Document:

EX-10.9

					
		 	 Confidential Materials omitted and filed separately with the

Securities and Exchange Commission. Double asterisks denote omissions.
	 	Exhibit 10.9

 LICENSE AGREEMENT 

between 
 AAVENUE
THERAPEUTICS 
 and 

THE CHILDREN’S HOSPITAL OF PHILADELPHIA® 

  
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 This License Agreement (the “Agreement”) is entered into this 14th day of October, 2013 (the “Effective Date”), by and between AAVenue Therapeutics, LLC, a limited liability company organized and existing under the laws of Delaware and having a principal
place of business at 34th and Civic Center Blvd, Philadelphia, PA 19104 (“Company”), and The Children’s Hospital of
Philadelphia®, a non-profit entity organized and existing under the laws of Pennsylvania and having a principal place of business at 34th
and Civic Center Boulevard, Philadelphia, PA 19104 (“CHOP”). Each of Company and CHOP may be referred to as a “Party” and both Company and Licensor may be collectively referred to as the “Parties”. 

CHOP and Company agree as follows: 
  

	1.	BACKGROUND 

  

	 	1.1	In the course of conducting biomedical and behavioral research, CHOP investigators made inventions that may have commercial applicability, related to the gene therapy treatments and vector manufacturing.

  

	 	1.2	By assignment of rights from CHOP employees and/or other inventors, CHOP owns intellectual property rights in any United States and foreign patent applications or patents corresponding to the assigned inventions. CHOP
also owns any tangible embodiments of these inventions actually reduced to practice by CHOP investigators. 

  

	 	1.3	CHOP desires to transfer these inventions to the private sector through commercialization licenses to facilitate the commercial development of products and services based on such intellectual property for public use and
benefit. 

  

	 	1.4	Company desires to exclusively acquire and license commercialization rights to certain of these inventions in order to develop services, methods, or marketable products for public sale, use, and benefit.

  

	 	1.5	Pursuant to a Technology Assignment Agreement dated as of October     , 2013, by and between Company and CHOP (the “Assignment Agreement”), which is included in Appendix C, CHOP has also
assigned to Company ownership of certain items and materials as set forth in the Assignment Agreement (as defined below). 

  

	2.	DEFINITIONS 

  

	 	2.1	“Affiliate(s)” shall mean any corporation, firm, partnership or other entity, which controls, is controlled by, or is under common control with, a Party. For purposes of this Paragraph 2.1, “control”
shall mean direct or indirect ownership of fifty percent (50%) or more of the outstanding stock or other voting rights entitled to elect directors thereof or the ability to otherwise control the management of such corporation, firm, partnership
or other entity. Notwithstanding the foregoing, for purposes of this Agreement, Company and CHOP shall be deemed not to be Affiliates of one another. 

  

	 	2.2	“Assigned Intellectual Property” means the certain items and materials assigned by CHOP to Company pursuant to the Assignment Agreement. 

 

	 	2.3	“CHOP Intellectual Property” means the Patent Rights, Gene Therapy Know-How, and Know-How. 

  
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	 	2.4	“Development Plan” means the written commercialization plan to be attached as Appendix B. 

  

	 	2.5	“First Commercial Sale” means, following regulatory approval of a Licensed Product, the initial transfer by or on behalf of Company or its Affiliates or sublicensees of such Licensed Product in exchange for
cash or some equivalent to which value can be assigned, excluding: (i) a transfers to a third party for purposes of clinical trials or other testing, or (ii) the use of Licensed Product by Company or any of its Affiliates or sublicensees
for research and development purposes. 

  

	 	2.6	“Gene Therapy Know-How” means technical information, know-how, process, procedure, composition, method, formula, protocol, technique, or data, other than Assigned Intellectual Property, owned or controlled by
CHOP and existing on the Effective Date, directly related only to the Assigned Intellectual Property or directly related only to adeno-associated viral vectors or CHOP’s lentiviral vector manufacturing capabilities. 

 

	 	2.7	“Know-How” means all rights, other than: (a) Patent Rights, (b) any patents and patent applications filed or to be filed by CHOP that are not included in the Patent Rights,(c) Gene Therapy Know-How
and (d) Assigned Intellectual Property, in any technical information, know-how, process, procedure, composition, method, formula, protocol, technique, or data, which rights are owned or controlled by CHOP and existing on the Effective Date and
which are necessary and/or useful for making, using, offering for sale, selling or importing Licensed Products. 

  

	 	2.8	“Licensed Field” means the field of gene therapy. 

  

	 	2.9	“Licensed Products” means any apparatus, device, system, product, article, appliance, method, process or other subject matter, covered in whole or in part by a pending claim in a pending application within the
Patent Rights or an unexpired claim in a patent within the Patent Rights, or described or enabled by the Gene Therapy Know-How. 

  

	 	2.10	“Licensed Territory” means worldwide. 

  

	 	2.11	“Net Sales” means the total gross receipts actually received for sales, including transfers of Licensed Products to others in a commercial transaction or making Licensed Products available to others in a
commercial transaction, whether invoiced or not, of Licensed Products by or on behalf of Company or its Affiliates or sublicensees less: 

  

	 	(a)	trade, quantity and cash discounts; 

  

	 	(b)	taxes, tariffs, customs duties, excises and other duties and governmental charges (other than taxes on income) levied on the sale, transportation or delivery of Licensed Products, if listed separately on invoices and
actually paid by Company or its Affiliates or sublicensees; 

  

	 	(c)	credits, chargebacks, retroactive price reductions, rebates and returns actually paid or allowed by Company or its Affiliates or sublicensees; and 

 

	 	(d)	transportation, insurance, packaging and postage charges, solely for shipment to a purchaser of a Licensed Product from Company or its Affiliates or sublicensees, if listed separately on invoices for payment by the
purchaser. 

  
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 Any discretionary rebates, discounts, adjustments or similar payments shall be commercially
reasonable and consistent with the normal course commercial practices of Company and its Affiliates and sublicensees. 
 No deductions shall
be made for commissions paid to any third party or individual sales representative, whether they be with independent sales agencies or regularly employed by Company, its Affiliates or sublicensees, and on its payroll, or for the cost of collections.

 Sales or transfers of Licensed Products among Company and its Affiliates and sublicensees for the purpose of subsequent resale to third
parties shall not be included in Net Sales. 
 Notwithstanding the foregoing, in the event a Licensed Products is sold as a Combination
Product, Net Sales shall be calculated by multiplying the Net Sales of the Combination Product by the fraction A/(A+B), where A is the gross invoice price of the Licensed Product if sold separately in a country and B is the gross invoice price of
the other product(s) included in the Combination Product if sold separately in such country. If no such separate sales are made by Company, its Affiliates or sublicensees in a country, Net Sales of the Combination Product shall be calculated in a
manner to be negotiated and agreed upon by the parties, reasonably and in good faith, which shall be based upon the relative value of the active components of such Combination Product. 

As used in this definition, “Combination Products” means any product that comprises a Licensed Products sold in conjunction with
another active component that is not a Licensed Product (whether packaged together or in the same formulation). 
  

	 	2.12	“Patent Rights” means (a) those issued patents and/or patent applications listed on Appendix A attached to this Agreement, (b) any patents or patent applications other than those described in the
foregoing clause (a) that claim inventions relating to Gene Therapy Know-How or Assigned Intellectual Property and were generated, conceived or otherwise made by Drs. Kathy High and Fraser Wright, and those working under their direct
supervision and directly engaged in CHOP gene therapy research, prior to the Effective Date, and (c) any patents issuing from such pending patent applications, or any related applications or patents claiming priority to any of such patent
applications or patents, as well as any continuations, divisionsals, reexaminations, reissues, substitutes, renewals or extensions of any of the foregoing patent applications or patents and counterparts of any of the foregoing in any country of the
world. Notwithstanding the foregoing, Company may elect within the first [**] days after the Effective Date to exclude any issued patents and/or patent applications from the Patent Rights by giving written notice to CHOP of election; provided that
such patents and/or patent application so removed shall be deemed to be removed from this Agreement ab initio and never to have been part of this Agreement. 

 

	3.	GRANT OF RIGHTS 

  

	 	3.1	 CHOP hereby grants and Company accepts, subject to the terms and conditions of this Agreement, a worldwide exclusive license in the Licensed Field,
with the right to sublicense, to use and practice the Patent Rights and Gene Therapy Know-How, and (ii) a worldwide non-exclusive license in the Licensed Field, with the right to sublicense, to use and practice the Know-How, in the case of both
(i) and (ii) to research, develop, make, have made, practice, use, import, lease, offer for sale, sell, and sublicense the Licensed Products within the Licensed Field (the “License”), subject, however, (a) to a reservation
of rights by CHOP 

  
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to research, make, have made, practice, have practiced, and use the Know-How for any purpose with no exclusions or exceptions and the CHOP Patent Rights and Gene Therapy Know-How solely for its
own academic and clinical research, and/or educational purposes excluding (I) use pursuant to any sponsored research or other funding agreement or arrangement with any commercial entity pursuant to which any commercial entity is granted any
right or interest with respect to the Patent Rights and Gene Therapy Know-How or research results generated through the use thereof, (II) the inclusion in any NDA, BLA or other application for marketing approval of any data comprised by the Patent
Rights and Gene Therapy Know-How and (III) any use of the Patent Rights and Gene Therapy Know-how for commercialization or licensing or transfer of rights therein for commercialization and(b) to any applicable reservation of rights by the U.S.
government. 

  

	 	3.2	This Agreement confers no license or rights by implication, estoppel, or otherwise under any patent applications or patents of CHOP other than Patent Rights regardless of whether such patents are dominant or subordinate
to the Patent Rights. 

  

	4.	SUBLICENSING 

  

	 	4.1	Company may enter into sublicensing agreements under the CHOP Intellectual Property, provided that Company shall notify CHOP of any such sublicensing agreements in which commercialization rights to Licensed Products are
granted. 

  

	 	4.2	Company agrees that any sublicenses granted by it shall provide that the obligations to CHOP of Paragraphs 4.4, 5.1-5.2, 7.1, 8.1, 10.1, 10.2, 10.3, 11.3-11.5, 12.5 and 12.6 of this Agreement shall be binding upon the
sublicensee as if it were a party to this Agreement. 

  

	 	4.3	Company’s execution of a sublicense agreement will not relieve Company of any of its obligations under this Agreement. Company is primarily liable to CHOP for any act or omission of an Affiliate or sublicensee of
Company that would be a breach of this Agreement if performed or omitted by Company, and Company will be deemed to be in breach of this Agreement as a result of such act or omission. 

 

	 	4.4	In the event this Agreement terminates, and such termination is not the result of any failure by a sublicensee to comply with the terms of this Agreement applicable to sublicensees, such sublicensee’s sublicense
shall survive such termination in respect of the sublicensee’s exercise of such sublicense rights provided, however, that CHOP shall not be obligated in any manner to perform any obligations of Company under the sublicense agreement beyond the
granting of rights to the sublicensee with respect to the Patent Rights and Gene Therapy Know-How. 

  

	 	4.5	Company agrees to forward to CHOP a copy of each fully executed sublicense agreement postmarked within [**] days of the execution of such agreement. To the extent permitted by law, CHOP agrees to maintain each such
sublicense agreement in confidence. 

  

	5.	STATUTORY AND CHOP REQUIREMENTS AND RESERVED GOVERNMENT RIGHTS 

  

	 	5.1	CHOP retains the ability to research, make, have made, practice, have practiced, and use CHOP Intellectual Property solely for its own research and/or educational purposes. In addition, all rights granted in this
Agreement are expressly granted subject to the rights of the U.S. Government pursuant to 35 U.S.C. Sections 200 et seq., as amended, (Patent Rights in Inventions Made with Federal Assistance) and the implementing regulations.

  
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	 	5.2	Company agrees that if any Patent Rights claiming inventions were supported by funding from a U.S. government agency, products used or sold in the United States embodying such Patent Rights shall be manufactured
substantially in the United States, unless a written waiver is obtained in advance from the appropriate government agency. 

  

	6.	CONSIDERATION 

  

	 	6.1	EQUITY 

 In partial consideration for CHOP entering into this Agreement, the Company is
issuing to CHOP [**] common share membership interests under the Common Share Membership Agreement of even date herewith, by and between Company and CHOP, as attached in Appendix C. 

 

	 	6.2	MILESTONES 

 Company shall pay to CHOP the applicable milestone payments listed in the
table below within [**] days after the first achievement of each milestone event for each category of Licensed Product by Company, its Affiliates, or sublicensees set forth below: 

 

									
	 	  	[**]	 	Hemophilia B	 	Hemophilia A	 	[**]
	 First patient treated in a US Phase III (or foreign equivalent) clinical trial for a Licensed Product
	  	[**]	 	$125k	 	[**]	 	[**]
	 First Commercial Sale of a Licensed Product in any country
	  	[**]	 	[**]	 	[**]	 	$5M

 For the avoidance of doubt, there will be no milestone payments for Licensed Products for [**]. 

 

	 	6.3	ROYALTY On a Licensed Product-by-Licensed Product and country-by-country basis, Company shall pay to CHOP, on a calendar quarterly basis, a royalty on Net Sales of each Licensed Product for an indication listed
within the immediately following table below at the rate specified in such table for such category of Licensed Product, during any period when the manufacture, use, offer for sale, sale or importation of such Licensed Product in the applicable
country would infringe any Patent Rights in the absence of the license granted to Company hereunder. 

  

									
	 	  	[**]	 	Hemophilia B	 	Hemophilia A	 	[**]
	 Patent Royalty
	  	[**]	 	[**]	 	[**]	 	[**]

 On a Licensed Product-by-Licensed Product and country-by-country basis, Company shall pay to CHOP, on a
calendar quarterly basis, a royalty on Net Sales of each Licensed Product that is not subject to royalties pursuant to the table above, at the rate specified in the table below for the Indication Prevalence Category applicable to such Licensed
Product, during any period when the manufacture, use, offer for sale, sale or importation of such Licensed Product in the applicable country would infringe any Patent Rights in the absence of the license granted to Company hereunder. 

  
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	 	  	INDICATION PREVALENCE CATEGORY
	  	COMMON	 	RARE	 	ULTRA-RARE
	 Patent Royalty
	  	[**]	 	[**]	 	[**]

 “Common” is defined as prevalence greater than 1 in 1,500. “Rare” is a prevalence less
than or equal to 1 in 1,500 but greater than 1 in 50,000. “Ultra-Rare” is a prevalence less than or equal to 1 in 50,000. 
 For
the avoidance of doubt, the royalty for Licensed Products directed to [**] will be [**]% 
 For the avoidance of doubt, the royalty for any
Licensed Product covered exclusively by Know-How will be [**]%. 
 For the further avoidance of doubt, no more than one of the foregoing
royalties shall be applicable to any given Licensed Product (i.e., under no circumstance shall the applicable royalty be calculated by applying two or more of the foregoing royalty rates to the same Licensed Product). 

Also, for the avoidance of doubt, Company shall pay to CHOP the royalties according to this Agreement on the Net Sales of the Licensed
Products made by sublicensee(s) or Affiliates as if such sales were Net Sales of Licensed Products by Company. 
  

	 	6.4	A claim of a patent or patent application licensed under this Agreement shall cease to fall within the Patent Rights for the purpose of computing the royalty payments in any given country on the earliest of the dates
that a) the claim has been abandoned but not continued, b) the patent expires or irrevocably lapses, or c) the claim has been held to be invalid or unenforceable by an unappealed or unappealable final decision of a court of competent jurisdiction or
administrative agency. 

  

	 	6.5	No multiple royalties shall be payable because any Licensed Products are covered by more than one claim, patent, or patent application of the Patent Rights. 

 

	 	6.6	On sales of Licensed Products by Company, its Affiliates or sublicensees (other than such sales made between or among Company, its Affiliates and sublicensees) made other than in an arm’s-length transaction, the
value of the Net Sales attributed under this Paragraph to such a transaction shall be that which would have been received in an arm’s-length transaction, based on sales of like quantity and quality products on or about the time of such
transaction. The following shall not be included in Net Sales, however: (i) a transfers to a third party for purposes of clinical trials or other testing, or (ii) the use of Licensed Product by Company or any of its Affiliates or
sublicensees for research and development purposes. 

  

	7.	RECORD KEEPING 

  

	 	7.1	 Company agrees to keep, and to require its Affiliates and sublicensees to keep, accurate and correct records of Licensed Products under this Agreement
appropriate to determine 

  
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the amount of royalties and payments due CHOP. Such records shall be retained for at least [**] years following a given reporting period. The records shall be made available, [**], at the request
of CHOP during normal business hours for inspection at the expense of CHOP by an accountant or other designated auditor selected by CHOP (and acceptable to Company) for the sole purpose of verifying reports and payments hereunder Company may only
object to an auditor selected by CHOP for good cause shown. If an inspection shows an underreporting or underpayment in excess of [**] percent ([**]%) for any twelve (12) month period, then Company shall reimburse CHOP for the cost of the
inspection at the time Company pays the unreported royalties, including any late charges as required by this Agreement. All payments required under this Paragraph shall be due within [**] days of the date CHOP provides Company notice of the payment
due. Late charges will be assessed by CHOP on any undisputed overdue payments at a rate of [**] percent ([**]%) per month. The payment of such late charges shall not prevent CHOP from exercising any other rights it may have as a consequence of the
lateness of any payment. 

  

	 	7.2	Company shall report to CHOP the date of the First Commercial Sale in each country in the Licensed Territory within [**] days of such occurrence. 

