Document:

EX-10.12

 Exhibit 10.12 

Confidential Materials omitted and filed separately with the 

Securities and Exchange Commission. Double asterisks denote omissions. 

LICENSE AGREEMENT 
 This Agreement, having
an effective date of October 14, 2013 (the “Effective Date”), is made and entered into by and between the University of Iowa Research Foundation (hereinafter “UIRF”) having offices at 112 North Capitol Street, 6
Gilmore Hall, Iowa City, Iowa 52242 and AAVenue Therapeutics, LLC, a Delaware limited liability company (hereinafter “LICENSEE”), having offices at c/o The Children’s Hospital of Philadelphia, 34th Street and Civic Center Blvd., Philadelphia, PA 19104. 
 WHEREAS, under the patent policy of The
University of Iowa (“UI”), all inventions and technology arising during the normal course of research and teaching at the UI are assigned and entrusted to the UIRF to obtain patent or other appropriate intellectual property protection and
license said technology; 
 WHEREAS, UIRF and The General Hospital Corporation d/b/a Massachusetts General Hospital, a not-for-profit Massachusetts
corporation, with a principal place of business at 55 Fruit Street, Boston, MA 02114 (“Institute”) are, therefore, co-owners by assignment of the PATENT RIGHTS (as defined below) from their respective inventors named therein
(“Inventor/s”) of their entire right, title and interest in the PATENT RIGHTS; 
 WHEREAS, LICENSEE wishes to obtain an exclusive world-wide
license in order to practice the above referenced invention covered by PATENT RIGHTS in the United States and in certain foreign countries, and to manufacture, use and sell in the commercial market the products made in accordance therewith; and 

WHEREAS, UIRF wishes to grant such a license to LICENSEE in accordance with the terms of this Agreement. 

NOW THEREFORE, in consideration of the foregoing premises, the parties agree as follows: 

ARTICLE I — DEFINITIONS 
 1.1 PATENT
RIGHTS shall mean (a) the patents and patent applications set forth in Appendix A, (b) all of UFs and UIRF’s interests in patents and patent applications, in addition to those set forth in Appendix A, claiming inventions conceived,
reduced to practice or otherwise generated in the conduct of activities pursuant to the UI-CHOP-HHMI RCA (defined below), whether prior to or after the Effective Date, and (c) the inventions described and claimed in any of the foregoing, and
any divisionals, continuations, continuations-in-part to the extent the claims are directed to subject matter specifically described in such patents and patent applications, patents issuing thereon, reexaminations and reissues thereof; and any and
all counterparts of such patents and patent applications in any country of the world; all of which will be automatically incorporated into and added to this Agreement and shall periodically be added to Appendix A attached to this Agreement and made
part thereof. 
 1.2 LICENSED PRODUCTS shall mean products the manufacture, use, offer for sale, sale or importation of which are covered by a VALID CLAIM,
products made in accordance with or by means of LICENSED PROCESSES or products that are discovered, developed, manufactured or commercialized through the use of RCA Results (defined below). 

 1.3 LICENSED PROCESSES shall mean processes the practice of which are covered by a VALID CLAIM. 

1.4 NET SALES shall mean amounts invoiced by LICENSEE, its AFFILIATES and sublicensees for sales of LICENSED PRODUCTS to third parties, less the following to
the extent applicable to such sales: 
 (i) trade, quantity and cash discounts and reasonable services fees paid to wholesalers and
distributors; 
 (ii) 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, listed separately on invoices and actually paid by LICENSEE or its AFFILIATES or sublicensees; 

(iii) credits, chargebacks, retroactive price reductions, rebates and returns actually paid or allowed by LICENSEE or its AFFILIATES or
sublicensees; 
 (iv) transportation, insurance, packaging and postage charges, if listed separately on invoices and actually paid by
LICENSEE or its AFFILIATES or sublicensees; 
 (v) negotiated payments made to private sector and government third party payors (e.g., PBMs,
HMOs and PPOs) and purchasers/providers (e.g., staff model HMOs, hospitals and clinics), regardless of the payment mechanism, including without limitation rebate, chargeback and credit mechanisms; 

(vi) discounts paid under discount prescription drug programs and reductions for coupon and voucher programs; and 

(vii) invoiced amounts that are subsequently written off as uncollectible. 

Sales or transfers of LICENSED PRODUCTS among LICENSEE 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 PRODUCT 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 LICENSEE, 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 PRODUCT” means any product that comprises a LICENSED PRODUCT sold in conjunction with another active component that is not a LICENSED PRODUCT (whether packaged together or in the same formulation).

 1.5 AFFILIATES shall mean corporations and non-corporate entities that control, are controlled by or are under
the common control with a party. A corporation or a non-corporate entity, as applicable, is deemed to be in control of another corporation if (a) it owns or directly or indirectly controls at least 50% of the voting stock of the other
corporation or (b) in the absence of ownership of at least 50% of the voting stock of a corporation, or in the case of a non-corporate entity, if it possesses directly or indirectly, the power to direct or cause the direction of the management
and policies of such corporation or non-corporate entity, as applicable. 
 1.6 FIELD shall mean all diagnostic, prophylactic and therapeutic uses. 

1.7 VALID CLAIM shall mean a claim of (a) any issued, unexpired patent included in the PATENT RIGHTS which (i) has not been finally cancelled,
withdrawn, abandoned, rejected, permanently revoked or nullified, held invalid or declared unpatentable or unenforceable by any court or other body of competent jurisdiction that is unappealable or unappealed within the time allowed for appeal,
(ii) has not been rendered unenforceable through disclaimer or otherwise, and (iii) is not lost through an interference or opposition proceeding; or (b) any patent application included in the PATENT RIGHTS that shall not have been
cancelled, withdrawn or abandoned. 
 1.8 UI-CHOP-HHMI RCA shall mean the Research Collaboration Agreement effective as of February 10, 2012, by and
among UI, on behalf of itself and its employee [**], Howard Hughes Medical Institute (“HHMI”) and The Children’s Hospital of Philadelphia (“CHOP”), as amended effective November 19, 2012 and September 3, 2013, a
copy of which is attached as Appendix C. 
 1.9 RCA Results shall mean all Research Project Results, as such term is defined in the UI-CHOP-HHMI RCA. 

ARTICLE II — GRANT 
 2.1 UIRF hereby
grants to LICENSEE and LICENSEE accepts, subject to the terms and conditions hereof, a worldwide, exclusive (subject to Paragraph 2.3) license in the FIELD under PATENT RIGHTS and RCA Results to make and have made, to use and have used, to offer for
sale, sell and have sold, and to import and have imported LICENSED PRODUCTS, and to practice LICENSED PROCESSES. Such license shall include the right to grant sublicenses. LICENSEE agrees during the period of exclusivity of this license in the
United States, that any LICENSED PRODUCT produced for sale in the United States will be manufactured substantially in the United States to the extent required under applicable law, such as 35 U.S.C. § 202 et seq. and the regulations pertaining
thereto. 
 2.2 The term of this Agreement, the exclusive license set forth in Paragraph 2.1 and LICENSEE’S royalty obligations pursuant to Paragraph
3.3 shall commence on the Effective Date and continue, on a LICENSED PRODUCT-by-LICENSED PRODUCT and LICENSED PROCESS-by-LICENSED PROCESS, and country-by-country, basis until the expiration of the last-to-expire VALID CLAIM covering the manufacture,
use, offer for sale, sale or importation of such LICENSED PRODUCT in such country. Following expiration of this Agreement with respect to a LICENSED PRODUCT or LICENSED PROCESS in a country, the exclusive

 
license set forth in Paragraph 2.1 with respect to RCA Results relating to such LICENSED PRODUCT or LICENSED PROCESS in such county shall convert to a fully paid-up, non-royalty-bearing,
perpetual exclusive license that shall thereafter survive any termination or expiration of this Agreement. 
 2.3 The granting and acceptance of this
license is subject to the following conditions: 
  

	 	(a)	The UI Patent Policy approved in 1983, Public Law 96-517 and Public Law 98-620. Any right granted in this Agreement greater than that permitted under Public Law 96-517 or Public Law 98-620 shall be subject to
modification as may be required to conform to the provision of that statute. 

  

	 	(b)	UIRF and Institute reserve for itself, Institute’s AFFILIATES, their respective Inventors, and future not-for-profit employers of their respective Inventors, the right to make and to use for educational, research,
and patient care and treatment purposes only, the subject matter covered by PATENT RIGHTS and RCA Results. UIRF further reserves the right to provide and to grant non-exclusive licenses to make and use subject matter covered by PATENT RIGHTS and RCA
Results to not-for-profit organizations and government entities, in each case for internal research and scholarly purposes only, as required under applicable law, such as 35 U.S.C. § 202 et seq. and the regulations pertaining thereto.

  

	 	(c)	LICENSEE shall pay all future costs connected with LICENSEE’S commercial development of the LICENSED PRODUCTS, including but not limited to the costs of complying with applicable governmental testing, approvals and
regulations. 

  

	 	(d)	LICENSEE shall use reasonable efforts to effect introduction of the LICENSED PRODUCTS into the commercial market as soon as practicable, consistent with sound and reasonable business practices and judgment; thereafter,
until the expiration of this Agreement, LICENSEE shall endeavor to keep LICENSED PRODUCTS reasonably available to the public, to the extent consistent with sound and reasonable business practices and judgment. 

 

	 	(e)	UIRF shall have the right to terminate or render this license non-exclusive at any time after five (5) years from the Effective Date if, at such time, both of the following conditions exist and LICENSEE does not
eliminate either of such conditions after written notice thereof from UIRF during the cure period set forth below: 

  

	 	(i)	has not put the licensed subject matter into commercial use in any country, directly or through an AFFILIATE or sublicensee, and 

  

	 	(ii)	is not demonstrably engaged in a research, development, manufacturing, marketing, or licensing program, as appropriate, directed toward this end. 

In order to exercise any right under this Paragraph 2.3(e), UIRF must give LICENSEE a one hundred twenty (120) day prior written notice of
such exercise 

 
referencing this Paragraph, and if LICENSEE eliminates either of the above conditions during such one hundred twenty (120) day written period, such exercise shall not take effect. 

 

	 	(f)	All sublicenses granted by LICENSEE hereunder shall include a requirement that the sublicensee use its reasonable efforts to bring the subject matter of the sublicenses into commercial use as quickly as is reasonably
practicable, consistent with sound and reasonable business practices and judgment, and otherwise be consistent with LICENSEE meeting LICENSEE’S obligations to UIRF under this Agreement. Copies of all sublicense agreements shall be provided to
UIRF within [**] days of execution, provided that UIRF shall hold such agreements in strict confidence and use them solely to monitor LICENSEE’S compliance with this Agreement. 

2.4 UIRF hereby grants to LICENSEE the right to extend the licenses granted or to be granted in Paragraph 2.1 to LICENSEE’S AFFILIATES and sublicensees
subject to the terms and conditions hereof. 
 2.5 All rights reserved to the United States Government and others under Public Law 96-517 and 98620 shall
remain and shall in no way be affected by this Agreement. 
 ARTICLE III — EQUITY INTEREST, ROYALTIES, PAYMENTS 

3.1 In partial consideration for the rights and licenses granted by UIRF to LICENSEE herein and for UIRF’s incurred, previously unreimbursed
out-of-pocket patent expenses for PATENT RIGHTS as of the Effective Date, concurrently with the execution of this Agreement, LICENSEE and UIRF are entering into a Common Share Membership Agreement (Appendix B) pursuant to which LICENSEE will issue
to UIRF [**] common membership interest shares in LICENSEE as set forth therein (the “Equity Consideration”). 
 3.2 LICENSEE shall reimburse UIRF
for [**] percent ([**]%) of UIRF’s incurred, previously unreimbursed out-of-pocket patent expenses for PATENT RIGHTS, which include services rendered through June 30, 2013 that UIRF has received invoices for as of August 6, 2013. For
avoidance of doubt, such amount constitutes a one-time, non-refundable payment of $[**], which LICENSEE shall pay to UIRF within [**] days after the Effective Date. In addition, [**] common membership interest shares of the Equity Consideration are
in consideration for the other [**] percent ([**]%) of UIRF’s incurred, previously unreimbursed out-of-pocket patent expenses for PATENT RIGHTS. 
 3.3
(a) LICENSEE shall pay directly to UIRF within [**] days after the end of each calendar quarter during which NET SALES occur, on a LICENSED PRODUCT-by-LICENSED PRODUCT and country-by-country basis, royalties of: 

 

	 	(i)	[**] percent ([**]%) of the NET SALES of all LICENSED PRODUCTS indicated for Huntington’s disease sold for Prophylactic and Therapeutic uses, sold by LICENSEE and its AFFILIATES and sublicensees; 

	 	(ii)	[**] percent ([**]%) of the NET SALES of all LICENSED PRODUCTS indicated for Huntington’s disease sold for Diagnostic uses, sold by LICENSEE and its AFFILIATES and sublicensees; 

 

	 	(iii)	[**] percent ([**]%) of the NET SALES of all LICENSED PRODUCTS indicated for any Lysosomal storage disease, including Batten’s disease, sold for Prophylactic and Therapeutic uses, sold by LICENSEE and its
AFFILIATES and sublicensees; 

  

	 	(iv)	[**] percent ([**]%) of the NET SALES of all LICENSED PRODUCTS indicated for any Lysosomal storage disease, including Batten’s disease, sold for Diagnostic uses, sold by LICENSEE and its AFFILIATES and
sublicensees; 

  

	 	(v)	[**] percent ([**]%) of the NET SALES of all LICENSED PRODUCTS indicated for any other disease sold for Prophylactic and Therapeutic uses, sold by LICENSEE and its AFFILIATES and sublicensees; and 

 

	 	(vi)	[**] percent ([**]%) of the NET SALES of all LICENSED PRODUCTS indicated for any other disease sold for Diagnostic uses, sold by LICENSEE and its AFFILIATES and sublicensees. 

 

	 	(b)	On sales among LICENSEE and its AFFILIATES and sublicensees for resale, such royalties shall not be paid, but such royalties shall thereafter be paid on NET SALES arising from the resale. 

 

	 	(c)	No more than one of the above royalties shall be payable on NET SALES arising from any given sale of a LICENSED PRODUCT, and if more than one of the above royalties would otherwise apply to a given sale of a LICENSED
PRODUCT, only the highest applicable royalty above shall apply to such sale. 

 3.4 LICENSEES shall pay UIRF the following amounts upon
LICENSEE or its AFFILIATES or sublicensees achieving the following milestones, which each shall be payable no more than once irrespective of how many times it is achieved or by how many LICENSED PRODUCTED it is achieved. Such payments are due within
[**] days after the end of the calendar quarter in which the milestone was achieved. 
  

	 	(a)	$[**] upon the [**]; 

  

	 	(b)	$[**] upon the [**]; 

  

	 	(c)	$[**] upon [**]; 

  

	 	(d)	$[**] upon the [**]; 

  

	 	(e)	$[**] upon the [**]; and 

  

	 	(f)	$[**] upon [**]. 

 3.5 Commencing in 2017, an annual license maintenance fee payment of $[**] shall be paid to UIRF. These annual
license maintenance fees shall be credited against any milestone payments and royalties that become payable to UIRF during the applicable calendar year for LICENSED PRODUCTS developed for Huntington’s disease but shall not be credited against
(a) any milestone payments or royalties accruing in any other period or (b) contract research funding payable to the University of Iowa pursuant to the terms of any sponsored research agreement. 

3.6 Commencing in 2017, an annual license maintenance fee payment of $[**] shall be paid to UIRF. These annual license maintenance fees shall be credited
against any milestone payments and royalties that become payable to UIRF during the applicable calendar year for LICENSED PRODUCTS developed for any Lysosomal storage disease including Batten’s disease but shall not be credited against
(a) any milestone payments or royalties accruing in any other period or (b) contract research funding payable to the University of Iowa pursuant to the terms of any sponsored research agreement. 

