EX-10.11



 Exhibit 10.11 

CONFIDENTIAL TREATMENT REQUESTED 

UNIVERSITY of PENNSYLVANIA 

Second Amendment to License Agreement 

This Second Amendment to License Agreement (this “Second Amendment”) effective as of September 9, 2014 (this “Second
Amendment Effective Date”), is made by and between The Trustees of the University of Pennsylvania (“Penn”) and ReGenX Biosciences, LLC (“Company”) (collectively, the “Parties”) and amends
the License Agreement between the Parties, which was effective as of February 24, 2009, as subsequently amended by a First Amendment dated March 6, 2009 (the “License Agreement”). All capitalized terms used but not defined
herein shall have the meaning set forth in the License Agreement. 
 BACKGROUND 

The License Agreement relates to certain intellectual property developed by Dr. James M. Wilson of Penn’s Perelman School of
Medicine, which intellectual property is the subject of patents or patent applications (the “Penn Dockets”). 
 WHEREAS, the Company has
elected to exercise certain of its option rights pursuant to the sponsored research agreement by and between the Parties effective as of February 24, 2009 and as subsequently amended with respect to certain Penn patentable inventions and patent
rights. Penn and Company have reached agreement on terms to extend such additional Penn patent rights to Company as set forth herein; 
 WHEREAS, The
Parties wish to amend the License Agreement to reflect these changes. 
 Now, therefore, in consideration of the promises and covenants contained in this
Second Amendment and for other good and valuable consideration the receipt and sufficiency of which is hereby acknowledged and intending to be legally bound, the Parties agree as follows: 

1) Exhibit A of the License Agreement is hereby amended and restated in its entirety by Exhibit A to this Second Amendment to add the
additional Patent Rights and Know-How designated therein as “Second Amendment Patent Rights and Know-How”. On the Second Amendment Effective Date, Company will reimburse Penn for all patent prosecution and maintenance expenses associated
with the Second Amendment Patent Rights that have not previously been reimbursed by Company. 
 2) The following definitions in
Section 1.2 of the License Agreement shall be amended and restated in their entirety as follows: 
 “Background Know-How”
means all Know-How that (a) was developed by Dr. Wilson, or other Penn researchers working under his direct supervision, at Penn, and (b) is related to the adeno associated virus technology platform discovered by Dr. Wilson at
Penn prior to February 24, 2009, or is related to the adeno associated virus technology platform discovered by Dr. Wilson at Penn after February 24, 2009 pursuant to the SRA, arid (c) is owned by Penn and available for licensing,


  
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and (d) is necessary or useful for the practice of the Patent Rights in connection with the manufacture, use, sale, importation and/or other exploitation of the Licensed Products or the
practice of the Licensed Processes in the Territory in the Field of Use, including, without, limitation, any Know-How necessary for the Company to manufacture or have manufactured the materials produced by the Penn Vector Core or
Dr. Wilson’s lab at Penn. 
 “Field of Use” means any and all fields of use, except with respect to the Patent Rights
listed in Exhibit A, Part 2 and all related Know-How and data, for which the Field of Use is limited to viral vector mediated gene therapy. 

“Know-How” means any and all information, discoveries, software, methods, works of authorship, techniques, formulae, data, biological
materials, processes, unpatentable inventions and other know-how, not including the Patent Rights, developed prior to the Effective Date or under the SRA. 

“Patent Rights” means (i) all of Penn’s patent rights represented by or issuing from the United States patents and patent
applications (including provisional patent applications) listed in Exhibit A, as well as any continuations, continuations-in-part (to the extent the inventions claimed or disclosed in any such patent or patent applications are directed to subject
matter specifically described in the patent or patent applications listed in Exhibit A), divisionals, reexaminations, renewals, re-issues, substitutions, extensions and foreign counterparts of any of the foregoing, and all other patents and patent
applications that claim priority from or have common priority with any of the foregoing patents and patent applications, (to the extent the inventions claimed or disclosed in any such patent or patent applications are directed to subject matter
specifically described in the patent or patent applications listed in Exhibit A) and including any patents issuing from any of the foregoing; and (ii) all patentable inventions (to the extent they are or become available for license) that
(a) were discovered by Dr. Wilson, or other Penn researchers working under his direct supervision, at Penn prior to the Second Amendment Effective Date, and (b) are related to the adeno associated virus technology platform discovered
by Dr. Wilson at Penn prior to the Effective Date or under the SRA, and (c) are owned by Penn and available for licensing. 