 

	 	7.3	Company shall submit to CHOP within [**] days after each calendar quarter ending March 31, June 30, September 30, and December 31 a royalty report setting forth for the preceding quarterly
period the amount of the Licensed Products sold by or on behalf of Company or by an Affiliate or a sublicensee in each country within the Licensed Territory the Net Sales, and the amount of royalty or other payment accordingly due. With each such
royalty report, Company shall submit payment of the earned royalties due. If no earned royalties are due to CHOP for any reporting period, the written report shall so state. The royalty report shall be certified as correct by an authorized officer
of Company and shall include a detailed listing of all deductions made under Paragraph 2.11 to determine Net Sales made under Article 6 to determine royalties due. 

 

	 	7.4	Company agrees to forward to CHOP a copy of quarterly royalty reports received by Company from its Affiliates and sublicensees during the preceding quarterly period as shall be pertinent to a royalty accounting to CHOP
by Company for activities under the sublicense. 

  

	 	7.5	Royalties and Milestones due under Articles 6 and 7 shall be paid in U.S. dollars. For conversion of foreign currency to U.S. dollars, the conversion rate shall be the New York stock exchange rate quoted in The Wall
Street Journal on the day that the payment is due. Any loss of exchange value taxes, or other expenses incurred in the transfer or conversion to U.S. dollars shall be paid entirely by Company. The royalty report required by paragraph 7.3 of this
Agreement shall accompany each such payment and a copy of such report shall also, be mailed to CHOP at its address for notices indicated on the Signature Page of this Agreement. 

 

	 	7.6	If Company is required by law to pay or withhold any income or other taxes on behalf of CHOP with respect to any monies payable to CHOP under this Agreement: 

 

	 	(a)	Company shall deduct them from the amount of such monies due; 

  

	 	(b)	any such tax required to be paid or withheld shall be an expense of and borne solely by CHOP; and 

  
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	 	(c)	Company promptly provide CHOP with a certificate or other documentary evidence to enable CHOP to support a claim for a refund or a foreign tax credit. 

 

	 	7.7	Company and CHOP agree to co-operate in all respects necessary to take advantage of any double taxation agreements or similar agreements as may, from time to time, be available in order to enable Company to make such
payments to CHOP without any deduction or withholding. 

  

	8.	PATENT FILING, PROSECUTION, AND MAINTENANCE 

  

	 	8.1	CHOP shall control the preparation, filing, prosecution, and maintenance of any and all patent applications or patents included in the Patent Rights and shall furnish copies of relevant patent-related documents to
Company. Notwithstanding this Paragraph 8.1, any opposition, validity challenge, interference, re-examination, reissue, derivation, supplemental examination, post-grant review, inter-parties review proceedings, negotiations or claims, in any forum
shall be handled in accordance with Paragraph 10.3. 

  

	 	8.2	Patent Costs 

  

	 	(a)	Commencing with the Effective Date, Company will reimburse CHOP for all documented attorneys’ fees, expenses, official fees and all other charges incurred on or after the Effective Date incident to the preparation,
filing, prosecution, and maintenance of the Patent Rights, within [**] days after Company’s receipt of invoices for such fees, expenses, and charges. Each party shall promptly inform the other as to all matters that come to its attention that
may affect the preparation, filing, prosecution, or maintenance of the Patent Rights. 

  

	 	(b)	Company will reimburse CHOP for all attorneys’ fees, expenses, official fees and all other charges incurred prior to the Effective Date incident to the preparation, filing, prosecution, and maintenance of the
Patent Rights by paying CHOP in [**] equal installments, with each installment due at [**] from the date of the First Commercial Sale of the first Licensed Product. The aggregate amount of such obligation (and corresponding installment amounts)
shall be determined based on the Patent Rights that remain after Company makes any elections that it is entitled to make in accordance with the definition of Patent Rights within the first [**] days after the Effective Date to exclude issued patents
and/or patent applications from the Patent Rights (i.e., such attorneys’ fees, expenses, official fees and other charges incurred prior to the Effective Date for which the reimbursement obligation set forth in this Paragraph 8.2(b) shall be
limited to those incurred with respect to the Patent Rights that remain Patent Rights beyond [**] days after the Effective Date). 

  

	 	8.3	 Throughout the prosecution of the Patent Rights, CHOP shall select patent counsel with CHOP continuing as the client of the patent counsel, provided
that CHOP shall consult with Company regarding its selection of such patent counsel and Company shall have the right to approve the patent counsel selected by CHOP. In addition, throughout the prosecution of the Patent Rights, Company shall have
adequate time to review and comment on all substantive communications and filings between the various U.S. and international patent offices and CHOP, and any reasonable suggestions by Company as to prosecution strategy shall be reasonably considered
by CHOP. CHOP shall not abandon or 

  
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fail to prosecute any patent applications or patents or specific claims therein in the Patent Rights without reasonable prior notice to Company. In the event that CHOP wishes to abandon
prosecution of any patent or patent application or specific claims therein in the Patent Rights, CHOP will allow Company to take over such prosecution if Company so indicates. CHOP shall have the final decision with respect to the preparation,
filing, prosecution and maintenance of the Patent Rights. Company may propose that CHOP adopt reasonable patent prosecution strategies for the Patent Rights, and CHOP shall not unreasonably refuse to incorporate any such measure or recommendation
proposed by Company. 

  

	 	8.4	If at any time during the term of this Agreement the Company opposes or contests the grant or validity of any Licensed Patent Right, or any claims thereof, CHOP will be entitled to terminate the license granted to
Company under Paragraph 3.1 of this Agreement with respect to such Patent Right, upon thirty (30) days’ prior written notice to Company. 

  

	9.	DILIGENCE 

  

	 	9.1	Company, together with its Affiliates and sublicensees, will use commercially reasonable best efforts, subject to the availability to Company of sufficient financing to do so, to develop and bring Licensed Products to
market through a diligent program of development, marketing and commercialization. Company will prepare and submit to CHOP within [**] months of the Effective Date of this Agreement a commercially reasonable development plan (the “Development
Plan”) for developing and bringing to market the currently envisioned the Licensed Products, which Development Plan shall include timelines for such activities, it being understood that the Development Plan may not address marketing plans for
early stage programs. The Development Plan will be incorporated into this Agreement into Appendix B. Company will provide CHOP with [**] reports of activities occurring during each period ending on [**], and any updates to the Development Plan for
the next [**] period. Company’s commercially reasonable efforts shall include, but shall not be limited to, an active, internal program directed toward developing, marketing and selling a Licensed Product. The efforts of a sublicensee or an
Affiliate shall be considered the efforts of Company. At CHOP’S request, the parties will discuss and use good faith efforts to resolve any reasonable concern raised by CHOP regarding Company’s efforts to develop and bring Licensed
Products to market. 

  

	 	9.2	Following the First Commercial Sale, Company shall use its commercially reasonable best efforts to make Licensed Products reasonably accessible to the public. 

 

	 	9.3	Company shall report to CHOP the date of the First Commercial Sale in each country in the Licensed Territory within [**] days of such occurrence. 

 

	 	9.4	All plans and reports required by this Article 9 and marked confidential by Company shall, to the extent permitted by law, be treated by CHOP as commercial and financial information obtained from a person and as
privileged and confidential pursuant to Article 13. 

  

	 	9.5	Company shall not make any willful false statement or any willful omission of any material fact in any report or document required to be provided to CHOP under this Agreement. 

  
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	10.	INFRINGEMENT AND PATENT ENFORCEMENT 

  

	 	10.1	CHOP and Company agree to notify each other promptly of each infringement or possible infringement of the Patent Rights, as well as any facts which may affect the validity, scope, or enforceability of the Patent Rights
of which either Party becomes aware. 

  

	 	10.2	Pursuant to this Agreement Company may a) bring suit in its own name, at its own expense, and on its own behalf for infringement of presumably valid claims in the Patent Rights or misappropriation of Gene-Therapy
Know-How; b) in any such suit, seek to enjoin infringement or misappropriation and collect damages, profits, and awards of whatever nature recoverable for such infringement; and c) settle any claim or suit for infringement of the Patent Rights or
misappropriation of Gene-Therapy Know-How. Company shall not settle any action that imposes any liability on CHOP or concedes the invalidity, enforceability or non-infringement of any of the Patent Rights or CHOP Intellectual Property without the
prior written consent of CHOP. If necessary or desirable to bring, maintain or prove damages in any such action, Company may require that CHOP join such suit. Should CHOP be made a party to any such suit at the request of Company, Company shall
reimburse CHOP for any costs, expenses, or fees, which CHOP incurs as a result of such action. In all cases, Company agrees to keep CHOP reasonably apprised of the status and progress of any litigation. Before Company commences an infringement
action, Company shall notify CHOP and give careful consideration to the views of CHOP. 

  

	 	10.3	In the event that a declaratory judgment action alleging invalidity or non-infringement of any of the Patent Rights or contesting the Gene Therapy Know-How rights (excluding any such action that is based on alleged
misappropriation by CHOP of Gene Therapy Know-How) shall be brought against Company or CHOP or raised by way of counterclaim or affirmative defense in an infringement suit brought by Company under Paragraph 10.2, pursuant to this Agreement, or any
opposition, validity challenge, interference, reexamination, reissue, derivation, supplemental examination, post-grant review, inter-parties review proceedings, negotiations or claims, in any forum relating to the Patent Rights, Company shall,
subject to the penultimate sentence of this Paragraph 10.3a) defend or handle the suit or proceeding, at its own expense, for presumably valid claims in the Patent Rights; and b) in any such suit or proceeding, ultimately seek to enjoin infringement
and to collect damages, profits, and awards of whatever nature recoverable from the party bringing such suit or otherwise defend the validity and enforceability of the Patent Rights. Company shall not settle any action that imposes any liability on
CHOP or concedes the invalidity, enforceability or non-infringement of any of the Patent Rights or CHOP Intellectual Property without the prior written consent of CHOP. If necessary or desirable to defend any such action, Company may require that
CHOP join such suit. Should CHOP be made a party to any such suit at the request of Company, Company shall reimburse CHOP for any costs, expenses, or fees which CHOP incurs as a result of such action. If Company elects not to initiate a defense
against such declaratory judgment action, CHOP at its option, may do so at its own expense and CHOP shall retain all recoveries from any such suit. In all cases, Company agrees to keep CHOP reasonably apprised of the status and progress of any
litigation. 

  

	 	10.4	In any action initiated or defended by Company under this Article 10 of this Agreement, any recovery will go first to reimburse any unreimbursed expenses of CHOP and Company in proportion to their respective
unreimbursed expenses and any remaining recovery shall go to Company and such remaining recovery shall be treated as a Net Sale for purpose of determining any royalty to CHOP. 

  
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	 	10.5	In any action instituted or defended by Company under Article 10 of this Agreement or caused by Company to be initiated against Company or CHOP in a Declaratory Judgment Action under Article 10 of this Agreement, CHOP
may elect to intervene or continue in such suit if Company ceases or fails to thereafter participate in such action for any reason and Company shall be liable for all judgments rendered against the Company in such actions and for all costs, expenses
or fees incurred by CHOP to continue any such actions. 

  

	 	10.6	The parties shall cooperate fully with one another in connection with any action under Paragraphs 10.2 or 10.3. CHOP agrees promptly to provide access to all necessary documents and to render reasonable assistance in
response to a request by Company. 

  

	11.	WARRANTIES, NEGATION OF WARRANTIES AND INDEMNIFICATION 

  

	 	11.1	CHOP offers no warranties other than those specified in this Article 11. 

  

	 	11.2	CHOP represents and warrants to Company that 

  

	 	(a)	as of the Signing Date, any patents in the Patent Rights are not invalid or unenforceable, to CHOP’s present knowledge, 

  

	 	(b)	CHOP has the right to grant to Company the license specified in Article 3 of this Agreement, and 

  

	 	(c)	By assignment of rights from CHOP employees and other inventors, CHOP owns intellectual property rights claimed in any United States and foreign patent applications or patents corresponding to the Patent Rights.

  

	 	11.3	CHOP does not warrant the validity of the Patent Rights and makes no representations whatsoever with regard to the scope of the Patent Rights, or that the Patent Rights may be exploited without infringing other patents
or other intellectual property rights of third parties. 

  

	 	11.4	NO WARRANTIES, EXPRESS OR IMPLIED, ARE OFFERED BY CHOP AS TO THE FITNESS FOR ANY PURPOSE OF THE MATERIALS OR INFORMATION PROVIDED TO COMPANY UNDER THIS AGREEMENT, OR THAT THE CHOP INTELLECTUAL PROPERTY MAY BE EXPLOITED
WITHOUT INFRINGING OTHER THIRD PARTY PATENT RIGHTS. COMPANY ACCEPTS THE CHOP INTELLECTUAL PROPERTY, INFORMATION AND THE MATERIALS “AS IS,” AND CHOP DOES NOT OFFER ANY GUARANTEE OF ANY KIND. 

 

	 	11.5	Except as otherwise set forth in Paragraphs 10.2 and 10.3, CHOP does not represent that it will commence legal actions against third parties infringing the Patent Rights. 

 

	 	11.6	 Company shall indemnify and hold CHOP and its Affiliates, and their respective directors, employees, Affiliates, students, fellows, agents, and
consultants harmless from and against all liability, demands, damages, expenses, and losses, including but not limited to, death, personal injury, illness, or property damage to the extent arising in connection with any activities related to CHOP
Intellectual Property and this Agreement by the Company and 

  
 12 

	 	
its Affiliates, and their respective sublicensees, directors, employees, agents, consultants or third parties, including, without limitation, the design, manufacture, distribution, sale, or use
of any Licensed Products, the use or exploitation of any CHOP Intellectual Property or in connection with any clinical trial in which Company participates utilizing the Licensed Products or the CHOP Intellectual Property, except to the extent
arising in connection with any material breach of this Agreement by, or the gross negligence or willful misconduct of, CHOP. Company agrees to maintain a liability insurance program consistent with sound business practice. 

 

	12.	TERM, TERMINATION, AND MODIFICATION OF RIGHTS 

  

	 	12.1	This Agreement is effective beginning with the Effective Date and shall extend to the expiration of the last to expire of the Patent Rights unless sooner terminated as provided in this Article 12. Upon termination of
this Agreement pursuant to this Article 12 as to a Licensed Product prior to expiration of Company’s royalty obligations with respect to such Licensed Product, Company’s rights under Article 3 of this Agreement shall cease, effective
immediately, with respect to such Licensed Product. After Company’s royalty obligations as to a Licensed Product have expired in a country, Company shall have a perpetual, full and unrestricted right to make, use, offer for sale, sell and
import such Licensed Product in such country under the Patent Rights, Gene-Therapy Know-How and Know-How. Following expiration of this Agreement in its entirety, Company’s rights under Article 3 of this Agreement shall convert to a fully
paid-up, non-royalty bearing, perpetual, unrestricted right to use the Patent Rights, Gene-Therapy Know-How and Know-How. 

  

	 	12.2	In the event that Company is in default in the performance of any material obligations under this Agreement, and if the default has not been remedied within [**] days after the date of notice in writing of such default,
CHOP may terminate this Agreement by written notice. 

  

	 	12.3	In the event that Company becomes insolvent, files a petition in bankruptcy, has such a petition filed against it, determines to file a petition in bankruptcy, or receives notice of a third party’s intention to
file an involuntary petition in bankruptcy, Company shall immediately notify CHOP in writing. 

  

	 	12.4	Company shall have a unilateral right to terminate this Agreement and/or any licenses in any country without cause by giving CHOP ninety (90) days prior written notice to that effect. 

 

	 	12.5	CHOP reserves the right of 35 U.S.C. §203 to terminate or modify this Agreement solely to the extent that such action is legally necessary to meet requirements of the applicable federal statutes or regulations and
such requirements are not reasonably satisfied by Company. Within [**] days of receipt of written notice of CHOP’s belief or notification from the government that it is legally necessary to modify or terminate this Agreement, Company shall, if
Company disagrees with such assessment, notify CHOP of such disagreement and the basis for Company’s position and this Agreement shall not be terminated or modified unless and until such disagreement is resolved in accordance with Paragraph
13.12 or by the exercise of the march-in-rights by the government. 

  
 13 

	 	12.6	Within [**] days of termination or expiration of this Agreement, a final report and all accrued payments shall be submitted by Company. If this Agreement is terminated under this Article 12, sublicenses may be converted
to direct licenses with CHOP pursuant to Paragraph 4.4. 

  

	13.	CONFIDENTIALITY 

  

	 	13.1	All plans and reports required to be provided by Company to CHOP shall, to the extent permitted by law, be treated by CHOP as commercial and financial information obtained from a person and as confidential.

  

	 	13.2	Unless otherwise provided in this Agreement, each Party agrees to hold in confidence, in accordance with this paragraph, any confidential information, marked or designated confidential, and disclosed to such Party
(“Receiving Party”) by the other Party (“Disclosing Party”) under this Agreement (hereinafter “Confidential Information”). For the purpose of this Agreement, “hold in confidence” means that the Receiving Party
will protect the Information in the same manner in which it protects its own confidential information, but in any event with no less than reasonable care, and will not disclose such Information (or permit the disclosure of such Information) except
to only those persons necessary for the performance of this Agreement or otherwise as reasonably necessary in the exercise of such Party’s rights or the performance of such Party’s obligations hereunder. The foregoing restriction shall not
apply to either Party disclosing such information to such of its directors, officers, employees, agents and/or representatives (“Party Persons”) as shall have a reason to be disclosed such information, provided that prior to any such
disclosure, the disclosing Party shall secure from each such Party Person to whom disclosure will be made an undertaking to conform to confidentiality requirements no less restrictive than those contained herein. 