3.7 The parties acknowledge that they may, by mutual agreement, substitute issuances to UIRF of membership interests in LICENSEE for some or all of the
payments described above in Paragraphs 3.4, 3.5 and 3.6. The parties will document any such agreement in writing and anticipate that the magnitude of any such additional equity issuance, if agreed, would be based on the payment amounts that are
eliminated by such agreement and the post-financing per share value of LICENSEE’S common equity immediately following the closing of LICENSEE’S then-most recent equity financing, which in turn would be based on the equity valuation model
used for such immediately prior equity financing. 
 3.8 Notwithstanding the above, should the LICENSEE (or any entity or person acting on its behalf) bring
any action or claim challenging the validity or enforceability of any PATENT RIGHTS in any forum (“Challenge”), the maintenance fees payable pursuant to Paragraphs 3.5 and 3.6 shall be doubled and all royalty rates applicable to LICENSED
PRODUCTS covered by such challenged PATENT RIGHTS shall be increased by [**]% of NET SALES following the date such claim is filed for the remaining term of this Agreement. 

3.9 In the event LICENSEE brings a Challenge during the term of this Agreement, LICENSEE agrees to pay directly to UIRF all royalties due under this Agreement
during the period of the Challenge. For the avoidance of doubt, LICENSEE shall not pay royalties into any escrow or other similar account. 
 3.10 In the
event that at least one claim of a patent that is subject to a Challenge survives the Challenge without being found invalid or unenforceable, the maintenance fees payable pursuant to Paragraphs 3.5 and 3.6 shall (disregarding the prior doubling
thereof pursuant to Paragraph 3.8) be tripled and all royalty rates (disregarding the prior increase in royalty rates under Paragraph 3.8) applicable to LICENSED PRODUCTS covered by such challenged PATENT RIGHTS shall be increased by [**]% of NET
SALES following the date of such finding for the remaining term of this Agreement. 
 3.11 LICENSEE will have no right to any refund or recovery of any
payments made under this Article III for any reason whatsoever. Further, LICENSEE acknowledges and agrees that the terms in this Article III reasonably reflect the value derived from this Agreement by LICENSEE in the event of a Challenge. 

 ARTICLE IV — REPORTING 

4.1 Prior to signing this Agreement, LICENSEE has provided to UIRF a written research and development plan under which LICENSEE intends to bring the subject
matter of the licenses granted hereunder into commercial use upon execution of this Agreement. 
 4.2 LICENSEE shall provide written annual reports within
[**] days after June 30 of each calendar year which shall include but not be limited to: reports of progress on research and development, regulatory approvals, manufacturing, sublicensing, marketing and sales during the preceding twelve
(12) months as well as plans for the coming year. If progress differs from that anticipated in the plan provided under 4.1, LICENSEE shall explain the reasons for the difference and propose a modified plan for UIRF’s review. LICENSEE shall
also provide reasonable additional data requested by UIRF to monitor LICENSEE’S performance. 
 4.3 LICENSEE shall report to UIRF the date of first
sale of LICENSED PRODUCTS in each country within [**] days of occurrence. 
 4.4 (a) LICENSEE agrees to submit to UIRF within [**] days after the calendar
quarters ending March 31, June 30, September 30, and December 31 following the first commercial sale of a LICENSED PRODUCT, reports setting forth for the preceding calendar quarter at least the following information:

  

	 	(i)	NET SALES of the LICENSED PRODUCTS sold by LICENSEE, its AFFILIATES and sublicensees in each country 

  

	 	(ii)	total invoiced amounts for such LICENSED PRODUCTS; 

  

	 	(iii)	deductions applicable to determine the NET SALES thereof; 

  

	 	(iv)	the amount of royalty due thereon; 

 and with each such royalty report to pay the amount of royalty due. Such
report shall be certified on behalf of LICENSEE as correct by an officer of LICENSEE and shall include a listing of all deductions by category from invoiced amounts as specified herein. If no royalties are due to UIRF for any reporting period, the
written report shall so state. 
  

	 	(b)	All payments due hereunder shall be payable in United States dollars. Conversion of foreign currency to U.S. dollars shall be made at the conversion rate existing in the United States (as reported in the Wall Street
Journal) on the last working day of each royalty period. Such payments shall be without deduction of exchange, collection or other charges. LICENSEE shall withhold from its payments to UIRF hereunder and pay to the applicable taxing authorities
foreign withholding taxes solely to the extent required under applicable law. 

 Please make checks payable to THE UNIVERSITY OF IOWA RESEARCH FOUNDATION, Federal Tax ID#
23-7436761, and mail to: 
 University of Iowa Research Foundation 

Attn: [**] 
 112 North Capitol
Street 
 6 Gilmore Hall 
 Iowa
City, IA 52242-5500 
 BANK WIRE PAYMENT INSTRUCTIONS: 

[**] 
 If you have questions,
please contact: [**]. 
  

	 	(c)	All such reports shall be maintained in confidence by UIRF, except as required by law, including Public Law 96-517 and 98-620. 

  

	 	(d)	Late payments shall be subject to an interest charge of [**] percent ([**]%) per annum calculated for the eperiods during which such payments are overdue. 

ARTICLE V — RECORD KEEPING 
 5.1
LICENSEE shall keep, and shall require its AFFILIATES and sublicensees to keep, accurate and correct records of LICENSED PRODUCTS made, used or sold under this Agreement, appropriate to determine the amount of royalties due hereunder to UIRF. Such
records shall be retained for at least [**] years following a given reporting period. They shall be available during normal business hours for inspection at the expense of UIRF by UIRF’s Internal Audit Department or by a Certified Public
Accountant selected by UIRF and approved by LICENSEE for the sole purpose of verifying reports and payments hereunder. Such accountant shall not disclose to UIRF any information other than information relating to accuracy of reports and payments
made under this Agreement. In the event that any such inspection shows an underreporting and underpayment in excess of [**] percent ([**]%) for any twelve (12) month period, then LICENSEE shall, subject to LICENSEE’S right to dispute any
such audit results, pay the cost of such examination as well as any additional sum that would have been payable to UIRF had the LICENSEE reported correctly, plus interest as set forth in Paragraph 4.4(d). 

ARTICLE VI — FILING, PROSECUTION AND MAINTENANCE OF PATENTS 

6.1 In addition to the reimbursement payment and portion of the Equity Consideration issued to UIRF for UIRF’s incurred, previously unreimbursed
out-of-pocket patent expenses for PATENT RIGHTS as set forth in Paragraph 3.2, which include services rendered through June 30, 2013 that UIRF has received invoices for as of August 6, 2013, LICENSEE shall reimburse UIRF for all reasonable
expenses UIRF incurs for the preparation, filing, prosecution and maintenance of PATENT RIGHTS following June 30, 2013 within [**] days after receipt of invoices from UIRF. Late payment of these invoices shall be subject to interest charges as
set forth in Paragraph 4.4(d). UIRF shall take responsibility for the preparation, filing, prosecution and maintenance of any and all patent applications and patents included in PATENT RIGHTS. 

 
UIRF shall promptly inform LICENSEE regarding all matters directly pertaining to prosecution of PATENT RIGHTS, and shall seek LICENSEE’S counsel in advance of making any filing or taking any
other action concerning all proposed courses of action affecting the PATENT RIGHTS, including but not limited to the geographic scope of patent protection that LICENSEE wishes to obtain, and all proposed courses of action in any re-examination,
post-grant review, inter partes review, supplemental examination, interference, derivation, opposition or cancellation proceedings. 
 6.2 UIRF and
LICENSEE shall cooperate fully in the preparation, filing, prosecution and maintenance of PATENT RIGHTS and of all patents and patent applications licensed to LICENSEE hereunder, executing all papers and instruments or requiring members of UIRF to
execute such papers and instruments as to enable UIRF to apply for, to prosecute and to maintain patent applications and patents in UIRF’s name in any country. Each party shall provide to the other notice at least [**] days prior to making any
filing or taking any other action as to all matters which come to its attention and which may affect the preparation, filing, prosecution or maintenance of any such patent applications or patents. 

6.3 If LICENSEE elects to no longer pay the expenses of a patent application or patent included with PATENT RIGHTS, LICENSEE shall notify UIRF not less than
[**] days prior to such action and shall thereby surrender its rights under such patent or patent application (in which case such patent or patent application shall thereafter be excluded from the PATENT RIGHTS). 

ARTICLE VII — MARKING 
 7.1 If a
licensed patent has been or is subsequently issued to UIRF covering any feature or features of the LICENSED PRODUCTS, LICENSEE agrees to mark each and every package or container in which the LICENSED PRODUCTS are used or sold by or for LICENSEE, if
and to the extent legally required, with marking complying with the provisions of Title 35, U.S. Code, Section 287 or any future equivalent provisions of law in the United States relating to the marking of patented devices, or with marking
complying with the law of the country where the LICENSED PRODUCTS are shipped, used or sold. 
 ARTICLE VIII — INFRINGEMENT 

8.1 With respect to any PATENT RIGHTS under which LICENSEE is exclusively licensed in the FIELD pursuant to this Agreement, LICENSEE or its sublicensee shall
have the right to prosecute in its own name and at its own expense any infringement of such patent, so long as such license is exclusive in the FIELD at the time of the commencement of such action. UIRF agrees to notify LICENSEE promptly of each
infringement of such patents of which UIRF is or becomes aware. Before LICENSEE or its sublicensees commences an action with respect to any infringement of such patents LICENSEE shall give reasonable consideration to the views of UIRF and to
potential effects on the public interest in making its decision whether or not to sue and in the case of such an action by a LICENSEE, its AFFILIATES or sublicensee, shall report such views to the AFFILIATES or sublicensee. 

8.2 If LICENSEE elects to sue for patent infringement, UIRF agrees to be named as nominal third party plaintiff if reasonably necessary to the commencement or
maintenance of any such 

 
action or to establish damages in such action, and further agrees to provide any information available to UIRF and needed by LICENSEE in prosecuting such action. LICENSEE shall reimburse UIRF for
any out-of-pocket costs it incurs as part of an action brought by LICENSEE or its sublicensee, irrespective of whether UIRF shall become a co-plaintiff. 

8.3 If LICENSEE or its AFFILIATES or sublicensee elects to commence an action as described above, LICENSEE may withhold up to [**] percent ([**]%) of the
royalty due to UIRF under this Agreement for any calendar year, during which such action is commenced, in order to cover [**] percent ([**]%) of the amount of the expenses and costs of such action, including attorney fees. In the event that such
[**] percent ([**]%) of such expenses and costs exceed the amount of royalties withheld by LICENSEE for any calendar year, LICENSEE may to that extent reduce the royalties due to UIRF from LICENSEE in succeeding calendar years, but never by more
than [**] percent ([**]%) of the royalty due in any one year. 
 8.4 No settlement, consent judgment or other voluntary final disposition of the suit may be
entered into without consent of UIRF and Institute, which consent shall not be unreasonably withheld; provided that, LICENSEE may enter into settlements, consent judgments or other voluntary final dispositions of suits without UIRF’s or
Institute’s consent if such dispositions do not admit wrongdoing of any kind of UIRF and/or Institute, do not impose any financial obligations on UIRF and/or Institute for which LICENSEE is not fully indemnifying UIRF and/or Institute, and such
dispositions do not admit any invalidity or unenforceability of the PATENT RIGHTS. 
 8.5 Recoveries, reimbursements, or any other form of award from such
action shall first be applied to reimburse LICENSEE and UIRF for litigation costs not paid from royalties in accordance with Paragraph 8.3 and then to reimburse UIRF for such royalties that were withheld. Any remaining recoveries, reimbursements, or
any other form of award shall be [**] percent ([**]%) retained by LICENSEE and [**] percent ([**]%) paid to UIRF. 
 8.6 In the event that LICENSEE and its
AFFILIATES and sublicensees, if any, elect not to exercise their right to prosecute an infringement of the PATENT RIGHTS pursuant to the above paragraphs, UIRF may do so at its own expense, controlling such action and retaining all recoveries
therefrom. 
 8.7 If a declaratory judgment action alleging invalidity of any of the PATENT RIGHTS is brought against LICENSEE or its AFFILIATES or
sublicensees, or UIRF, other than as a counterclaim in a suit brought by LICENSEE or its AFFILIATES or sublicensees pursuant to Paragraph 8.1, then UIRF, at its sole option, shall have the right to intervene and take over the sole defense of the
action at its own expense. Before UIRF so intervenes, UIRF shall give reasonable consideration to the views of LICENSEE, its AFFILIATES and sublicensees in defending such action. 

8.8 UIRF shall have no obligation to defend any action for infringement brought against LICENSEE or its AFFILIATES or sublicensees by a third party. In the
event LICENSEE or its AFFILIATES or sublicensees is sued by a third party, and as a result of the settlement of such suit is required to pay a royalty to a third party on a LICENSED PRODUCT, the amount of royalty paid will be deducted from the
royalty payment due to the UIRF for that LICENSED 

 
PRODUCT. In the event the settlement prevents the LICENSEE or its AFFILIATES or sublicensees from continuing sales of a LICENSED PRODUCT, no additional royalties and/or minimum royalties will
apply for that LICENSED PRODUCT. 
 8.9 Sections 8.1, 8.2, 8.4., 8.5, 8.6, 8.7, 8.8, and 8.9 shall survive termination or expiration of this Agreement. 

ARTICLE IX — TERMINATION OF AGREEMENT 

9.1 Upon any termination of this Agreement, and except as provided herein to the contrary, all rights and obligations of the parties hereunder shall cease,
except as follows: 
  

	 	(a)	UIRF’s right to receive or recover and LICENSEE’S obligation to pay royalties accrued or accruable for payment at the time of any termination; 

 

	 	(b)	LICENSEE’S obligation to maintain records and UIRF’s right to conduct a final audit as provided in Article V of this Agreement; and 

 

	 	(c)	Any cause of action or claim of UIRF, accrued or to accrue because of any breach or default by LICENSEE. 

 9.2
In the event LICENSEE fails to make payments due hereunder, UIRF shall have the right to terminate this Agreement upon [**] days written notice, unless LICENSEE makes such payments plus interest within the [**] day notice period. If payments are not
so made, UIRF may immediately terminate this Agreement; provided that, if during such [**] day period LICENSEE notifies UIRF that LICENSEE disputes in good faith any such payment amount and pays UIRF all undisputed amounts, then such [**] day period
shall be tolled until such dispute is resolved. 
 9.3 In the event that LICENSEE shall be in material default in the performance of LICENSEE’S
obligations under this Agreement (other than as provided in 9.2 above which shall take precedence over any other default), and if the default has not been remedied within [**] days after the date of notice in writing of such default, UIRF may
terminate this Agreement immediately by written notice; provided that, if during such [**] day period LICENSEE notifies UIRF that LICENSEE disputes in good faith that it is in material default, then such [**] day period shall be tolled until such
dispute is resolved. 
 9.4 This Agreement will automatically terminate if LICENSEE becomes insolvent, makes an assignment for the benefit of creditors,
files or has filed against it a petition in bankruptcy or seeking reorganization, has a receiver appointed, or institutes any proceedings for liquidation or winding-up. Upon any termination as defined herein, LICENSEE’S license under the PATENT
RIGHTS and RCA Results shall terminate. 
 9.5 Any sublicense granted by LICENSEE under this Agreement shall provide for termination or assignment to UIRF,
at the option LICENSEE, of LICENSEE’S interest therein upon termination of this Agreement. 
 9.6 LICENSEE shall have the right to terminate this
Agreement, either in its entirety or on a LICENSED PRODUCT-by-LICENSED PRODUCT basis, by giving ninety (90) days advance 

 
written notice to UIRF to that effect. Upon termination, a final report shall be submitted and any royalty payments and unreimbursed patent expenses due to UIRF become immediately payable with
respect to any LICENSED PRODUCT affected by such termination, and if such termination is a termination of all of LICENSEE’S licenses as to LICENSED PRODUCTS in either the category referenced in Paragraph 3.5 or the category referenced in
Paragraph 3.6, then no further payments under Paragraph 3.5 or Paragraph 3.6, as applicable, shall be payable after the effective date of such termination. 

