Section 6.6, “Patent Challenge” means any action against Licensor, The Trustees of the
University of Pennsylvania, or the ReGenX Licensors, including an action for declaratory judgment, to declare or render invalid or unenforceable the Licensed Patents, or any claim thereof. 

6.7 Effects of Termination. The effect of termination pursuant to Section 6.2, by Licensee pursuant to Section 6.3, by either Party, as
applicable, under Section 6.4, or by Licensor pursuant to Section 6.5 or 6.6 shall be as follows: 
 6.7.1 The licenses granted by
Licensor hereunder shall terminate, and Licensee, its Affiliates, and (unless the sublicense agreement is assigned pursuant to Section 6.7.2) all Sublicensees shall cease to make, have made, use, import, sell, and offer for sale all AAV
Materials or Licensed Products and shall cease to otherwise practice the Licensed Patents; provided that Licensee, its Affiliates, and Sublicensees shall have the right to continue to sell their existing inventories of Licensed Products for a period
not to exceed **** after the effective date of such termination; 
 6.7.2 Licensee shall assign to Licensor any or all sublicenses granted
to Third Parties to the extent of the rights licensed to Licensee hereunder and sublicensed to the Sublicensee; provided that (i) prior to such assignment, Licensee shall advise Licensor whether such Sublicensee is then in full compliance with
all terms and conditions of its sublicense and continues to perform thereunder, and, if such Sublicensee is not in full compliance or is not continuing to perform, Licensor may elect not to have such sublicense assigned, in which event such
sublicense shall terminate; (ii) such Sublicensee must agree in writing to assume Licensee’s terms, conditions, and obligations to Licensor set forth in this Agreement, including all payment obligations; and (iii) following such
assignment, Licensor shall not be liable to such Sublicensee with respect to any obligations of Licensee to the Sublicensee that are not consistent with, or not required by, Licensor’s obligations to Licensee under this Agreement; and all
sublicenses not assigned to Licensor as provided in this Section 6.7.2 shall terminate; 
 6.7.3 If termination is by Licensee pursuant
to Section 6.3 or by Licensor pursuant to Section 6.4, 6.5, or 6.6, then, effective as of such termination of this Agreement, Licensee shall grant, and hereby grants, to Licensor a non-exclusive, perpetual, irrevocable, worldwide,
royalty-free, transferable, sublicensable license under any patentable modifications or improvements (and any intellectual property rights with respect thereto) developed, during the term of this Agreement, by Licensee, any Affiliates, or any
Sublicensees to any vector that is the subject of a claim within any of the Licensed Patents, for use by Licensor (and its sublicensees) for the research, development, and commercialization of products in any therapeutic indication; 

  
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 provided that the categorization of patentable modifications or improvements that are subject to this
Section 6.7.3 will be subject to the same exclusions applicable to “Licensed Back Improvements” under Sections 2.7.5 and 2.7.6. 

6.7.4 Licensee shall pay all monies then-owed to Licensor under this Agreement; 

6.7.5 Each Receiving Party shall, at the other Party’s request, return all Confidential Information of the Disclosing Party.
Notwithstanding the foregoing, one copy may be kept by either Party for a record of that Party’s obligations; and 
 6.7.6 If
termination is only with respect to a particular Disease Indication within the Research Field or the Commercial Field, but not all Disease Indications, then the provisions of this Section 6.7 shall only apply with respect to the terminated
Disease Indications, and this Agreement shall continue as provided herein with respect to the non-terminated Disease Indications. 
 6.8 Survival.
Licensee’s obligation to pay all monies due and owed to Licensor under this Agreement which have matured as of the effective date of termination or expiration shall survive the termination or expiration of this Agreement. In addition, the
provisions of Article 1 (Definitions), Section 2.4, (Retained Rights), Section 2.5 (Government Rights), Section 2.7 (Improvements), Section 3.1 (Initial Fee), Article 3 (Consideration) (with respect to any final reports or to the
extent any amounts are due but unpaid), Section 3.7 (Reports and Records), Article 5 (Confidentiality), Section 6.7 (Effects of Termination), Section 6.8 (Survival), Section 8.3 (Disclaimer of Warranties, Damages),
Section 8.4 (Indemnification), Section 8.5 (Insurance), Article 9 (Use of Name), and Article 10 (Additional Provisions) shall survive such termination or expiration of this Agreement in accordance with their respective terms. 

ARTICLE 7: PATENT MAINTENANCE; PATENT INFRINGEMENT 

7.1 Prosecution of Licensed Patents. As between Licensor and Licensee, but subject to any obligations of Licensor to the ReGenX Licensors, the Parties
agree as follows: 
 7.1.1 Licensor shall have the sole right, but not the obligation, to Prosecute patent applications and issued patents
within Licensed Patents, in Licensor’s sole discretion. 
 7.1.2 Nothing in this Agreement obligates Licensor to continue to Prosecute
any patent applications or issued patents, and Licensee acknowledges that Licensor shall have no obligation to undertake any inter-party proceedings, such as oppositions or interferences, or to undertake any re-examination or re-issue proceedings,
in either case, with respect to the Licensed Patents. 
 7.2 Infringement Actions Against Third Parties. 