 

	 	13.3	The non-disclosure provision of Paragraph 12.1 shall not apply to: 

  

	 	(a)	Confidential Information in the public domain otherwise than by breach of this Agreement; 

  

	 	(b)	Confidential Information in the lawful possession of a Party prior to disclosure by any other Party as evidenced by written records; 

 

	 	(c)	Confidential Information that was created independent of disclosure as evidenced by written records; 

  

	 	(d)	Confidential Information obtained from a third party who is free to divulge the same; or 

  

	 	(e)	Confidential Information which is properly disclosed pursuant to a statutory obligation, an order or rules of a court of competent jurisdiction or that of a competent regulatory authority. 

 

	 	13.4	A Party will be entitled to make a disclosure or public statement concerning the subject matter or any term of this Agreement, or to disclose Confidential Information that the Receiving Party is required to make or
disclose only pursuant to: 

  

	 	(a)	a valid order of a court or governmental authority; or 

  
 14 

	 	(b)	any other requirement of law or any securities or stock exchange; provided that if the Receiving Party becomes legally required to make such announcement, public statement or disclosure hereunder, the Receiving Party
shall give the other Party prompt notice of such fact to enable the other Party to seek a protective order or other appropriate remedy concerning any such announcement, public statement or disclosure, including confidential treatment and/or
appropriate redactions. 

  

	 	13.5	The obligations of Receiving Party to maintain confidentiality of Information under this Agreement will survive its expiration or termination and will endure for [**] years from the date of termination of this
Agreement. The Parties may disclose the existence of this Agreement and that Company is licensed under CHOP patents or technology. 

  

	14.	GENERAL PROVISIONS 

  

	 	14.1	Neither Party may waive or release any of its rights or interests in this Agreement except in writing. The failure of a Party to assert a right hereunder or to insist upon compliance with any term or condition of this
Agreement shall not constitute a waiver of that right by such Party or excuse a similar subsequent failure to perform any such term or condition by the other Party. 

 

	 	14.2	This Agreement, and the Agreements specified herein, constitutes the entire agreement between the Parties relating to the subject matter of the grant of the CHOP Intellectual Property, and all prior negotiations,
representations, agreements, and understandings are merged into, extinguished by, and completely expressed by this Agreement. 

  

	 	14.3	The provisions of this Agreement are severable, and in the event that any provision of this Agreement shall be determined to be invalid or unenforceable under any controlling body of law such determination shall not in
any way affect the validity or enforceability of the remaining provisions of this Agreement. 

  

	 	14.4	If either Party desires a modification to this Agreement, the Parties shall, upon reasonable notice of the proposed modification by the Party desiring the change, confer in good faith to determine the desirability of
such modification. No modification will be effective until a written amendment is signed by the signatories to this Agreement or their designees. 

  

	 	14.5	The construction, validity, performance, and effect of this Agreement shall be governed by the Laws of the Commonwealth of Pennsylvania and any actions involving this Agreement shall only be brought within a
jurisdiction in Pennsylvania. 

  

	 	14.6	All notices required or permitted by this Agreement shall be given by prepaid, first class, registered or certified mail, overnight carrier properly addressed to the other Party at the address designated on the
following Signature Page, or to such other address as may be designated in writing by such other Party, and shall be effective as of the date of the postmark of such notice. 

 

	 	14.7	This Agreement shall not be assigned by Company except a) with the prior written consent of CHOP, such consent not to be withheld unreasonably; or b) to an Affiliate or as part of a sale or transfer of all or
substantially all of the business or assets of Company relating to operations which concern this Agreement. Company shall notify CHOP in writing within [**] days of any assignment of this Agreement by Company. 

  
 15 

	 	14.8	Company agrees in its use of any CHOP supplied materials to comply with all applicable statutes, regulations, and guidelines, including Public Health Service and National Institutes of Health regulations and guidelines.
Company agrees not to use the materials for research involving human subjects or clinical trials in the United States without complying with 21 CFR Part 50 and 45 CFR Part 46 and any and all applicable laws or regulations. Company agrees not to use
the materials for research involving human subjects or clinical trials outside of the United States without complying with the applicable laws and regulations of the appropriate national control authorities. 

 

	 	14.9	Company acknowledges that it is subject to and agrees to abide by the United States laws and regulations (including the Export Administration Act of 1979 and Arms Export Control Act) controlling the export of technical
data, computer software, laboratory prototypes, biological material, and other commodities. The transfer of such items may require a license from the cognizant Agency of the U.S. Government or written assurances by Company that it shall not export
such items to certain foreign countries without prior approval of such agency. CHOP neither represents that a license is not required or that, if required, it shall be issued. 

 

	 	14.10	To the extent legally required, Company agrees to mark the Licensed Products or their packaging sold in the United States with all applicable U.S. patent numbers and similarly to indicate “Patent Pending”
status. To the extent legally required, all Licensed Products manufactured in, shipped to, or sold in other countries shall be marked in such a manner as to preserve CHOP patent rights in such countries. 

 

	 	14.11	By entering into this Agreement, CHOP does not directly or indirectly endorse any product or service provided, or to be provided, by Company whether directly or indirectly related to this Agreement. Company shall not
state or imply that this Agreement is an endorsement by CHOP or their employees in any advertising, promotional, or sales literature without the prior written consent of CHOP. 

 

	 	14.12	The Parties agree to attempt to settle amicably any controversy or claim arising under this Agreement or a breach of this Agreement. Thereafter, either Party may exercise any administrative or judicial remedies that may
be available. 

  

	 	14.13	Neither party shall issue any press releases or public disclosure relating to this Agreement without the prior written consent of the other party, which consent shall not be unreasonably withheld or delayed. Neither
Party shall use the name or logo of the other party, and Company shall not use the name of past or present CHOP employees, in any advertising, promotional or sales activities without prior written consent obtained from such employees in each
separate case, except as otherwise provided in this Agreement. 

  

	 	14.14	Nothing relating to the grant of a license, nor the grant itself, shall be construed to confer upon any person any immunity from or defenses under the antitrust laws or from a charge of patent misuse, and the
acquisition and use of rights pursuant to 37 CFR Part 404 shall not be immunized from the operation of state or Federal law by reason of the source of the grant. 

  

	 	14.15	Paragraphs 3.2, 4.4, 7.1, 7.3, 10.5, 11.6, 12.1, 12.6, 13.5, 14.5, 14.11, and 14.13 of this Agreement shall survive termination of this Agreement. 

  
 16 

 SIGNATURES BEGIN ON FOLLOWING PAGE 

  
 17 

 CHOP PATENT EXCLUSIVE LICENSE AGREEMENT 

SIGNATURE PAGE 
 IN WITNESS WHEREOF, duly authorized
representatives of the Parties have duly executed this Agreement to be effective as of the Effective Date. 
 For THE CHILDREN’S HOSPITAL OF
PHILADELPHIA: 
  

					
	 /s/ Steven M. Altschuler
	 		 	 October 14, 2013

	Signature of Authorized CHOP Official	 		 	Date
			
	 Steven M. Altschuler
	 		 	
	Printed Name	 		 	
			
	 President & CEO
	 		 	
	Title	 		 	

 Mailing Address for Notices: 

 

					
	 The Children’s Hospital of Philadelphia

Office of Technology Transfer
 Colket Translational Research
Building
 Suite 2200
 3615 Civic Center Boulevard

Philadelphia, PA 19104
 Attention: Director, Technology
Transfer
	 		 	 With copy to:
 Office of General Counsel

34th and Civic Center Blvd.

Philadelphia, PA 19104

For AAVENUE THERAPEUTICS: 
  

					
	 /s/ Jeffrey D. Marrazzo
	 		 	 October 14, 2013

	Signature of Authorized Official	 		 	Date
			
	 Jeffrey D. Marrazzo
	 		 	
	Printed Name	 		 	
			
	 President and Chief Executive Officer
	 		 	
	Title	 		 	

 Mailing Address for Notices: 

Aavenue Therapeutics, LLC 
 c/o The Children’s Hospital of
Philadelphia® 
 34th and Civic Center Blvd.

 Philadelphia, PA 19104 

  
 18 

 APPENDIX A — Patent(s) or Patent Application(s) 

Confidential Materials omitted and filed separately with the Securities and Exchange Commission. A total of two pages were omitted. [**] 

  
 19 

 APPENDIX B — Development Plan 

  
 20 

 Appendix C – Related Agreements 
  

	 	1.	Technology Assignment Agreement dated October 14, 2013 between the Company and The Children’s Hospital of Philadelphia: Incorporated by reference to Exhibit 10.8 to the Company’s Registration
Statement on Form S-1 

  

	 	2.	Master Research Services Agreement dated October 14, 2013 between the Company and The Children’s Hospital of Philadelphia: Incorporated by reference to Exhibit 10.9 to the Company’s Registration
Statement on Form S-1 

  

	 	3.	Services Agreement dated December 26, 2013 between the Registrant and The Children’s Hospital of Philadelphia: Incorporated by reference to Exhibit 10.10 to the Company’s Registration Statement on
Form S-1 

  

	 	4.	Common Share Agreement dated October 14, 2013 between the Company and The Children’s Hospital of Philadelphia: attached below 

 

	 	5.	Common Share Agreement dated October 14, 2013 between the Company and The Children’s Hospital of Philadelphia: attached below 

  
 21 

 Common Share Membership Agreement 

This Common Share Membership Agreement (this “Agreement”) is made as of October 14, 2013 (the “Effective
Date”), by and between AAVenue Therapeutics, LLC (the “Company”) and The Children’s Hospital of Philadelphia (the “Member”). 

For valuable consideration, receipt of which is acknowledged, the parties hereto agree as follows: 

 

	 	1.	Defined Terms. 

 (a) Capitalized terms used but not otherwise defined herein shall have
the meaning assigned to such terms in the Amended and Restated Limited Liability Company Agreement of the Company, dated as of October 14, 2013, as amended from time to time (the “LLC Agreement”). 

(b) For purposes of this Agreement: 

“Member Shares” means [**] Common Shares. 
  

	 	2.	Sale of Member Shares; Continuation as Member of the Company. 

 (a) As of the Effective
Date, the Company hereby issues to the Member, and the Member hereby accepts from the Company, subject to the terms and conditions set forth in this Agreement and in the LLC Agreement, the Member Shares in consideration for entering into a License
Agreement attached hereto as Exhibit A, dated the date hereof (the “License Agreement”). Upon execution of this Agreement and the License Agreement, the Member shall continue as a member of the Company effective as of the
Effective Date. The number of Member Shares acquired by the Member shall be reflected on Schedule A to the LLC Agreement opposite such Member’s name (together any other Common Shares held by the Member). The Member Shares are hereby
designated in accordance with the LLC Agreement as Series 1 Common Shares. 
 3. Agreement to be Bound by LLC Agreement. The Member
agrees to be bound by the terms and conditions of the LLC Agreement and authorizes the signature page of this Agreement to be attached to the LLC Agreement, or counterparts thereof. The Member acknowledges receipt of a copy of the LLC Agreement.

 4. “Market Stand-off” Agreement. The Member hereby agrees that it will not,
without the prior written consent of the managing underwriter, during the period commencing on the date of the final prospectus relating to the initial registration by the Company of the Surviving Corporation Shares or any other equity securities on
a registration statement under the Securities Act (the “IPO”), and ending on the date specified by the Company and the managing underwriter (such period not to exceed 180 days), or such other period as may be requested by the
Company or an underwriter to accommodate regulatory restrictions on (1) the publication or other distribution of research reports and (2) analyst recommendations and opinions, including, but not limited to, the restrictions contained in
FINRA Rule 2711(f)(4) or NYSE Rule 472(f)(4), or any successor provisions or amendments thereto), (i) lend; offer; pledge; sell; contract to sell; sell any option or contract to purchase; purchase any option or contract to sell; grant any
option, right, or warrant to 

 
purchase; or otherwise transfer or dispose of, directly or indirectly, any Surviving Corporation Shares or any securities convertible into or exercisable or exchangeable (directly or indirectly)
for Surviving Corporation Shares (whether such shares or any such securities are then owned by the Member or are thereafter acquired) or (ii) enter into any swap or other arrangement that transfers to another, in whole or in part, any of the
economic consequences of ownership of such securities, whether any such transaction described in clause (i) or (ii) above is to be settled by delivery of Surviving Corporation Shares or other securities, in cash, or otherwise. The
foregoing provisions of this Section 4 shall apply only to the IPO, shall not apply to the sale of any securities to an underwriter pursuant to an underwriting agreement, and shall be applicable to the Member only if all officers and
directors are subject to the same restrictions and the Company uses commercially reasonable efforts to obtain a similar agreement from all stockholders individually owning more than 1% of the outstanding Surviving Corporation Shares (after giving
effect to conversion into Surviving Corporation Shares of all outstanding Preferred Shares). The underwriters in connection with such registration are intended third-party beneficiaries of this
Section 4 and shall have the right, power, and authority to enforce the provisions hereof as though they were a party hereto. The Member further agrees to execute such agreements as may be reasonably requested by the underwriters in
connection with such registration that are consistent with this Section 4 or that are necessary to give further effect thereto. 
  

	 	5.	Miscellaneous. 

 (a) Construction. For purposes of this Agreement, references to
Common Shares shall include references to any securities issued in respect of Common Shares in connection with any reorganization of the Company, reclassification of the Common Shares or other similar transaction, including in connection with the
conversion of the LLC into a Corporation pursuant to Section 12.04 of the LLC Agreement. For the avoidance of doubt, any and all new, substituted or additional securities to which the Member is entitled by reason of his ownership of the Member
Shares shall be immediately subject to the provisions of this Agreement in the same manner and to the same extent as the Member Shares. 

(b) Separability of Provisions. Each provision of this Agreement shall be considered separable. To the extent that any provision of
this Agreement is prohibited or ineffective under the Act, this Agreement shall be considered amended to the smallest degree possible in order to make this Agreement effective under the Act (and, if the Act is subsequently amended or interpreted in
such manner as to make effective any provision of this Agreement that was formerly rendered invalid, such provision shall automatically be considered to be valid from the effective date of such amendment or interpretation). 

(c) Waiver; Amendment. Any provision for the benefit of the Company contained in this Agreement may be waived, either generally or in
any particular instance, by the Board. The terms of this Agreement may be amended only by a written instrument duly executed by the Company and the Member. 

(d) Binding Effect. This Agreement shall be binding upon and inure to the benefit of the Company and the Member and their respective
heirs, legal representatives, successors and assigns, subject to the terms of this Agreement, the CHOP Agreements and the LLC Agreement. 

 (e) Notice. All notices required or permitted hereunder shall be delivered in accordance
with the provisions of the LLC Agreement. 
 (f) Applicable Law. This Agreement shall be construed and enforced in accordance with
the laws of the State of Delaware, including the Act, as interpreted by the courts of the State of Delaware, notwithstanding any rules regarding conflicts or choice of law to the contrary. 

(g) Entire Agreement. This Agreement, the CHOP Agreements and the LLC Agreement constitute the full and entire understanding and
agreement between the parties with respect to the subject matter hereof, and any other written or oral agreement relating to the subject matter hereof existing between the parties is expressly canceled. 

[Remainder of Page Intentionally Left Blank] 

 Executed, in counterpart, as of the Effective Date. 

 

			
	MEMBER:
	THE CHILDREN’S HOSPITAL OF
	PHILADELPHIA	 	
		
	By:	 	 /s/ Steven M. Altschuler

		
	Name:	 	 Steven M. Altschuler

		
	Title:	 	 President & CEO

  

			
	ACCEPTED AND AGREED:
	
	AAVENUE THERAPEUTICS, LLC
		
	By:	 	 /s/ Jeffrey D. Marrazzo

	Name:	 	 Jeffrey D. Marrazzo

	Title:	 	 Chief Executive Officer

 EXHIBIT A 

License Agreement 
 Incorporated by Reference to
Exhibit 10.7 to the Company’s Registration Statement on Form S-1 

 Common Share Membership Agreement 

This Common Share Membership Agreement (this “Agreement”) is made as of October 14, 2013 (the “Effective
Date”), by and between AAVenue Therapeutics, LLC (the “Company”) and The Children’s Hospital of Philadelphia (the “Member”). 

For valuable consideration, receipt of which is acknowledged, the parties hereto agree as follows: 

 

	 	1.	Defined Terms. 

 (a) Capitalized terms used but not otherwise defined herein shall have
the meaning assigned to such terms in the Amended and Restated Limited Liability Company Agreement of the Company, dated as of October 14, 2013, as amended from time to time (the “LLC Agreement”). 

(b) For purposes of this Agreement: 

“Member Shares” means [**] Common Shares. 
  

	 	2.	Sale of Member Shares; Continuation as Member of the Company. 

 (a) As of the Effective
Date, the Company hereby issues to the Member, and the Member hereby accepts from the Company, subject to the terms and conditions set forth in this Agreement and in the LLC Agreement, the Member Shares in consideration for the transfer of
(A) certain intellectual property rights as set forth in that certain Technology Assignment Agreement attached hereto as Exhibit A (the “Technology Assignment Agreement”) and (B) certain contracts as set forth in
that certain Assignment of Contracts attached hereto as Exhibit B, each dated the date hereof (together with the Technology Assignment Agreement, the “CHOP Agreements”). Upon execution of this Agreement and the CHOP
Agreements, the Member shall continue as a member of the Company effective as of the Effective Date. The number of Member Shares acquired by the Member shall be reflected on Schedule A to the LLC Agreement opposite such Member’s name
(together with any other Common Shares held by the Member). The Member Shares are hereby designated in accordance with the LLC Agreement as Series 1 Common Shares. 