9.7 LICENSEE shall have the right during a period of [**] months following the effective date of such termination to sell or otherwise dispose of the LICENSED
PRODUCT existing at the time of such termination, and shall make a final report and payment of all royalties related thereto within [**] days following the end of such period or the date of the final disposition of such inventory, whichever first
occurs. 
 9.8 Termination of this Agreement as to a LICENSED PRODUCT shall not alter the rights and obligations of the parties to the remaining LICENSED
PRODUCTS. 
 ARTICLE X — ASSIGNMENT 

10.1 This Agreement is binding upon and shall inure to the benefit of the respective successors and assigns of the parties hereto. This Agreement may not be
assigned by LICENSEE without prior written consent of UIRF, such consent not to be unreasonably withheld; provided, however, that no such written consent of UIRF shall be required to assign this Agreement (a) to an AFFILIATE of LICENSEE, or
(b) in connection with the merger or sale or other transfer of all or substantially all of the business or assets of LICENSEE to which this Agreement relates. LICENSEE agrees to provide UIRF, within [**] days of any such assignment, with
contact information for the assignee. 
 ARTICLE XI — REPRESENTATIONS AND WARRANTIES; LIMITATION OF LIABILITY 

11.1 Nothing in this agreement shall be construed as conferring a right to use in advertising, publicity or otherwise the name of the UI or UIRF, or the
inventors. Unless required by law, or unless specifically approved in advance in writing by UIRF, LICENSEE’S use of the name “The University of Iowa” or the name of any University of Iowa college, department, or inventor in
advertising, publicity or other promotional activities is expressly prohibited, provided that LICENSEE may publicly disclose that it has been granted a license under the PATENT RIGHTS and RCA Results and its research, development and
commercialization activities with respect to LICENSED PRODUCTS. 
 11.2 UIRF represents that it has the right to grant the licenses and rights contemplated
under this Agreement. 
 11.3 UIRF has not received written notice of any pending or threatened claims or actions seeking to invalidate any PATENT RIGHTS.

 11.4 EXCEPT AS MAY BE EXPRESSLY SET FORTH HEREIN, UIRF AND INSTITUTE EXPRESSLY DISCLAIM ANY AND ALL IMPLIED OR EXPRESS WARRANTIES AND

 
MAKES NO EXPRESS OR IMPLIED REPRESENTATIONS OR WARRANTIES OF ANY KIND CONCERNING THE PATENT RIGHTS OR RCA RESULTS AND THE RIGHTS GRANTED HEREUNDER, INCLUDING WITHOUT LIMITATION, WARRANTIES OF
MERCHANTABILITY, FITNESS FOR ANY PARTICULAR PURPOSE OF THE PATENT RIGHTS, RCA RESULTS, LICENSED PROCESSES OR LICENSED PRODUCTS CONTEMPLATED BY THIS AGREEMENT, NONINFRINGEMENT, VALIDITY OF PATENT RIGHTS CLAIMS, WHETHER ISSUED OR PENDING, AND THE
ABSENCE OF LATENT OR OTHER DEFECTS, WHETHER OR NOT DISCOVERABLE, AND HEREBY DISCLAIMS THE SAME. SPECIFICALLY, AND NOT TO LIMIT THE FOREGOING, UIRF AND INSTITUTE MAKE NO WARRANTY OR REPRESENTATION (i) REGARDING THE VALIDITY OR SCOPE OF ANY OF
THE CLAIM(S), WHETHER ISSUED OR PENDING, OF ANY OF THE PATENT RIGHTS, and (ii) THAT THE EXPLOITATION OF THE PATENT RIGHTS, RCA RESULTS OR ANY PRODUCT WILL NOT INFRINGE ANY PATENTS OR OTHER INTELLECTUAL PROPERTY RIGHTS OF UIRF, INSTITUTE, OR OF
ANY THIRD PARTY. 
 11.5 UIRF and Institute assume no responsibilities whatever with respect to design, development, manufacture, use, sale or other
disposition by LICENSEE or AFFILIATES of LICENSED PRODUCTS or LICENSED PROCESSES. The entire risk as to the design, development, manufacture, offering for sale, sale, or other disposition and performance of LICENSED PRODUCTS and LICENSED PROCESSES
is assumed by LICENSEE and AFFILIATES. 
 11.6 IN NO EVENT SHALL UIRF, INSTITUTE, LICENSEE OR ANY OF THEIR AFFILIATES OR ANY OF THEIR RESPECTIVE TRUSTEES,
DIRECTORS, OFFICERS, MEDICAL AND PROFESSIONAL STAFF, EMPLOYEES AND AGENTS BE LIABLE TO ANY OTHER PARTY OR TO ANY OTHER PARTY’S AFFILIATES, SUBLICENSEES, OR DISTRIBUTORS FORINDIRECT, SPECIAL, INCIDENTAL OR CONSEQUENTIAL DAMAGES OF ANY KIND
ARISING IN ANY WAY OUT OF THIS AGREEMENT OR THE LICENSE RIGHTS GRANTED HEREUNDER, HOWEVER CAUSED AND ON ANY THEORY OF LIABILITY, INCLUDING WITHOUT LIMITATION ECONOMIC DAMAGES OR INJURY TO PROPERTY OR LOST PROFITS, REGARDLESS OF WHETHER UIRF,
INSTITUTE OR LICENSEE, AS APPLICABLE, SHALL BE ADVISED, SHALL HAVE OTHER REASON TO KNOW, OR IN FACT SHALL KNOW OF THE POSSIBILITY OF THE FOREGOING; PROVIDED THAT, NOTHING IN THIS SECTION 11.6 SHALL LIMIT LICENSEE’S INDEMNIFICATION
OBLIGATIONS UNDER THIS AGREEMENT WITH RESPECT TO THIRD PARTY CLAIMS. 
 11.7 LICENSEE, AFFILIATES, and sublicensees shall not use the name of the Institute
or of any trustee, director, officer, staff member, employee, student, or agent of the Institute or any adaption thereof in any advertising, promotional or sales literature, publicity, or in any document employed to obtain funds or financing without
the prior written approval of the Institute or the individual whose name is to be used. For Institute, such approval shall be obtained from Institute’s Chief Public Affairs Officer. 

11.8 This Section 11 shall survive termination or expiration of this Agreement. 

 ARTICLE XII — GENERAL 

 

	12.1    (a)	LICENSEE shall indemnify, defend and hold harmless UIRF, the University of Iowa, Institute and its AFFILIATES, and their respective current and former employees, trustees, directors, officers, medical and professional
staff, and agents, the Board of Regents — State of Iowa, and their respective successors, heirs and assigns (the “Indemnitees”), against any liability, damage, loss or expenses (including reasonable attorneys’ fees and expenses
of litigation) incurred by or imposed upon the Indemnitees or any one of them in connection with any claims, suits, actions, demands or judgments arising out any theory of product liability (including, but not limited to, actions in the form of
tort, warranty, or strict liability) concerning any product, process or service made, used or sold pursuant to any right or license granted under this Agreement. 

  

	           (b)	LICENSEE agrees, at its own expense, to provide attorneys reasonably acceptable to UIRF and to Institute, to defend against any actions brought or filed against any party indemnified hereunder with respect to the
subject of indemnity contained herein, whether or not such actions are rightfully brought; provided, however, that any Indemnitees shall have the right to retain its own counsel, at the expense of Licensee, if representation of such Indemnitees by
counsel retained by Licensee would be inappropriate because of actual or potential differences in the interests of such Indemnitees and any other party represented by such counsel. Licensee agrees to keep UIRF and Institute informed of the progress
in the defense and disposition of such claim and to consult with UIRF and Institute prior to any proposed settlement. 

  

	           (c)	 Beginning at the time as any such product, process or service is being commercially distributed, sold, leased or otherwise transferred, or performed
or used (other than for the purpose of obtaining regulatory approvals) by LICENSEE or AFFILIATE or agent of LICENSEE or sublicensee, LICENSEE shall, at its sole cost and expense procure and maintain Commercial General Liability insurance in amounts
not less than $[**] per occurrence and $[**]annual aggregate and naming the Indemnitees as additional insureds. During clinical trials of any such product, process or service LICENSEE shall, at its sole cost and expense, procure and maintain
Commercial General Liability insurance in such equal or lesser amounts as UIRF shall require, naming the Indemnitees as additional insureds. Such Commercial General Liability insurance shall provide (i) product liability coverage and
(ii) broad form contractual liability coverage for Licensee’s indemnification under Section 12.1 (a) of this Agreement, which includes coverage for bodily injury and property damage, including completed operations, personal
injury, coverage for contractual employees, blanket contractual and products and completed operations. Following LICENSEE’S acquisition by a pharmaceutical company having (together with its AFFILIATES) more than one billion U.S. dollars in
annual revenues, LICENSEE may elect to self-insure all or part of the limits described above, provided that, if LICENSEE elects to self-insure all or part of the limits described above (including deductibles or retentions which are in
excess of $[**] annual aggregate), such self-insurance 

	 	
program must be reasonably acceptable to UIRF, Institute and the Risk Management Foundation. The minimum amounts of insurance coverage required under this Section 12.1 (c) shall not be
construed to create a limit of LICENSEE’S liability with respect to its indemnification under Section 12.1 (a) of this Agreement. Policy shall contain a severability of interests provision. 

 

	           (d)	LICENSEE shall provide UIRF and Institute with a certificate of liability insurance in accordance with Section 12.1(c) at such time as any product, process or service is commercially distributed, sold, leased or
otherwise transferred, or performed or used. LICENSEE shall provide UIRF and Institute with written notice at least [**] days prior to the cancellation, non-renewal or material change in such insurance; if LICENSEE does not obtain replacement
insurance providing comparable coverage within such [**] day period, UIRF shall have the right to terminate this Agreement effective at the end of such [**] day period without notice or any additional waiting periods. 

 

	           (e)	LICENSEE shall maintain such Commercial General Liability insurance beyond the expiration or termination of this Agreement during (i) the period that any product, process, or service, relating to, or developed
pursuant to, this Agreement is being commercially distributed, sold, leased, or otherwise transferred, or performed or used (other than for the purpose of obtaining regulatory approvals) by LICENSEE or by a sublicensee, AFFILIATE or agent of
LICENSEE and (ii) a reasonable period after the period referred to in (e)(i) above which in no event shall be less than [**] years. 

  

	           (f)	Sections 12.1 (c), 12.1 (d), and 12.1 (e) shall survive termination or expiration of this Agreement. 

12.2 In the event of any controversy or claim arising out of or relating to any provision of this Agreement or the breach thereof, the parties shall try to
settle such conflicts amicably between themselves. Subject to the limitation stated in the final sentence of this section, any such conflict which the parties are unable to resolve may be settled through arbitration conducted in accordance with the
rules of the American Arbitration Association. In the event a dispute is arbitrated, the demand for arbitration shall be filed within a reasonable time after the controversy or claim has arisen, and in no event after the date upon which institution
of legal proceedings based on such controversy or claim would be barred by the applicable statutes of limitation. Such arbitration shall be held in Chicago, Illinois. The award through arbitration shall be final and binding. Either party may enter
any such award in a court having jurisdiction or may make application to such court for judicial acceptance of the award and an order of enforcement, as the case may be. Notwithstanding the foregoing, either party may, without recourse to
arbitration, assert against the other party a third-party claim or cross-claim in any action brought by a third party, to which the subject matter of this Agreement may be relevant. 

12.3 Should a court of competent jurisdiction later consider any provision of this Agreement to be invalid, illegal, or unenforceable, it shall be considered
severed from this Agreement. All other provisions, rights and obligations shall continue without regard to the severed provision, provided that the remaining provisions of this Agreement are in accordance with the intention of the parties. 

 12.4 No waiver by a party of any breach of this Agreement, no matter how long continuing or how often repeated,
shall be deemed a waiver of any subsequent breach thereof, nor shall any delay or omission on the part of a party to exercise any right, power or privilege hereunder be deemed a waiver of such right, power or privilege. 

12.5 The relationship between the parties is that of independent contractor and contractee. LICENSEE shall not be deemed to be an agent of UIRF in connection
with the exercise of any rights hereunder, and shall not have any right or authority to assume or create any obligation or responsibility on behalf of UIRF. 

12.6 No party hereto shall be deemed to be in default of any provision of this Agreement, or for any failure in performance, resulting from acts or events
beyond the reasonable control of such party, such acts of God, acts of civil or military authority, civil disturbance, war, strikes, fires, power failures, natural catastrophes or other “force majeur” events. 

ARTICLE XIII — NOTICES; APPLICABLE LAW 

13.1 Any notice, report or payment provided for in this Agreement shall be deemed sufficiently given if in writing and when sent by express courier, certified
or registered mail addressed to the party for whom intended at the address set forth below, or to such address as either party may hereafter designate in writing to the other: 
  

					
	(a)	  	For the UIRF:	  	University of Iowa Research Foundation
		  		  	Attn: Executive Director
		  		  	112 North Capitol Street
		  		  	6 Gilmore Hall
		  		  	Iowa City, Iowa 52242-1320
			
	(b)	  	For the LICENSEE:	  	AAVenue Therapeutics, LLC,
		  		  	c/o The Children’s Hospital of Philadelphia
		  		  	34th Street and Civic Center Blvd., Philadelphia, PA 19104
		  		  	Attn: Chief Executive Officer
			
		  	With a copy to:	  	The Children’s Hospital of Philadelphia
		  		  	34th Street and Civic Center Blvd.,
		  		  	Philadelphia, PA 19104
		  		  	Attn: General Counsel

 13.2 This Agreement shall be construed, interpreted, and applied in accordance with the laws of the State of Iowa without
regard to any conflicts of law principles that would require the application of the laws of another jurisdiction. 
 13.3 LICENSEE agrees to comply with all
applicable laws and regulations. In particular, it is understood and acknowledged that the transfer of certain commodities and technical data is subject to United States laws and regulations controlling the export of such commodities and technical
data, including all Export Administration Regulations of the United States Department 

 
of Commerce. These laws and regulations among other things, prohibit or require a license for the export of certain types of technical data to certain specified countries. LICENSEE hereby agrees
and gives written assurance that it will comply with all United States laws and regulations controlling the export of commodities and technical data, that it will be solely responsible for any violation of such by LICENSEE or its AFFILIATES or
sublicensees, and that it will defend and hold UIRF harmless in the event of any legal action of any nature occasioned by such violation. 

ARTICLE XIV — INTEGRATION 
 14.1 This
Agreement constitutes the final and entire agreement between the parties, and supersedes all prior written agreements and any prior or contemporaneous oral understanding regarding the subject matter hereof. Any representation, promise or condition
in connection with such subject matter which is not incorporated in this agreement shall not be binding on either party. No modification, renewal, extension or termination of this agreement or any of its provisions shall be binding upon the party
against whom enforcement of such modification, renewal, extension or termination is sought, unless made in writing and signed on behalf of such party by a duly authorized officer. 

IN WITNESS WHEREOF, each of the parties have caused this agreement to be executed by its duly authorized representative. 

 

									
	LICENSOR	 		 	LICENSEE
	The University of Iowa Research Foundation	 		 	AAVenue Therapeutics, LLC
					
	By:	 	 /s/ Zev Sunleaf
	 		 	By:	 	 /s/ Jeffrey D. Marrazzo

									
	Name:	 	Zev Sunleaf	 		 	Name:	 	Jeffrey D. Marrazzo

									
	Title:	 	Executive Director	 		 	Title: 	 	President and CEO
			
	 9/5/13
	 		 	 10/14/13

	Date	 		 	Date

 Appendix A 

Patent Rights 
  

																	
	 UIRF
Invention
No.
	 	Title	 	Application
No.	 	Patent /
IP No.	 	Application
Filing Date	 	Issue
Date of
Patent	 	Country of
Filing	 	UIRF Patent
No.	 	VHP Ref.
No.