7.2.1 Licensee is responsible for notifying Licensor promptly of any infringement of Licensed Patents within the Disease Indications (other
than Retained Rights) that may come to Licensee’s attention. 
 7.2.2 As between Licensor and Licensee, but subject to any obligations
of Licensor to the ReGenX Licensors, Licensor shall have the sole right, but not the obligation, to prosecute any 

  
 23 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 such infringement **** recovered in connection therewith. In any action to enforce any of the Licensed
Patents, Licensee, at the request and expense of Licensor, shall cooperate to the fullest extent reasonably possible. Nothing in this Agreement obligates Licensor to bring or prosecute lawsuits against Third Parties for infringement of any Licensed
Patents. 
 7.2.3 Licensee shall have no right to undertake prosecution of any such infringement. 

7.3 Defense of Infringement Claims. In the event Licensee or Licensor becomes aware that Licensee’s or any of its Affiliates’ or any
Sublicensees’ practice of the Licensed Patents is the subject of a claim for patent infringement by a Third Party, that Party shall promptly notify the other, and the Parties shall consider the claim and the most appropriate action to take.
Licensee shall cause each of its Affiliates and each Sublicensee to notify Licensee promptly in the event such entity becomes aware that its practice of the Licensed Patents is the subject of a claim of patent infringement by another. To the extent
Licensor takes any action, Licensor (or the ReGenX Licensors) shall have the right to require Licensee’s reasonable cooperation in any such suit, upon written notice to Licensee; and Licensee shall have the obligation to participate upon
Licensor’s request, in which event, Licensor shall bear the cost of Licensee’s participation. Without Licensor’s prior written permission, Licensee must not settle or compromise any such suit in a manner that imposes any material
obligations or restrictions on Licensor or the ReGenX Licensors or grants any rights to the Licensed Patents other than rights that Licensee has the right to grant under this Agreement. 

ARTICLE 8: REPRESENTATIONS AND WARRANTIES; INDEMNIFICATION 

8.1 Representations and Warranties by Licensor. Licensor represents and warrants to Licensee as of the Effective Date: 

8.1.1 Licensor has the right, power, and authority to enter into this Agreement and to grant to Licensee the licenses specified in this
Agreement; 
 8.1.2 This Agreement when executed shall become the legal, valid, and binding obligation of it, enforceable against it, in
accordance with its terms; 
 8.1.3 There are no actions, suits, proceedings, or arbitrations pending or, to Licensor’s knowledge,
threatened against Licensor relating to the Licensed Research Patents that would be inconsistent with the rights granted to Licensee under this Agreement; 

8.1.4 To Licensor’s Knowledge, Licensor has not received any written notice from the ReGenX Licensors informing Licensor that there are
any actions, suits, proceedings, or arbitrations pending against the ReGenX Licensors relating to the Licensed Research Patents that would be inconsistent with the rights granted to Licensee under this Agreement; 

8.1.5 To Licensor’s knowledge, (a) the Licensed Research Patents are solely owned by the Trustees of the University of Pennsylvania,
and (b) no Third Party (other than the ReGenX Licensors) has any right, interest, or claim in or to such Licensed Research Patents with respect to the Disease Indications that are inconsistent with those granted to Licensee with respect to the
Disease Indications; 

  
 24 

 

	****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE
COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 8.1.6 To Licensor’s knowledge, GSK Agreement and Penn Agreement are in full force and
effect; 
 8.1.7 To Licensor’s knowledge, no Third Party is infringing any of the Licensed Research Patents in a manner that is
inconsistent with the scope of rights granted to Licensee with respect to the Disease Indications; and 
 8.1.8 Licensor has not received
any written notice from any Third Party patentee alleging infringement of such Third Party’s patents by the practice of the Licensed Research Patents with respect to the Disease Indications. 

8.2 Representations and Warranties by Licensee. Licensee represents and warrants to Licensor as of the Effective Date that: 

8.2.1 Licensee has the right, power, and authority to enter into this Agreement and to grant the licenses granted by it hereunder; 

8.2.2 This Agreement when executed shall become the legal, valid, and binding obligation of it, enforceable against it, in accordance with its
terms; 
 8.2.3 Licensee has the ability and the resources, including financial resources, necessary to carry out its obligations under this
Agreement; and 
 8.2.4 There are no actions, suits, proceedings, or arbitrations pending or, to Licensee’s knowledge, threatened
against Licensee that would impact activities under this Agreement. 
 8.3 Disclaimer of Warranties, Damages. 

