EX-10.16



 Exhibit 10.16 

CONFIDENTIAL TREATMENT REQUESTED 

LICENSE AGREEMENT 
 This LICENSE AGREEMENT
(“Agreement”) is entered into as of March 21, 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 AveXis, Inc. (formerly known as BioLife Cell Bank, Inc.), a corporation organized under the laws of the State of Delaware, with offices at 4925 Greenville Avenue,
Suite 604, Dallas, TX 75206 (“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 9; 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
“AAV9” means (a) the recombinant adeno-associated virus serotype 9 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype 9 vector that are
covered by the claims of the Licensed Patents. 
 1.2 “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. 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. 
 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 
  
 ****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 

 
 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, 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 spinal muscular atrophy in humans by in
vivo gene therapy using AAV9. 
 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, to the extent they cover AAV9, (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 AAV9 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 granted 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 import, including products manufactured 
  

****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 

 
 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 sold by Licensee, its Affiliates, and any of its or their Sublicensees with respect to the administration of any AAV9 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, inter partes review, and interferences. 

  
 3 

 ****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 

 
 1.17 “Receiving Party” has the meaning set forth in Section 5.1.

 1.18 “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). 
 1.19 “Retained Rights” has the meaning set forth in
Section 2.2. 
 1.20 “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.

 1.21 “Third Party” means any person or entity other than a Party to this Agreement or Affiliates of a Party to this Agreement. 

1.22 “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, 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 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 outside of the Field. Without limiting the foregoing, and
notwithstanding anything in this Agreement to the contrary, Licensee acknowledges and agrees that the following rights are retained by Licensor and the ReGenX 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 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
AAV9. 

  
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 CONFIDENTIAL TREATMENT REQUESTED 

 
 2.2.2 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 AAV9; and 

  

	 	(b)	A non-exclusive right for the ReGenX Licensors (which right is sublicensable by the ReGenX Licensors) to use the Licensed Patents for non-commercial research purposes and to use the Licensed Patents for such ReGenX
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 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. 
 2.3 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. 

  
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 CONFIDENTIAL TREATMENT REQUESTED 

 
 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 only grant sublicenses pursuant to a written sublicense agreement with the Sublicensee; ****. Licensor must receive written notice as soon as practicable following execution of any such sublicenses. Any
further sublicenses granted by any Sublicensees (to the extent permitted hereunder) must comply with the provisions of this Section 2.4 (including Section 2.4.2) to the same extent as if Licensee granted such sublicense directly.


  

	 	(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 the ReGenX 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 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. 

  

	 	(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: 

  
 6 

 ****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 

 
  

	 	(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 AAV9, 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 2.5.1(b), to practice the Licensed Back Improvements in the Field. 


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. 

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 $2,000,000, which shall be payable as follows: (i)**** upon the Effective Date, (ii) **** within **** after the
Effective Date, and (iii) **** within **** after the Effective Date; provided that any unpaid portion of the initial fee will be immediately payable upon any termination of this Agreement. 

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 of the **** human subject in a clinical trial (i.e., **** patient, first dose)	  	 	****	  
	2. First treatment in Phase 3 Clinical Trial (i.e., first patient, first dose)	  	 	****	  
	3. First NDA submission for a Licensed Product in the United States	  	 	****	  
	4. First NDA submission for a Licensed Product in the European Union	  	 	****	  
	5. First NDA approval for a Licensed Product in the United States	  	 	****	  


  
 7 

 ****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 

 

					
	6. First NDA approval for a Licensed Product in the European Union	  	 	****	  
		  	  
	  
	 
	 Total:
	  	$	12,250,000.00	  
		  	  
	  
	 

 For clarity, the milestone payments set forth in this Section 3.3 are payable **** with respect to each milestone event,
****. 
 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, subject to the reductions in royalty rates set forth in Section 3.4.1: 
  

					
	 Cumulative Annual Net Sales of all Licensed Products Worldwide
	  	Royalty
Percentage	 
	Portion of Net Sales in a calendar year less than ****	  	 	****	  
	Portion of Net Sales in a calendar year between (and including) **** through (and including) ****	  	 	****	  
	Portion of Net Sales in a calendar year greater than ****	  	 	****	  

 By way of example only, if Licensee receives $700,000,000 in cumulative Net Sales of all Licensed Products in
a calendar year, then the royalties payable by Licensee to Licensor under this Section 3.4 during such calendar year would be calculated as follows: 

= (****)(****) + (****)(****) + (****)(****) 

= (****) + (****) + (****) 
 =
**** 
 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, 

  
 8 

 ****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 

 
 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 = **** – **** = **** (but subject to the cap described below)

 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 the Licensed Product ceases to
infringe or be covered by a Valid Claim in that country. 
 3.5 Sublicense Fees. 