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

 Appendix B 

Common Share Membership Agreement 

 Common Share Membership Agreement 

This Common Share Membership Agreement (this “Agreement”) is made as of October 14, 2013 (the “Admission
Date”), by and between AAVenue Therapeutics, LLC (the “Company”) and the University of Iowa Research Foundation (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; Admission as Member of the Company. 

(a) As of the Admission 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 become a member of the Company effective as of the Admission 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. 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 License Agreement 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 License Agreement 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 Admission Date. 

 

					
	 MEMBER:

	
	THE UNIVERSITY OF IOWA RESEARCH FOUNDATION
		
	 By: 
	 	 /s/ Zev Sunleaf

		 	Name:	 	  Zev Sunleaf
		 	Title:	 	  Executive Director

  

			
	 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 10.11 to the Company’s Registration Statement on Form S-1 

 Appendix C 

Research Collaboration Agreement effective as of February 10, 2012, by and among UI, on behalf of itself and its employee [**], Howard
Hughes Medical Institute (“HHMI”) and The Children’s Hospital of Philadelphia (“CHOP”), as amended effective November 19, 2012 and September 3, 2013 

 September     , 2013 

Jeffrey D. Marrazzo 
 President & CEO 

AAVenue Therapeutics, LLC 
 c/o The Children’s Hospital of
Philadelphia 
 34th Street and Civic Center Blvd., Philadelphia, PA 19104 
  

	RE:	Research Collaboration Agreement (RCA), effective February 10, 2012 and amended November 19, 2012 and September 3, 2013, by and among The University of Iowa, on behalf of itself and its employee, [**]
(“Iowa”). Howard Hughes Medical Institute (“HHMI”), and The Children’s Hospital of Philadelphia (“Hospital”) 

Dear Dr. Marrazzo: 
 This Memorandum of
Understanding (MOU) serves as a follow up to our conversations regarding the above referenced Research Collaboration Agreement (RCA), which is attached herein. 

The University of Iowa Research Foundation (UIRF) understands and acknowledges that the intent is to replace the RCA with a Sponsored Research
Agreement (SRA) between Iowa and AAVenue Therapeutics, LLC (AAVenue). In the meantime, from now until the SRA becomes fully executed, the UIRF agrees to the following: 
  

	 	•	 	For any “Sole Inventions” owned by Iowa, as defined in the RCA, in the event that the UIRF files a patent application claiming such Sole Invention, the UIRF agrees to incorporate such patent application under
the PATENT RIGHTS as defined in Paragraph 1.1. of the License Agreement between UIRF and AAVenue, effective October 14, 2013, a copy of which is attached herein. 

 

	 	•	 	For any “Joint Inventions” that are jointly owned by Iowa and Hospital or HHMI, as defined in the RCA, in the event that the UIRF or Hospital files a patent application claiming such Joint Invention, and to
the extent that Hospital agrees to assign or license its interest, if any, in such patent rights to AAVenue, the UIRF agrees to incorporate such patent application under the PATENT RIGHTS as defined in Paragraph 1.1. of the License Agreement between
UIRF and AAVenue, effective October 14, 2013. 

 If these provisions are acceptable to you, please sign and date below.
Then return a copy of this letter. 
 Best Regards, 
 /s/ Zev
Sunleaf 
 Zev Sunleaf 
 Executive Director 

University of Iowa Research Foundation 

 ACKNOWLEDGED AND AGREED: 
  

									
	By:	 	 /s/ Jeffrey D. Marrazzo
	  	        Date:	  	 October 14, 2013
	  	
	 Jeffrey D. Marrazzo
 President &
CEO, AAVenue Therapeutics
	  		  		  	

 RESEARCH COLLABORATION AGREEMENT 

This Research Collaboration Agreement (the “Agreement”), having an Effective Date of February 10, 2012 is made “between The University of
Iowa on behalf of itself and its employee, [**] (“Iowa”), and Howard Hughes Medical Institute (“HHMI”) and The Children’s Hospital of Philadelphia (the “Hospital”) under the following terms and conditions. HHMI and
the Hospital are referred to collectively in this Agreement as the “Institutions” and, unless specifically named, are treated as a single party. 

1. Research Project. Iowa and the Institutions desire to undertake collaborative research activities for the purpose of developing a
protocol using Adeno-Associated Virus (“AAV”) gene therapy as a potential treatment for subjects diagnosed with Huntington’s and Batten’s disease (“Research Activities”). Iowa and the Institutions will work jointly to
develop a safe and effective AAV vector for future testing in animal models and ultimately in human subjects (the “Research Project”). The respective contributions of Iowa and the Institutions to the Research Project are described in the
Statement of Work set forth on Attachment A to this Agreement, which is incorporated herein by reference. The Principal Investigator for the Institutions will be [**]. The Principal Investigator for Iowa will be [**]. The Research Project shall not
exceed the scope of work set forth on Attachment A, provided that [**] may from time to time, agree to certain modifications of the Scope of Work detailed in Attachment A herein that do not materially alter its scope, as they believe appropriate.
Any significant changes must be in writing and must be approved by Iowa, HHMI, and the Hospital. Each party will bear all of its own costs and expenses in connection with the Research Project. 

All parties agree that clinical trials will not be performed under this Agreement. A separate agreement between the parties is required for
the conduct of any future clinical trials. In addition, Iowa agrees that CHOP will be the sponsor of any clinical trial directly resulting from Research A divides under this Agreement. The parties agree that in consideration of Iowa’s
performance of Research Activities under this Agreement, any and all subsequent clinical trials resulting from such Research Activities shall be offered for placement at Iowa. Such clinical trials shall be conducted by Iowa under die direction of an
Iowa employee acting as principal investigator. 
 2. Transfer of Materials Among Parties. Biological and other research materials,
as hereinafter defined, may be transferred between Iowa and the Institutions in connection with the Research Project. The following terms shall govern any transfer of materials pursuant to the Research Project. 

a. In General. It is expected that Iowa will transfer to the Institutions materials developed outside the course of the Research
Project as set forth in Attachment A, and the Institutions will transfer to Iowa materials developed outside the course of the Research Project as set forth in Attachment A. In addition, other materials developed during the course of the Research
Project may be transferred between the parties as part of the Research Project. Materials developed solely by Iowa, whether developed before or after the Effective Date, together with progeny and unmodified derivatives, will be owned solely by Iowa
(“Iowa Materials”); materials developed solely by Institutions, whether before or after the Effective Date, together with progeny and unmodified derivatives, will be owned solely by Institutions (“Institutions Materials”);
materials developed jointly by researchers at Iowa and the Institutions 

 
in the course of the Research Project will be owned jointly (“Jointly Developed Materials”). Iowa Materials, Institutions Materials and Jointly Developed Materials are sometimes
hereinafter referred to as “Research Materials,” singly or collectively. 
 b. No Warranties. All Research
Materials transferred in connection with the Research Project are experimental in nature and shall be used with prudence and appropriate caution, since not all of their characteristics are known. ALL RESEARCH MATERIALS ARE PROVIDED WITHOUT
WARRANTY OF MERCHANTABILITY OR FITNESS FOR A PARTICULAR PURPOSE OR ANY OTHER WARRANTY, EXPRESS OR IMPLIED. A party providing its Research Materials makes no representation or warranty to the receiving party that the use of such Research
Materials will not infringe any patent, copyright, trademark or other proprietary right. 
 c. Legal Title; Use. Legal
title to any Research Materials transferred hereunder shall be unaffected by this Agreement or the transfer made hereunder. The Institutions agree that the Iowa Materials shall be used only in the course of the Research Project and only in [**]
laboratory in research by laboratory personnel under her immediate and direct control. Iowa will use Institutions Materials only in the course of the Research Project, and only in [**] laboratory in research by laboratory personnel under her
immediate and direct control. In addition, during the term of the Research Project, any Jointly Developed Materials will not be used by the parties for any purpose other than in the Research Project. 

d. Limitations. Research Materials transferred under this Agreement are provided only for use in vitro. Research Materials
transferred under this Agreement will not be used in humans, including for purposes of diagnostic testing. Any use of Iowa Materials by the Institutions, or of Institutions Materials by Iowa, or of Jointly Developed Materials by a party,
other than in accordance with this paragraph 2, is a material breach of this Agreement for purposes of the termination provisions of paragraph 8, below. 

e. Recipient Rights in Transferred Materials. The transfer of Iowa Material to the Institutions, and the transfer of
Institutions Materials to Iowa, gives the recipient no rights to such Iowa Materials or Institutions Materials other than those specifically set forth in this Agreement. 

3. Confidentiality. Subject to paragraph 5, below, during the term of this Agreement and for a period of [**] years thereafter, each
party shall keep confidential all written and oral information that is disclosed to it by the other party in connection with the Research Project that is marked “Confidential” and/or identified in writing as “Confidential” by the
disclosing parry (“Confidential Information”) and treat the Confidential Information according to the same internal security procedures and with the same degree of care regarding its confidentiality as the party receiving the disclosure
treats similar information of its own within its organization. Confidential Information does not include information that: (i) is or later becomes available to the public through no breach of this Agreement; (ii) is obtained from a third
party who had the legal right to disclose the information; (iii) as of the date of disclosure, is already in the possession of the party to whom disclosure is made; or (iv) is required to be disclosed by law, government regulation, or
court order. Receiving party may retain one (1) copy of all Confidential Information for archival purposes. 

 4. Results of Research Project. 

a. In General. Each party will keep the other parties informed of research results obtained from its work in connection
with the Research Project (“Research Project Results”). Confidential Information shared in accordance with this paragraph shall be treated as Confidential by the party to which it is disclosed, and shall be handled by that party in
accordance with, the terms of paragraph 3, above until such Research Project Results are published in accordance with Section 5 herein. Following the collaboration, each party shall have an unrestricted right to use for its own internal
research purposes all Research Project Results, including without limitation any Sole Invention of any party and any Joint Invention (as such terms are defined below), obtained from the Research Project. The party generating any tangible Research
Project Results will make a reasonable number of samples of such Results available to the other parties as soon as the Research Project Results have been generated. 

b. Inventions. For purposes of this Agreement, an “Invention” is any invention or discovery, whether patentable
or nonpatentable, or copyrightable or non-copyrightable, that is conceived or reduced to practice in the course of the Research Project. Inventorship of Inventions will be determined in accordance with principles of U.S. patent law. In the case of a
non-patentable Invention, inventorship will be determined under such principles by treating such Invention as if it were patentable. If an Invention is made by one or more inventors all of whom are required to assign rights in the Invention to a
single party (a “Sole Invention”), the Sole Invention shall be the property of that party. If an Invention is made by more than one inventor, and at least one inventor is required to assign rights in the Invention to Iowa, and at least one
inventor is required to assign rights in the Invention either to HHMI or to the Hospital, the Invention shall be jointly owned by the parties who are assigned rights in the Invention (each, a “Joint Invention”). However, HHMI will assign
its rights in any Sole Inventions and Joint Inventions to the Hospital pursuant to the collaborative arrangements between them, subject, however, to a research-use license retained by HHMI. In the event of such Joint Inventions, the Hospital and
Iowa may pursue joint patent protection of Joint Inventions. 
 c. Licensing of Sole Inventions. Each of Iowa and the
Institutions separately reserve the right to license their interest(s) in any Sole or Joint Invention, subject to the other party’s right to use the Sole or Joint Invention for their own internal research or academic purposes, and the
Institutions or Iowa, as the case may be, shall have no right to compensation in connection with any such license granted by the other party to any third party. 

5. Publication. It is contemplated that the Research Project Results will be jointly published; however, the Institutions and Iowa each
separately reserve the right to publish Research Project Results generated in the course of the Research Project. The parties agree to abide by the policies of major medical journals in which publications will appear and the International Committee
of Medical Journal Editors (“ICMJE”) as to such matters as the public release or availability of data or biological materials relating to the publication and to abide by the HHMI Research Tools and Sharing Policies (found at
http://www.hhmi.org/about/research/policies.html#sharing.) Authorship of the Research Project Results will be determined in accordance with academic standards and custom. Proper acknowledgment will be made for the contributions of each party to the
Research Project Results 

 
being published. If a proposed publication is not a joint publication, the party wishing to make the publication shall provide a copy of the manuscript or abstract to the other party at least
[**] days prior to publication in order to allow the other party an opportunity to protect Confidential Information or intellectual property that might be disclosed by the manuscript or abstract. In addition, a party will not publish Confidential
Information received from the other party (not to include Project Research Results) without such other party’s written consent. Upon publication of Research Project Results, the confidentiality restrictions of this Agreement shall no longer
apply to them. In addition, each party shall make samples of its Research Materials (including Jointly Developed Materials) disclosed in the publication available upon request (supplies permitting) to scientists at non-profit institutions, provided
that the recipient scientist agrees in writing that such Research Materials (i) will be used for research in the recipient scientist’s laboratory only, (ii) will not be used for any commercial purpose, (iii) will not be used for
work on human subjects, and (iv) will not be distributed to other laboratories. 
 6. Responsibilities of the
Parties. Each party is an independent contractor and has no authority to bind or act on behalf of another party. Each party is responsible and liable to the other parties only for the negligent acts and omissions of its employees acting
within the scope of his/her employment relating to the Research Project or to any Research Materials that have been transferred to it in connection with the Research Project, to the full extent permitted by the Iowa Tort Claims Act which is the
exclusive remedy for processing tort claims against the State of Iowa. 
 7. Compliance with Laws and Regulations. All
research done in connection with the Research Project, including all use of Research Materials transferred hereunder, will be done in compliance with all applicable federal, state or local laws, governmental regulations of the United States,
including without limitation current NIH regulations or regulations pertaining to research with recombinant DNA that may be applicable. 

8. Term of Agreement; Duration of Research Project. This Agreement shall go into effect on the Effective Date and shall
continue in effect until the Research Project is completed or terminated. It is expected that the Research Project will be completed within approximately three years of the Effective Date. However, Iowa, HHMI, or the Hospital may terminate the
Research Project and this Agreement at any time upon 30 days’ written notice to the other parties, regardless of whether the Research Project has been completed. In addition, in the event of a material breach of this Agreement by a party, any
other party may terminate the Research Project and this Agreement immediately upon written notice to both other parties. If the Research Project and this Agreement are terminated, Iowa Materials received pursuant to this Agreement by the
Institutions shall, at the request of Iowa, be returned to Iowa or properly destroyed, and Institution Materials received pursuant to this Agreement by Iowa shall, at the request of the Institutions, be returned to the Institutions or properly
destroyed. The terms of paragraphs 2, 3, 4, 5, 6, and 7, this sentence, and the preceding sentence shall survive any termination of this Agreement. 

9. Assignment. This Agreement is not assignable by a party, whether by operation of law or otherwise, either in whole or in
part, without the prior written consent of the other parties. 

 10. Counterparts. This Agreement may be executed in counterparts, each of which shall be
an original, but which counterparts shall together constitute one and the same instrument. This Agreement may also be delivered electronically, and an electronic copy of an executed signature page or pages shall be as binding as originals. 

11. Entire Agreement. This Agreement (including Attachment A hereto), and any other documents executed in connection herewith by
authorized representatives of the parties, contain the entire agreement between the parties relating to the subject matter contained herein, and supersede all prior or contemporaneous agreements, written or oral, with respect thereto. 