8.3.1 EXCEPT AS SET FORTH IN SECTION 8.1, THE LICENSED PATENTS, AAV MATERIALS, LICENSED PRODUCTS, AND ALL RIGHTS LICENSED UNDER THIS AGREEMENT
ARE PROVIDED ON AN “AS IS” BASIS, AND LICENSOR MAKES NO REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, WITH RESPECT THERETO. BY WAY OF EXAMPLE BUT NOT OF LIMITATION, LICENSOR MAKES NO REPRESENTATIONS OR WARRANTIES, AND HEREBY DISCLAIMS
ALL EXPRESS AND IMPLIED REPRESENTATIONS AND WARRANTIES, (i) OF COMMERCIAL UTILITY, ACCURACY, COMPLETENESS, PERFORMANCE, MERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE, VALIDITY OR ENFORCEABILITY OF THE LICENSED PATENTS, AND PROFITABILITY; OR
(ii) THAT THE USE OF THE LICENSED PATENTS, AAV MATERIALS, OR LICENSED PRODUCTS WILL NOT INFRINGE ANY PATENT, COPYRIGHT, TRADEMARK, OR OTHER PROPRIETARY RIGHTS OF THIRD PARTIES. 

8.3.2 EXCEPT AS SET FORTH HEREIN, NONE OF LICENSOR OR THE REGENX LICENSORS SHALL BE LIABLE TO LICENSEE, LICENSEE’S SUCCESSORS OR ASSIGNS,
ANY SUBLICENSEES, OR ANY THIRD PARTY WITH RESPECT TO ANY CLAIM ARISING FROM USE OF THE LICENSED PATENTS, AAV MATERIALS, LICENSED PRODUCTS, AND ANY OR ALL RIGHTS LICENSED UNDER THIS 

  
 25 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
AGREEMENT OR FROM THE DEVELOPMENT, TESTING, MANUFACTURE, USE, OR SALE OF AAV MATERIALS OR LICENSED PRODUCTS 

8.3.3 NEITHER PARTY SHALL BE LIABLE TO THE OTHER PARTY OR ITS SUCCESSORS OR ASSIGNS, ANY SUBLICENSEE, OR THIRD PARTY AND NEITHER OF THE REGENX
LICENSORS SHALL BE LIABLE TO LICENSEE, LICENSEE’S SUCCESSORS OR ASSIGNS, ANY SUBLICENSEES, OR ANY THIRD PARTY WITH RESPECT TO ANY CLAIM FOR LOSS OF PROFITS, LOSS OR INTERRUPTION OF BUSINESS, OR FOR INDIRECT, SPECIAL, INCIDENTAL, PUNITIVE, OR
CONSEQUENTIAL DAMAGES OF ANY KIND, INCLUDING ANY ARISING FROM OR RELATING TO ANY BREACH OF THIS AGREEMENT OR THE EXERCISE OF RIGHTS HEREUNDER, REGARDLESS OF ANY NOTICE OF SUCH DAMAGES; PROVIDED THAT NOTHING IN THIS SECTION 8.3.3 IS INTENDED TO LIMIT
OR RESTRICT THE INDEMNIFICATION RIGHTS OR OBLIGATIONS OF EITHER PARTY UNDER SECTION 8.4 OR TO LIMIT A PARTY’S LIABILITY FOR BREACHES OF ITS OBLIGATION REGARDING CONFIDENTIALITY UNDER ARTICLE 5. 

8.4 Indemnification. 
 8.4.1 By
Licensee. Licensee shall defend, indemnify, and hold harmless Licensor, the ReGenX Licensors, and their respective shareholders, members, officers, directors, trustees, faculty, students, contractors, agents, and employees (individually, a
“Licensor Indemnified Party” and, collectively, the “Licensor Indemnified Parties”) from and against any and all Third Party liability, loss, damage, action, claim, fee, cost, or expense (including attorneys’
fees) (individually, a “Third Party Liability” and, collectively, the “Third Party Liabilities”) suffered or incurred by the Licensor Indemnified Parties from claims of such Third Parties that result from or arise
out of: ****; provided, however, that Licensee shall not be liable for claims to the extent based on any breach by Licensor of the representations, warranties, or obligations of this Agreement or the gross negligence or intentional misconduct of any
of the Licensor Indemnified Parties. Without limiting the foregoing, Licensee must defend, indemnify, and hold harmless the Licensor Indemnified Parties from and against any Third Party Liabilities resulting from: 

 

	 	(a)	any **** or other claim of any kind related to the **** by a Third Party of a Licensed Product that was **** by Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors; 

 

	 	(b)	any claim by a Third Party that the ****; and 

  
 26 

 

	****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE
COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

	 	(c)	**** conducted by or on behalf of Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors relating to the Licensed Patents, AAV Materials, or Licensed Products, including any claim by or on
****. 

 8.4.2 By Licensor. Licensor shall defend, indemnify, and hold harmless Licensee, its shareholders, members,
officers, directors, contractors, agents, and employees (individually, a “Licensee Indemnified Party” and, collectively, the “Licensee Indemnified Parties”) from and against any and all Third Party Liabilities
suffered or incurred by the Licensee Indemnified Parties from claims of such Third Parties to the extent that such claims result from or arise out of the ****; provided, however, that Licensor shall not be liable for claims to the extent based on
any breach by Licensee of the representations, warranties, or obligations of this Agreement or the gross negligence or intentional misconduct of any of the Licensee Indemnified Parties. 

