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Exhibit 10.26

LICENSE AGREEMENT
 
This LICENSE AGREEMENT (“Agreement”) is entered into as of November 19, 2018
(“Effective Date”) by and between REGENXBIO Inc., a corporation organized under
the laws of the State of Delaware, with offices at 9600 Blackwell Road, Suite
210, Rockville, MD 20850 (“Licensor”), and Rocket Pharmaceuticals, Ltd., a
Cayman Islands exempted company, with offices at 350 Fifth Avenue, Suite 7530,
New York, NY 10118 (“Licensee”).  Licensor and Licensee are hereinafter referred
to individually as a “Party” and collectively as the “Parties.”
 
WHEREAS, Licensor has rights under certain Licensed Patents (as defined herein)
pertaining to certain recombinant adeno-associated virus vectors; and
 
WHEREAS, Licensee desires to obtain from Licensor certain licenses under the
Licensed Patents under the terms set forth herein;
 
NOW, THEREFORE, in consideration of the promises and covenants contained in this
Agreement, and intending to be legally bound, the Parties hereby agree as
follows:
 
ARTICLE 1:  DEFINITIONS
 
1.1          “* * *” means (a) the recombinant adeno-associated virus serotype *
* * vector with the specified sequence set forth in GenBank (* * *) and (b) any
recombinant adeno-associated virus derivatives of such serotype * * * vector
that are covered by the claims of the Licensed Patents.
 
1.2          “AAV9” means (a) the recombinant adeno-associated virus serotype 9
vector with the specified sequence set forth in GenBank (protein id * * *) and
(b) any recombinant adeno-associated virus derivatives of such serotype 9 vector
that are covered by the claims of the Licensed Patents.
 
1.3          “* * *” means (a) the recombinant adeno-associated virus serotype *
* * vector with the specified sequence set forth in GenBank (* * *) and (b) any
recombinant adeno-associated virus derivatives of such serotype * * * vector
that are covered by the claims of the Licensed Patents.
 
1.4          “Affiliate” means any legal entity directly or indirectly, during
the term of this Agreement, controlling, controlled by, or under common control
with another entity.  For purposes of this Agreement, “control” means the direct
or indirect ownership of more than 50% of the outstanding voting securities of a
legal entity, or the right to receive more than 50% of the profits or earnings
of a legal entity, or the right to control the policy decisions of a legal
entity.  An entity may be or become an Affiliate of an entity and may cease to
be an Affiliate of an entity, in each case, during the term of this Agreement.
 
1.5          “BLA” means a Biological License Application (BLA) pursuant to 21
C.F.R. § 601.2 or a New Drug Application filed with the FDA as described in 21
C.F.R. § 314.
 
1.6          “Calendar Quarter” means each three-month period or any portion
thereof, beginning on January 1, April 1, July 1, and October 1.
 

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1.7          “Change of Control” means (i) any transaction or series of related
transactions following which the holders of Licensee’s capital stock or
membership or equity interests immediately prior to such transaction or series
of related transactions collectively are the owners of less than 50% of the
outstanding equity interests of Licensee entitled to (a) vote with respect to
the election of directors (or positions having a similar function) or (b)
receive the proceeds upon any sale, liquidation or dissolution of Licensee; 
(ii) a sale, transfer, or other disposition, in a single transaction or series
of related transactions, of all or a material portion of Licensee’s interest in
the Licensed Products; (iii) a sale, transfer, or other disposition, in a single
transaction or series of related transactions, of all or a material portion of
Licensee’s right title, or interest in its assets taken as a whole; or (iv) the
merger of Licensee with a Third Party by operation of law or otherwise.
 
1.8          “Commercial Option” has the meaning set forth in Section 2.2.
 
1.9          “Confidential Information” means and includes all technical
information, inventions, developments, discoveries, software, know-how, methods,
techniques, formulae, animate and inanimate materials, data, processes,
finances, business operations or affairs, and other proprietary ideas, whether
or not patentable or copyrightable, of either Party that are (a) marked or
otherwise identified as confidential or proprietary at the time of disclosure in
writing; or (b) if disclosed orally, visually, or in another non-written form,
identified as confidential at the time of disclosure and summarized in
reasonable detail in writing as to its general content within 30 days after
original disclosure.  The Parties acknowledge that (i) the terms and conditions
of this Agreement and (ii) the records and reports referred to in Section 3.7
will be deemed the Confidential Information of both Parties, regardless of
whether such information is marked or identified as confidential.  In addition,
information provided to Licensee pursuant to the provisions of Section 7.1 will
be deemed the Confidential Information of Licensor, regardless of whether such
information is marked or identified as confidential.  Notwithstanding the
foregoing, Confidential Information will not include the following, in each
case, to the extent evidenced by competent written proof of the Receiving Party:
 