12. Export Control. Prior to transferring any materials which are subject to export control, a party shall notify the receiving party
in writing of the nature and extent of the export control. 
 Agreed by: 
  

			
	HOWARD HUGHES MEDICAL INSTITUTE

  

			
	By:	 	 /s/ Jack E. Dixon

 

			
	Name:	 	 Jack E. Dixon, PhD

 

			
	Title:	 	 Vice President and Chief Scientific
Officer

  

			
	Date:	 	 2-10-2012

 

			
	Children’s Hospital of Philadelphia, The

  

			
	By:	 	 /s/ Ellen Purpus

 

			
	Name:	 	 Ellen Purpus, Ph.D.

 

			
	Title:	 	 Director of Technology Transfer

 

			
	Date:	 	 15 Feb 2012

 

			
	The University of Iowa

  

			
	By:	 	 /s/ Wendy Beaver

 

			
	Name:	 	 Wendy Beaver

 

			
	Title:	 	Senior Associate Director
		 	 Division of Sponsored Programs

 

			
	Date:	 	 2-23-2012

					
	Read and acknowledged:
		
		 	[**]
			
		 	Date:	 	 Feb 14, 2012

		
		 	[**]
			
		 	Date:	 	 2/22/2012

 Attachment A 

Research Collaboration Agreement 

STATEMENT OF WORK 
 The Institutions will
supply Iowa with the following biological or other materials developed outside of the Research Project: [**]. 
 Iowa will supply the Institutions with the
following biological or other materials developed outside of the Research Project: [**]. 
 Iowa will supply Institutions with the following biological or
other materials developed during the course of the Research Project: [**]. 
 Description of the research that will be done by the Institutions in the
course of the Research Project: [**]. 
 Description of the research that will be done by Iowa in the course of the Research Project: [**]. 

Clinical trials will not be performed under this Agreement. A separate agreement between the parties is required for the conduct of any future clinical trial.
In addition, Iowa agrees that CHOP will be the sponsor of any clinical trial directly resulting from Research Activities under this Agreement. The parties agree that in consideration of Iowa’s performance of Research Activities under this
Agreement, any and all subsequent clinical trials resulting from such Research Activities shall be offered for placement at Iowa. Such clinical trials shall be conducted by Iowa under the direction of an Iowa employee acting as principal
investigator. 

 CONTRACT AMENDMENT #1 

This Amendment issued this 19th day of November, 2012, serves as modification to the Research
Collaborative Agreement (the “Agreement”), made between The University of Iowa on behalf of itself and its employee, [**] (“Iowa”), and Howard Hughes Medical Institute (“HHMI) and The Children’s Hospital of Philadelphia
(the “Hospital”) dated February 10, 2012. 
 The terms of the Agreement are hereby modified as follows: 

 

	 	(1)	Paragraph 4(c) is amended to include: Each of Iowa and the Institutions agree not to license their interest(s) in any Sole or Joint Inventions until the earlier of the following: (1) Submission of an IND to the FDA
based on the Research Project; or (2) until at least [**] days after the final results of the Research Project funded by Hospital have been received by both Parties. The foregoing is subject to each party’s right to use the Sole or Joint
Invention for its own internal research or academic purposes following the collaboration. 

  

	 	(2)	The following Paragraph (d) is added to Section 4 Results of Research Project: 

 4(d).
Licensing of Intellectual Property in the field of Huntington’s Disease. Iowa and its designated manager of intellectual property, The University of Iowa Research Foundation, agree not to commercially license the following intellectual property
in the field of Huntington’s Disease and Batten’s Disease therapeutics, without Hospital’s approval, until at least [**] days after the final results of the Research Project funded by Hospital have been received by both Parties. 

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

 

	 	(3)	A Section 13 entitled Payment and Billings shall be added to the Agreement and shall state: 

  

	 	13.	Payment and Billings. It is agreed to and understood by the parties hereto that, total costs to Hospital for Iowa’s performance of the project outlined in Exhibit A, parts I and II, entitled Iowa-CHOP HD Program
and Iowa-CHOP Batten’s Disease Program, respectively, shall not exceed the sum of [**] Dollars ($[**]) and [**] dollars ($[**]), respectively. A detailed budget for the whole Statement of Work is described in Exhibit B, parts I and II. On a
quarterly basis following execution of this amendment, Iowa shall submit an invoice to Hospital reporting the work performed in the calendar quarter immediately preceding the date of the invoice, and Hospital shall pay said invoice within [**] days
of receipt. Invoices shall be submitted to [**]. The period of performance for the Statement of Work described in Exhibit B, parts I and 11 shall be 2 years from the date of execution of this amendment. The budgets described in Exhibit B may be
modified by mutual consent of both Iowa and Hospital. 

  

	 	(4)	Exhibits A and B, attached hereto, shall be added to the Statement of Work of the Research Collaboration Agreement. 

 All other terms and conditions of the Agreement remain in full force and effect. 

IN WITNESS WHEREOF, the parties hereto, represented by officials authorized to bind them, have caused this Amendment to be executed in duplicate as of the
date(s) set forth below. 
  

					
	HOWARD HUGHES MEDICAL INSTITUTE

  

					
	By:	 		 	 /s/ Jack E. Dixon

 

					
	Name:	 		 	 Jack E. Dixon, PhD

 

					
	Title:	 		 	 Vice President and Chief Scientific Officer

 

					
	Date:	 		 	 12/7/12

 

					
	Children’s Hospital of Philadelphia, The

  

					
	By:	 		 	 /s/ Ellen Purpus

 

					
	Name:	 		 	 Ellen Purpus, Ph.D.

 

					
	Title:	 		 	 Director of Technology Transfer

 

					
	Date:	 		 	 27 Nov 2012

 

					
	The University of Iowa

  

					
	By:	 		 	 /s/ Jennifer Lassner

 

					
	Name:	 		 	 Jennifer Lassner

 

					
	Title:	 		 	 Executive Director of Sponsored Programs

 

					
	Date:	 		 	 11/20/12

 

					
	Read and acknowledged:

  

					
		 	[**]	 	

  

					
		 	Date:	 	 Nov 28, 2012

 

					
		 	[**]	 	

  

					
		 	Date:	 	 Nov 16, 2012

 Exhibit A 

Part I 
 Iowa-CHOP HD Program 

[**]. 
 Part II 

Iowa-CHOP Batten’s Disease Program 
 Confidential
Materials omitted and filed separately with the Securities and Exchange Commission. A total of two pages were omitted. [**] 

 Exhibit B 

Budget 
 Part I —
Huntinton’s Disease Budget 
 Confidential Materials omitted and filed separately with the Securities and Exchange Commission. A total of three pages
were omitted. [**] 

 Part II — Batten’s Disease 

Budget for Batten Project — [**] 
 Confidential Materials
omitted and filed separately with the Securities and Exchange Commission. A total of one page was omitted. [**] 

 CONTRACT AMENDMENT #2 

This Amendment issued this 3rd day of September, 2013, serves as a modification to the Research Collaboration Agreement (the “Agreement”), made
among The University of Iowa on behalf of itself and its employee, [**] (“Iowa”), and Howard Hughes Medical Institute (“HHMI”) and The Children’s Hospital of Philadelphia (the “Hospital”) dated February 10,
2012, as previously amended in Contract Amendment #1 thereto, issued November 19, 2012. 
 The terms of the Agreement are hereby modified as follows:

  

	 	(1)	The amendment to paragraph 4(c) created as part of Contract Amendment #1 in November 2012 is amended to: Each of Iowa and the Institutions agree not to license their interest(s) in any Sole or Joint Inventions to any
company other than AAVenue Therapeutics, LLC, a Delaware limited liability company (“AAVenue”), until the earlier of the following: (1) Submission of an IND to the FDA based on the Research Project; or (2) until at least [**]
days after the final results of the Research Project funded by Hospital have been received by both Parties. The foregoing is subject to each party’s right to use the Sole or Joint Invention for its own internal research or academic purposes
following the collaboration. 

  

	 	(2)	The Paragraph (d) added to Section 4, Results of Research Project, created as part of Contract Amendment #1 in November 2012 is amended to: 

4(d). Licensing of Intellectual Property in the field of Huntington’s Disease and Batten’s Disease Therapeutics. Iowa and its
designated manager of intellectual property, The University of Iowa Research Foundation, agree not to commercially license the following intellectual property in the field of Huntington’s Disease and Batten’s Disease therapeutics to any
company other than AAVenue, until at least [**] days after the final results of the Research Project funded by Hospital have been received by both Parties. 

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

As amended hereby, the terms and conditions of the Agreement, as previously amended, remain in full force and effect. 

IN WITNESS WHEREOF, the parties hereto, represented by officials authorized to bind them, have caused this Amendment to be executed in duplicate as of the
date(s) set forth below. 
 HOWARD HUGHES MEDICAL INSTITUTE 
  

			
	By:	 	 /s/ Erin K. O’Shea

 

			
	Name:	 	 Erin K. O’Shea, Ph.D.

 

			
	Title:	 	 Vice President and Chief Scientific Officer

  

			
	Date:	 	 8/28/13

			
	Children’s Hospital of Philadelphia, The

  

			
	By:	 	 /s/ Ellen Purpus

 

			
	Name:	 	 Ellen Purpus, Ph.D.

 

			
	Title:	 	 Director of Technology Transfer

 

			
	Date:	 	 3 Sept 2013

			
	The University of Iowa

  

			
	By:	 	 /s/ Jennifer Lassner

 

			
	Name:	 	 Jennifer Lassner

 

			
	Title:	 	 Executive Director of Sponsored Programs

 

			
	Date:	 	 8/26/13

 

			
	Read and acknowledged:

  

					
		 	[**]
			
		 	Date:	 	  

		
		 	[**]
			
		 	Date:	 	 8/23/13

 LICENSE AGREEMENT AMENDMENT 

This Amendment (hereinafter “Amendment”) serves as a modification to the License Agreement, having an effective date of October 14, 2013, made
by and between the University of Iowa Research Foundation, an Iowa Corporation having its principal office at 6 Gilmore Hall, 112 North Capitol Street, Iowa City, Iowa 52242 (hereinafter “UIRF”), and Spark Therapeutics, LLC, formerly known
as AAVenue Therapeutics, LLC (hereinafter “Licensee”), having offices at c/o The Children’s Hospital of Philadelphia, 34th Street and Civic Center Blvd., Philadelphia, PA 19104
(hereinafter “License”). 
 WHEREAS, UIRF and Licensee desire that the Agreement be amended in order to add patents and patent applications
described and disclosed as part of: 
 [**]. 

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

	1.	Appendix A “Patent Rights” shall be amended to add the following patents and patent applications: 

  

																	
	 UIRF
Invention
No.
	 	Title	 	Application
No.	 	Patent /
IP No.	 	Application
Filing Date	 	Issue
Date of
Patent	 	Status	 	Country of
Filing	 	UIRF Patent
No.
	[**]	 	[**]	 	[**]	 	[**]	 	[**]	 	[**]	 	[**]	 	[**]	 	[**]

 Confidential Materials omitted and filed separately with the Securities and Exchange Commission. A total of one
page was omitted. [**] 
  

	2.	In accordance with Section 6.1 of the License, which states that “LICENSEE shall reimburse UIRF for all reasonable expenses UIRF incurs for the preparation, filing, prosecution and maintenance of PATENT
RIGHTS”, Licensee shall pay to UIRF within [**] days, in the amount of [**] Dollars and [**] cents ($[**]) for previously unreimbursed out-of-pocket patent expenses relating to the patents and patent applications specified in paragraph 1 of
this Amendment, which includes services rendered through October 29, 2013 that the UIRF has received invoices for as of October 31, 2013. 

IN WITNESS WHEREOF, the parties hereto have executed this Amendment in duplicate originals by their duly authorized officer or representative. The effective
date of this Amendment is December 27, 2013. 
  

											
	University of Iowa Research Foundation	  		  		  	Spark Therapeutics, LLC
						
	By:	 	 /s/ Zev Sunleaf
	  		  		  	By:	  	 /s/ Jeffrey D. Marrazzo

											
	Name:	 	Zev Sunleaf	  		  		  	Name	  	Jeffrey D. Marrazzo

											
	Title:	 	Executive Director	  		  		  	Title:	  	President and CEO

											
	Date:	 	  
	  		  		  	Date:EX-10.13

 Exhibit 10.13 

Confidential Materials omitted and filed separately with the 

Securities and Exchange Commission. Double asterisks denote omissions. 

Final Execution Copy 

UNIVERSITY OF PENNSYLVANIA 

PATENT LICENSE AGREEMENT 

This Patent License Agreement (this “Agreement”) is between The Trustees of the University of Pennsylvania, a Pennsylvania nonprofit
corporation (“Penn”), and AAVenue Therapeutics, LLC, a Delaware limited liability company (“Company). This Agreement is being signed on October 14, 2013 (the “Execution Date”). This Agreement will
become effective on October 14, 2013 (the “Effective Date”). 
 BACKGROUND 

Penn jointly owns with Cornell University (“CU”) and University of Florida (“UFLA”) certain intellectual property developed by Drs. Jean
Bennett, Albert J. Maguire, Samuel G. Jacobson and Gustavo Aguirre of Penn’s Perelman School of Medicine, and its School of Veterinary Medicine and Gregory M. Acland of CU and William W. Hauswirth of UFLA. The intellectual property is managed
under an Inter-Institutional Agreement (IIA) by and among Penn, CU and UFLA (each individually, an “Institution” and collectively the “Institutions”) under which Penn has, with consultation with the other Institutions, the
authority to negotiate and grant exclusive and nonexclusive licenses on behalf of the Institutions. The intellectual property relates to methods of treating diseases and conditions that may result in blindness, including, in particular, Lebers
Congenital Amaurosis (LCA). The Institutions jointly own certain letters patent and/or applications for letters patent relating to the intellectual property as set forth in Exhibit A below. Company desires to obtain a co-exclusive license under the
aforementioned patent rights and related intellectual property to commercially exploit the same. The Institutions have determined that the co-exclusive commercial exploitation of the patent rights and related intellectual property by Company is in
the best interest of the Institutions and is consistent with their educational and research missions and goals. 
 In consideration of the mutual
obligations contained in this Agreement, and intending to be legally bound, the parties agree as follows: 
 1. LICENSE 

1.1 License Grant. Penn grants to Company a co-exclusive, world-wide license (the “License”) to make, have made, use,
import, offer for sale and sell Licensed Products in the Field of Use during the Term (as such terms may be defined in Sections 1.2 and 6.1). The License includes the right to sublicense as permitted by this Agreement. No other rights or licenses
are granted by Penn. 
 1.2 Related Definitions. The term “Licensed Products” means products that are made, made for,
used, imported, offered for sale or sold by Company or its Affiliates or sublicensees and that would (i) in the absence of the License, infringe (or, in the case of pending patent applications, upon issuance, would infringe) at least one
unexpired claim of the Patent Rights or (ii) use a process or machine covered by a claim of Patent Rights, whether the claim is issued or pending. The term “Patent Rights” means all of Penn’s, CU’s and UFLA’s
patent rights represented by or 

 
issuing from: (a) the United States patents and patent applications listed in Exhibit A; (b) any continuation, divisional and re-issue applications of (a); and (c) any foreign
counterparts and extensions of (a) or (b). The term “Affiliate” means a legal entity that is controlling, controlled by or under common control with Company and that has executed either this Agreement or a written joinder
agreement agreeing to be bound by all of the terms and conditions of this Agreement. For purposes of this Section 1.2, the word “control” means (x) the direct or indirect ownership of more than fifty percent (50%) of
the outstanding voting securities of a legal entity, (y) the right to receive fifty percent (50%) or more of the profits or earnings of a legal entity, or (z) the right to determine the policy decisions of a legal entity. The term
“Field of Use” means research, development, manufacture and commercialization for the diagnosis, treatment, amelioration and prevention of human and animal diseases. The term “co-exclusive” means that, other than the
license granted to Company hereunder, during the term of this Agreement, no more than one license granted by the Institutions to no more than one third party licensee (other than rights granted to non-commercial entities for educational and research
purposes pursuant to Section 1.3) under the Patent Rights will be in effect at any given time. 
 1.3 Reservation of Rights by
Penn. Penn reserves the right for each of the Institutions to use, and to permit other non-commercial entities to use, the Patent Rights for educational and research purposes. 