3. Agreement to be Bound by LLC Agreement. The Member agrees to be bound by the terms and conditions of the LLC Agreement and
authorizes the signature page of this Agreement to be attached to the LLC Agreement, or counterparts thereof. The Member acknowledges receipt of a copy of the LLC Agreement. 

4. “Market Stand-off” Agreement. The Member hereby agrees that it will not, without
the prior written consent of the managing underwriter, during the period commencing on the date of the final prospectus relating to the initial registration by the Company of the Surviving Corporation Shares or any other equity securities on a
registration statement under the Securities Act (the “IPO”), and ending on the date specified by the Company and the managing underwriter (such period not to exceed 180 days), or such other period as may be requested by the Company
or an underwriter to accommodate regulatory restrictions on (1) the publication or other distribution of research reports and (2) analyst recommendations and opinions, including, but not limited to, the restrictions contained in FINRA Rule
2711(f)(4) or NYSE Rule 472(f)(4), or any successor provisions or amendments thereto), (i) lend; offer; pledge; sell; contract to sell; sell any option or contract to purchase; purchase any option or contract to sell; grant any option, right,
or warrant to purchase; or otherwise transfer or dispose of, directly or indirectly, any Surviving Corporation Shares or any securities convertible into or exercisable or exchangeable (directly or indirectly) for Surviving Corporation Shares
(whether such shares or any such securities are then owned by the Member or 

 
are thereafter acquired) or (ii) enter into any swap or other arrangement that transfers to another, in whole or in part, any of the economic consequences of ownership of such securities,
whether any such transaction described in clause (i) or (ii) above is to be settled by delivery of Surviving Corporation Shares or other securities, in cash, or otherwise. The foregoing provisions of this Section 4 shall apply
only to the IPO, shall not apply to the sale of any securities to an underwriter pursuant to an underwriting agreement, and shall be applicable to the Member only if all officers and directors are subject to the same restrictions and the Company
uses commercially reasonable efforts to obtain a similar agreement from all stockholders individually owning more than 1% of the outstanding Surviving Corporation Shares (after giving effect to conversion into Surviving Corporation Shares of all
outstanding Preferred Shares). The underwriters in connection with such registration are intended third-party beneficiaries of this Section 4 and shall have the right, power, and authority to
enforce the provisions hereof as though they were a party hereto. The Member further agrees to execute such agreements as may be reasonably requested by the underwriters in connection with such registration that are consistent with this
Section 4 or that are necessary to give further effect thereto. 
  

	 	5.	Miscellaneous. 

 (a) Construction. For purposes of this Agreement, references to
Common Shares shall include references to any securities issued in respect of Common Shares in connection with any reorganization of the Company, reclassification of the Common Shares or other similar transaction, including in connection with the
conversion of the LLC into a Corporation pursuant to Section 12.04 of the LLC Agreement. For the avoidance of doubt, any and all new, substituted or additional securities to which the Member is entitled by reason of his ownership of the Member
Shares shall be immediately subject to the provisions of this Agreement in the same manner and to the same extent as the Member Shares. 

(b) Separability of Provisions. Each provision of this Agreement shall be considered separable. To the extent that any provision of
this Agreement is prohibited or ineffective under the Act, this Agreement shall be considered amended to the smallest degree possible in order to make this Agreement effective under the Act (and, if the Act is subsequently amended or interpreted in
such manner as to make effective any provision of this Agreement that was formerly rendered invalid, such provision shall automatically be considered to be valid from the effective date of such amendment or interpretation). 

(c) Waiver; Amendment. Any provision for the benefit of the Company contained in this Agreement may be waived, either generally or in
any particular instance, by the Board. The terms of this Agreement may be amended only by a written instrument duly executed by the Company and the Member. 

(d) Binding Effect. This Agreement shall be binding upon and inure to the benefit of the Company and the Member and their respective
heirs, legal representatives, successors and assigns, subject to the terms of this Agreement, the CHOP Agreements and the LLC Agreement. 

(e) Notice. All notices required or permitted hereunder shall be delivered in accordance with the provisions of the LLC Agreement. 

(f) Applicable Law. This Agreement shall be construed and enforced in accordance with the laws of the State of Delaware, including the
Act, as interpreted by the courts of the State of Delaware, notwithstanding any rules regarding conflicts or choice of law to the contrary. 

(g) Entire Agreement. This Agreement, the CHOP Agreements and the LLC Agreement constitute the full and entire understanding and
agreement between the parties with respect to the subject matter hereof, and any other written or oral agreement relating to the subject matter hereof existing between the parties is expressly canceled. 

[Remainder of Page Intentionally Left Blank] 

 Executed, in counterpart, as of the Effective Date. 

 

			
	MEMBER:
	THE CHILDREN’S HOSPITAL OF PHILADELPHIA
		
	By:	 	 /s/ Steven M. Altschuler

		
	Name:	 	 Steven M. Altschuler

		
	Title:	 	 President & CEO

  

			
	ACCEPTED AND AGREED:
	
	AAVENUE THERAPEUTICS, LLC
		
	By:	 	 /s/ Jeffrey D. Marrazzo

	Name:	 	Jeffrey D. Marrazzo
	Title:	 	Chief Executive Officer

 EXHIBIT A 

Technology Assignment Agreement 
 Incorporated by
reference to Exhibit 10.8 to the Company’s Registration Statement on Form S-1 

 EXHIBIT B 

Assignment of Contracts 

 ASSIGNMENT OF CONTRACTS 

This Assignment of Contracts (this “Assignment”) dated as of October 14, 2013 is entered into between The
Children’s Hospital of Philadelphia (“Assignor”), and AAVenue Therapeutics, LLC (“Assignee”). All capitalized words and terms used in this Assignment and not defined herein shall have the respective meanings
ascribed to them in Common Share Membership Agreement dated the date hereof, between Assignor and Assignee (the “Agreement”). 

WHEREAS, the Assignor and Assignee have entered into a Common Share Membership Agreement, dated the date hereof (the “Membership
Agreement”), pursuant to which the Assignee is issuing Series 1 Common Shares (as defined in the Membership Agreement) to the Assignor in partial consideration for entering into this Assignment; and 

WHEREAS, pursuant to the Membership Agreement, Assignor has agreed to convey, assign, transfer and deliver to Assignee the contracts of
Assignor, including pending contracts, identified on Schedule A attached hereto (the “Assigned Contracts”), and Assignee has agreed to assume all rights and obligations under the Assigned Contracts and to perform and
discharge the obligations of Assignor thereunder. 
 NOW, THEREFORE, in consideration of the mutual promises set forth in the Membership
Agreement and other good and valuable consideration, the receipt and sufficiency of which are hereby acknowledged, Assignor and Assignee hereby agree as follows: 

1. Assignor hereby conveys, assigns, transfers and delivers, and Assignee hereby accepts, all of Assignor’s right, title and interest in
and to the Assigned Contracts. Assignor will use its commercially reasonable efforts to obtain all necessary third party or governmental consents necessary to consummate the transactions provided for in this Agreement to the extent not otherwise
obtained by the date hereof. Assignor will execute and deliver such further instruments and take such further actions as may be required to effect the provisions of this Assignment. 

2. Assignee accepts all the right, title and interest in the Assigned Contracts and assumes and agrees to observe, perform and be bound by all
of the terms of the Assigned Contracts. 
 3. This Assignment shall be governed by and construed in accordance with the internal laws of the
State of Delaware without giving effect to any choice or conflict of law provision or rule (whether of the State of Delaware or any other jurisdiction) that would cause the application of laws of any jurisdiction other than those of the State of
Delaware. 
 [REMAINDER OF PAGE INTENTIONALLY LEFT BLANK] 

  
 - 32 - 

 IN WITNESS WHEREOF, Assignor and Assignee have caused this Assignment to be duly executed as of
and on the date first above written. 
  

			
	ASSIGNOR:
	
	THE CHILDREN’S HOSPITAL OF PHILADELPHIA
		
	By:	 	 /s/ Steven M. Altschuler

		
	Name:	 	 Steven M. Altschuler

		
	Title:	 	 President & CEO

	
	ASSIGNEE:
	
	AAVENUE THERAPEUTICS, LLC
		
	By:	 	 /s/ Jeffrey D. Marrazzo

	Name:	 	Jeffrey D. Marrazzo
	Title:	 	Chief Executive Officer

  
 - 33 - 

 SCHEDULE A 

Assigned Contracts 
 Research
Collaboration Agreement, dated May 9, 2013, by and between Oregon Health and Science University and the Assignor. 
 Research Collaboration Agreement,
dated February 10, 2012, as amended to date, by and among The University of Iowa, Howard Hughes Medical Institute and the Assignor. 
 Clinical Trial
Agreement, dated February 15, 2010, as amended to date, by and between University of Iowa and the Assignor. 
 Master Consulting Agreement, dated
September 24, 2009, as amended to date, by and between Statistics Collaborative, Inc. and the Assignor. 
 All work orders under Master Consulting
Agreement, dated September 24, 2009, as amended to date, by and between Statistics Collaborative, Inc. and the Assignor. 
 Master Services Agreement,
dated November 1, 2010, as amended to date, by and between RRD International, LLC and the Assignor. 
 All work orders under Master Services Agreement,
dated November 1, 2010, as amended to date, by and between RRD International, LLC and the Assignor. 
 Consulting Agreement, dated July 11, 2012,
as amended to date, by and between Biologics Consulting Group, Inc. and the Assignor. 
 All work orders under Consulting Agreement, dated July 11,
2012, as amended to date, by and between Biologics Consulting Group, Inc. and the Assignor. 
 Confidential Disclosure Agreement, dated August 26,
2009, by and between Alan Boyd Consultants Ltd. and the Assignor. 
 Consultancy Agreement, dated October 23, 2009, as amended to date, by and between
Alan Boyd Consultants Ltd and the Assignor. 
 All work orders (annexes) under Consultancy Agreement, dated October 23, 2009, as amended to date, by
and between Alan Boyd Consultants Ltd and the Assignor. 
 Master Contract Research Organization Agreement, effective November 1, 2011, as amended to
date, by and between Westat Inc. and the Assignor. 
 All task orders under Master Contract Research Organization Agreement, effective November 1,
2011, as amended to date, by and between Westat Inc. and the Assignor. 
 Master Business Associate Agreement, dated January 31, 2012, by and between
Westat Inc. and the Assignor. 

  
 - 34 - 

 Purchase Service Agreement, dated November 4, 2011, by and between Westat Inc. and the Assignor. 

Pending Contracts: 
 Legal Representative Agreement
by and between Alan Boyd Consultants Ltd. and the Assignor. 
 Clinical Trial Agreement by and between Sydney Local Health District (SLHD) and the Assignor.
(Project: AAV8-hFIX19-101 Study) 
 Clinical Trial Agreement by and between St. James’s Hospital (Dublin, Ireland) and the Assignor. (Project:
AAV8-hFIX19-101 Study) 
 Clinical Trial Agreement by and between The State University of Campinas (Brazil) and the Assignor. (Project: AAV8-hFIX19-101
Study) 
 Clinical Trial Agreement by and between University of California, San Francisco and the Assignor. (Project: AAV8-hFIX19-101 Study) 

Master Service Agreement by and between Quintiles, Inc. and the Assignor. (Project: AAV8-hFIX19-101 Study International Site Monitoring) 

Business Associate Agreement by and between Quintiles, Inc. and the Assignor. (Project: AAV8-hFIX19-101 Study International Site Monitoring) 

Contract Service Agreement by and between Calvert Laboratories, Inc. and the Assignor. (Project: AAV2-hCHM-101 Study) 

  
 - 35 - 

 LICENSE AGREEMENT AMENDMENT 

This Amendment (hereinafter “Amendment”), with an effective date of
December 26th, 2013 (the “Effective Date”) serves as a modification to the License Agreement, having an effective date of October 14, 2013 (the “Agreement”), made by
and between Spark Therapeutics, LLC, (formerly known as AA Venue Therapeutics, LLC) a limited liability company organized and existing under the laws of Delaware and having a principal place of business at 34th and Civic Center Blvd, Philadelphia, PA 19104 (“Company”) and The Children’s Hospital of Philadelphia, a non-profit entity organized and existing under the laws of Pennsylvania
and having a principal place of business at 34th and Civic Center Boulevard, Philadelphia, PA 19104 (“CHOP”). 

WHEREAS, CHOP and Company desire that the Agreement be amended in order to add certain patents to the Agreement 

NOW, THEREFORE, in consideration of the mutual covenants and promises contained herein, CHOP and Company, intending to be bound, hereby
mutually agree to the following: 
  

	1.	Appendix A - Patent(s) or Patent Application(s) of the Agreement shall be deleted and replaced in its entirety with Appendix A-l hereto and all references in the Agreement to Appendix A shall, after the effective date
of this Amendment, be deemed to be references to Appendix A-l. 

  

	2.	The construction, validity, performance, and effect of this Amendment shall be governed by the Laws of the Commonwealth of Pennsylvania and any actions involving this Agreement shall only be brought within a
jurisdiction in Pennsylvania. 

  

	3.	This Agreement may be executed in multiple counterparts, each of which shall be deemed an original, but all of which together shall constitute one and the same instrument. 

[Signatures on next page.] 

  
 - 36 - 

 IN WITNESS WHEREOF, the parties hereto have executed this Amendment in duplicate originals by their duly
authorized officer or representative. 
  

									
	The Children’s Hospital of Philadelphia	 		 	Spark Therapeutics, LLC
					
	By:	 	 /s/ Ellen Purpus
	 		 	By:	 	 /s/ Jeffrey D. Marrazzo

	Name:	 	Ellen Purpus	 		 	Name:	 	Jeffrey D. Marrazzo
	Title:	 		 		 	Title:	 	President and CEO
					
	Date:	 	 12/30/13
	 		 	Date:	 	 12/26/13

  
 - 37 - 

 APPENDIX A-l - Patent(s) or Patent Application(s) 

Confidential Materials omitted and filed separately with the Securities and Exchange Commission. A total of two pages were omitted. [**] 

  
 - 38 - 

 LICENSE AGREEMENT AMENDMENT NO. 2 

This Amendment No. 2 (hereinafter “Amendment”), with an effective date of May 16, 2014 (the “Effective Date”)
serves as a modification to the License Agreement, having an effective date of October 14, 2013 as amended by that certain License Agreement Amendment, dated December 26, 2013 (together, the “Agreement”), made by and between
Spark Therapeutics, Inc.,(formerly known as AAVenue Therapeutics LLC) a corporation organized and existing under the laws of Delaware and having a principal place of business at 34th and Civic
Center Blvd. Philadelphia, PA 19104 (“Company”) and The Children’s Hospital of Philadelphia’®, a non-profit entity organized and existing under the laws of Pennsylvania and
having a principal place of business at 34lh and Civic Center Boulevard, Philadelphia, PA 19104 (“CHOP”). 

WHEREAS, CHOP and Company desire that the Agreement be amended in order to add certain patents to the Agreement 

NOW, THEREFORE, in consideration of the mutual covenants and promises contained herein, CHOP and Company, intending to be bound, hereby
mutually agree to the following: 
  

	1.	Appendix A - Patent(s) or Patent Application(s) of the Agreement shall be deleted and replaced in its entirety with Appendix A-l hereto and all references in the Agreement to Appendix A shall, after the effective date
of this Amendment, be deemed to be references to Appendix A-l. 

  

	2.	The construction, validity, performance, and effect of this Amendment shall be governed by the Laws of the Commonwealth of Pennsylvania and any actions involving this Agreement shall only be brought within a
jurisdiction in Pennsylvania. 

  

	3.	This Agreement may be executed in multiple counterparts, each of which shall be deemed an original, but all of which together shall constitute one and the same instrument. 

[Signatures on next page.] 

 IN WITNESS WHEREOF, the parties hereto have executed this Amendment in duplicate originals by their duly
authorized officer or representative. 
  

									
	The Children’s Hospital of Philadelphia	 		 	Spark Therapeutics, Inc.
					
	By:	 	 /s/ Mary Tomlinson
	 		 		 	
	Name:	 	Mary Tomlinson	 		 	By:	 	 /s/ Jeffrey D. Marrazzo

	Title:	 	 Sr. Vice-president, Research

Administration
	 		 	Name:	 	Jeffrey D. Marrazzo
		 		 		 	Title:	 	President and CEO
					
	Date:	 	 5/16/14
	 		 	Date:	 	 5/16/14

 APPENDIX A-l 

 Patents or Patent Applications Licensed of Spark THx 

 

																			
	 Tech ID
	  	Internal
ID	  	Country	  	File Date	  	Serial No.	  	Patent
No.	  	Issue
Date	  	App Type	  	Title	  	Inventors

 Confidential Materials omitted and filed
separately with the Securities and Exchange Commission. A total of seven pages were omitted. [**] 

  
 1 

 THIRD AMENDMENT TO LICENSE AGREEMENT 

This Amendment (hereinafter “Amendment”) to the License Agreement (as defined herein) is made as of the date of the last signature in the signature
lines below, by and between Spark Therapeutics, Inc. (formerly known as AAVenue Therapeutics, LLC), a Delaware corporation with an address of 3737 Market Street, Suite 1300, Philadelphia, PA 19104 (“Company”), and The Children’s
Hospital of Philadelphia, with an address of 34th Street and Civic Center Blvd., Philadelphia, PA 19104 (“CHOP”). 