1.9.1          information that was already known to the Receiving Party, other
than under an obligation of confidentiality, at the time of disclosure by the
Disclosing Party;
 
1.9.2          information that was generally available to the public or
otherwise part of the public domain at the time of its disclosure to the
Receiving Party;
 
1.9.3          information that became generally available to the public or
otherwise part of the public domain after its disclosure, other than through any
act or omission of the Receiving Party in breach of this Agreement;
 
1.9.4          information that is independently discovered or developed by the
Receiving Party without the use of Confidential Information of the Disclosing
Party; or
 
1.9.5          information that was disclosed to the Receiving Party, other than
under an obligation of confidentiality, by a Third Party who had no obligation
to the Disclosing Party not to disclose such information to others.
 
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1.10        “Disclosing Party” has the meaning set forth in Section 5.1.
 
1.11        “Domain Antibody” means * * *.
 
1.12        “EMA” means the European Medicines Agency and any successor
agency(ies) in the European Union with responsibilities comparable to those of
the European Medicines Agency.
 
1.13        “FDA” means the United States Food and Drug Administration, or a
successor agency in the United States with responsibilities comparable to those
of the United States Food and Drug Administration.
 
1.14        "Field" means the treatment of Danon Disease in humans by in vivo
gene therapy using the Licensed Vector(s) to deliver any known LAMP2 transgene
isoforms * * * and all possible combinations of LAMP2 transgene isoforms.
 
1.15        “GSK Agreement” means that certain License Agreement entered into
between Licensor and SmithKline Beecham Corporation, effective on March 6, 2009,
as amended by that certain Amendment to License Agreement dated April 15, 2009,
and as further amended from time to time.
 
1.16        “Know-How” means any and all ideas, information, know-how, data,
research results, writings, inventions, discoveries, and other technology
(including any proprietary materials), whether or not patentable or
copyrightable.
 
1.17        “Licensed Back Improvements” means any patentable modifications or
improvements developed by Licensee, any Affiliates, or any Sublicensees to any
vector that is the subject of a claim within the Licensed Patents.
 
1.18        “Licensed Know-How” means any Know-How Licensor provides to Licensee
pursuant to Section 2.8.
 
1.19        “Licensed Patents” means, on a Licensed Vector-by-Licensed Vector
basis, to the extent they cover such Licensed Vector, (a) all United States
patents and patent applications listed in Exhibit A-1 (for AAV9), Exhibit A-2
(if the Licensed Vector is * * *), or Exhibit A-3 (if the Licensed Vector is * *
*), including patents arising from such patent applications; and (b) any
re-examination certificates thereof, and their foreign counterparts and
extensions, continuations, divisionals, and re-issue applications; provided that
“Licensed Patents” will not include any claim of a patent or patent application
covering any Manufacturing Technology.
 
1.20          “Licensed Product” means (a) any product using the applicable
Licensed Vector that is made, made for, used, sold, offered for sale, or
imported by Licensee, its Affiliates, and any of its or their Sublicensees,
where (i) the manufacture, use, sale, offer for sale, or import of which
product, in the absence of the license granted pursuant to this Agreement, would
infringe or is covered by at least one Valid Claim of the Licensed Patents in
the country of manufacture, use, sale, offer for sale, or import, including
products manufactured by a process that would infringe or is covered by at least
one Valid Claim of the Licensed Patents in the country of manufacture, use,
sale, offer for sale, or import or (ii) that incorporates, was developed using,
or is produced or manufactured through the use of, or with respect to which
Licensee otherwise acquired a license to, Licensed Know-How; or (b) any service
sold by Licensee, its Affiliates, and any of its or their Sublicensees with
respect to the administration of any product using a Licensed Vector to patients
that (i) in the absence of the license granted pursuant to this Agreement, would
infringe or is covered by at least one Valid Claim of the Licensed Patents in
the country of sale or (ii) that incorporates, was developed using, or is
produced or manufactured through the use of, or with respect to which Licensee
otherwise acquired a license to, Licensed Know-How.
 
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1.21        “Licensed Program” means the program relating to * * * and/or * * *
for which Licensee has exercised a Commercial Option in accordance with Section
2.2.1.
 
1.22        “Licensed Technology” means, collectively, the Licensed Patents and
Licensed Know-How.
 
1.23        “Licensed Vectors” means AAV9 and, if and when a Commercial Option
is exercised, the vector(s) that is/are selected by Licensee pursuant to Section
2.2 (each vector, a “Licensed Vector”).
 
1.24        “Licensee Inventions” means: (i) any new or improved * * *, AAV9,
and * * * capsid-related Know-How that is related specifically to the capsid of
* * *, AAV9, and/or * * * and that is first discovered, produced, conceived, or
reduced to practice by or on behalf of Licensee, its Affiliates, or any of its
or their Sublicensees and used specifically in the development of Licensed
Products in the exercise of any rights granted under this Agreement.
 