1.4 U.S. Government Rights. The parties acknowledge that the United States government retains rights in intellectual property funded
under any grant or similar contract with a Federal agency. The License is expressly subject to all applicable United States government rights, including, but not limited to, any applicable requirement that products, which result from such
intellectual property and are sold in the United States, must be substantially manufactured in the United States. 
 1.5 Sublicense
Conditions. The Company’s right to sublicense granted by Penn under the License is subject to each of the following conditions: 

(a) In each sublicense agreement, Company will (i) prohibit the sublicensee from further sublicensing under the License , provided that
such prohibition shall not apply to further sublicensing by any entity that (together with its affiliates) had [**] U.S. dollars or more in worldwide drug product revenues in the calendar year most recently completed as of the grant of the
sublicense; and (ii) require the sublicensee to comply with the terms and conditions of this Agreement applicable to sublicensees. For purposes of Sections 1.5 (a) and (c) and 13.5, “affiliates” shall mean a legal entity
that is controlling, controlled by or under common control with sublicensee. For purposes of these Sections, the word “control” means (x) the direct or indirect ownership of more than fifty percent (50%) of the outstanding
voting securities of a legal entity, (y) the right to receive fifty percent (50%) or more of the profits or earnings of a legal entity, or (z) the right to determine the policy decisions of a legal entity. 

(b) Within [**] days after Company enters into a sublicense agreement, Company will deliver to Penn a complete and accurate copy of the entire
sublicense agreement written in the English language. Penn’s receipt of the sublicense agreement, however, will constitute neither an approval of the sublicense nor a waiver of any right of Penn or obligation of Company under this Agreement.

  
 2 

 (c) In the event that Company causes or experiences a Trigger Event (as defined in
Section 6.4), all payments due to Company from its Affiliates or sublicensees under the sublicense agreement will, upon notice from Penn to such Affiliate or sublicensee, become payable directly to Penn for the account of Company and, subject
to such notice and (except with respect to any sublicensee that is an entity that (together with its affiliates) had [**] U.S. dollars or more in worldwide drug product revenues in the calendar year most recently completed as of the grant of the
sublicense, for which such consent shall not be required) the written consent of Penn, any sublicenses granted to any such Affiliate or sublicensee shall, subject to such continued payments, remain in effect. Upon receipt of any such funds, Penn
will remit to Company the amount by which such payments exceed the amounts owed by Company to Penn. If Penn does not consent to survival of any sublicenses, then (except with respect to any sublicensee that is an entity that (together with its
affiliates) had [**] U.S. dollars or more in worldwide drug product revenues in the calendar year most recently completed as of the grant of the sublicense, for which such consent shall not be required) they terminate along with this Agreement
according to Section 6.3. 
 (d) Company’s execution of a sublicense agreement will not relieve Company of any of its obligations
under this Agreement. Company is primarily liable to Penn 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. 
 1.6 No License by Implication. Nothing in this Agreement confers by estoppel,
implication or otherwise, any license or rights under any Penn patent other than the Patent Rights, regardless whether such patents are dominant or subordinate to the Patent Rights. Nothing in this Agreement confers by estoppel, implication or
otherwise, any license or rights under any Company patent, regardless whether such patents are dominant or subordinate to the Patent Rights, or any authorization under any regulatory approval or orphan drug designation held by Company. 

2. DILIGENCE 
 2.1 Development
Plan. Company will deliver to Penn, a copy of an initial development plan for the Patent Rights (the “Development Plan”). The purpose of the Development Plan is (a) to demonstrate Company’s capability to bring the
Patent Rights to commercialization, (b) to project the timeline for completing the necessary tasks, and (c) to measure Company’s progress against the projections. Thereafter, Company will deliver to Penn an annual updated Development
Plan no later than [**] of each year during the Term. The Development Plan will include, at a minimum, the information listed in Exhibit B. 

2.2 Company’s Efforts. Company will use commercially reasonable efforts to develop, commercialize, market and sell Licensed
Products in a manner consistent with the Development Plan. 
 2.3 Diligence Events. The Company will use commercially reasonable
efforts to achieve each of the diligence events by the applicable completion date listed in the table below for the first Licensed Product. 

  
 3 

					
	 DILIGENCE EVENT
	  	COMPLETION DATE	 
	 [**]
	  	 	[	**] 
	 [**]
	  	 	[	**] 
	 [**]
	  	 	[	**] 

 [**]. 
 2.4
Diligence Resources. Until the filing of the first BLA for the first Licensed Product, Company will expend resources in the development and commercialization of the Licensed Products of amounts not less than the diligence minimums specified
in the table below in each 12-month period following the Effective Date. If Company’s total expenditures for development and commercialization of Licensed Products in any 12-month period do not meet or exceed the applicable diligence minimum,
then Company will pay to Penn the amount of the shortfall. Company will make any payments of the shortfall to Penn within together with the next Development Plan due to Penn under Section 2.1. 

 

													
	 ANNIVERSARY:
	  	First	 	 	Second	 	 	Third and thereafter	 
	 LICENSE DILIGENCE FEE:
	  	 	[	**] 	 	 	[	**] 	 	 	[	**] 

 3. FEES AND ROYALTIES 

3.1 Milestone Payments. In partial consideration of the License, Company will pay to Penn the applicable milestone payment listed in the
table below within [**] days after achievement of each milestone event for each Licensed Product, regardless of whether such milestone was achieved by Company, its Affiliates or sublicensees. Company will provide Penn with written notice within [**]
days after achieving each milestone for each Licensed Product. 
  

					
	 MILESTONE
	  	PAYMENT	 
	 First Commercial Sale of a Licensed Product in the US
	  	$	2,000,000	  
	 First Commercial Sale of a Licensed Product anywhere within the European Union
	  	$	1,750,000	  

 The term “European Union” means the European Union as it is constituted as of the time of the relevant First
Commercial Sale. For the purposes of this Section 3.1 only, the term “First Commercial Sale” shall mean the first sale by Company, its Affiliates or a sublicensee, whether at retail, wholesale or otherwise, of any Licensed
Product following marketing approval in the country of sale to a third party that is not an Affiliate or a sublicensee (a “Commercial Sale”). The following are not Commercial Sales: (i) a transfer or sale by Company to a
sublicensee hereunder or by a sublicensee hereunder to another such sublicensee, unless any such sublicensee is the end user of the Licensed Product, in which case such transfer shall be deemed to be a Commercial Sale; (ii) a transfer by
Company, or any Affiliate or any sublicensee hereunder, to a third party for purposes of clinical trials, as free samples, or under compassionate use, patient assistance, named patient or other similar programs or studies where the Licensed Product
is supplied and/or delivered without charge, or for other testing, or a commercially reasonable number of units of Licensed Product transferred for no consideration for marketing purposes (e.g., samples), but not for resale by the third party;
(iii) the use of Licensed Product by Company or any of its Affiliates or sublicensees for research and development purposes; or (iv) sales made to a distributor prior to commercial launch of a Licensed Product, until the earlier of such
time as Company recognizes the revenue for such transfers pursuant to US GAAP or such time as the distributor makes any sale of such Licensed Product. 

  
 4 

 For clarity, each time a milestone is achieved with respect to a Licensed Product, then any other milestone
payments with respect to earlier milestones that have not yet been paid will be due and payable together with the milestone payment for the milestone that is actually achieved. For additional clarity, milestones are due and payable on Licensed
Products and on products that, upon FDA approval, would become Licensed Products. 
 With Penn’s written concurrence and consent (upon consultation
with the other Institutions), Company may substitute issuance to Penn of Company’s most recently issued preferred membership interests for up to 50% of any of the payments described above in this Section 3.1. If Company requests such
option and Penn (upon consultation with the other Institutions) agrees, the membership interests issued to the Institutions would be based on the payment amount(s) that are eliminated by such issuance and the post-financing per share value of
Company’s most recent preferred membership interests immediately following the closing of Company’s then-most recent equity financing. In connection with any such issuance, the Institutions will enter into a preferred share membership
agreement with Company on terms and conditions substantially the same as the terms and conditions of Company’s other most recent preferred share membership agreements, and such other documents as the parties mutually agree (“Equity
Document(s)”). 
 3.2 Earned Royalties. In partial consideration of the License, Company will pay to Penn a royalty of [**]
percent ([**]%) of Net Sales in the US and [**] percent ([**]%) of Net Sales in countries outside the US during the Quarter with no minimum royalty obligations. Earned royalty payments shall be due and payable regardless of whether they are
triggered by Company, its Affiliates and or its Sublicensees. Company has the right to reduce royalty payments hereunder by amounts paid to third parties for licenses to third party IP by up to [**]% on a country by country basis, if a license to
third party IP is required to sell a Licensed Product. In no event shall royalties to Penn be reduced below [**]% in any country. 
 3.3
Related Definitions. The term “Sale” means any bona fide transaction for the sale, use, lease, transfer or other disposition of a Licensed Product to a third party for which consideration is received or expected by Company or
its Affiliate or sublicensee. A Sale is deemed completed at the time that Company or its Affiliate or sublicensee invoices, ships or receives payment for a Licensed Product, whichever occurs first. The term “Quarter” means each
three-month period beginning on January 1, April 1, July 1 and October 1. The term “Net Sales” means the consideration received or expected from, or the fair market value attributable to, each Sale,
less Qualifying Costs that are directly attributable to a Sale, specifically identified on an invoice or other documentation and actually borne by Company or its Affiliates or sublicensees. For purposes of determining Net Sales, the words
“fair market value” mean the cash consideration that Company or its Affiliates or sublicensees would realize from an unrelated buyer in an arms’ length sale of an identical item sold in the same quantity and at the time and
place of the transaction. The term “Qualifying Costs” means: (a) customary trade, cash and quantity discounts and inventory management fees paid to wholesalers and distributors; (b) credits, chargebacks, retroactive price
reductions, rebates, refunds or claims or returns that do not exceed the original invoice amount; (c) outbound transportation expenses and transportation insurance premiums; (d) sales and use taxes, tariffs, customs duties, excises and
other taxes and fees imposed 

  
 5 

 
by and indefeasibly paid to a governmental agency (other than taxes on income), (e) negotiated payments made to private sector and government third party payors (e.g., PBMs, HMOs and PPOs)
and purchasers/providers (e.g., staff model HMOs, hospitals and clinics), regardless of the payment mechanism, including without limitation rebate, chargeback and credit mechanisms; and (f) discounts under discount prescription drug programs
and reductions for coupon and voucher programs. 
 3.4 CHOP. In consideration of the terms of this Agreement, Company represents and
warrants as of the Effective Date and covenants during the Term, that the Children’s Hospital of Philadelphia (“CHOP”) [**] as of the Effective Date [**]; provided that, for the avoidance of doubt, [**]. 

4. REPORTS AND PAYMENTS 
 4.1 Royalty
Reports. Within [**] days after the end of each Quarter following the First Commercial Sale, Company will deliver to Penn a report, certified on behalf of the Company by the chief financial officer of Company, detailing the calculation of all
royalties, fees and other payments due to Penn for such Quarter. The report will include, at a minimum, the following information for the Quarter, each listed by product, by country: (a) the number of units of Licensed Products constituting
Sales; (b) the gross consideration invoiced, billed or received for Sales; (c) Qualifying Costs, listed by category of cost; (d) Net Sales; (e) the royalties, fees and other payments owed to Penn, listed by category; and
(f) the computations for any applicable currency conversions. Each royalty report will be substantially in the form of the sample report attached as Exhibit C. 

4.2 Payments. Company will pay all royalties due to Penn under Section 3.2 within [**] days after the end of the Quarter in which
the royalties accrued along with any diligence payments due under Section 2.4. 
 4.3 Records. Company will maintain, and will
cause its Affiliates and sublicensees to maintain, complete and accurate books, records and related background information to verify Sales, Net Sales, and all of the royalties, fees, and other payments due or paid under this Agreement, as well as
the various computations reported under Section 4.1. The records for each Quarter will be maintained for at least [**] years after submission of the applicable report required under Section 4.1. 

4.4 Audit Rights. Upon reasonable prior written notice to Company, Company and its Affiliates and sublicensees will provide an
independent accounting firm designated by Penn and reasonably acceptable to Company, which independent accounting firm shall be required to enter into a reasonable confidentiality agreement with Company with access to all of the books, records, key
personnel and related background information required to conduct a review or audit of Sales, Net Sales, and all of the royalties, fees, and other payments payable under this Agreement. Access will be made available: (a) during normal business
hours; (b) in a manner reasonably designed to facilitate such review or audit without unreasonable disruption to Company’s business; and (c) no more than [**] during the Term (as defined below) and for a period of [**] years
thereafter. Penn’s independent accounting firm will disclose to Penn the discrepancies in the amounts paid by Company to Penn identified in such review or audit and such underlying books, records or

  
 6 

 
background information necessary or useful in such determination. Company will promptly pay to Penn the amount of any underpayment determined by the review or audit, plus accrued interest. If the
review or audit determines that Company has underpaid any payment by [**] percent ([**]%) or more, then Company will also promptly pay the costs and expenses of Penn and its accountants in connection with the review or audit. 

4.5 Information Rights. In the event that, in response to a proposal from Company, the Institutions elect to receive equity in Company
as described in Section 3.1 above, then, thereafter, until the earlier of the closing of the Company’s initial public offering or such time as Penn, CU and UFLA no longer hold such equity, Company will provide to the Institutions, at least
as frequently as the following reports are distributed to the Board of Directors or management of Company, copies of: (a) all Board and managerial reports that relate to the Patent Rights or the Licensed Products; and (b) all business
plans, projections and financial statements for Company that are distributed to the Board of Directors or management of Company. For clarity, in the event that, in response to a proposal from Company, the Institutions elect to not receive equity in
Company, then until the closing of the Company’s initial public offering, Company will provide to Perm, at least as frequently as the following reports are distributed to the Board of Directors or management of Company, copies of all Board and
managerial reports that relate to the Patent Rights or the Licensed Products. After the closing of the Company’s initial public offering, Company will provide to Penn, promptly after filing, a copy of each annual report, proxy statement, 10-K,
10-Q and other material report filed with the U.S. Securities and Exchange Commission. 
 4.6 Currency. All dollar amounts referred to
in this Agreement are expressed in United States dollars. All payments will be made in United States dollars. If Company receives payment from a third party in a currency other than United States dollars for which a royalty or fee is owed under this
Agreement, then (a) the payment will be converted into United States dollars at the conversion rate for the foreign currency as published in the eastern edition of the Wall Street Journal as of the last business day of the Quarter in which the
payment was received by Company, and (b) the conversion computation will be documented by Company in the applicable report delivered to Penn under Section 4.1. 

  
 7 

 4.7 Place of Payment. All payments by Company are payable to “The Trustees of the
University of Pennsylvania” and will be made to the following addresses: 
  

					
	 Bv ACH/Wire:
	  	 Bv Check (direct mail):
	  	 Bv Check (lockbox):

	 [**]
  

Payment should include the
 necessary amount to
cover
 any bank charges incurred
	  	 The Trustees of the
 University of

Pennsylvania c/o Center
 for Technology Transfer

Attention: Financial
 Coordinator
	  	 The Trustees of the
 University of

Pennsylvania
 c/o Center for

Technology Transfer
 PO Box 785546

Philadelphia, PA
 19178-5546

 4.8 Interest. All amounts that are not paid by Company when due will accrue interest from the date due
until paid at a rate equal to [**] percent ([**]%) per month (or the maximum allowed by law, if lower). 
 5. CONFIDENTIALITY AND USE OF PENN’S NAME

 5.1 Confidentiality Agreement. If Company and Penn entered into one or more Confidential Disclosure Agreements prior to the
Effective Date, then such agreements will continue to govern the protection of confidential information under this Agreement, and each Affiliate and sublicensee of Company will be bound to Company’s obligations under such agreements. If,
however, no Confidential Disclosure Agreement has been entered into between Company and Penn prior to the Effective Date, then in connection with the execution of this Agreement, the parties will enter into a Confidential Disclosure Agreement
substantially similar to Penn’s standard form. Notwithstanding the foregoing, Penn shall be entitled to share any such Confidential Information with CU and UFLA under terms of confidentiality at least as restrictive as those set forth in the
Confidentiality Agreement. The term “Confidentiality Agreement” means all Confidential Disclosure Agreements between the parties that remain in effect after the Effective Date. 