8.4.3 Indemnification Procedure. Each Party, as an indemnifying party (an “Indemnifying Party”), shall not be
permitted to settle or compromise any claim or action giving rise to Third Party Liabilities in a manner (a) that imposes any restrictions or obligations on any indemnified party (an “Indemnified Party”) without the Indemnified
Party’s prior written consent, (b) if Licensee is the Indemnifying Party, that imposes any restrictions or obligations on the ReGenX Licensors or grants any rights to the Licensed Patents, AAV Materials, or Licensed Products other than
those Licensee has the right to grant under this Agreement without Licensor’s prior written consent, or (c) if Licensor is the Indemnifying Party, that grants any rights to the Licensed Back Improvements other than those Licensor has the
right to grant under this Agreement without Licensee’s prior written consent. The Indemnifying Party shall be permitted to control any litigation or potential litigation involving the defense of any claim subject to indemnification pursuant to
this Section 8.4, including the selection of counsel, with the reasonable approval of the Indemnified Party. If an Indemnifying Party fails or declines to assume the defense of any such claim or action within **** after notice thereof, the
Indemnified Party may assume the defense of such claim or action at the cost and risk of the Indemnifying Party, and any Third Party Liabilities related thereto shall be conclusively deemed a Third Party Liability of the Indemnifying Party. The
indemnification rights of a Indemnified Party contained in this Agreement are in addition to all other rights that such Indemnified Party may have at law or in equity or otherwise. The Indemnifying Party will pay directly all Third Party Liabilities
incurred for defense or negotiation of any claim hereunder or will reimburse the Indemnified Party for all documented Third Party Liabilities incident to the defense or negotiation of any such claim within **** after the Indemnifying Party’s
receipt of invoices for such fees, expenses, and charges. 
 8.5 Insurance. Licensee will procure and maintain insurance policies for the following
coverages with respect to product liability, personal injury, bodily injury, and property damage arising out of Licensee’s (and its Affiliates’ and any Sublicensees’) performance under this Agreement: (a) during the term of this
Agreement, comprehensive general liability, including broad form and contractual liability, in a minimum amount of **** combined single limit 

  
 27 

 

	****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE
COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 per occurrence (or claim) and in the aggregate annually; (b) prior to the commencement of clinical
trials involving Licensed Products and thereafter for a period of not less than **** (or such longer period as Licensee is required by applicable law to continue to monitor the participants in the clinical trial), clinical trials coverage in amounts
that are reasonable and customary in the U.S. pharmaceutical industry, subject always to a minimum limit of **** combined single limit per occurrence (or claim) and in the aggregate annually; and (c) from prior to the first commercial sale of a
Licensed Product until **** after the last sale of a Licensed Product, product liability coverage, in amounts that are reasonable and customary in the U.S. pharmaceutical industry, subject always to a minimum limit of **** combined single limit per
occurrence (or claim) and in the aggregate annually. Licensor may review periodically the adequacy of the minimum amounts of insurance for each coverage required by this Section 8.5, and Licensor reserves the right to require Licensee to adjust
the limits accordingly. The required minimum amounts of insurance do not constitute a limitation on Licensee’s liability or indemnification obligations to the Licensor Indemnified Parties under this Agreement. The policies of insurance required
by this Section 8.5 will be issued by an insurance carrier with an A.M. best rating of **** or better and will name Licensor as an additional insured with respect to Licensee’s performance (and its Affiliates’ and any
Sublicensees’) under this Agreement. Licensee will provide Licensor with insurance certificates evidencing the required coverage within **** after the Effective Date and the commencement of each policy period and any renewal periods. Each
certificate will provide that the insurance carrier will notify Licensor in writing at least **** prior to the cancellation or material change in coverage. Licensee will cause all Sublicensees to comply with the terms of this Section 8.5 to the
same extent as Licensee. 
 ARTICLE 9: USE OF NAME 

Licensee, its Affiliates, any Sublicensees, and all of its and their employees and agents must not use Licensor’s, the University of Pennsylvania’s,
or SmithKline Beecham Corporation’s name, seal, logo, trademark, or service mark (or any adaptation thereof) or the name, seal, logo, trademark, or service mark (or any adaptation thereof) of any of such entities’ representative, school,
organization, employee, or student in any way without the prior written consent of Licensor or such entity, as applicable; provided, however that Licensee may acknowledge the existence and general nature of this Agreement, subject to
Section 5.3. 
 ARTICLE 10: ADDITIONAL PROVISIONS 