1.25        “Manufacturing Technology” means any and all patents, patent
applications, Know-How, and all intellectual property rights associated
therewith that are owned or controlled by Licensor, and including all tangible
embodiments thereof, that claim, cover or relate to the manufacture of:
adeno-associated viruses, adeno-associated virus vectors, research or commercial
reagents related thereto, Licensed Products, or other products, including
manufacturing processes, technical information relating to the methods of
manufacture, protocols, standard operating procedures, batch records, assays,
manufacture formulations, quality control data, specifications, scale up
methods, any and all manufacture improvements, modifications, and changes
thereto, and any and all activities associated with such manufacture.  Any and
all chemistry, manufacturing, and controls (CMC), drug master files (DMFs), or
similar materials provided by Licensor to regulatory authorities and the
information contained therein are deemed Manufacturing Technology.
 
1.26        “Marketing Authorization” means all approvals, licenses,
registrations or authorizations of any federal, state or local regulatory
agency, department, bureau or other governmental entity, necessary for the
manufacturing, use, storage, import, transport, marketing and sale of Licensed
Products in a country or regulatory jurisdiction.
 
1.27        “Net Sales” means the gross receipts from sales or other disposition
of a Licensed Product (including fees for services within the definition of
“Licensed Product”) by Licensee and/or its Affiliates and/or any Sublicensees to
Third Parties less the following deductions that are directly attributable to a
sale, specifically and separately identified on an invoice or other
documentation and actually borne by Licensee, its Affiliates, or any
Sublicensees:  * * *.  In the event consideration other than cash is paid to
Licensee, its Affiliates, or any Sublicensees, for purposes of determining Net
Sales, the Parties shall use the cash consideration that Licensee, its
Affiliates, or any Sublicensees would realize from an unrelated buyer in an
arm’s length sale of an identical item sold in the same quantity and at the time
and place of the transaction, as determined jointly by Licensor and Licensee
based on transactions of a similar type and standard industry practice, if any.
 
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1.28        “Option Term” means the period beginning on the Effective Date and
ending on the fourth anniversary of the Effective Date.
 
1.29        “Penn Agreement” means that certain License Agreement entered into
between Licensor and The Trustees of the University of Pennsylvania, effective
on February 24, 2009, as amended by that letter agreement dated March 6, 2009,
by that certain Second Amendment to License Agreement effective on September 9,
2014, and by that certain Third Amendment to License Agreement effective on
April 29, 2016, and as further amended from time to time.
 
1.30        “Phase 3 Clinical Trial” means a pivotal clinical trial in humans
performed to gain evidence with statistical significance of the efficacy of a
product in a target population, and to obtain expanded evidence of safety for
such product that is needed to evaluate the overall benefit-risk relationship of
such product, to form the basis for approval of an BLA and to provide an
adequate basis for physician labeling, as described in 21 C.F.R. § 312.21(c) or
the corresponding regulation in jurisdictions other than the United States.
 
1.31        “Prosecute” means preparation, filing, and prosecuting patent
applications and maintaining patents, including any reexaminations, reissues,
oppositions, inter partes review, and interferences.
 
1.32        “Receiving Party” has the meaning set forth in Section 5.1.
 
1.33        “REGENXBIO Licensors” means SmithKline Beecham Corporation (or any
successor thereto under the GSK Agreement) and The Trustees of the University of
Pennsylvania (or any successor thereto under the Penn Agreement), if any
Licensed Technology is sublicensed from the Penn Agreement.
 
1.34        “Retained Rights” has the meaning set forth in Section 2.3.
 
1.35        “Sublicensee” means (i) any Third Party or Affiliate to whom
Licensee grants a sublicense of some or all of the rights granted to Licensee
under this Agreement as permitted by this Agreement; and (ii) any other Third
Party or Affiliate to whom a sublicensee described in clause (i) has granted a
further sublicense as permitted by this Agreement.
 
1.36        “Third Party” means any person or entity other than a Party to this
Agreement or Affiliates of a Party to this Agreement.
 
1.37        “Valid Claim” means a claim of an issued and unexpired patent
(including any patent claim the term of which is extended by any extension,
supplementary protection certificate, patent term restoration, or the like)
included within the Licensed Patents or a claim of a pending patent application
included within the Licensed Patents, that has not lapsed, been abandoned, been
held revoked, or been deemed unenforceable or invalid by a non-appealable
decision or an appealable decision from which no appeal was taken within the
time allowed for such appeal of a court or other governmental agency of
competent jurisdiction and has not been pending for more than fifteen (15)
years.
 