“SRA” means the each of: 1) the Sponsored Research Agreement between the Company and Penn effective as of February 24, 2009, as
subsequently amended and 2) the Sponsored Research Agreement between the Company and Penn effective as of November 1, 2013 and any future amendments thereof 

3) Section 1.5 (a) of the License Agreement shall be amended and restated in its entirety as follows: 

“1.5(a) Within **** after Company enters into a sublicense agreement, Company will deliver to Penn a complete and accurate copy of the
entire executed sublicense agreement written in the English language.” 

  

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COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
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 4) Section 2.3 of the License Agreement shall be amended and restated in its entirety as
follows: 
 “Company is deemed to have fully satisfied all of its obligations under this Section 2.” 

5) Section 3.2 is hereby amended to add the following language at the end: 

“Notwithstanding anything herein to the contrary, in no event shall Penn receive a royalty of less than **** (****) of Net Sales of
Licensed Pharmaceutical Products sold by ****, where such Licensed Pharmaceuticals Products are not also covered by GSK Licensed Patents. For clarity, **** include ****.” 

6) Section 6.1 of the License Agreement shall be amended and restated in its entirety as follows: 

“6.1 Term. This Agreement will commence on Effective Date and end upon the expiration of the Royalty Term (the
“Term”). Earned royalties pursuant to Section 3.4 shall only be payable hereunder during the Royalty Term. Upon expiration of the Agreement, Company’s license under Section 1.1(c) will become perpetual, irrevocable,
royalty-free, transferable, sublicensable, and fully paid-up.” 
 7) Section 6.3 of the License Agreement shall be amended and
restated in its entirety as follows: 
 “6.3 Early Termination by Penn. Penn may terminate this Agreement if: (a) Company
is more than thirty (30) days late in paying to Penn any amounts owed under this Agreement and does not pay Penn in full within thirty (30) days after receipt of written notice indicating such default and demanding payment, including accrued
interest (a “Payment Default”); (b) other than a Payment Default, Company or its Affiliate fails to achieve a diligence event on or before the applicable completion date or otherwise breaches this Agreement and does not cure
such failure or breach within sixty (60) days after written notice of the breach; or (c) Company or its Affiliate experiences a Trigger Event.” 

8) Section 6.4 of the License Agreement shall be amended and restated in its entirety as follows: 

“6.4 Trigger Event. The term “Trigger Event” means any of the following: (a) if Company or its Affiliate
(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 

  

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appointed without its consent, not discharged within ****, (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 ****; (b) the institution or commencement by Company or its Affiliate of any
proceeding under any law related to bankruptcy, insolvency, liquidation or the reorganization, readjustment or release of debtors; (c) the entering of any order for relief relating to any of the proceedings described in Section 6.4(a) or
(b) above; (d) the calling by Company or its Affiliate of a meeting of its creditors with a view to arranging a composition or adjustment of its debts; (e) the act or failure to act by Company or its Affiliate indicating its consent
to, approval of or acquiescence in any of the proceedings described in Section 6.4(b) (d) above; or (f) the commencement by Company of any action against Penn, including an action for declaratory judgment, to declare or render invalid
or unenforceable the Patent Rights, or any claim thereof.” 
 9) Section 6.5 of the License Agreement shall be amended and
restated in its entirety as follows: 
 “6.5 Effect of Termination. Upon the early termination of this Agreement pursuant to
Section 6.2 or 6.3: (a) the Licenses shall terminate; (b) Company and all its Affiliates will cease all making, having made, using, importing, offering for sale and selling all Licensed Products and practicing the Licensed Processes;
(c) Company shall assign and Penn will accept the assignment of all sublicenses granted to sublicensees to the extent related solely to the Patent Rights or the Licensed Products; provided, however, that any such sublicensee (i) is not in
breach of any provision of this Agreement or the applicable sublicense agreement as of the effective date of such termination and (ii) upon request by Penn, agrees in writing that such sublicensee will perform all obligations of Company under
this Agreement that are applicable to the sublicensed rights; (d) Company will pay to Penn all amounts, including accrued interest, owed to Penn under this Agreement and any Sponsored Research Agreement related to the Patent Rights, through the
date of termination, including royalties on Licensed Products invoiced or shipped through the date of termination when such payments are received, whether or not payment is received prior to termination; (e) each Party will, at the other
Party’s request, return to such Party all confidential information of such Party; and (0 except as provided in Section 6.6, all rights and duties of Penn and the Company under this Agreement immediately terminate without further action
required by either Penn or Company.” 
 10) The second sentence of Section 13.5 is amended and restated as follows: 

  

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COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
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 Notwithstanding the foregoing, provided that Company is not in breach of any provisions of
this Agreement, Company shalt be permitted to assign this Agreement, without the prior written consent of Penn, pursuant to a merger or sale of all or substantially all of the assets to which the Agreement relates to a company in the business of
developing and commercializing pharmaceutical products that has, together with its affiliates, a market value or, in the case of a publicly traded company listed on a nationally recognized exchange, market capitalization, of at least $250,000,000.