WHEREAS, CHOP and Company are parties to a License Agreement, dated October 14, 2013 (the “License Agreement” or
“Agreement”) under which CHOP has granted Company an exclusive license to certain patent rights and know-how related to technology developed at least in part by Katherine High, MD, as defined in the Agreement; 

WHEREAS, the Agreement was amended by Company and CHOP on December 23, 2013 to replace Appendix A to the Agreement; 

WHEREAS, the Agreement was further amended by Company and CHOP on May 16, 2014 to replace Appendix A to the Agreement; 

WHEREAS, Katherine High, MD, was an investigator of the Howard Hughes Medical Institute (“HHMI”) at her laboratory at CHOP when she
created certain of the technology embodied in the licensed patent rights; 
 WHEREAS, HHMI owned a part of the CHOP Intellectual Property
and assigned those rights to CHOP; 
 NOW, THEREFORE, CHOP and the Company agree to amend the Agreement as follows: 

 

	 	1.	Section 1.6 is hereby added to the Agreement as follows: 

 A subset of the CHOP
Intellectual Property was created, at least in part, by Dr. Katherine High, MD, while she was an employee of the Howard Hughes Medical Institute (“HHMI”) at her laboratory at CHOP. 

 

	 	2.	Section 2.12 is hereby amended to read, in its entirety, as follows: 

 “Patent
rights” means: the United States patents and or patent applications listed in Appendix A; United States patents issued from the applications listed in Appendix A and from divisionals and continuations of these applications and any reissues of
such United States patents; claims of continuation-in-part applications and patents directed to subject matter specifically described in the applications listed in Appendix A; and claims of all foreign patent applications, patents, and other
intellectual property which are directed to subject matter specifically 

 
described in the United States and/or patent applications listed in Appendix A. Notwithstanding the foregoing, Company may elect within the first [**] days after the Effective Date to exclude any
issued patents and/or patent applications from the Patent Rights by giving written notice to CHOP of election; provided that such patents and/or patent application so removed shall be deemed to be removed from this Agreement ab initio and
never to have been part of this Agreement. 
  

	 	3.	Section 3.1 is hereby amended to read, in its entirety, as follows: 

 CHOP hereby grants
and Company accepts, subject to the terms and conditions of this Agreement, (i) a worldwide exclusive license in the Licensed Field, with the right to sublicense, to use and practice the Patent Rights and Gene Therapy Know-How, and (ii) a
worldwide non-exclusive license in the Licensed Field, with the right to sublicense, to use and practice the Know-How, in the case of both (i) and (ii) to research, develop, make, have made, practice, use, import, lease, offer for sale,
sell, and sublicense the Licensed Products within the Licensed Field (the “License”), subject, however, (a) to a reservation of rights by CHOP to research, make, have made, practice, have practiced, and use the Know How for any
purpose with no exclusions or exceptions and the CHOP Patent Rights and Gene Therapy Know-How solely for its own academic and clinical research, and/or educational purposes, and use by other academic and non-profit entities for academic and clinical
research, and/or educational purposes, excluding (I) use pursuant to any sponsored research or other funding agreement or arrangement with any commercial entity pursuant to which any commercial entity is granted any right or interest with
respect to the Patent Rights and Gene Therapy Know-How or research results generated through the use thereof, (II) the inclusion in any NDA, BLA or other application for marketing approval of any data comprised by the Patent Rights and Gene Therapy
Know-How and (III) any use of the Patent Rights and Gene Therapy Know-How for commercialization or licensing or transfer of rights therein for commercialization and (b) to any applicable reservation of rights by the U.S. government. 

 

	 	4.	Section 3.3 is hereby added to the Agreement, as follows: 

 Company acknowledges that it
has been informed that certain of the CHOP Intellectual Property was developed, at least in part, by employees of HHMI and that HHMI has a paid up, non-exclusive, irrevocable license to use such CHOP Intellectual Property for HHMI’s research
purposes, but with no right to assign or sublicense (the “HHMI License”). This license is explicitly made subject to the HHMI License. 

  
 2 

	 	5.	Section 3.4 is hereby added to the Agreement, as follows: 

 CHOP retains the rights for
academic and non-profit entities to research, make, have made, practice, have practiced, and use the CHOP Intellectual Property for academic and research purposes. 
  

	 	6.	Section 4.2 is hereby amended by adding the following sentence to the end of the Section: 

Each sublicensee must be subject to a written agreement that contains obligations, terms and conditions in favor of HHMI or the HHMI
Indemnitees, as applicable, that are substantially similar to those undertaken by Company in favor of HHMI or the HHMI Indemnitees, as applicable, under this Agreement and intended for the protection of the HHMI Indemnitees, including, without
limitation, the obligations, terms and conditions regarding indemnification, insurance and HHMI’s third party beneficiary status. 
  

	 	7.	Section 11.7 is hereby added to the Agreement, as follows: 

 HHMI and its trustees,
officers, employees, and agents (collectively, “HHMI Indemnitees”), will be indemnified, defended by counsel acceptable to HHMI, and held harmless by Company from and against any claim, liability, cost, expense, damage, deficiency, loss,
or obligation, of any kind or nature (including, without limitation, reasonable attorneys’ fees and other costs and expenses of defense) (collectively, “Claims”), based upon, arising out of, or otherwise relating to this Agreement,
the Assignment Agreement, or any sublicense, including without limitation any cause of action relating to product liability. The previous sentence will not apply to any Claim that is determined with finality by a court of competent jurisdiction to
result solely from the gross negligence or willful misconduct of an HHMI Indemnitee. Notwithstanding any other provision of this Agreement, Company’s obligation to defend, indemnify and hold harmless the HHMI Indemnitees under this paragraph
will not be subject to any limitation or exclusion of liability or damages or otherwise limited in any way. 
  

	 	8.	Section 14.7 is hereby amended by adding the following sentence to the end of the Section: 

Such assignment will only be valid if the recipient of the assignment agrees in writing to CHOP to be bound by the obligations of this
Agreement as if it were Company. 
  

	 	9.	Section 14.13 is hereby amended by adding the following sentence to the end of the Section: 

Company acknowledges that under HHMI policy, Company may not use the name of HHMI or of any HHMI employee in a manner that reasonably could
constitute an endorsement of a commercial product or service; but that use for other purposes, even if commercially motivated, is permitted provided that (1) the 

  
 3 

 
use is limited to accurately reporting factual events or occurrences, and (2) any reference to the name of HHMI or any HHMI employees in press releases or similar materials intended for
public release is approved by HHMI in advance. 
  

	 	10.	Section 14.15 is hereby amended to read, in its entirety, as follows: 

 Paragraphs
3.2, 4.4, 7.1, 7.3, 10.5, 11.6, 11.7, 12.1, 12.6, 13.5, 14.5, 14.11, 14.13, and 14.16 of this Agreement shall survive termination of this Agreement. 
  

	 	11.	Section 14.16 is hereby added to the Agreement, as follows: 

 HHMI is not a party to the
Agreement and has no liability to any licensee, sublicensee, or user of anything covered by this Agreement, but HHMI is an intended third-party beneficiary of this Agreement and certain of its provisions are for the benefit of HHMI and are
enforceable by HHMI in its own name. 
  

	 	12.	Section 14.17 is hereby added to the Agreement, as follows: 

 In the event that CHOP or
Company determines that any patents or patent applications other than those described on the appendices hereto claim inventions relating to the Gene Therapy Know-How or Assigned Intellectual Property, which inventions were conceived or reduced to
practice prior to the Effective Date, CHOP and Company shall amend this Agreement to include such patents or patent applications under this Agreement. 

  
 4 

 IN WITNESS WHEREOF, the parties have hereunto set their hands and duly executed this Amendment to
License Agreement on the date set forth below. 
  

									
	The Children’s Hospital of Philadelphia	 	 	 	Spark Therapeutics, Inc.
					
	By:	 	 /s/ Thomas J. Todorow
	 		 	By:	 	 /s/ Joseph La Barge

					
	Title:	 	 Executive Vice President and CFO
	 		 	Title:	 	 GC & Head of Business Administration

					
	Date:	 	 12/5/14
	 		 	Date:	 	 December 5, 2014

  
 5EX-10.15

					
		 	 Confidential Materials omitted and filed separately with the

Securities and Exchange Commission. Double asterisks denote omissions.
	 	Exhibit 10.15

 LICENSE AGREEMENT 

between 
 GENABLE
TECHNOLOGIES LIMITED 
 and 

SPARK THERAPEUTICS, LLC 

 This License Agreement (the “Agreement”) is entered into this 18th day of March, 2014 (the “Effective Date”), by and between Genable Technologies Limited, organized and existing under the laws of Ireland, and having a principal place of
business at Media House, South County Business Park, Leopardstown, Dublin 18, Ireland (“Genable”) and Spark Therapeutics, LLC, organized and existing under the laws of Delaware, USA, and having a principal place of business at 3501
Civic Center Boulevard, Philadelphia, PA 19104, USA (“Spark”). 
 Genable and Spark may collectively be referred to as the
“Parties” and each a “Party”. 
 Spark and Genable agree as follows: 

 

	1.	BACKGROUND 

  

	 	1.1	Pursuant to agreements dated October 14, 2013, Spark has acquired or licensed from The Children’s Hospital of Philadelphia (“CHOP”) certain patent rights and confidential and trade secret
information relating to the manufacture of adeno-associated virus vectors as therapeutic agents for various indications and conditions. 

  

	 	1.2	Genable desires to obtain from Spark an exclusive right and license under such patent rights and confidential and trade secret information for the development and commercialization of therapeutic agents for the
treatment of rhodopsin-linked, autosomal dominant retinitis pigmentosa (“RHO-adRP”). Spark is willing to grant such right and license to Genable. 

 

	 	1.3	Simultaneous with this Agreement, Spark and Genable have entered into the Development Agreement and the Manufacturing Agreement. 

  

	2.	DEFINITIONS 

  

	 	2.1	“Affiliate(s)” shall mean any corporation, firm, partnership or other entity, which controls, is controlled by, or is under common control with, a Party. For purposes of this Paragraph 2.1,
“control” shall mean direct or indirect ownership of fifty percent (50%) or more of the outstanding stock or other voting rights entitled to elect directors thereof or the ability to otherwise control the management of such
corporation, firm, partnership or other entity. Notwithstanding the foregoing, CHOP shall be deemed not to be an Affiliate of Spark. 

  

	 	2.2	“Commercially Reasonable Efforts” means the carrying out of applicable obligations under this Agreement in a commercially reasonable manner in good faith using all such efforts and resources consistent
with the practice of comparable biological development companies of a similar size and resources, both financial and otherwise, to those of Genable, that would be used by such companies were they developing a comparable biological product;

  

	 	2.3	“CHOP License Agreement” means the License Agreement dated as of October 14, 2013, by and between Spark (f/k/a AAVenue Therapeutics, LLC) and CHOP. 

 

	 	2.4	 “Clinical Trial” means an investigation in human subjects and/or patients intended to discover or verify the clinical,
pharmacological and/or other pharmacodynamic effects of a Licensed Product, and/or to identify any adverse reactions to a 

  
 2 

	 	
Licensed Product, and/or to study absorption, distribution, metabolism, and/or excretion of a Licensed Product with the objective of ascertaining its safety, activity and/or efficacy.

  

	 	2.5	“Confidential Know-How” means any and all rights, other than Patent Rights, in any scientific, pharmaceutical or technical information, know-how, discovery, invention, process, procedure, composition,
method, formula, protocol, technique, or data owned or controlled by Spark and maintained in confidence or as a trade secret by Spark which are not covered by the Patent Rights and which are used for practicing or manufacturing the Licensed Products
and/or relating to the Licensed Field. Confidential Know-How includes but is not limited to the information set out in Appendix A. 

  

	 	2.6	“Development Consultancy Agreement” means the development consultancy agreement of even date entered into by the Parties, subject to terms and conditions set out therein. 

 

	 	2.7	“EMA” means the European Medicines Agency or any successor agency thereof having the authority to regulate the sale of medicinal or pharmaceutical products in the European Union through marketing
approval, not including any governmental authority with responsibility solely for pricing or reimbursement approvals. 

  

	 	2.8	“FDA” means the United States Food and Drug Administration or any other successor agency whose approval is necessary to market the Licensed Product in the USA. 

 

	 	2.9	“First Commercial Sale” means the first sale during a full scale commercial launch by or on behalf of Genable or its sublicensees of Licensed Products in an arm’s length transaction to an
independent third party in any country in the Territory after all applicable required Regulatory Approvals in such country, in exchange for cash or some cash equivalent to which value can be assigned for the purpose of determining Net Sales.

  

	 	2.10	“GT038” means a gene therapeutic comprising an AAV vector containing DNA encoding an RNAi targeting rhodopsin in combination with an AAV vector containing DNA encoding a rhodopsin gene for the treatment
of RHO-adRP, which is in development by Genable as of the Effective Date, and as such gene therapeutic may be modified after the Effective Date. 

  

	 	2.11	“Improvements” means any and all improvements, developments, adaptions, enhancements, alterations or modifications to the Spark Intellectual Property, whether or not patentable, which are either made,
owned or licensed by or on behalf of Spark. 

  

	 	2.12	“Licensed Field” means adeno-associated virus (“AAV”) based therapeutic agents for the treatment of RHO-adRP. 

 

	 	2.13	 “Licensed Products” means any product incorporating GT038 in the Licensed Field sold by or on behalf of Licensee, its Affiliates,
licensees or its sublicensees, the manufacture, use or sale of which is covered by a claim of the Patent Rights or utilizes Confidential Know-How of Spark in the country of such manufacture, use or

  
 3 

	 	
sale but for the license granted herein. For the avoidance of doubt, all products supplied by Spark to Genable pursuant to the Manufacturing Agreement shall be deemed to utilize Confidential
Know-How of Spark. 

  

	 	2.14	“Licensed Territory” means worldwide. 

  

	 	2.15	“Manufacturing Agreement” means the manufacturing agreement of even date entered into by the Parties pursuant to which Spark is appointed the exclusive manufacturer of the Licensed Product for Genable,
subject to terms and conditions set out therein. 

  

	 	2.16	“Net Sales” means the total gross receipts invoiced by Genable, its Affiliates, licensees and sublicensees for sales, including transfers of Licensed Products to others for value or making Licensed
Products available to others for value, of Licensed Products by or on behalf of Genable, its Affiliates, licensees or sublicensees, less: 

  

	 	(a)	sales returns and allowances actually given to third parties, including, trade, quantity and cash discounts and other adjustments (retroactive or otherwise), including, but not limited to, those granted on account of
price adjustments, billing errors, rejected goods, damaged or defective goods, recalls, returns, rebates, stocking allowances, reimbursements or similar payments actually made to customers, wholesalers or distributors, provided however that any
discretionary rebates, discounts, adjustments or similar payments shall be commercially reasonable and consistent with standard industry practices; 

  

	 	(b)	insurance and freight charges and transportation costs actually paid to third parties for the shipment of Licensed Products; 

  

	 	(c)	customs or excise duties, sales tax, consumption tax and other taxes (except income taxes) or duties relating to sales of Licensed Products to third parties that are actually paid by Genable; and 

 

	 	(d)	invoiced amounts that are subsequently written off as uncollectible, provided that if any such amounts are collected after having been written off, such amounts shall thereupon be reincluded in Net Sales.

 No deductions shall be made for commissions paid to any third party or individual (whether they be with independent sales
agencies or regularly employed by Genable, its Affiliates, licensees or sublicensees, and on its payroll) or for the cost of collections. 

Notwithstanding the foregoing, “Net Sales” shall not include amounts (i) for any Licensed Product furnished to a third party
for use in Clinical Trials, for compassionate use or as promotional samples, in either case for which payment (other than the cost of the Licensed Product) is not intended to be received or (ii) from sales or other dispositions of Licensed
Products among Licensee and any of its Affiliates, licensees or sublicensees, unless the Affiliate, licensee or sublicensee, as the case may be, is an end-customer of such Licensed Product. 

  
 4 

	 	2.17	“Patent Rights” means all patent applications and/or patents, now existing, currently pending, or, if associated with Improvements, hereinafter filed, owned and/or controlled by Spark, with respect to
the manufacture, purification, process development and preparation of proviral plasmids and recombinant AAV, including the application and/or patents set forth on Appendix A, and any foreign counterparts thereof and all patents issuing therefrom, as
well as any continuations, continuations-in-part, divisions, reexaminations, reissues, substitutes, renewals or extensions thereof, and any foreign counterparts thereof. 

 

	 	2.18	“Regulatory Application” means any regulatory application or any other application for marketing approval for the Licensed Product, which Licensee will file in the Territory, including any supplements
or amendments thereto which Licensee may file. 

  

	 	2.19	“Regulatory Approval” means the final approval to market the Licensed Product in any country of the Territory, including pricing and reimbursement approval and any other approval which is required to
launch the Licensed Product in the normal course of business. 

  

	 	2.20	“Spark Intellectual Property” means the Patent Rights and Confidential Know-How and Improvements. 

  

	 	2.21	“Year” means each twelve-month period beginning on January 1 and ending on 31 December during the term of this Agreement. 

 

	 	2.22	“$” means United States Dollars. 

  

	3.	GRANT OF RIGHTS 

  

	 	3.1	Spark hereby grants and Genable accepts, subject to the terms and conditions of this Agreement, a worldwide, royalty-bearing exclusive license (even as to Spark) to the Spark Intellectual Property with the right to
sublicense, to research, develop, import, use, have used, make and have made, market, offer for sale, distribute, sell, and have sold, the Licensed Products within the Licensed Field in the Licensed Territory (the “License”).