10.1 Relationship. Nothing in this Agreement shall be deemed to establish a relationship of principal and agent between Licensee and Licensor, nor any
of their agents or employees for any purpose whatsoever, nor shall this Agreement be construed as creating any other form of legal association or arrangement which would impose liability upon one Party for the act or failure to act of the other
Party. 
 10.2 Assignment. The rights and obligations of Licensee and Licensor hereunder shall inure to the benefit of, and shall be binding upon,
their respective permitted successors and assigns. Licensee may not assign this Agreement or any of its rights or obligations under this Agreement 

  
 28 

 

	****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE
COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
without the prior written consent of Licensor, which consent may not be unreasonably withheld, conditioned, or delayed. Notwithstanding the foregoing, Licensee may assign this Agreement without
Licensor’s consent, (a) to an Affiliate; provided that such Affiliate will continue to have to perform under Section 4.1 with at least the same level of efforts that Licensee would have been required to exercise; or (b) pursuant
to a sale or merger of Licensee or the transfer of substantially all of the assets of Licensee’s business to which this Agreement relates (whether by sale, merger, reorganization, consolidation, or otherwise); provided that, as part of any
permitted assignment, (i) Licensee provides Licensor with written notice of such assignment at least five business days prior to the effectiveness of such assignment; (ii) Licensee requires any such assignee to agree in writing to be
legally bound by this Agreement to the same extent as Licensee and provides Licensor with a copy of such assignee undertaking; and (iii) if such assignment is to an Affiliate, Licensee remains responsible for the performance of this Agreement
by such Affiliate. An assignment to an Affiliate will terminate, and all rights assigned will revert to Licensee, if and when such Affiliate ceases to be an Affiliate of Licensee, and Licensee will provide Licensor written notice of such assignment
within five business days of such event. In addition, Licensee will provide Licensor with written notice of any Change of Control (for purposes of this Agreement, the term “Change of Control” means the acquisition by a person or group of
“control” of Licensee, as defined in Section 1.3, whether or not the person or group acquiring control would be deemed an “Affiliate” under such Section 1.3) of Licensee at least five business days prior to the
effectiveness of such Change of Control. Licensor may assign this Agreement and its rights and obligations without the consent of Licensee. No assignment shall relieve the assigning Party of responsibility for the performance of any accrued
obligations which it has prior to such assignment. Any attempted assignment by Licensee in violation of this Section 10.2 shall be null and void and of no legal effect. 

10.3 Waiver. A waiver by either Party of a breach of any provision of this Agreement will not constitute a waiver of any subsequent breach of that
provision or a waiver of any breach of any other provision of this Agreement. 
 10.4 Notices. Notices, payments, statements, reports, and other
communications under this Agreement shall be in writing and shall be deemed to have been received as of the date received if sent by public courier (e.g., Federal Express), sent by Express Mail, receipt requested, delivered in person, or sent
by facsimile (with a copy of such facsimile also sent by one of the other methods of delivery) and addressed as follows: 
  

			
	If for Licensor:	  	with a copy to:
		
	 ReGenX Biosciences, LLC
 750 17th Street, NW

Suite 1100
 Washington, DC 20006

USA
 Attn: Chief Executive Officer

Telephone: 202-785-7438
 Facsimile: 202-785-7439
	  	 ReGenX Biosciences, LLC
 750 17th Street,
NW
 Suite 1100
 Washington, DC 20006

USA
 Attn: General Counsel

Telephone: 202-785-7438
 Facsimile:
202-785-7439

 If for Licensee: 

  
 29 

 CONFIDENTIAL TREATMENT REQUESTED 

 

	
	 Voyager Therapeutics, Inc.
 75 Sidney Street

Cambridge, MA 02139
 Attn: Chief Executive Officer

Telephone: 857-259-5340
 Facsimile: 617-621-2971

 Either Party may change its official address upon written notice to the other Party. 

10.5 Applicable Law. This Agreement shall be construed and governed in accordance with the laws of the State of Delaware, without giving effect to
conflict of law provisions that may require the application of the laws of another jurisdiction. Subject to Section 10.6, the Parties hereby submit to the exclusive jurisdiction of and venue in the courts located in the State of Delaware with
respect to any and all disputes concerning the subject of this Agreement. 
 10.6 Dispute Resolution. In the event of any controversy or claim
arising out of or relating to this Agreement, the Parties shall first attempt to resolve such controversy or claim through good faith negotiations for a period of not less than **** following notification of such controversy or claim to the other
Party. If such controversy or claim cannot be resolved by means of such negotiations during such period, then such controversy or claim shall be resolved by binding arbitration administered by the American Arbitration Association
(“AAA”) in accordance with the Commercial Arbitration Rules of the AAA in effect on the date of commencement of the arbitration, subject to the provisions of this Section 10.6. The arbitration shall be conducted as follows:

 10.6.1 The arbitration shall be conducted by three arbitrators, each of whom by training, education, or experience has knowledge of the
research, development, and commercialization of biological therapeutic products in the United States. The arbitration shall be conducted in English and held in New York, New York. 