5.2 Other Confidential Matters. Penn is not obligated to accept any confidential information from Company, except for the reports
required by Sections 2.1, 4.1, 4.4 and 6.6. Penn, acting through its Center for Technology Transfer and finance offices, will use reasonable efforts not to disclose to any third party outside of Penn (other than CU and UFLA) any confidential
information of Company contained in those reports other than Penn’s, CU’s and UFLA’s accountants and advisors under appropriate confidentiality obligations, for so long as such information remains confidential. Each of Penn, CU and
UFLA bear no institutional responsibility for maintaining the confidentiality of any other information of Company. Company may elect to enter into confidentiality agreements with individual investigators at Penn, CU or UFLA that comply with the
internal policies of Penn, CU and UFLA, as applicable. 
 5.3 Use of Name. Company and its Affiliates, sublicensees, employees, and
agents may not use the name, logo, seal, trademark, or service mark (including any adaptation of them) of Penn, CU or UFLA or any Penn, CU or UFLA school, organization, employee, student or representative, without the prior written consent of such
Institution(s). 

  
 8 

 6. TERM AND TERMINATION 

6.1 Term. This Agreement will commence on Effective Date and terminate upon the expiration or abandonment of the last patent to expire
or become abandoned of the Patent Rights (the “Term”). 
 6.2 Early Termination by Company. Company may terminate this
Agreement at any time effective upon completion of each of the following conditions: (a) providing at least sixty (60) days’ prior written notice to Penn of such intention to terminate; (b) ceasing to make, have made, use,
import, offer for sale and sell all Licensed Products; (c) terminating all sublicenses and causing all Affiliates and sublicensees to cease making, having made, using, importing, offering for sale and selling all Licensed Products; and
(d) paying all amounts owed to Penn, CU and/or UFLA, as applicable, under this Agreement and any Sponsored Research Agreement between Company and any or all of the Institutions related to the Patent Rights, through the effective date of
termination. 
 6.3 Early Termination by Penn. Penn may terminate this Agreement if: (a) Company is more than [**] days late in
paying to Penn,, as applicable, any amounts owed under this Agreement and does not pay Penn, as applicable, in full, including accrued interest, within [**] days following written notice of such payment default (a “Payment Default”);
(b) other than a Payment Default, Company or its Affiliate or sublicensee breaches this Agreement and does not cure the breach within [**] days after written notice of the breach; or (c) Company or its Affiliate or sublicensee experiences
a Trigger Event. 
 6.4 Trigger Event. The term “Trigger Event means any of the following: (a) in the event that
Penn, CU and/or UFLA, as applicable, receive equity in Company under this Agreement, a material default by Company under any Equity Document, to the extent applicable, that is not cured within any cure period specified in the Equity Document(s), or
within thirty (30) days of written notice, if no cure period is specified; (b) Company (i) becomes insolvent, bankrupt or generally fails to pay its debts as such debts become due, (ii) is adjudicated insolvent or bankrupt,
(iii) admits in writing its inability to pay its debts, (iv) suffers the appointment of a custodian, receiver or trustee for it or its property and, if appointed without its consent, such appointment is not discharged within thirty
(30) days, (v) makes an assignment for the benefit of creditors, or (vi) suffers proceedings being instituted against it under any law related to bankruptcy, insolvency, liquidation or the reorganization, readjustment or release of
debtors and, if contested by it, not dismissed or stayed within ten (10) days; (c) the institution or commencement by Company or its Affiliates of any proceeding under any law related to bankruptcy, insolvency, liquidation or the
reorganization, readjustment or release of debtors; (d) the entering of any order for relief relating to any of the proceedings described in Section 6.4(b) or (c) above; (e) the calling by Company or its Affiliates of a meeting
of its creditors with a view to arranging a composition or adjustment of its debts; (f) the act or failure to act by Company or its Affiliates indicating its consent to, approval of or acquiescence in any of the proceedings described in
Section 6.4(b) – (e) above; (g) dissolution of Company or termination of Company’s LLC Agreement (unless the entity survives as a S or C corporation); or (h) the commencement by Company of any action against Penn, CU or
UFLA, including an action for declaratory judgment, to declare or render invalid or unenforceable the Patent Rights, or any claim thereof. 

  
 9 

 6.5 Effect of Termination. Upon the termination of this Agreement for any reason:
(a) the License terminates; (b) Company and, subject to Section 1.5(c), all its Affiliates and sublicensees will cease all making, having made, using, importing, offering for sale and selling all Licensed Products, except to extent
permitted by Section 6.6; (c) Company will pay to Penn all amounts, including accrued interest, owed to Penn under this Agreement related to the Patent Rights, through the date of termination, including royalties on Licensed Products
invoiced or shipped through the date of termination and any sell off period permitted by Section 6.6, whether or not payment is received prior to termination or expiration of the sell off period permitted by Section 6.6; (d) Company
will, at Penn’s request, return to Penn all confidential information of Penn; and (e) in the case of termination under Section 6.3, all duties of Penn and all rights (but not duties) of Company under this Agreement immediately
terminate without further action required by either Penn or Company. 
 6.6 Inventory & Sell Off. Upon the termination of
this Agreement for any reason, Company will cause physical inventories to be taken immediately of: (a) all completed Licensed Products on hand under the control of Company or its Affiliates or sublicensees; and (b) such Licensed Products
as are in the process of manufacture and any component parts on the date of termination of this Agreement. Company will deliver promptly to Penn a copy of the written inventory, certified by an officer of the Company. Upon termination of this
Agreement for any reason, Company will promptly remove, efface or destroy all references to Penn from any advertising, labels, web sites or other materials used in the promotion of the business of Company or its Affiliates or sublicensees, and
Company and its Affiliates and sublicensees will not represent in any manner that it has rights in or to the Patent Rights or the Licensed Products. Upon the termination of this Agreement for any reason other than pursuant to Section 6.3,
Company may sell off its inventory of Licensed Products existing on the date of termination for a period of [**] months and pay Penn royalties on Sales of such inventory within [**] days following the expiration of such [**] month period. 

6.7 Survival. Company’s obligation to pay all amounts, including accrued interest, owed to Penn under this Agreement will survive
the termination of this Agreement for any reason. Sections 13.10 and 13.11 and Articles 4, 5, 6, 9, 10, and 11 will survive the termination of this Agreement for any reason in accordance with their respective terms. 

7. PATENT PROSECUTION AND MAINTENANCE 

7.1 Patent Control. Penn controls the preparation, prosecution and maintenance of the Patent Rights and the selection of patent counsel,
with input from Company. For purposes of this Article 7, the word “maintenance” includes any interference negotiations, claims, or proceedings, in any forum, brought by Penn, CU, UFLA, Company, a third party, or the United States
Patent and Trademark Office relating to the Patent Rights, and any requests by Penn, CU, UFLA or Company that the United States Patent and Trademark Office reexamine or reissue any patent in the Patent Rights. 

7.2 Payment and Reimbursement. Within [**] days after the Effective Date, Company will reimburse Penn$[**], representing approximately
fifty percent (50%) of all historically accrued attorneys’ fees, expenses, official fees and all other charges accumulated prior to the Effective Date incident to the preparation, filing, prosecution and maintenance of the Patent

  
 10 

 
Rights. Company will reimburse Penn for all documented attorneys’ fees, expenses, official fees and all other charges accumulated on or after the Effective Date incident to the maintenance
of U.S. Patent No. [**], within [**] days after Company’s receipt of invoices for such fees, expenses and charges. Penn will be solely responsible for all attorneys’ fees, expenses, official fees and all other charges accumulated on or
after the Effective Date incident to the preparation, filing, prosecution, and maintenance of all other Patent Rights. Company will be responsible for patent expenses for U.S. Patent No. [**] under this Section 7.2 until such time as there is
another co-exclusive licensee in which case Company will be responsible for 50% of ongoing patent expenses for such patent with other such co-licensee. Penn reserves the right to require the Company to provide a deposit in advance of incurring out
of pocket patent expenses for U.S. Patent No. [**] estimated by counsel to exceed $[**]. If Company fails to reimburse patent expenses under this Section 7.2, or provide a requested deposit with respect to U.S. Patent No. [**], then Penn will
be free at its discretion and expense to either abandon U.S. Patent No. [**] or to continue such maintenance activities, and U.S. Patent No. [**] and any continuation will be automatically excluded from the term “Patent Rights” hereunder.
Penn is free at its discretion and expense to either abandon or continue the preparation, filing, prosecution, and maintenance of all Patent Rights other than U.S. Patent No. [**] at any time. 

8. INFRINGEMENT 
 8.1 Notice.
Company, Penn, CU and UFLA will notify each other promptly of any infringement of the Patent Rights that may come to their attention. Company, Penn, CU and UFLA will consult each other in a timely manner concerning any appropriate response to the
infringement. 
 8.2 Prosecution of Infringement. During such time as Company is the sole licensee under the Patent Rights, Company
may prosecute any infringement of the Patent Rights at Company’s expense, including defending against any counterclaims or cross claims brought by any party against Company, Penn, CU or UFLA regarding the Patent Rights and defending against any
claim that the Patent or Patent Rights are invalid in the course of any infringement action or in a declaratory judgment action. Each of Penn, CU and UFLA reserves the right to intervene voluntarily and join Company in any such infringement
litigation. If Penn, CU or UFLA chooses not to intervene voluntarily, but is a necessary party to the action brought by Company, then Company may join such Institution(s) in the infringement litigation. If Company decides not to prosecute any
infringement of the Patent Rights, then any of Penn, CU or UFLA may elect to prosecute such infringement independently of Company in its or their sole discretion. 

8.3 Cooperation. In any litigation under this Article 8, Company, Penn, CU or UFLA, at the request and sole expense of any other
requesting party, will cooperate to the fullest extent reasonably possible. This Section 8.3 will not be construed to require any of Company, Penn, CU or UFLA 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, any of Company, Penn, CU or UFLA is required to undertake any activity, including legal discovery, in any litigation or potential litigation (other
than litigation or potential litigation to which Penn, CU and/or UFLA is a voluntary party) brought by, or otherwise related to, Company in accordance with this Article 8, as a right of lawful process of a court of competent jurisdiction, then,
subject to Section 8.4, Company will pay all expenses incurred by Penn, CU and/or UFLA, as applicable, in 

  
 11 

 
undertaking such required activities; provided that, if any such litigation or potential litigation is not brought by Company, then (a) if such litigation or potential litigation is brought
by a third party licensee of Penn, CU and/or UFLA with respect to the Patent Rights, then Company shall not be required to pay such expenses incurred by Penn, CU and/or UFLA and (b) if such litigation or potential litigation is not brought by a
third party licensee of Penn, CU and/or UFLA with respect to the Patent Rights, but is also otherwise related to such a third party licensee, then Company shall only be required to pay fifty percent (50%) of such expenses incurred by Penn, CU
and/or UFLA. 
 8.4 Control of Litigation. Company may control any litigation or potential litigation involving the prosecution of
infringement claims regarding the Patent Rights in which none of the Institutions is a party, including the selection of counsel, all with input from Penn, CU, and/or UFLA, as applicable, provided that Company is the sole licensee under the Patent
Rights. Penn, CU, and/or UFLA, as applicable, controls any litigation or potential litigation involving the prosecution of infringement claims regarding the Patent Rights in all other instances, including where Penn, CU, and/or UFLA has elected to
prosecute the infringement independently of Company or has voluntarily or involuntarily joined Company in the infringement litigation, including the selection of counsel, all with input from Company. Company must not settle or compromise any such
litigation in a manner that imposes any obligations or restrictions on Penn, CU or UFLA or grants any rights to the Patent Rights, other than any permitted sublicenses, without the prior written permission of Penn, CU and/or UFLA, as applicable. In
all instances in which Penn, CU and/or UFLA, as applicable, is a party, Penn, CU and/or UFLA reserve the right to select its own counsel. If Penn, CU and/or UFLA is involuntarily joined as a party, each of Penn, CU and UFLA may elect to be
represented by Company’s counsel at Company’s expense if Company is a party to such litigation or potential litigation and retains the right to select its own counsel, and will be responsible for all litigation expenditures with respect to
any such separate representation as set forth in Section 8.5, provided that if, in any such litigation or potential litigation brought by, or otherwise related to, Company, the interests of Penn, CU and/or UFLA, as applicable, and Company are
so adverse as to prohibit counsel from jointly representing Penn, CU and/or UFLA, as applicable, and Company, the Company shall be responsible for all litigation expenditures of such separate representation(s). 

8.5 Recoveries from Litigation. If Company prosecutes any infringement claims either without Penn, CU or UFLA as a party or with Penn,
CU or UFLA involuntarily joined as a party, then Company will reimburse each of Penn, CU and UFLA, as applicable, for its litigation expenditures, including any attorneys’ fees, expenses, official fees and other charges incurred by such
Institutions, even if there are no financial recoveries from the infringement action. Company will reimburse the Institutions within [**] days after receiving each invoice for such amounts incurred by the Institutions. After reimbursing each of
Penn, CU and UFLA, as applicable, for its expenditures, Company will use the financial recoveries from such claims, if any, (a) first, to reimburse Company for its litigation expenditures; and (b) second, to retain any remainder but to
treat the remainder as Net Sales for the purpose of determining the royalties due under Section 3.2. If Company prosecutes any infringement claims with Penn, CU or UFLA joined as a voluntary party, then any financial recoveries from such claims
will be (x) first, shared between Company, Penn, CU and UFLA to reimburse them for their respective shares of the aggregate litigation expenditures; and (y) second, shared equally by Company, on the one hand, and Penn, CU and UFLA, on the
other hand, as to any remainder after Company and each of the Institutions have fully recovered their aggregate litigation expenditures. If Penn, CU and/or UFLA, as applicable, 

  
 12 

 
prosecutes any infringement claims independent of Company, then Penn, CU and/or UFLA, as applicable, will prosecute such infringement at its or their, as applicable, expense, will reimburse
Company for Company’s litigation expenditures, including any attorneys’ fees, expenses, official fees and other charges to the extent incurred by Company in cooperating with Penn, CU and/or UFLA, as applicable, at its or their request, as
applicable, in such prosecuting such claims, and will retain the balance of any financial recoveries in their entirety. 
 9. DISCLAIMER OF WARRANTIES

 9.1 Disclaimer. THE PATENT RIGHTS, LICENSED PRODUCTS AND ANY OTHER TECHNOLOGY LICENSED UNDER THIS AGREEMENT ARE PROVIDED ON AN
“AS IS” BASIS. EACH OF PENN, CU AND UFLA MAKES NO REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, INCLUDING BUT NOT LIMITED TO ANY WARRANTY OF ACCURACY, COMPLETENESS, PERFORMANCE, MERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE,
COMMERCIAL UTILITY, NON-INFRINGEMENT OR TITLE. 
 10. LIMITATION OF LIABILITY 