 11. For clarity, Exhibit A now includes, in Part 2, all available intellectual property and technology owned and controlled by Penn and
created at Penn in the laboratory of Dr. James M. Wilson, MA., Ph.D. through the Second Amendment Effective Date in the disease indications of familial hypercholesterolemia (FH) and ornithine transcarbarnylase deficiency (OTC), including
any related data and know-how. 
 12. This Second Amendment, together with the License Agreement and First Amendment, constitute the entire
agreement between the Parties. All other terms and provisions of the License Agreement, except as expressly amended by this Second Amendment, remain in full force and effect. 

13. This Second Amendment may be executed in two or more counterparts, each of which shall be deemed an original and together shall be deemed
one and the same instrument. 
 IN WITNESS WHEREOF, the Parties, intending to be legally bound, have caused this Second Amendment to be executed by their
duly authorized representatives. 
  

			
	THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
		
	By:	 	 /s/ John S. Swartley, Ph.D.

	Name:	 	 John S. Swartley, Ph.D.

	Title:	 	 Associate Vice President for Research

		 	 Executive Director, PCI

	Date:	 	 September 9, 2014

	
	REGENX BIOSCIENCES, LLC
		
	By:	 	 /s/ Kenneth T. Mills

	Name:	 	Kenneth T. Mills
	Title:	 	President and CEO
	Date:	 	 September 9, 2014



  
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 Exhibit A 

Patents and Patent Applications in the Patent Rights 

Part 1; No Field of Use Limitation 
  

							
	 Penn # 
	  	 Disclosure Title
	  	 US Patents
	  	 Foreign Patents

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


  

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



  
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COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
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	 Docket V5085
	  	 Combination method to generate novel and/or hybrid coding sequences within
the defined domains

	 Serial No
	  	 Patent No
	    	 App Type
	    	 File Date
	    	 Country
	    	 Issue Date

	****	  		    	****	    	****	    	****	    	
	****	  		    	****	    	****	    	****	    	
		
	 Docket V5131
	  	 A domain of the adeno-associated virus stereotype 6 capsid that confers
transduction of conducting
airway epithelium

	 Serial No
	  	 Patent No
	    	 App Type
	    	 File Date
	    	 Country
	    	 Issue Date

	****	  		    	****	    	****	    	****	    	
	****	  		    	****	    	****	    	****	    	
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	****	  		    	****	    	****	    	****	    	
	****	  		    	****	    	****	    	****	    	
		
	 Docket X5836
	  	 The receptor for AAV9 and its modification in vivo to improve lung gene
therapy

	 Serial No
	  	 Patent No
	    	 App Type
	    	 File Date
	    	 Country
	    	 Issue Date

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


  

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



  
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	 Docket Z6622
	  	 Treatment of MPS1

	 Serial No
	  	 Patent No
	    	 App Type
	    	 File Date
	    	 Country
	    	 Issue Date

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

 Exhibit A, Part 2 

Field of Use limited to viral vector mediated delivery of gene therapy product 

 

											
	 Docket 14-7025
	  	 Improved AAV-LDLR for treating human disease

	 Serial No
	  	 Patent No
	    	 App Type
	    	 File Date
	    	 Country
	    	 Issue Date

	****	  		    	****	    	****	    	****	    	
	****	  		    	****	    	****	    	****	    	
		
	 Docket 14-7037
	  	 AAV OTC for treating human disease

	 Serial No
	  	 Patent No
	    	 App Type
	    	 File Date
	    	 Country
	    	 Issue Date

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

 Penn / Wilson Lab Know-How for the Familial Hypercholesterolemia and Onithine Transcarbamylase Deficiency (OTC) Programs

 FH Know-How (associated with ****) 
 **** 

  

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COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED
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 OTC Know-How (associated with ****) 

**** 

  

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



  
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