3.5.1 In further consideration of the license granted to Licensee under Section 2.1, Licensee will pay Licensor **** of any sublicense
fees (****) received by Licensee or its Affiliates from a Third Party for the Licensed Patents from any Sublicensee or from any Third Party granted any option to obtain a sublicense. 

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 or its Affiliates corresponding directly to the development of Licensed Products pursuant to a specific agreement;


  

	 	(b)	Any and all amounts paid to Licensee or its Affiliates by a Sublicensee as royalties on sales of Licensed Product sold by the Sublicensee under a sublicense agreement; and 


 

	 	(c)	Consideration received for the purchase of an equity interest in Licensee or its Affiliates at fair market value. 


  
 9 

 ****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 

 
 3.5.3 If Licensee or its Affiliates 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 3.5 (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 that is not an arm’s length transaction, fees due under this Section 3.5 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. 

3.5.4 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 3.3, then the amount of the payment 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. 

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: 

 

	 	(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; 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. 

  
 10 

 ****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 

 
 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. 

3.6.4 All financial reports under this Section 3.6 will be certified by the chief financial officer of Licensee or Licensee’s
qualified financial representative. 
 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 the ReGenX 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 five years 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. If the review or audit determines that Licensee has overpaid any payment, then Licensor shall refund the overpayment to
Licensee. 
 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 

  
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 ****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 

 
 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 ****. 

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; 

  
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 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 2.5.3. 
 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) protect the Confidential Information of the Disclosing Party with at least the same degree of care as it protects its own
confidential and proprietary information, and in any event with not less than a reasonable degree of care; (c) 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 (d) 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. 

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 

  
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 CONFIDENTIAL TREATMENT REQUESTED 

 
 related to Licensee’s Licensed Product or AAV9 program that have been publicly
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 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 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 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 six months’ 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 payment in full of the initial fee
under Section 3.1, such termination notice shall be accompanied by Licensee’s payment of all unpaid amounts 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 

  
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 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 any Trigger Event. In addition, if the
Sublicensee’s experiencing of a Trigger Event gives a ReGenX Licensor a right of termination under the Penn Agreement or GSK Agreement, 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, any of
its Affiliates, or any Sublicensee of a Patent Challenge. 

  
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 6.5.2 For purposes of this Section 6.5, “Patent
Challenge” means any action against Licensor or the ReGenX Licensors, including an action for declaratory judgment, to declare or render invalid or unenforceable the Licensed Patents, or any claim thereof. 

6.6 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: 
 6.6.1 The licenses granted by Licensor hereunder shall terminate,
and Licensee, its Affiliates, and (unless the sublicense agreement is assigned pursuant to Section 6.6.2) all Sublicensees 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 shall have the right to continue to sell its existing inventories of Licensed Products for a period not to exceed **** after the effective date of such termination; 

6.6.2 If termination is by Licensor pursuant to Section 6.3, 6.4, or 6.5, then, at Licensor’s request, 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; 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; and all sublicenses not requested to be assigned to Licensor shall terminate. If termination is for any other reason, then all sublicenses shall terminate; 

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 

6.6.5 Each Receiving Party shall, at the Disclosing 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 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), Article 3 (Consideration) (with respect to any final reports or to the extent any amounts are due but unpaid), Section 3.6 (Reports 

  
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 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 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 Trustees of the University of Pennsylvania control 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 the ReGenX Licensors set forth in the GSK Agreement and Penn Agreement 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 the GSK Agreement and the Penn
Agreement. 
 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. However, Licensee is under no obligation to search for potential infringers. 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 any
such Licensed Patent and such Licensed Patent 

  
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 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 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 The Party not controlling the action under this Section 7.2 shall be entitled to independent counsel in such proceedings but at its
own expense, not subject to reimbursement by the other Party and not subject to any offset against any damages received by the Party bringing suit under Section 7.2.5. The controlling Party shall keep the cooperating party reasonably informed
of the progress of the action proceedings. 
 7.2.5 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 the ReGenX Licensors set
forth in the GSK Agreement and the Penn Agreement, as follows: (a) 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
(b) the balance remaining, if any, from any such recovery shall be ****. 
 7.2.6 Licensee acknowledges and agrees that (a) the
rights and obligations under this Section 7.2 are subject to the rights of the ReGenX Licensors under the GSK Agreement and Penn Agreement (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 the GSK Agreement and the Penn Agreement. Furthermore, Licensee
acknowledges the following: 
 7.2.6.1 All monies recovered upon the final judgment or settlement of any action with respect
to Competitive Infringement will also need to be allocated to the ReGenX 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. 