10.1 Limitation of Liability. NONE OF PENN, CU OR UFLA WILL BE LIABLE TO COMPANY, ITS AFFILIATES, SUBLICENSEES, SUCCESSORS OR ASSIGNS,
OR ANY THIRD PARTY WITH RESPECT TO ANY CLAIM: ARISING FROM COMPANY’S OR ITS AFFILIATES’ OR SUBLICENSEES’ USE OF THE PATENT RIGHTS, LICENSED PRODUCTS OR ANY OTHER TECHNOLOGY LICENSED UNDER THIS AGREEMENT; OR ARISING FROM THE
DEVELOPMENT, TESTING, MANUFACTURE, USE OR SALE OF LICENSED PRODUCTS. NONE OF PENN, CU, OR UFLA WILL BE LIABLE TO COMPANY, ITS AFFILIATES, SUBLICENSEES, SUCCESSORS OR ASSIGNS, OR ANY THIRD PARTY FOR LOST PROFITS, BUSINESS INTERRUPTION, OR INDIRECT,
SPECIAL OR CONSEQUENTIAL DAMAGES OF ANY KIND. EXCEPT FOR COMPANY’S INDEMNIFICATION OBLIGATIONS UNDER ARTICLE 11 OF THIS AGREEMENT, COMPANY WILL NOT BE LIABLE TO PENN, CU, OR UFLA OR TO ANY THIRD PARTY, FOR ANY LOST PROFITS, BUSINESS INFORMATION
OR INDIRECT, SPECIAL OR CONSEQUENTIAL DAMAGES OF ANY KIND. 
 11. INDEMNIFICATION 

11.1 Indemnification. Company will defend, indemnify, and hold harmless each Indemnified Party from and against any and all Liabilities
with respect to an Indemnification Event. The term “Indemnified Party” means each of Penn, CU and UFLA, and their respective trustees, officers, faculty, students, employees, contractors, and agents. The term
“Liabilities’” means all damages, awards, deficiencies, settlement amounts, defaults, assessments, fines, dues, penalties, costs, fees, liabilities, obligations, taxes, liens, losses, lost profits and expenses (including, but
not limited to, court costs, interest and reasonable fees of attorneys, accountants and other experts) that are incurred by an Indemnified Party or awarded or otherwise required to be paid to third parties by an Indemnified Party. The term
“Indemnification Event” means; (a) any Claim by a third party against one or more Indemnified Parties arising out of or resulting from the development, testing, use, manufacture, promotion, sale or other disposition of any
Patent Rights 

  
 13 

 
or Licensed Products by Company, its Affiliates, sublicensees, assignees or vendors or third parties, including, but not limited to, (x) any product liability or other Claim of any kind
related to use by a third party of a Licensed Product, (y) any Claim by a third party that the practice of any of the Patent Rights or the design, composition, manufacture, use, sale or other disposition of any Licensed Product infringes or
violates any patent, copyright, trade secret, trademark or other intellectual property right of such third party, and (z) any Claim by a third party relating to clinical trials or studies for Licensed Products; (b) any Claim by a third
party against one or more Indemnified Parties arising out of or resulting from any material breach of this Agreement by Company or its Affiliates or sublicensees; and (c) the enforcement of this Article 11 by any Indemnified Party. The term
“Claim” means any charges, complaints, actions, suits, proceedings, hearings, investigations, claims or demands. 
 11.2
Reimbursement of Costs. Company will pay directly all Liabilities incurred for defense or negotiation of any Claim pursuant to this Article 11 or will reimburse Penn, CU or UFLA, as applicable, for all documented Liabilities incident to the
defense or negotiation of any Claim pursuant to this Article 11 within [**] days after Company’s receipt of invoices for such fees, expenses and charges. 

11.3 Control of Litigation. Company controls any litigation or potential litigation involving the defense of any Claim pursuant to this
Article 11, including the selection of counsel, with input from Penn, CU and UFLA. Each of Penn, CU and UFLA, as applicable, reserves the right to protect its interest in defending against any Claim pursuant to this Article 11 by selecting its own
counsel, with any attorneys’ fees and litigation expenses paid for by Penn, CU and/or UFLA, as applicable, pursuant to Sections 11.1 and 11.2, unless Penn, CU and/or UFLA, as applicable, takes such action due to a conflict of interest that
makes separate representation of Penn, CU and/or UFLA, as applicable, and Company necessary, in which case Company will pay such attorneys’ fees and litigation expenses. 

11.4 Other Provisions. Company will not settle or compromise any Claim pursuant to this Article 11 giving rise to Liabilities in any
manner that imposes any restrictions or obligations on Penn, CU or UFLA or grants any rights to the Patent Rights or the Licensed Products, other than permitted sublicenses, without prior written consent of Penn, CU and/or UFLA, as applicable. If
Company fails or declines to assume the defense of any Claim pursuant to this Article 11 within [**] days after notice of such Claim, or fails to reimburse an Indemnified Party for any Liabilities pursuant to Sections 11.1 and 11.2 within the [**]
day time period set forth in Section 11.2, then Penn, CU or UFLA, as applicable, may assume the defense of such Claim for the account and at the risk of Company. The indemnification rights of the Indemnified Parties under this Article 11 are in
addition to all other rights that an Indemnified Party may have at law, in equity or otherwise. 
 12. INSURANCE 

12.1 Coverages. Company will procure and maintain insurance policies for coverages with respect to personal injury, bodily injury and
property damage arising out of Company’s performance under this Agreement and reasonably adequate to fulfill any potential obligation to the Indemnified Party, but not less than: (a) during the Term, comprehensive general liability,
including broad form and contractual liability, in a minimum amount of $ [**] per occurrence and $[**] in the aggregate; (b) prior to the commencement of clinical trials involving Licensed 

  
 14 

 
Products, clinical trials coverage in a minimum amount of $[**] combined single limit per occurrence and in the aggregate; and (c) prior to the Sale of the first Licensed Product, product
liability coverage, in a minimum amount of $[**] combined single limit per occurrence and in the aggregate. The required minimum amounts of insurance do not constitute a limitation on Company’s liability or indemnification obligations under
this Agreement. 
 12.2 Other Requirements. The policies of insurance required by Section 12.1 will be issued by an insurance
carrier with an A.M. Best rating of “A” or better and will name Penn, CU and UFLA as an additional insured with respect to Company’s performance under this Agreement. Company will provide Penn with insurance certificates evidencing
the required coverage within [**] days after the Effective Date and the commencement of each policy period and any renewal periods. Each certificate will provide that the insurance carrier will notify Penn in writing at least [**] days prior to the
cancellation or material change in coverage. 
 13. ADDITIONAL PROVISIONS 

13.1 Independent Contractors. The parties are independent contractors. Nothing contained in this Agreement is intended to create an
agency, partnership or joint venture between the parties. At no time will either party make commitments or incur any charges or expenses for or on behalf of the other party. 

13.2 No Discrimination. Neither Penn nor Company will discriminate against any employee or applicant for employment because of race,
color, sex, sexual or affectional preference, age, religion, national or ethnic origin, handicap, or veteran status. 
 13.3 Compliance
with Laws. Company must comply with all prevailing laws, rules and regulations that apply to its activities or obligations under this Agreement. For example, Company will comply with applicable United States export laws and regulations. The
transfer of certain technical data and commodities may require a license from the applicable agency of the United States government and/or written assurances by Company that Company will not export data or commodities to certain foreign countries
without prior approval of the agency. Penn does not represent that no license is required, or that, if required, the license will issue. 

13.4 Modification, Waiver & Remedies. This Agreement may only be modified by a written amendment that is executed by an
authorized representative of each party. Any waiver must be express and in writing. No waiver by either party of a breach by the other party will constitute a waiver of any different or succeeding breach. Unless otherwise specified, all remedies are
cumulative. 
 13.5 Assignment & Hypothecation. Company may not assign this Agreement or any part of it, either directly or
by merger or operation of law, without the prior written consent of Penn, provided that Company may assign this Agreement to an Affiliate, or to a third party that is an entity that (together with its affiliates) had one billion U.S. dollars or more
in worldwide drug product revenues in the calendar year most recently completed as of the date of such assignment in connection with a sale or transfer of all or substantially all of Company’s business or assets, provided that: (a) the
assignee agrees in writing to be legally bound by this Agreement and to deliver to Penn an updated Development Plan within [**] days after the closing of the proposed 

  
 15 

 
transaction; and (b) Company provides Penn with a copy of assignee’s undertaking. Any permitted assignment will not relieve Company of responsibility for performance of any obligation
of Company that has accrued at the time of the assignment. Company will not grant a security interest in the License or this Agreement during the Term, except in connection with a royalty sale or similar royalty monetization transaction. Any
prohibited assignment or security interest will be null and void. 
 13.6 Notices. Any notice or other required communication (each, a
“Notice”) must be in writing, addressed to the party’s respective notice address listed on the signature page, and delivered: (a) personally; (b) by certified mail, postage prepaid, return receipt requested;
(c) by recognized overnight courier service, charges prepaid; or (d) by facsimile. A Notice will be deemed received: if delivered personally, on the date of delivery; if mailed, five (5) days after deposit in the United States mail;
if sent via courier, one (1) business day after deposit with the courier service; or if sent via facsimile, upon receipt of confirmation of transmission provided that a confirming copy of such Notice is sent by certified mail, postage prepaid,
return receipt requested. 
 13.7 Severability & Reformation. If any provision of this Agreement is held to be invalid or
unenforceable by a court of competent jurisdiction, then the remaining provisions of this Agreement will remain in full force and effect. Such invalid or unenforceable provision will be automatically revised to be a valid or enforceable provision
that comes as close as permitted by law to the parties’ original intent. 
 13.8 Headings & Counterparts. The headings
of the articles and sections included in this Agreement are inserted for convenience only and are not intended to affect the meaning or interpretation of this Agreement. This Agreement may be executed in several counterparts, all of which taken
together will constitute the same instrument. 
 13.9 Governing Law. This Agreement will be governed in accordance with the laws of
the Commonwealth of Pennsylvania, without giving effect to the conflict of laws provisions. 
 13.10 Dispute Resolution. If a dispute
arises between the parties concerning any right or duty under this Agreement, then the parties will confer, as soon as practicable, in an attempt to resolve the dispute. If the parties are unable to resolve the dispute amicably, then the parties
will submit to the exclusive jurisdiction of, and venue in, the state and Federal courts located in the Eastern District of Pennsylvania with respect to all disputes arising under this Agreement. 

13.11 Integration. This Agreement with its Exhibits contains the entire agreement between the parties with respect to the Patent Rights
and the License and supersedes all other oral or written representations, statements, or agreements with respect to such subject matter, including but not limited to the Term Sheet. 

Each party has caused this Agreement to be executed by its duly authorized representative. 

  
 16 

									
	 THE TRUSTEES OF THE

UNIVERSITY OF PENNSYLVANIA
	 		 	 AAVENUE THERAPEUTICS

LLC

					
	By:	 	 /s/ John S. Swartley
	 		 	By:	 	 /s/ Jeffrey D. Marrazzo

	Name:	 	John S. Swartley, PhD	 		 	Name:	 	Jeffrey D. Marrazzo
	Title:	 	 Executive Director,
 Center for Technology
Transfer
	 		 	Title:	 	President & Chief Executive Officer

									
		 		 		 		 	
	Address:	 	 Center for Technology Transfer
 University of
Pennsylvania
 3160 Chestnut Street, Suite 200
 Philadelphia, PA
19104-6283
 Attention: Executive Director
	 		 	Address:	 	  
 c/o The Children’s Hospital of Phila.

34th St. & Civic Center Blvd.
 Philadelphia, PA
19104

					
	Required copy to:	 	 University of Pennsylvania
 Office of General
Counsel
 133 South 36th Street, Suite 300

Philadelphia, PA 19104-3246
 Attention: General Counsel
	 		 		 	

  
 17 

 EXHIBIT INDEX 
  

	Exhibit  A	Patents and Patent Applications in Patent Rights 

  

	Exhibit  B	Minimum Contents of Development Plan 

  

	Exhibit  C	Format of Royalty Report 

  
 18 

 Exhibit A 

Patents and Patent Applications in Patent Rights 
  

																									
	 Tech ID: N2514
	  	Method of Treating or Retarding the Development of Blindness	 
	 Serial No.
	  	Patent
No.	 	 	App Type	 	 	File Date	 	 	Status	 	 	Country	 	 	Issue Date	 
	 [**]
	  	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 
	 [**]
	  	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 
	 [**]
	  	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 
	 [**]
	  	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 	 	 	[	**] 

  
 19 

 Exhibit B 

Minimum Contents of Development Plan 
 The
initial Development Plan and each update to the Development Plan will include, at a minimum, the following information: 
  

	 	•	 	The date of the Development Plan and the reporting period covered by the Development Plan. 

  

	 	•	 	Identification and nature of each active relationship between Company and its Affiliates, sublicensees or subcontractors in the research, development or commercialization of Licensed Products or Patent Rights

  

	 	•	 	Significant projects completed during the reporting period by Company or its Affiliates, sublicensees or subcontractors in the research, development or commercialization of Licensed Products or Patent Rights.

  

	 	•	 	Significant projects currently being performed by Company or its Affiliates, sublicensees or subcontractors in the research, development or commercialization of Licensed Products or Patent Rights. 

 

	 	•	 	Future projects expected to be undertaken during the next reporting period by Company or its Affiliates, sublicensees or subcontractors in the research, development or commercialization of Licensed Products or Patent
Rights. 

  

	 	•	 	Projected timelines to product launch of each Licensed Product prior to first Sale. 

  

	 	•	 	Projected annual Net Sales for each Licensed Product after first Sale. 

  

	 	•	 	Significant changes to the current Development Plan since the previous Development Plan and the reasons for the changes. 

  

	 	•	 	Significant assumptions underlying the Development Plan and the future variables that may cause significant changes to the Development Plan. 

 

	 	•	 	Copies of all reports required by Section 4.1 of this Agreement that have not already been delivered to Penn. 

  
 20 

 Exhibit C 
  

			
	

	  	 Center for Technology Transfer

University of Pennsylvania
 Royalty
Report

  

							
	Licensee:	  	  
	  	Agreement:	  	  

	Inventor:	  	  
	  	Patent #:	  	  

	Period Covered: From:	  	                      /             
         /                        	  	Through:	  	                      /             
         /                      
	Prepared By	  	  
	  	Date:	  	  

	Approved By:	  	  
	  	Date:	  	  

 If License covers several major product lines, please prepare a separate for each line. Then combine all
product lines into a summary report. 
  

	Report  Type:	 ̈ Single Product Line Report: 
 ̈ Multi-product Summary Report: Page 1 of
             Pages 
 ̈ Product Line Detail: Line:             
Trade name:                  Page:              

	Report  Currency:	 ̈ U.S. Dollars                  ̈
Other
                                         
                            

 

													
	 Country
	  	Gross
Sales	  	*Less:
Allowances	  	Net
Sales	  	Royalty
Rate	  	Period Royalty Amount
	  	 	  	 	  	 	  	 	  	This Year	  	Last Year
	 U.S.A
	  		  		  		  		  		  	
	 Canada
	  		  		  		  		  		  	
	 Europe
	  		  		  		  		  		  	
		  		  		  		  		  		  	
		  		  		  		  		  		  	
		  		  		  		  		  		  	
		  		  		  		  		  		  	
		  		  		  		  		  		  	
	 Japan
	  		  		  		  		  		  	
	 Other
	  		  		  		  		  		  	
		  		  		  		  		  		  	
		  		  		  		  		  		  	
		  		  		  		  		  		  	
		  		  		  		  		  		  	
		  		  		  		  		  		  	
	 Total:
	  		  		  		  		  		  	

 Total Royalty:
                     Conversion Rate:
                     Royalty in U.S. Dollars $
                         

The following royalty forecast is non-binding and for CTT internal planning purposes only: 

Royalty Forecast Under this agreement: Next Quarter:                 
Q2:                  Q3:                  Q4:
                 

 

On a separate page, please indicate the reasons for returns or other adjustments if significant. Also note any unusual occurrences that
affected royalty amounts during this period. To assist CTT’s forecasting, please comment on any significant expected trends in sales volume 

  
 21

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