10.6.2 In its demand for arbitration, the Party initiating the arbitration shall provide a statement setting forth the nature of the dispute,
the names and addresses of all other parties, an estimate of the amount involved (if any), the remedy sought, otherwise specifying the issue to be resolved, and appointing one neutral arbitrator. In an answering statement to be filed by the
responding Party within **** after confirmation of the notice of filing of the demand is sent by the AAA, the responding Party shall appoint one neutral arbitrator. Within **** from the date on which the responding Party appoints its neutral
arbitrator, the first two arbitrators shall appoint a chairperson. 
 10.6.3 If a Party fails to make the appointment of an arbitrator as
provided in Section 10.6.2, the AAA shall make the appointment. If the appointed arbitrators fail to appoint a chairperson within the time specified in Section 10.6.2 and there is no agreed extension of time, the AAA shall appoint the
chairperson. 
 10.6.4 The arbitrators will render their award in writing and, unless all Parties agree otherwise, will include an
explanation in reasonable detail of the reasons for their award. Judgment upon the award rendered by the arbitrators may be entered in any court having 

  
 30 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 
jurisdiction thereof, including in the courts described in Section 10.5. The arbitrators will have the authority to grant injunctive relief and other specific performance; provided that the
arbitrators will have no authority to award damages in contravention of this Agreement, and each Party irrevocably waives any claim to such damages in contravention of this Agreement. The arbitrators will, in rendering their decision, apply the
substantive law of the State of Delaware, without giving effect to conflict of law provisions that may require the application of the laws of another jurisdiction. The decision and award rendered by the arbitrators will be final and non-appealable
(except for an alleged act of corruption or fraud on the part of the arbitrator). 
 10.6.5 The Parties shall use their reasonable efforts
to conduct all dispute resolution procedures under this Agreement as expeditiously, efficiently, and cost-effectively as possible. 
 10.6.6
All expenses and fees of the arbitrators and expenses for hearing facilities and other expenses of the arbitration will be borne equally by the Parties unless the Parties agree otherwise or unless the arbitrators in the award assess such expenses
against one of the Parties or allocate such expenses other than equally between the Parties. Each of the Parties will bear its own counsel fees and the expenses of its witnesses except to the extent otherwise provided in this Agreement or by
applicable law. 
 10.6.7 Compliance with this Section 10.6 is a condition precedent to seeking relief in any court or tribunal in
respect of a dispute, but nothing in this Section 10.6 will prevent a Party from seeking equitable or other interlocutory relief in the courts of appropriate jurisdiction, pending the arbitrators’ determination of the merits of the
controversy, if applicable to protect the confidential information, property, or other rights of that Party or to otherwise prevent irreparable harm that may be caused by the other Party’s actual or threatened breach of this Agreement. 

10.7 No Discrimination. Licensee, its Affiliates, and any Sublicensees, in their respective activities under this Agreement, shall not discriminate
against any employee or applicant for employment because of race, color, sex, sexual, or affectional preference, age, religion, national, or ethnic origin, handicap, or because he or she is a disabled veteran or a veteran (including a veteran of the
Vietnam Era). 
 10.8 Compliance with Law. Licensee (and its Affiliates’ and any Sublicensees’) must comply with all prevailing laws,
rules, and regulations that apply to its activities or obligations under this Agreement. Without limiting the foregoing, it is understood that this Agreement may be subject to United States laws and regulations controlling the export of technical
data, computer software, laboratory prototypes, and other commodities, articles, and information, including the Arms Export Control Act as amended in the Export Administration Act of 1979 and that Licensee’s obligations are contingent upon
compliance with applicable United States export laws and regulations. The transfer of certain technical data and commodities may require a license from the cognizant agency of the United States Government and/or written assurances by Licensee that
Licensee shall not export data or commodities to certain foreign countries without prior approval of such agency. Licensor neither represents that a license is not required nor that, if required, it will issue. 

  
 31 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 10.9 Entire Agreement. This Agreement embodies the entire understanding between the Parties relating
to the subject matter hereof and supersedes all prior understandings and agreements, whether written or oral, including that certain Mutual Confidentiality Agreement effective as of December 12, 2013 between the Parties. All “Confidential
Information” disclosed by the Parties pursuant to such Mutual Confidentiality Agreement shall be deemed “Confidential Information” under this Agreement (unless and until it falls within one of the exclusions set forth in
Section 1.7). This Agreement may not be varied except by a written document signed by duly authorized representatives of both Parties. 
 10.10
Marking. Licensee, its Affiliates, and any Sublicensees shall mark any Licensed Product (or their containers or labels) made, sold, or otherwise distributed by it or them under this Agreement with any notice of patent rights necessary or (to
the extent commercially feasible and consistent with prevailing business practices) desirable under applicable law to enable the Licensed Commercial Patents to be enforced to their full extent in any country where Licensed Products are made, used,
sold, offered for sale, or imported. 
 10.11 Severability and Reformation. If any provision of this Agreement is held to be invalid or unenforceable
by a court of competent jurisdiction, then such invalid or unenforceable provision will be automatically revised to be a valid or enforceable provision that comes as close as permitted by law to the Parties’ original intent; provided that, if
the Parties cannot agree upon such valid or enforceable provision, the remaining provisions of this Agreement will remain in full force and effect, unless the invalid or unenforceable provisions are of such essential importance to this Agreement
that it is to be reasonably assumed that the Parties would not have entered into this Agreement without the invalid or unenforceable provisions. 
 10.12
Further Assurances. Each Party hereto agrees to execute, acknowledge, and deliver such further instruments, and to do all other acts, as may be necessary or appropriate in order to carry out the purposes and intent of this Agreement. 