  

	 	3.2	Pursuant to the Manufacturing Agreement, Genable shall appoint Spark as the exclusive manufacturer of the Licensed Product. Genable shall not exercise its license to manufacture Licensed Products other than to the
extent permitted by the Manufacturing Agreement. 

  

	 	3.3	Spark will provide a third party nominated by Genable and reasonably acceptable to Spark, pursuant to an appropriate material custody agreement, with sufficient cells from Spark’s Master Cell Bank (MCB) comprised
of [**] cells and any additional Working Cell Banks (WCB) that may be used by Spark for the manufacture of Licensed Product(s) in the future, in order that the third party may established a second MCB in order to comply with regulatory requirements.
Subject to the terms of the Manufacturing Agreement, Genable shall be entitled to have the third party transfer this second MCB to a third party contract manufacturer in order to transfer the manufacturing process(es) for the Licensed Product(s) to
a third party contract manufacturer reasonably acceptable to Spark in accordance with an appropriate material custody agreement between the third party contract manufacturer and Spark. 

  
 5 

	 	3.4	For the avoidance of doubt, Spark shall not license to, nor permit the use by, any third party of the Spark Intellectual Property for the sale or distribution of Licensed Products in the Licensed Field in the Licensed
Territory. 

  

	 	3.5	Spark acknowledges that Genable has considerable proprietary know-how and data related to GT038 and has filed patent applications and obtained patents world-wide in relation to GT038. Nothing in this Agreement shall
grant Spark any rights of any nature whatsoever to GT038, provided that, Genable hereby grants to Spark a license to such know-how, data and patent rights, on a non-exclusive, non-sub-licensable, non-royalty-bearing basis solely to the extent
necessary for Spark to perform its obligations under the Manufacturing Agreement and the Development Consultancy Agreement. 

  

	 	3.6	The Licensed Field may be expanded or modified by mutual written agreement between the Parties. 

  

	 	3.7	Spark will in good faith promptly disclose in writing to Genable any Improvements having application to Licensed Products in the Licensed Field. Any such Improvement shall be automatically included in the Spark
Intellectual Property licensed to Genable Licensee pursuant to Paragraph 3.1 of this Agreement. 

  

	 	3.8	Genable’s rights under this Agreement with respect to Patent Rights licensed by Spark from CHOP shall be subject to Genable’s satisfaction, as a sublicensee of Spark under the CHOP License Agreement, of the
obligations set forth on Appendix B. Such obligations shall be binding upon Genable with respect to the Patent Rights licensed by Spark from CHOP to the extent Spark is required to impose such obligations on its sublicensees pursuant to the CHOP
License Agreement. For the avoidance of doubt, (a) the “Company” referred to in the language from the CHOP License Agreement quoted in Appendix B is Spark, but under the CHOP License Agreement Spark is obligated to impose its
obligations under the quoted provisions on its sublicensees to the same extent such obligation apply to Spark and (b) the Patent Rights identified in Appendix A are licensed by Spark from CHOP and are therefore subject to this
Paragraph 3.8. 

  

	4.	SUBLICENSING 

  

	 	4.1	Genable may exercise its rights under this Agreement by an Affiliate and any action of such Affiliate shall be deemed for all purposes to be an action of Genable. 

 

	 	4.2	Genable may enter into sublicensing agreements for the Licensed Products to and only to the extent of any license granted to Genable under this Agreement. 

 

	 	4.3	Genable agrees that any sublicenses granted by it shall contain provisions which are equivalent to Paragraphs 3.8, 4.5 and 7.1 of this Agreement. 

 

	 	4.4	Genable’s execution of a sublicense agreement will not relieve Genable of any of its obligations under this Agreement. Genable is primarily liable to Spark for any act or omission of an Affiliate or sublicensee of
Genable that would be a breach of this Agreement if performed or omitted by Genable, and Genable will be deemed to be in breach of this Agreement as a result of such act or omission. 

 

	 	4.5	Termination of the license granted to Genable by Spark under this Agreement will terminate all sublicenses which may have been granted by Genable. 

  
 6 

	5.	PAYMENTS 

  

	 	5.1	In consideration of the license of the Spark Intellectual Property, Genable shall pay to Spark a once-off upfront non-refundable fee of twenty thousand dollars ($20,000), due within [**] days of the Effective Date.

  

	 	5.2	In further consideration of the license of the Spark Intellectual Property, Genable shall pay to Spark the applicable milestone payments listed in the table below within [**] days after each milestone event for the
first Licensed Product is achieved by Genable, its Affiliates, licensees or sublicensees: 

  

			
	[**]	  	[**]
	[**]	  	[**]
	[**]	  	[**]

  

	 	5.3	For the avoidance of doubt: 

  

	 	(i)	each of the milestone payments set out in Paragraph 5.2 shall be payable no more than once; 

  

	 	(ii)	all clinical milestones refer to [**]; 

  

	 	(iii)	the Licensee shall have no obligation to pay any milestones other than those listed in Paragraph 5.2; and 

  

	 	(iv)	none of the foregoing milestones may be skipped. If a milestone is not achieved for any reason, but the subsequent milestone is achieved, the prior unachieved milestone shall be deemed achieved and the corresponding
milestone payment shall be payable with the milestone payment for the achieved subsequent milestone. In addition, with respect to the [**] milestone, if a [**]. 

  

	 	5.4	In consideration of the license of the Spark Intellectual Property, the royalty payable by Genable to Spark on Net Sales of the Licensed Product shall be calculated by reference to the table set out below:

  

			
	 Annual Net Sales
	  	 Applicable Royalty Rate

	First $[**]	  	[**]% of Net Sales
	Increments above $[**]	  	[**]% of Net Sales

  
 7 

	 	5.5	Sublicense Revenues: 

  

	 	5.5.1	For any licenses or sublicenses granted by Genable during the term of this Agreement, Genable shall pay to Spark the milestones and royalties according to this Agreement on milestone achievements and Net Sales of the
Licensed Products by Affiliates, licensee(s) and sublicensee(s) as if such milestone achievements and sales were milestone achievements and Net Sales from Licensed Products by Genable. 

 

	 	5.5.2	For the avoidance of doubt, any payments received by Genable from a licensee or sublicensee such as for the funding of research and/or development, or for the granting of any commercialization rights including any
milestone or other upfront payments, shall not be considered to be part of Net Sales. 

  

	 	5.6	No multiple royalties shall be payable because any Licensed Products are covered by more than one patent, or patent application of the Patent Rights. 

 

	 	5.7	On sales of Licensed Products by Genable or Affiliates, licensees or sublicensees made other than in an arm’s-length transaction, the value of the Net Sales attributed under this Article 5 to such a transaction
shall be that which would have been received in an arm’s-length transaction, based on sales of like quantity and quality products on or about the time of such transaction. 

 

	 	5.8	The payments due to Spark from Genable pursuant to Paragraphs 5.1 and 5.2 shall be payable as set forth in such Paragraphs. Royalty payments due to Spark from Genable pursuant to Paragraph 5.3 shall be payable within
[**] days of the end of the calendar quarter in which the applicable Net Sales occur. 

  

	 	5.9	Payments made by Genable to Spark shall be delivered by wire transfer in U.S. Dollars (unless otherwise specifically agreed by the parties in writing) to the designated bank account of Spark in accordance with such
timely written instructions as Spark shall from time to time provide. 

  

	6.	PATENT FILING, PROSECUTION, AND MAINTENANCE 

  

	 	6.1	Spark shall control the preparation, filing, prosecution, and maintenance of any and all patent applications or patents included in the Patent Rights and shall furnish copies of relevant patent-related documents to
Genable. 

  

	 	6.2	Spark shall have sole discretion whether to prepare, file, prosecute and maintain any and all patent applications or patents included in the Patent Rights in the Territory. 

 

	 	6.3	If at any time during the term of this Agreement Genable opposes or contests the grant or validity of the Patent Rights licensed herein, or any claims thereof, Spark will be entitled to terminate the license granted to
Genable under Paragraph 3.1 of this Agreement, upon thirty (30) days’ prior written notice to Genable. 

  

	7.	RECORD KEEPING 

  

	 	7.1	 Genable agrees to keep accurate and correct records of Licensed Products appropriate to determine the amount of royalties due Spark. Such records
shall be 

  
 8 

	 	
retained for at least [**] years following a given reporting period. The records shall be available, [**], during normal business hours for inspection at the expense of Spark by an accountant or
other designated auditor selected by Spark (and reasonably acceptable to Genable) for the sole purpose of verifying reports and payments hereunder. Genable may only object to an auditor selected by Spark for good cause shown. The accountant or
auditor shall only disclose to Spark information relating to the accuracy of reports and payments made under this Agreement. If an inspection shows an underreporting or underpayment in excess of [**] percent ([**]%) for any twelve (12) month
period, then Genable shall reimburse Spark for the reasonable cost of the inspection at the time Genable pays the unreported royalties, including any late charges as required by Paragraph 7.2 of this Agreement. All payments required under this
Paragraph 7.1 shall, if not disputed by Genable, be due within [**] days of the date Spark provides Genable notice of the payment due. 

  

	 	7.2	Late charges will be assessed by Spark on any undisputed overdue payments, and on all disputed overdue payments that are determined not to have been correctly disputed, at a rate of [**] percent ([**]%) per month. The
payment of such late charges shall not prevent Spark from exercising any other rights it may have as a consequence of the lateness of any payment. 

  

	8.	PERFORMANCE REQUIREMENTS AND REPORTS 

  

	 	8.1	Genable will use Commercially Reasonable Efforts to develop, bring to market and commercialize the Licensed Products through a diligent program of development, marketing and commercialization, in both [**]. Within [**]
of the Effective Date, Genable will prepare an outline development plan for developing and bringing the Licensed Product to market in both [**] (the “Commercial Development Plan”) and provide a copy to Spark. The Commercial
Development Plan may be changed by Genable from time to time based on medical, regulatory and commercial considerations then pertaining. 

  

	 	8.2	Genable shall report to Spark the date of the First Commercial Sale in each country in the Licensed Territory within [**] days of such occurrence. 

 

	 	8.3	Genable shall submit to Spark within [**] days after each calendar quarter ending March 31, June 30, September 30, and December 31 a royalty report setting forth for the preceding quarterly
period the amount of the Licensed Products sold by or on behalf of Genable or by an Affiliate, licensee or sublicensee in each country within the Licensed Territory, the Net Sales, and the amount of royalty or other payment accordingly due. With
each such royalty report, Genable shall submit payment of the earned royalties due. If no earned royalties are due to Spark for any reporting period, the written report shall so state. The royalty report shall be certified as correct by an
authorized officer of Genable and shall include a detailed listing of all deductions made under Paragraph 2.15 to determine Net Sales made under Article 6 to determine royalties due. 

 

	 	8.4	 Royalties due under Article 5 shall be paid in U.S. dollars. For conversion of foreign currency to U.S. dollars, the conversion rate shall be the New
York foreign exchange rate quoted in The Wall Street Journal on the day that the payment is due. Any loss of exchange, value taxes, or other expenses incurred in the transfer

  
 9 

	 	
or conversion to U.S. dollars shall be paid entirely by Genable. The royalty report required by Paragraph 8.3 of this Agreement shall accompany each such payment and a copy of such report shall
also be mailed to Spark at its address for notices indicated in Paragraph 14.6 of this Agreement. 

  

	 	8.5	All plans and reports required by this Article 8 and marked confidential by Genable shall be treated by Spark as commercial and financial information obtained from a person and as privileged and confidential.

  

	 	8.6	If applicable laws of Ireland require that taxes be withheld with respect to any payments by Genable to Spark under this Agreement, Genable will: (a) deduct those taxes from the remittable payment, (b) pay the
taxes to the proper taxing authority, and (c) send evidence of the obligation together with proof of tax payment to Spark on a timely basis following that tax payment. If Spark is a taxable entity in the United States and is therefore entitled
to the benefits of the double taxation treaty between Ireland and the United States, and Spark provides Genable with a Form 6166 from the United States Internal Revenue Service with respect to such taxable status, at or prior to the time of any
payment potentially subject to the Irish withholding tax is made hereunder, then payments made by Genable to Spark hereunder shall be made without withholding tax; provided that, if such double taxation treaty is modified after the Effective Date so
that payments to Spark hereunder are subject to withholding taxes, Genable shall give notice to Spark of such change and shall pay to Spark such additional amount as may be necessary so that Spark shall receive, after deduction of such withholding
tax, the amount which Spark would have received in the absence of such withholding tax less [**] percent ([**]%) of the withholding tax amount (i.e., the Parties [**] percent ([**]%) of the withholding tax amount). If Spark is not able to meet the
above criteria for withholding tax treaty benefits (e.g., by ceasing to be, or by assigning its interest in this Agreement to an entity that is not, a taxable entity in the United States entitled to the benefits of the double taxation treaty between
Ireland and the United States), then Genable shall make payments less any required withholding tax, and such withholding taxes required under Irish law shall be borne solely by Spark. If Genable or any successor or assign of Genable makes any
payment to Spark hereunder in a manner that subjects such payment to a withholding tax obligation under the laws of any jurisdiction other than those of Ireland (i.e., either by such entity being or becoming domiciled in any jurisdiction other than
Ireland or by such entity making any payment to Spark from a jurisdiction outside of Ireland), then Genable shall give notice to Spark of such requirement and shall pay to Spark such additional amount as may be necessary so that Spark shall receive,
after deduction of such withholding tax, the amount which Spark would have received in the absence of such withholding tax. Each Party agrees to cooperate with the other Party in claiming refunds or exemptions from such deductions or withholdings
under any relevant agreement or treaty which is in effect (e.g., Genable shall not withhold Irish withholding tax without first confirming with Spark that Spark is not able to provide the documentation of its taxable status as described above). The
Parties shall discuss and cooperate regarding applicable mechanisms for minimizing such taxes to the extent possible in compliance with applicable law. In addition, the Parties shall cooperate in accordance with applicable law to minimize indirect
taxes (such as value added tax, sales tax, consumption tax and other similar taxes) in connection with this Agreement. 

  
 10 

	9.	INFRINGEMENT AND PATENT ENFORCEMENT 

  

	 	9.1	Spark and Genable agree to notify each other promptly of each infringement or possible infringement of the Patent Rights within the Licensed Field, as well as any facts which may affect the validity, scope, or
enforceability of the Patent Rights of which either Party becomes aware. 

  

	 	9.2	If infringement of the Patent Rights occurs, or if infringement may occur, Spark shall have sole discretion to charge a third party with infringement and shall have the sole discretion to institute an infringement
action. Spark may decline to charge infringement or institute an infringement action at Spark’s sole discretion. If Spark elects to charge a third party with infringement or elects to institute an infringement action, Spark shall bear the cost
of such action and shall retain all recovery. 

  

	 	9.3	If Spark elects not to charge a third party with infringement or elects not to institute an infringement action (such decision to be made promptly by Spark), Genable shall, subject to the terms of the CHOP License
Agreement with respect to Patent Rights licensed from CHOP, if applicable, have the right (but not the obligation) to bring such suit. If Genable so elects to charge a third party with infringement or to institute an infringement action, Genable
shall bear all costs in such action or any resulting defense or declaratory judgment action for non-infringement, invalidity or unenforceability of the Patent Rights and Genable shall not settle any such action in a manner that imposes any liability
on Spark or imposes a material detriment to any Patent Rights without express permission by Spark (such permission not to be unreasonably withheld, delayed or conditioned). If Genable elects to charge a third party with infringement or elects to
institute an infringement action, Genable shall, after payment to CHOP of any share thereof payable to CHOP pursuant to the CHOP License Agreement with respect to Patent Rights licensed from CHOP, if applicable, retain all recovery.

  

	 	9.4	In any action instituted by a third party to contest the validity or unenforceability of the Patent Rights, Spark shall have the sole discretion to defend such action at its own expense. If Spark desires not to defend
said action, Genable shall, subject to the terms of the CHOP License Agreement with respect to Patent Rights licensed from CHOP, if applicable, have the right (but not the obligation) to defend such suit. If Genable so decides to defend them such
defense shall be conducted by Genable at Genable’s sole expense and Genable shall not settle any such action in a manner that imposes any liability on Spark or imposes any material detriment to any Patent Rights without express permission by
Spark (such permission not to be unreasonably withheld, delayed or conditioned). If Genable elects to defend such an action, Genable shall, after payment to CHOP of any share thereof payable to CHOP pursuant to the CHOP License Agreement with
respect to Patent Rights licensed from CHOP, if applicable, retain all recovery. 

  

	 	9.5	In any litigation under this Article 9, either Party, at the request and sole expense of the other Party, will cooperate to the fullest extent reasonably possible. This Paragraph 9.5 will not be construed to require
either Party to undertake any activities, including legal discovery, at the request of any third party, except as may be required by lawful process of a court of competent jurisdiction. If, however, either Party is required to undertake any
activity, including legal discovery, as a right of lawful process of a court of competent jurisdiction, then the litigating Party will pay all expenses incurred by the other Party. 

  
 11 

	10.	WARRANTIES AND INDEMNIFICATION 

  

	 	10.1	Spark and Genable offer no warranties other than those specified in this Article 10. 

  

	 	10.2	Spark represents and warrants to Genable that, as of the Effective Date: 

  

	 	(a)	the issued Patent Rights are not invalid or unenforceable, to Spark’s present knowledge, 

  

	 	(b)	Spark has the right to grant to Genable the license specified in Article 3 of this Agreement, 

  

	 	(c)	Spark owns or has a valid license under any United States and foreign patent applications or patents corresponding to the Patent Rights, 

 

	 	(d)	this Agreement has been duly executed and delivered by a duly authorized officer of Spark and constitutes the valid and legally binding obligations of Spark enforceable against Spark according to its terms except as
enforceability of this Agreement may be limited by applicable bankruptcy, insolvency, reorganization, moratorium or other similar laws affecting creditors’ rights generally, and 

 

	 	(e)	no proceedings or written notices have been received by Spark from any third party alleging that the Spark Intellectual Property infringes any third party intellectual property. 