7.2.6.2 The ReGenX 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. 
 7.2.6.3 In any infringement prosecuted by the ReGenX
Licensors, all financial recoveries will be ****. 

  
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 7.2.6.4 In any infringement prosecuted by the ReGenX
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). 
 7.2.6.5 The written consent of the ReGenX Licensors will be required (a) for any decision that
would have a materially adverse affect on the validity, 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 either
of the ReGenX 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 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, which shall not be unreasonably denied, Licensee must not settle or compromise any such suit in a manner that imposes any material obligations or restrictions on Licensor or either of
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: 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 rights 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 Patents that would be inconsistent with the rights granted to Licensee under this Agreement; 

8.1.4 To Licensor’s knowledge, (a) the Licensed 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 Patents in the Field that are inconsistent with those granted to Licensee in the Field under this Agreement; and 

  
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 CONFIDENTIAL TREATMENT REQUESTED 

 
 8.1.5 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. 
 8.2 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 rights 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. 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 REPRESENTATIONS OR WARRANTIES, AND HEREBY
DISCLAIMS ALL EXPRESS AND IMPLIED REPRESENTATIONS AND WARRANTIES, (i) OF COMMERCIAL UTILITY, ACCURACY, COMPLETENESS, PERFORMANCE, TITLE, 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 AND THE REGENX
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, EXEMPLARY, 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. 

  
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 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, 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 **** by Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors; 


 

	 	(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 Indemnification Procedure. Licensee, 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 Licensor, the ReGenX Licensors, or any indemnified party (an “Indemnified
Party”) without Licensor’s prior written consent or that 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 

  
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 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 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 
 9.1
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 

  
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 ****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 

 
 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 5.2 or 5.3, as applicable. 
 9.2 Licensor and all of its employees
and agents must not use Licensee’s name, seal, logo, trademark, or service mark (or any adaptation thereof) in any way without the prior written consent of Licensee; provided, however that Licensor may acknowledge the existence and general
nature of this Agreement, subject to Section 5.2 or 5.3, as applicable, and refer to Licensee as a licensee of Licensor. 
 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 without the prior written consent of Licensor. Such prohibition
on assignment of this Agreement shall apply even with respect to a sale or merger of Licensee, the transfer of substantially all of Licensee’s business assets, or the sale of a majority of the capital stock of Licensee. 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
	 	 ReGenX Biosciences, LLC

	 750 17th Street, NW
	 	 750 17th Street, NW

	 Suite 1100
	 	 Suite 1100

	 Washington, DC 20006
	 	 Washington, DC 20006

	 USA
	 	 USA

	 Attn: Chief Executive Officer
	 	 Attn: General Counsel

	 Telephone: 202-785-7438
	 	 Telephone: 202-785-7438



  
 23 








 CONFIDENTIAL TREATMENT REQUESTED 

 
  

			
	 Facsimile: 202-785-7439
	 	 Facsimile: 202-785-7439

		
	 If for Licensee:
	 	
		
	 AveXis, Inc.
	 	
	 4925 Greenville Avenue, Suite 604
	 	
	 Dallas, TX 75206
	 	
	 Attn: Chief Executive Officer
	 	
	 Telephone: 972-725-7797
	 	
	 Facsimile: 516-619-0412
	 	

 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 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 New York 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. 

  
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 ****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 

 
 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, 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 

  
 25 








 CONFIDENTIAL TREATMENT REQUESTED 

 
 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, dated February 6, 2014, between Licensor and Licensee and that certain Mutual
Non-Disclosure Agreement, dated March 29, 2013, between Licensor and Licensee (who was then known as BioLife Cell Bank, Inc.). All “Confidential Information” disclosed by the Parties pursuant to such Confidential 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 to 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. 
 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


  
 26 








 CONFIDENTIAL TREATMENT REQUESTED 

 
 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. 
 [REMAINDER OF PAGE INTENTIONALLY LEFT BLANK] 

  
 27 








 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
	  	AVEXIS, INC.
				