10.13 Interpretation; Construction. The captions to the several Articles and Sections of this Agreement are included only for convenience of reference
and shall not in any way affect the construction of, or be taken into consideration in interpreting, this Agreement. In this Agreement, unless the context requires otherwise, (a) the word “including” shall be deemed to be followed by
the phrase “without limitation” or like expression; (b) references to the singular shall include the plural and vice versa; (c) references to masculine, feminine, and neuter pronouns and expressions shall be interchangeable;
(d) the words “herein” or “hereunder” relate to this Agreement; (e) “or” is disjunctive but not necessarily exclusive; (f) the word “will” shall be construed to have the same meaning and effect
as the word “shall”; (g) all references to “dollars” or “$” herein shall mean U.S. Dollars; (h) unless otherwise provided, all reference to Sections, Articles, and exhibits in this Agreement are to Sections,
Articles, and exhibits of and in this Agreement; and (i) whenever this Agreement refers to a number of days, such number shall refer to calendar days unless business days are specified. Business days shall mean a day on which banking
institutions in Washington, D.C. are open for business. Each Party represents that it has been represented by legal counsel in connection with this Agreement and acknowledges that it has participated in the drafting hereof. In interpreting and
applying the terms and provisions of this Agreement, the Parties agree that no presumption will apply against the Party which drafted such terms and provisions. 

  
 32 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 10.14 Cumulative Rights and Remedies. The rights and remedies provided in this Agreement and all other
rights and remedies available to either Party at law or in equity are, to the extent permitted by law, cumulative and not exclusive of any other right or remedy now or hereafter available at law or in equity. Neither asserting a right nor employing
a remedy shall preclude the concurrent assertion of any other right or employment of any other remedy, nor shall the failure to assert any right or remedy constitute a waiver of that right or remedy. 

10.15 Counterparts. This Agreement may be executed in one or more counterparts, each of which will be deemed an original, but all of which together
will constitute one and the same instrument. 
 [REMAINDER OF PAGE INTENTIONALLY LEFT BLANK] 

  
 33 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 IN WITNESS WHEREOF, the Parties, intending to be legally bound, have caused this License
Agreement to be executed by their duly authorized representatives. 
  

									
	REGENX BIOSCIENCES, LLC	 		 	VOYAGER THERAPEUTICS, INC.
					
	By:	 	 /s/ Kenneth T. Mills
	 		 	By:	 	 Mark Levin

	Name:	 	Kenneth T. Mills	 		 	Name:	 	Mark Levin
	Title:	 	President & CEO	 		 	Title:	 	CEO

  

 CONFIDENTIAL TREATMENT REQUESTED 

 

 Exhibit A 

Licensed Research Patents 
  

									
	 Application #
	  	 Title
	  	 Inventors
	  	 Nos.
	  	 Penn Docket

#

	 ****
	  	****	  	****	  	****	  	****
	 ****
	  	****	  	****	  	****	  	****
	 ****
	  	****	  	****	  	****	  	****
	 ****
	  	****	  	****	  	****	  	****
	 ****
	  	****	  	****	  	****	  	****
	 ****
	  	****	  	****	  	****	  	****
	 ****
	  	****	  	****	  	****	  	****
	 ****
	  	****	  	****	  	****	  	****

  
  

	****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE
COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
PORTIONS. 

 CONFIDENTIAL TREATMENT REQUESTED 

 

 Exhibit B 

Press Release 
  

			
	

	 	

 REGENX BIOSCIENCES AND VOYAGER THERAPEUTICS ANNOUNCE LICENSE AGREEMENT 

 

	 	•	 	Voyager acquires rights to REGENX’s proprietary NAV® vectors in multiple CNS disorders 

 

	 	•	 	Eighth third-party commercial license of REGENX’s NAV vectors since 2010 

  

	 	•	 	REGENX to receive undisclosed upfront payment, milestones and royalties in exchange for non-exclusive worldwide license 