 

	 	10.3	Spark further represents and warrants to Genable as of the Effective Date: 

  

	 	(a)	the CHOP License Agreement is valid and in full force and effect; and 

  

	 	(b)	there are no existing or claimed defaults by Spark, and to Spark’s best knowledge, by CHOP under the CHOP License Agreement, and no event, act or omission has occurred that (with or without notice, lapse of time or
the happening or occurrence of any other event) would result in a default under the CHOP License Agreement. 

  

	 	10.4	Spark covenants to Genable that during the Term: 

  

	 	(a)	Spark will fully comply with all of the terms and conditions of the CHOP License Agreement and will reasonably enforce its rights under the CHOP License Agreement and Spark will not assign its rights under the CHOP
License Agreement except where this Agreement is also being assigned in accordance with Paragraph 14.7, provided that, Spark shall not be responsible for any failure to comply with the CHOP License Agreement that results from Genable’s
failure to fully comply with all of the terms and conditions of this Agreement; and 

  
 12 

	 	(b)	Spark will keep Genable reasonably informed with respect to Spark’s transactions, arrangements and business under the CHOP License Agreement that relate to Genable and/or the transactions contemplated hereunder,
and Spark shall provide Genable with any written notices delivered by Spark or CHOP as appropriate thereunder that relate to Genable and/or the obligations or rights under this Agreement that may adversely affect Genable; and 

 

	 	(c)	Spark shall not amend, modify, or waive any of its rights under the CHOP License Agreement, if such amendment, modification or waiver would adversely affect Genable and/or Genable’s rights and/or obligations or
under this Agreement, without the prior written consent of Genable. For the avoidance of doubt, Spark shall not terminate any of its rights under the CHOP License Agreement without the prior written consent of Genable if such termination would
adversely affect Genable and/or Genable’s rights and/or obligations or under this Agreement. 

  

	 	10.5	Save for the warranties set forth in Paragraphs 10.2 and 10.3, Spark does not warrant the validity of the Patent Rights and makes no representations whatsoever with regard to the scope of the Patent Rights, or that the
Patent Rights may be exploited without infringing other patents or other intellectual property rights of third parties. 

  

	 	10.6	NO WARRANTIES, EXPRESS OR IMPLIED, ARE OFFERED BY SPARK AS TO THE FITNESS FOR ANY PURPOSE OF THE MATERIALS OR INFORMATION PROVIDED TO GENABLE UNDER THIS AGREEMENT, OR THAT THE SPARK INTELLECTUAL PROPERTY MAY BE
EXPLOITED WITHOUT INFRINGING OTHER THIRD PARTY RIGHTS. GENABLE ACCEPTS THE SPARK INTELLECTUAL PROPERTY, INFORMATION AND THE MATERIALS “AS IS,” AND SPARK DOES NOT OFFER ANY GUARANTEE OF ANY KIND. 

 

	 	10.7	Spark does not represent that it will commence legal actions against third parties infringing the Spark Intellectual Property. 

  

	 	10.8	Genable warrants that to the best of its knowledge it has the necessary patent rights, licenses and other intellectual property required to develop GT038. 

 

	 	10.9	Genable represents and warrants to Spark that this Agreement has been duly executed and delivered by a duly authorized officer of Genable and constitutes the valid and legally binding obligations of Genable enforceable
against Genable according to its terms except as enforceability of this Agreement may be limited by applicable bankruptcy, insolvency, reorganization, moratorium or other similar laws affecting creditors’ rights generally. 

 

	 	10.10	 Genable shall indemnify and hold Spark, its employees, affiliates, agents, and consultants harmless from and against all liability, demands, damages,
expenses, and losses, including but not limited to, death, personal injury, illness, or property damage to the extent arising in connection with any activities related to Licensed Products under this Agreement by Genable, its directors, employees,
agents and its sublicensees, including any of their customers, patients, or end-users, including, without limitation, the research, development, importation, exportation, 

  
 13 

	 	
design, manufacture, distribution, offering for sale, sale, or use of any Licensed Products or in connection with any end user or clinical trial in which Genable participates utilizing Licensed
Products, except to the extent arising (i) in connection with any breach of this Agreement by Spark, or (ii) from the gross negligence or willful misconduct of Spark, its employees, affiliates, agents, and consultants. 

 

	 	10.11	Spark shall indemnify and hold Genable, its employees, affiliates, agents, and consultants harmless from and against all liability, demands, damages, expenses, and losses arising in connection with (i) any breach
of this Agreement by Spark, or (ii) the gross negligence or willful misconduct of Spark, its employees, affiliates, agents, and consultants. 

  

	 	10.12	Each Party shall when seeking an indemnity pursuant to Paragraphs 10.9 and 10.10 shall: 

  

	 	(a)	fully and promptly notify the indemnifying Party of any claim or proceedings, or threatened claim or proceedings; 

  

	 	(b)	permit the indemnifying Party to take full control of such claim or proceedings, with counsel of such indemnifying Party’s choice, provided that the indemnifying Party shall reasonably and regularly consult with
the indemnified Party in relation to the progress and status of such claim or proceedings; 

  

	 	(c)	co-operate in the investigation and defense of such claim or proceedings; and 

  

	 	(d)	take all reasonable steps to mitigate any loss or liability in respect of any such claim or proceedings. 

The indemnifying Party may settle a claim on terms which provide only for monetary relief and do not include any admission of liability. Save
as aforesaid, neither the indemnifying Party nor the Party to be indemnified shall acknowledge the validity of, compromise or otherwise settle any claim without the prior written consent of the other, which shall not be unreasonably withheld or
delayed. 
  

	 	10.13	NOTWITHSTANDING ANYTHING TO THE CONTRARY IN THIS AGREEMENT, NEITHER SPARK NOR GENABLE SHALL BE LIABLE TO THE OTHER BY REASON OF ANY REPRESENTATION OR WARRANTY, CONDITION OR OTHER TERM OR ANY DUTY OF COMMON LAW, OR UNDER
THE EXPRESS TERMS OF THIS AGREEMENT, FOR ANY INDIRECT, CONSEQUENTIAL, SPECIAL, INCIDENTAL OR PUNITIVE LOSS OR DAMAGE (OR FOR ANY LOSS OF CURRENT OR FUTURE PROFITS, LOSS OF ENTERPRISE VALUE) LOSS OF USE, LOSS OF SAVINGS OR ANTICIPATED SAVING, LOSS OF
GOODWILL, LOSS OF DATA OR LOSS OF BUSINESS OR ANTICIPATED BUSINESS, WHETHER OCCASIONED BY THE NEGLIGENCE OF THE RESPECTIVE PARTIES, THEIR EMPLOYEES OR AGENTS OR OTHERWISE. 

 

	 	10.14	Nothing in this Agreement shall have the effect of excluding or limiting any liability for death or personal injury caused by negligence or for fraud. 

  
 14 

	11.	CONFIDENTIALITY 

  

	 	11.1	Each Party undertakes with the other that it shall keep, and that it shall procure that its respective directors and employees keep secret and confidential all know-how, technical, business and other information that
has the quality of confidentiality and that is communicated to it by the other Party under or in respect of this Agreement or acquired from any other Party as a result of this Agreement (“Confidential Information”) and shall not
disclose the same or any part of the same to any person whatsoever SAVE THAT either Party may disclose Confidential Information to its Affiliates and sublicensees and any of its directors, employees or consultants who are directly or indirectly
legitimately involved with the Spark Intellectual Property and its exploitation and who require the said Confidential Information for the purposes of the said involvement. 

 

	 	11.2	The non-disclosure provision of Paragraph 11.1 shall not apply to: 

  

	 	(a)	Confidential Information in the public domain otherwise than by breach of this Agreement; 

  

	 	(b)	Confidential Information in the lawful possession of a Party prior to disclosure by any other Party as evidenced by written records; 

 

	 	(c)	Confidential Information that was created independent of disclosure as evidenced by written records; or 

  

	 	(d)	Confidential Information obtained from a third party who is free to divulge the same. 

  

	 	11.3	The obligations of each Party under this Article 11 shall continue in force notwithstanding the termination of this Agreement. 

  

	 	11.4	Any Confidential Information disclosed by the disclosing Party shall be used by the receiving Party exclusively for the purposes of fulfilling the receiving Party’s rights and obligations under this Agreement and
for no other purpose. 

  

	 	11.5	A Party (the “Required Party”) will be entitled to make a disclosure or public statement concerning the existence, subject matter or any term of this Agreement, or to disclose Confidential Information
that the Required Party is required to make or disclose pursuant to: 

  

	 	(a)	a valid order of a court or governmental authority; or 

  

	 	(b)	any other requirement of law or any securities or stock exchange; 

 provided that if the
Required Party becomes legally required to make such announcement, public statement or disclosure hereunder, the Required Party shall (to the extent possible) give the other Party prompt notice of such fact to enable the other Party to seek a
protective order or other appropriate remedy concerning any such announcement, public statement or disclosure, including confidential treatment and/or appropriate redactions. 
  

	 	11.6	The Required Party shall fully co-operate with the other Party in connection with that other Party’s efforts to obtain any such order or other remedy. If any such order or other remedy does not fully preclude
announcement, public statement or disclosure, the Required Party shall make such announcement, public statement or disclosure only to the extent that the same is legally required. 

  
 15 

	12.	TERM AND TERMINATION 

  

	 	12.1	This Agreement is effective beginning with the Effective Date and shall extend on a country-by-country basis until the later of a) the date of the expiration of the last to expire of the Patent Rights in the country of
sale or the country of manufacture or b) the tenth anniversary of the First Commercial Sale in the country of sale, unless sooner terminated as provided in this Article 12. 

 

	 	12.2	Upon termination of this Agreement, Genable’s rights under Article 3 of this Agreement shall cease, effective immediately. On a country-by-country basis, after this Agreement has expired pursuant to Paragraph 12.1,
if this Agreement shall not have earlier terminated, Genable’s license in such country of the Territory shall remain in force on a fully paid-up, non-royalty-bearing basis, provided, however, that Genable may still have obligations to Spark in
countries in which this Agreement has not expired. 

  

	 	12.3	In the event that Genable is in material default in the payment of any money to Spark under this Agreement, and if the default has not been remedied within [**] days after the date of notice in writing of such default,
Spark may terminate this Agreement by written notice, provided that, if Genable in good faith disputes any such amount, provides notice of such dispute to Spark, institutes dispute resolution pursuant to Article 13 and pays all undisputed amounts
prior to the end of such [**] day period, this Agreement shall not terminate if Genable pays all amounts finally determined to be payable in such dispute resolution within [**] days after such final determination. In the event that Genable is in
material default in the performance of any other obligations under this Agreement, and if the default has not been remedied within [**] days after the date of notice in writing of such default, Spark may terminate this Agreement by written notice.
Spark shall also have the unilateral right and option to terminate this Agreement under Paragraph 6.4 upon [**] days prior notice to Genable. 

  

	 	12.4	In the event that Genable becomes insolvent, files a petition in bankruptcy, has such a petition filed against it, determines to file a petition in bankruptcy, or receives notice of a third party’s intention to
file an involuntary petition in bankruptcy, Genable shall immediately notify Spark in writing and Spark shall thereupon have the right to terminate this Agreement upon thirty (30) days’ notice to Genable. 

 

	 	12.5	Genable shall have a unilateral right to terminate this Agreement and/or any licenses in any country without cause by giving Spark ninety (90) days prior written notice to that effect. In the event that Spark
becomes insolvent, files a petition in bankruptcy, has such a petition filed against it, determines to file a petition in bankruptcy, or receives notice of a third party’s intention to file an involuntary petition in bankruptcy, Spark shall
immediately notify Genable in writing and Genable shall thereupon have the right to terminate this Agreement upon thirty (30) days’ notice to Spark. 

  

	 	12.6	 Upon termination of this Agreement for any reason, Genable, its Affiliates, licensees and sublicensees shall cease all development, use, distribution,
and sale of Licensed Products, provided however, that Genable and its Affiliates, licensees and sublicensees shall be permitted for a period not exceeding [**] 

  
 16 

	 	
months to exhaust their stocks of the Licensed Products, provided that this Agreement has not been terminated by Spark due to a breach of this Agreement by Genable or an Affiliate, licensee or
sublicensee or under Paragraph 6.3 of this Agreement, and any such post-termination sales by Genable, its Affiliates, licensees and sublicensees shall be subject to a surviving royalty obligation under Paragraph 5.4. 

 

	 	12.7	Within [**] days of the final sale of Licensed Product under this Article 12, a final report shall be submitted by Genable together with any royalty payments or other amounts due to Spark. 

 

	 	12.8	Termination of this Agreement will not relieve any party from any obligation that has accrued prior to termination. 

  

	 	12.9	The following provisions of this Agreement shall survive termination: Section 4.5, Article 7, Sections 8.3 - 8.6, Sections 10.10 - 10.14, Article 11, Sections 12.2,12.6 - 12.9, 14.5, 14.9 and 14.13.

  

	13.	DISPUTES 

  

	 	13.1	If a dispute arises which cannot be resolved in the normal course of events, any Party to the dispute may give notice in writing to the others specifying the subject matter of the dispute and its proposal for its
resolution. The Parties must procure that the dispute is considered by their respective authorized representatives and that such authorized representatives use all reasonable endeavors, in good faith, to resolve the dispute within [**] days of the
date of the notice specifying the dispute. If the authorized representatives reach agreement on the matter in dispute in the period specified in this Paragraph 13.1, the Parties shall procure that their respective representatives sign a joint
memorandum to that effect recording the resolution and procure that such agreement is fully and promptly carried into effect. 

  

	 	13.2	If the authorized representatives fail to reach agreement, any Party may refer the matter to the Chief Executive Officers of the Parties (together the “Senior Officers”). The Parties shall respectively procure
that the Senior Officers attempt in good faith to resolve the dispute. If the Senior Officers reach agreement on the matter in dispute within [**] days of the dispute being referred to them (or such other period as the Parties may mutually agree in
writing) the Parties shall procure that their respective Senior Officers shall sign a joint memorandum to that effect recording the resolution and procure that such agreement is promptly and fully carried into effect. 

 

	 	13.3	The dispute resolution procedure shall have been exhausted if the matter in dispute: 

  

	 	(a)	has not been resolved in accordance with Paragraph 13.1 within the relevant period and is not referred to the Senior Officers within the relevant period; or 

 

	 	(b)	where it is so referred, has not been resolved in accordance with Paragraph 13.2 within the relevant period. 

  
 17 

	 	13.4	For the avoidance of doubt, the fact that the dispute resolution procedure has been exhausted without resolution shall not prevent the Parties from agreeing that the dispute be referred to an independent alternative
form of dispute resolution and/or to arbitration. 

  

	 	13.5	The foregoing provisions shall not prevent either Party from commencing legal proceedings or applying to the court for injunctive or other interim relief at any time. 

 

	 	13.6	Any controversy or claim related to or arising out of this Agreement (other than a patent dispute) shall be settled by arbitration conducted on a confidential basis under the Commercial Arbitration Rules of the
International Centre for Dispute Resolution (“ICDR”) in effect at the time of the arbitration (“Rules”). Any arbitration shall be held in Manhattan, New York before one disinterested arbitrator selected by mutual agreement of the
Parties; provided, however, that if the Parties are unable to agree on the arbitrator within [**] days, the arbitrator shall be appointed in accordance with the Rules. Any Party desiring arbitration shall serve on the other Party pursuant to
Section 13.6 and the regional case management center of the ICDR administering cases for such location in accordance with the aforesaid Rules, its notice of intent to arbitrate (“Arbitration Notice”). All arbitrations shall be
administered by the ICDR. 

  

	 	13.7	The arbitrator shall have no authority to award damages expressly precluded under this Agreement. The award of the arbitrator shall be final and binding upon the Parties and judgment upon such award may be entered and
enforced in any court of competent jurisdiction. Unless the arbitrator for good cause determines otherwise, the costs and expenses of the arbitrator shall be shared equally by the Parties and each Party will bear its own attorneys’ fees and
other costs associated with the arbitration proceeding. If court proceedings to stay litigation or compel arbitration are necessary, the Party that unsuccessfully opposes such proceedings will pay all associated costs, expenses and attorneys’
fees that are reasonably incurred by the other Party. 

  

	14.	GENERAL PROVISIONS 

  

	 	14.1	Neither Party may waive or release any of its rights or interests in this Agreement except in writing. The failure of either Party to assert a right hereunder or to insist upon compliance with any term or condition of
this Agreement shall not constitute a waiver of that right by such Party or excuse a similar subsequent failure to perform any such term or condition by Genable. 

  

	 	14.2	This Agreement, together with the Manufacturing Agreement and Development Agreement, constitutes the entire agreement between the Parties relating to the subject matter, and all prior negotiations, representations,
agreements, and understandings are merged into, extinguished by, and completely expressed by such agreements. 

  

	 	14.3	The provisions of this Agreement are severable, and in the event that any provision of this Agreement shall be determined to be invalid or unenforceable under any controlling body of law such determination shall not in
any way affect the validity or enforceability of the remaining provisions of this Agreement. 