	 By:
	  	 /s/ Kenneth Mills
	  	By:	  	 /s/ John A. Carbona

				
	 Name:
	  	 Kenneth Mills
	  	 Name:
	  	 John A. Carbona

				
	 Title:
	  	 President & CEO
	  	Title:	  	 CEO


  
 ****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 A 

Licensed Patents 
  

									
	 Application #
	  	 Patent #
	  	 Filing Date
	  	 Country
	  	 Status

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

  
 ****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 SUBJECT TO FINAL REVIEW AND APPROVAL 
  

REGENX BIOSCIENCES AND AVEXIS ENTER INTO LICENSE AGREEMENT FOR DEVELOPMENT OF TREATMENTS FOR SPINAL MUSCULAR ATROPHY USING NAV® rAAV9 VECTORS 
 Washington, DC and Dallas, TX — REGENX Biosciences, LLC (REGENX) and AveXis,
Inc. (AveXis) announce that they have entered into an agreement for the development and commercialization of products to treat Spinal Muscular Atrophy (SMA) using NAV rAAV9 vectors. 

Under the terms of the agreement, REGENX granted AveXis an exclusive worldwide license, with rights to sublicense, to REG ENX’s NAV rAAV9 vector for
treatment of SMA disease in humans. In return for these rights, REGENX receives an up-front payment, certain milestone fees and royalties on net sales of products incorporating NAV rAAV9. 

“We believe this exclusive license agreement is important to the successful development of NAV-based gene delivery treatments for patients with
SMA,” said Ken Mills, President and CEO of REGENX. “As a leader in gene therapy, we are pleased to be formally collaborating with AveXis which has assembled a world class team of scientific and clinical experts in SMA, led by Brian Kaspar,
Ph.D. and his colleagues at Nationwide Children’s Hospital and The Ohio State University, who have demonstrated tremendous dedication to the development of innovative gene therapy treatments for patients with SMA.” 

“AveXis is committed to the development of new treatments for patients with SMA using NAV-vector technology and we feel rAAV9 is the most promising
vector to achieve this goal, something we like to call our ‘special snowflake’. We believe the unique properties of rAAV9 will allow us to effectively develop novel treatments, and is at the center of research being done at the Kaspar
Laboratory in Columbus, Ohio,” said John A. Carbona, CEO of AveXis. “Everyone associated with our SMA program is very pleased to establish this agreement withREGENX, which provides an important foundation for our team to continue to
develop novel therapies for patients with all types of SMA.” 







 CONFIDENTIAL TREATMENT REQUESTED 

DRAFT SUBJECT TO FINAL REVIEW AND APPROVAL 
  

About Spinal Muscular Atrophy 
 Spinal muscular atrophy
(SMA) is an autosomal-recessive genetic disorder characterized by progressive weakness of the lower motor neurons. SMA is caused by a genetic defect in the SMN1 gene which codes SMN, a protein necessary for survival of motor neurons. SMA kills more
infants than any other genetic disease in today’s world. 
 About REGENX Biosciences 

REGENX Biosciences (www.regenxbio.com) 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’s leadership in AAV gene therapy and corresponding intellectual property has enabled it to establish collaborations with leading global partners including Chatham
Therapeutics, Fondazione Telethon, Audentes Therapeutics, Lysogene, and Esteve. 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. For more information regarding REGENX, please visit www.regenxbio.com. 
 About AveXis 

Based in Dallas, Texas, AveXis is a clinic-ready synthetic biology platform company establishing unique industry alliances to create innovative treatments for
people with unmet medical needs. Spinal muscular atrophy is the company’s first focus. 
 For more information regarding AveXis, please visit
www.avexisinc.com. 
 Contacts: 
 REGENX Biosciences

 Vit Vasista, 202-785-7438 

vvasista@regenxbio.com 







 CONFIDENTIAL TREATMENT REQUESTED 

DRAFT SUBJECT TO FINAL REVIEW AND APPROVAL 
  

AveXis 
 Corporate Contact: 

John A. Carbona, Chief Executive Officer 

972-725-7797 or jcPavexisinc.com 
 Media
Contact: 
 Jillian Bowman, Administrative Specialist 

972-725-7797 or iillianb@avexisinc.com