WASHINGTON, DC and CAMBRIDGE, Mass. June 2, 2014 – REGENX Biosciences, LLC and Voyager Therapeutics today announced that they have
entered into a license agreement for use of REGENX’s proprietary NAV® vectors for the development and commercialization of gene therapies to treat Amyotrophic Lateral Sclerosis (ALS),
Friedreich’s ataxia (FA) and Huntington’s disease (HD). 
 Under the terms of the agreement, REGENX has granted Voyager a non-exclusive worldwide
license, as well as sublicensing rights, to REGENX’s NAV vectors for the treatment of ALS, FA and HD. In exchange for these rights, REGENX will receive an undisclosed upfront payment, ongoing fees, milestone payments, and royalties on net sales
of products incorporating NAV vectors. REGENX will also receive a share of certain sublicensing revenues. 
 “This license agreement serves as further
validation of our proprietary NAV vector technology platform, and is an important step towards the successful development of NAV-based gene delivery treatments for patients afflicted with the serious and debilitating rare diseases to which Voyager
is committed,” said Ken Mills, President and CEO of REGENX. “As the leader in next-generation AAV gene therapy, REGENX is pleased to be collaborating with Voyager, which is well-positioned to develop innovative treatments through the
application of our NAV technology.” 
 Mark Levin, Interim CEO of Voyager, commented, “Voyager is the leading AAV gene therapy company focused on
developing life-changing treatments for patients with devastating CNS disorders. We are committed to advancing the AAV gene therapy field via broad-based investment in a number of key technological areas. In addition to providing a valuable addition
to Voyager’s intellectual property portfolio, the rights to use REGENX’s NAV vectors will position us to rapidly advance the development of breakthrough CNS gene therapies.” 

About Amyotrophic Lateral Sclerosis 
 Amyotrophic
Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive, fatal neurodegenerative disease that leads to muscle weakness, loss of mobility, impaired speech, and difficulty breathing and swallowing. Most ALS patients only
live three to five years after initial 

  
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symptoms appear, and it is estimated that as many as 30, 000 patients in the United States and 450,000 worldwide are living with the disease. Familial ALS accounts for 5 to 10 percent of ALS
cases, including an estimated 20 percent of familial ALS cases caused by toxic gain of function mutations in the SOD1 gene. 
 About Friedreich’s
Ataxia 
 Friedreich’s ataxia (FA) is the most common hereditary ataxia, with approximately 8,000 patients living with the disease in the United
States and Europe. FA patients have a genetic mutation in the FXN gene, which limits the production of the protein frataxin, causing a variety of debilitating symptoms and complications, loss of coordination and balance, muscle weakness, impaired
vision, hearing and speech, scollosis, diabetes and cardiomyopathy. 
 About Huntington’s Disease 

Huntington’s disease (HD) is an inherited neurodegenerative disorder where symptoms typically become noticeable between 30 and 50 years of age. HD is
caused by a genetic mutation in the huntingtin gene, which leads to the production of a mutated huntingtin protein, resulting in symptoms such as chorea, rigidity, abnormal posturing, cognitive impairment and psychiatric symptoms, and difficulty
with speech and swallowing. It is estimated that 1 in every 10,000 Americans has HD and more than 250,000 others are at-risk of having inherited the HD genetic mutation. 

About REGENX Biosciences 
 ReGenX Biosciences is
the leading next-generation AAV gene therapy company, developing a new class of personalized therapies based on its proprietary NAV® vector technology platform for a range of severe diseases
with serious unmet needs. NAV vector technology includes novel AAV vectors rAAV7, rAAV8, rAAV9 and rAAVrh10. The company’s treatments in development include programs addressing lysosomal storage disorders and ocular diseases. ReGenX’s
leadership in AAV gene therapy and corresponding intellectual property has enabled it to establish collaborations with leading global partners including Baxter Healthcare, Fondazione Telethon, Audentes Therapeutics, Lysogene, Esteve, AveXis and
AAVLife. In addition, together with Fidelity Biosciences, ReGenX formed Dimension Therapeutics, a company focused on the development and commercialization of AAV gene therapies for rare diseases. 

For more information regarding ReGenX, please visit www.regenxbio.com. 

About Voyager Therapeutics 
 Voyager Therapeutics
is a gene therapy company developing life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS). Voyager is committed to advancing the field of AAV (adeno-associated virus) gene therapy through innovation and
investment in vector optimization and engineering, dosing techniques, as well as process development and production. The company’s initial pipeline is focused on CNS diseases in dire need of effective new therapies,

  
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including Parkinson’s disease, a monogenic form of amyotrophic lateral sclerosis (ALS), and Friedreich’s ataxia. Founded by scientific and clinical leaders in the fields of AAV gene
therapy, expressed RNA interference and neuroscience, Voyager Therapeutics was launched in 2014 with funding from leading life sciences investor Third Rock Ventures and is headquartered in Cambridge, Mass. For more information, please visit
www.voyagertherapeutics.com. 
 ### 

Contact: 
 REGENX Biosciences 

Vit Vasista, 202-785-7438 
 vvasista@regenxbio.com 

REGENX Biosciences (Media) 
 Annie Starr, 973-415-8838 

astarr@6degreespr.com 
 Voyager Therapeutics (Media) 

Katie Wilson Engleman 
 Pure Communications, Inc. 

910-509-3977 

  
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