  
 18 

	 	14.4	If either Party desires a modification to this Agreement, the Parties shall, upon reasonable notice of the proposed modification by the Party desiring the change, confer in good faith to determine the desirability of
such modification. No modification will be effective until a written amendment is signed by the signatories to this Agreement or their designees. 

  

	 	14.5	The construction, validity, performance, and effect of this Agreement shall be governed by the Laws of the State of New York and, subject to Article 13, any and all actions or proceedings relating to this Agreement
shall be brought and pursued exclusively in the federal or state courts sitting in United States District Court for the Southern District of the State of New York. 

 

	 	14.6	Any notice required to be given under this Agreement shall be in writing and shall be delivered personally, or sent by pre-paid post or recorded delivery or by commercial courier, to each Party required to receive the
notice at its address as set out below: 

 Spark: 

Spark Therapeutics, LLC 
 3501
Civic Center Blvd., 5th Floor 
 Philadelphia, PA 19104 

USA 
 Attention: Jeffrey D.
Marrazzo, CEO 
 Genable: 

Genable Technologies Limited 

c/o Delta Partners 
 Media
House, South County Business Park 
 Leopardstown, Dublin 18 

Ireland 
 Attention: Jason
Loveridge, CEO 
 or as otherwise specified by the relevant Party by notice in writing to each other Party. 

Any notice shall be deemed to have been duly received: 
  

	 	(i)	if delivered personally, when left at the address and for the contact referred to in this Paragraph 14.6, or 

  

	 	(ii)	if delivered by commercial courier, on the date and at the time that the courier’s delivery receipt is signed. 

A notice required to be given under this Agreement shall not be validly given if sent by email. The provisions of this Paragraph 14.6 shall
not apply to the service of any proceedings or other documents in any legal action. 
  

	 	14.7	This Agreement, or any obligations of a party under this Agreement, may not be assigned except as expressly provided in this Agreement. This Agreement may be assigned by either Party as part of a sale or transfer of
substantially the entire business of the assigning Party relating to operations which concern this Agreement, provided that the assigning Party notifies the other Party in writing within [**] days of any assignment of this Agreement by the assigning
Party. 

  
 19 

	 	14.8	Genable acknowledges that it is subject to and agrees to abide by the United States laws and regulations (including the Export Administration Act of 1979 and Arms Export Control Act) controlling the export of technical
data, computer software, laboratory prototypes, biological material, and other commodities. The transfer of such items may require a license from the relevant Agency of the U.S. Government or written assurances by Genable that it shall not export
such items to certain foreign countries without prior approval of such agency. Spark neither represents that a license is not required or that, if required, it shall be issued. 

 

	 	14.9	Promptly after execution of this Agreement, the Development Agreement and the Manufacturing Agreement, the Parties shall issue the press release attached hereto as Appendix C. Neither Party shall issue any press
releases or public disclosure relating to this Agreement, other than the aforementioned initial press release and any public disclosures that may be required pursuant to applicable securities laws and regulations, without the prior written consent
of the other Party, which consent shall not be unreasonably withheld or delayed. Neither Party shall use the name or logo of the other Party, and Genable shall not use the name of past or present Spark employees, in any advertising, promotional or
sales activities without prior written consent obtained from the other Party in each separate case, except as otherwise provided in this Agreement. 

  

	 	14.10	Neither Party to this Agreement shall be liable for delay in the performance of any of its obligations hereunder if such delay results from causes beyond its reasonable control, including, without limitation, acts of
God, fires, strikes, acts of war, or intervention of a government authority, non-availability of raw materials, but any such delay or failure shall be remedied by such Party as soon as practicable. 

 

	 	14.11	This Agreement may be executed in any number of counterparts, each of which when so executed shall be deemed to be an original and all of which when taken together shall constitute this Agreement. 

 

	 	14.12	Both Parties are independent contractors under this Agreement. Nothing herein contained shall be deemed to create an employment, agency, joint venture or partnership relationship between the Parties hereto or any of
their agents or employees. Neither Party shall have any express or implied power to enter into any contracts or commitments or to incur any liabilities in the name of, or on behalf of, the other Party, or to bind the other Party in any respect
whatsoever. 

  

	 	14.13	During the term of this Agreement and for a period of [**] months thereafter, neither Party shall solicit an employee of the other Party who is or has been involved in the performance or oversight of any development,
manufacturing or regulatory activity with respect to any Licensed Product to terminate his or her employment and accept employment or work as a consultant with the soliciting Party. Notwithstanding the foregoing, nothing herein shall restrict or
preclude the Parties’ right to make generalized searches for employees by way of a general solicitation for employment placed in a trade journal, newspaper or website. 

 

	 	14.14	No provision of this Agreement is intended to confer any rights, benefits, remedies, obligations, or liabilities hereunder upon any third party beneficiary or on any person other than the Parties and their respective
affiliates, successors and assigns. 

  
 20 

 IN WITNESS WHEREOF, duly authorized representatives of the Parties have duly executed this Agreement to be
effective as of the Effective Date. 
 SPARK THERAPEUTICS, LLC: 

 

					
	 /s/ Jeffrey D. Marrazzo
	 		  	 March 18, 2014

	Signature of Authorized Official	 		  	Date
			
	 Jeffrey D. Marrazzo
	 		  	
	Printed Name	 		  	
			
	 President & CEO
	 		  	
	Title	 		  	
			
	GENABLE TECHNOLOGIES LIMITED:	 		  	
			
	 /s/ Jason Loveridge
	 		  	 18 March 2014

	Signature of Authorized Official	 		  	Date
			
	 Jason Loveridge
	 		  	
	Printed Name	 		  	
			
	 CEO
	 		  	
	Title	 		  	

  
 21 

 APPENDIX A — Patent(s) or Patent Application(s) and Confidential Information 

 

					
	 Spark

Tech ID
Number
	  	 Patent
Application/
Publication/
Granted No.
	  	 Title

	[**]	  	[**]	  	[**]
	[**]	  	[**]	  	[**]

 [**] 
 Intellectual property
owned or controlled by Spark with respect to: 
 [**]. 

  
 22 

 APPENDIX B - CHOP License Agreement Terms Applicable to Genable 

Provisions quoted below in this Appendix B are copied verbatim from the CHOP License Agreement. Capitalized terms in language quoted below shall have the
meanings ascribed to them in the CHOP License Agreement and Paragraph cross-references therein shall refer to Paragraphs in the CHOP License Agreement. 

“4.4 In the event this Agreement terminates, and such termination is not the result of any failure by a sublicensee to comply with the terms of this
Agreement applicable to sublicensees, such sublicensee’s sublicense shall survive such termination in respect of the sublicensee’s exercise of such sublicense rights provided, however, that CHOP shall not be obligated in any manner to
perform any obligations of Company under the sublicense agreement beyond the granting of rights to the sublicensee with respect to the Patent Rights and Gene Therapy Know-How. 
  

... 
 5.1 CHOP retains the ability to research, make, have
made, practice, have practiced, and use CHOP Intellectual Property solely for its own research and/or educational purposes. In addition, all rights granted in this Agreement are expressly granted subject to the rights of the U.S. Government pursuant
to 35 U.S.C. Sections 200 et seq., as amended, (Patent Rights in Inventions Made with Federal Assistance) and the implementing regulations. 
 5.2 Company
agrees that if any Patent Rights claiming inventions were supported by funding from a U.S. government agency, products used or sold in the United States embodying such Patent Rights shall be manufactured substantially in the United States, unless a
written waiver is obtained in advance from the appropriate government agency. 
  
 ...

 7.1 Company agrees to keep, and to require its Affiliates and sublicensees to keep, accurate and correct records of Licensed Products under this
Agreement appropriate to determine the amount of royalties and payments due CHOP. Such records shall be retained for at least [**] years following a given reporting period. The records shall be made available, [**], at the request of CHOP during
normal business hours for inspection at the expense of CHOP by an accountant or other designated auditor selected by CHOP (and acceptable to Company) for the sole purpose of verifying reports and payments hereunder. Company may only object to an
auditor selected by CHOP for good cause shown. If an inspection shows an underreporting or underpayment in excess of [**] percent ([**]%) for any twelve (12) month period, then Company shall reimburse CHOP for the cost of the inspection at the
time Company pays the unreported royalties, including any late charges as required by this Agreement. All payments required under this Paragraph shall be due within [**] days of the date CHOP provides Company notice of the payment due. Late charges
will be assessed by CHOP on any undisputed overdue payments at a rate of [**] percent ([**]%) per month. The payment of such late charges shall not prevent CHOP from exercising any other rights it may have as a consequence of the lateness of any
payment. 
  
 ... 

8.1 CHOP shall control the preparation, filing, prosecution, and maintenance of any and all patent applications or patents included in the Patent Rights and
shall furnish copies of relevant patent-related documents to Company. Notwithstanding this Paragraph 8.1, any opposition, validity challenge, interference, re-examination, reissue, derivation, supplemental examination, post-grant review,
inter-parties review proceedings, negotiations or claims, in any forum shall be handled in accordance with Paragraph 10.3. 

  
 23 

  
 ... 

10.1 CHOP and Company agree to notify each other promptly of each infringement or possible infringement of the Patent Rights, as well as any facts which may
affect the validity, scope, or enforceability of the Patent Rights of which either Party becomes aware. 
 10.2 Pursuant to this Agreement Company may a)
bring suit in its own name, at its own expense, and on its own behalf for infringement of presumably valid claims in the Patent Rights or misappropriation of Gene-Therapy Know-How; b) in any such suit, seek to enjoin infringement or misappropriation
and collect damages, profits, and awards of whatever nature recoverable for such infringement; and c) settle any claim or suit for infringement of the Patent Rights or misappropriation of Gene-Therapy Know-How. Company shall not settle any action
that imposes any liability on CHOP or concedes the invalidity, enforceability or non-infringement of any of the Patent Rights or CHOP Intellectual Property without the prior written consent of CHOP. If necessary or desirable to bring, maintain or
prove damages in any such action, Company may require that CHOP join such suit. Should CHOP be made a party to any such suit at the request of Company, Company shall reimburse CHOP for any costs, expenses, or fees, which CHOP incurs as a result of
such action. In all cases, Company agrees to keep CHOP reasonably apprised of the status and progress of any litigation. Before Company commences an infringement action, Company shall notify CHOP and give careful consideration to the views of CHOP.

 10.3 In the event that a declaratory judgment action alleging invalidity or non-infringement of any of the Patent Rights or contesting the Gene Therapy
Know-How rights (excluding any such action that is based on alleged misappropriation by CHOP of Gene Therapy Know-How) shall be brought against Company or CHOP or raised by way of counterclaim or affirmative defense in an infringement suit brought
by Company under Paragraph 10.2, pursuant to this Agreement, or any opposition, validity challenge, interference, re-examination, reissue, derivation, supplemental examination, post-grant review, inter-parties review proceedings, negotiations or
claims, in any forum relating to the Patent Rights, Company shall, subject to the penultimate sentence of this Paragraph 10.3a) defend or handle the suit or proceeding, at its own expense, for presumably valid claims in the Patent Rights; and b) in
any such suit or proceeding, ultimately seek to enjoin infringement and to collect damages, profits, and awards of whatever nature recoverable from the party bringing such suit or otherwise defend the validity and enforceability of the Patent
Rights. Company shall not settle any action that imposes any liability on CHOP or concedes the invalidity, enforceability or non-infringement of any of the Patent Rights or CHOP Intellectual Property without the prior written consent of CHOP. If
necessary or desirable to defend any such action, Company may require that CHOP join such suit. Should CHOP be made a party to any such suit at the request of Company, Company shall reimburse CHOP for any costs, expenses, or fees which CHOP incurs
as a result of such action. If Company elects not to initiate a defense against such declaratory judgment action, CHOP at its option, may do so at its own expense and CHOP shall retain all recoveries from any such suit. In all cases, Company agrees
to keep CHOP reasonably apprised of the status and progress of any litigation. 
  
 ...

 11.3 CHOP does not warrant the validity of the Patent Rights and makes no representations whatsoever with regard to the scope of the Patent Rights, or
that the Patent Rights may be exploited without infringing other patents or other intellectual property rights of third parties. 

  
 24 

 11.4 NO WARRANTIES, EXPRESS OR IMPLIED, ARE OFFERED BY CHOP AS TO THE FITNESS FOR ANY PURPOSE OF THE MATERIALS OR
INFORMATION PROVIDED TO COMPANY UNDER THIS AGREEMENT, OR THAT THE CHOP INTELLECTUAL PROPERTY MAY BE EXPLOITED WITHOUT INFRINGING OTHER THIRD PARTY PATENT RIGHTS. COMPANY ACCEPTS THE CHOP INTELLECTUAL PROPERTY, INFORMATION AND THE MATERIALS “AS
IS,” AND CHOP DOES NOT OFFER ANY GUARANTEE OF ANY KIND. 
 11.5 Except as otherwise set forth in Paragraphs 10.2 and 10.3, CHOP does not represent that
it will commence legal actions against third parties infringing the Patent Rights. 
  

... 
 12.5 CHOP reserves the right of 35 U.S.C. §203 to
terminate or modify this Agreement solely to the extent that such action is legally necessary to meet requirements of the applicable federal statutes or regulations and such requirements are not reasonably satisfied by Company. Within [**] days of
receipt of written notice of CHOP’S belief or notification from the government that it is legally necessary to modify or terminate this Agreement, Company shall, if Company disagrees with such assessment, notify CHOP of such disagreement
and the basis for Company’s position and this Agreement shall not be terminated or modified unless and until such disagreement is resolved in accordance with Paragraph 13.12 or by the exercise of the march-in-rights by the government. 

12.6 Within [**] days of termination or expiration of this Agreement, a final report and all accrued payments shall be submitted by Company. If this Agreement
is terminated under this Article 12, sublicenses may be converted to direct licenses with CHOP pursuant to Paragraph 4.4.” 

  
 25 

 APPENDIX C - Press Release 

 

			
	

	  	

	 Media Inquiries:
 Jessica
Rowlands
 202-729-4089

Jessica.Rowlands(S)fkhealth.com
	  	 Dr. Jason Loveridge
 + 33 674177812

jloveridge@genable.net

 Spark Therapeutics and Genable Technologies Announce Collaboration to Advance a Gene Therapy Treatment for a
Rare Form of Retinitis Pigmentosa 
 PHILADELPHIA, Penn., Mar. 18, 2014 — Spark Therapeutics and Genable Technologies announced today
that they have entered into a collaboration agreement for Genable’s lead therapeutic to treat rhodopsin linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038. Under the terms of the collaboration, Genable will license certain
adeno-associated virus (AAV) vector manufacturing patents from Spark. The parties have entered into a broad agreement in which Spark will be the exclusive manufacturer for the product and provide development advice and expertise to Genable to help
in the ongoing development of GT038. Spark will receive milestone payments and royalties on future sales of GT038 as well as near-term revenue from the manufacture and supply of product. 

“We are excited to apply our deep expertise in AAV clinical development and manufacturing to augment Genable’s great work, and expand the number of
debilitating diseases of the eye that can be addressed through gene therapy,” said Jeffrey D. Marrazzo, Spark Therapeutics co-founder, president and CEO. 

Dr. Jason Loveridge, CEO of Genable Technologies commented “We have chosen Spark as our partner to advise, lend their experience and manufacture
GT038 based on their broad expertise in gene therapy. We see them as a world-class organization and we are excited to be advancing our novel therapy GT038 into the clinic”. 

GT038 is a potential treatment for rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP), an inherited retinal dystrophy that leads to
blindness in most cases. There is currently no approved pharmacologic treatment for adRP, which affects an estimated 30,000 patients worldwide. GT-038 utilizes AAV vectors with an established safety and efficacy profile to deliver RNA interference
(RNAi) molecules to suppress the expression of faulty and normal copies of RHO and restore normal gene expression. GT038 has been granted Orphan Drug Designation in both the USA and Europe. 

About Genable Technologies 
 Genable Technologies Ltd. is
a privately held, venture capital backed Dublin (Ireland) based bio-pharmaceutical company. The company is developing new gene therapies to treat “dominant” genetic diseases. The company has received significant support and investment form
Fountain Healthcare Partners, Delta Partners, Fighting Blindness Ireland, Foundation Fighting Blindness Clinical Research Institute (USA) and Enterprise Ireland. To learn more please visit www.genable.net 

  
 26 

 About Spark Therapeutics 

Spark Therapeutics is developing potentially curative, one-time gene therapy products to transform the lives of patients and re-imagine the treatment of
debilitating diseases. Spark’s lead gene therapy candidate, for RPE65-related blindness, is currently in Phase 3 clinical trials with the potential to be the first approved gene therapy in the U.S., and the first treatment to address the
significant unmet needs of patients living with blindness due to inherited retinal dystrophies. 
 Spark’s founding team includes scientists who led
the movement to develop gene therapy as a new treatment paradigm, establishing clinical proof of concept in the eye and liver and contributing key insights to the field that have resulted in a resurgence of industry interest in gene-based medicines.
In addition to the Phase 3 program in /?PE65-related blindness, the company has a Phase 1/2 program in hemophilia B, and preclinical programs to address neurodegenerative diseases and other inherited retinal dystrophies and hematologic disorders.
Spark has rights to a proprietary manufacturing platform that has an unparalleled track record of success in supporting clinical studies across diverse therapeutic areas and routes of administration. The company’s expertise across research,
clinical, regulatory and manufacturing builds on a legacy of innovation and excellence in gene therapy established by Spark’s team while at The Children’s Hospital of Philadelphia Center for Cellular and Molecular Therapeutics. To learn
more visit www.sparktx.com. 
 # # # 

  
 27

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