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ARTICLE 2:  LICENSE GRANTS
 
2.1          License Grant.  Subject to the terms and conditions of this
Agreement, including the Retained Rights, Licensor hereby grants to Licensee an
exclusive, sublicensable (as provided in Section 2.5 only), non-transferable
(except as provided in Section 10.2), royalty-bearing, worldwide license under
the Licensed Technology to make, have made, use, import, sell, and offer for
sale Licensed Products using AAV9 solely in the Field, including, for the
avoidance of doubt, the right to conduct research and development.
 
2.2          Commercial License Option.  Subject to the terms and conditions of
this Agreement, Licensor hereby grants to Licensee during the Option Term two
distinct exclusive options, one of which is using * * * in the Field and one of
which is using * * * in the Field, exercisable at Licensee’s sole discretion, to
obtain an exclusive, sublicensable (as provided in Section 2.5 only),
non-transferable (except as provided in Section 10.2), royalty-bearing,
worldwide license under the Licensed Technology to make, have made, use, import,
sell, and offer for sale Licensed Products (each such right, a “Commercial
Option”) in accordance with the following provisions:
 
2.2.1          Method of Exercise.  To exercise a Commercial Option, Licensee
must, prior to the end of the Option Term, (i) provide written notice to
Licensor, which written notice must specify which Commercial Option is being
exercised and (ii) pay the fee specified in Section 3.2.
 
2.2.2          License Grant Upon Exercise.  If Licensee exercises a Commercial
Option, effective upon Licensor’s receipt of the notice and, the fee described
in Section 2.2.1 (the “Grant Date” for the applicable Licensed Program), subject
to the terms and conditions of this Agreement, including the Retained Rights,
Licensor shall be deemed to have granted to Licensee an exclusive, sublicensable
(as provided in Section 2.5 only), non-transferable (except as provided in
Section 10.2), royalty-bearing, worldwide license under the Licensed Technology
to make, have made, use, import, sell, and offer for sale Licensed Products in
the Field that are the subject of the Licensed Program, including, for the
avoidance of doubt, the right to conduct research and development.
 
2.2.3          Exercised Commercial Option.  For the avoidance of doubt, the
foregoing license granted pursuant to Section 2.2.2 will be deemed granted on
the Grant Date on a Licensed Program-by-Licensed Program basis, solely with
respect to the Licensed Program for which a particular Commercial Option was
exercised under this Section 2.2.  The Parties acknowledge that there may be
different Grant Dates for each Licensed Program, depending on when and if
Licensee exercises a Commercial Option for the applicable Licensed Program. 
Each Commercial Option will terminate at the end of the Option Term if Licensee
fails to exercise such Program Option prior to the end of the Option Term, and
Licensee will have no further rights under this Agreement to exercise any
unexercised Program Option.
 
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2.3          Retained Rights.  Except for the rights and licenses specified in
Section 2.1 and, if applicable, Section 2.2.2, no license or other rights are
granted to Licensee under any intellectual property of Licensor, whether by
implication, estoppel, or otherwise and whether such intellectual property is
subordinate, dominant, or otherwise useful for the practice of the Licensed
Technology.  Notwithstanding anything to the contrary in this Agreement,
Licensor may use and permit others to use the Licensed Technology for any
research, development, commercial, or other purposes outside of the Field. 
Without limiting the foregoing, and notwithstanding anything in this Agreement
to the contrary, Licensee acknowledges and agrees that the following rights are
retained by Licensor and the REGENXBIO Licensors (individually and collectively,
the “Retained Rights”), whether inside or outside the Field:
 
2.3.1          The rights and licenses granted in Section 2.1 and, if
applicable, Section 2.2.2, shall not include any right (and Licensor and the
REGENXBIO Licensors retain the exclusive (even as to Licensee), fully
sublicensable right) under the Licensed Technology to make, have made, use,
sell, offer to sell, and import Domain Antibodies that are expressed by an
adeno-associated vector, including any Licensed Vector.
 
2.3.2          Licensor and the REGENXBIO Licensors retain the following rights
with respect to the Licensed Technology:
 

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

 

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

 
2.3.3          The rights and licenses granted in Section 2.1 and, if
applicable, Section 2.2.2, shall not include any right (and Licensor retains the
exclusive (even as to Licensee), fully sublicensable right) under the Licensed
Technology:
 

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

 

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

 
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2.3.4          Licensor retains the fully sublicensable right under the Licensed
Technology to grant non-exclusive research and development licenses to
Affiliates and Third Parties; provided that such research and development rights
retained by Licensor shall not include the right to conduct (or permit others to
conduct) clinical trials in humans in the Field or any rights to use the
Licensed Technology to sell products in the Field.
 
2.3.5          The Trustees of the University of Pennsylvania may use and permit
other non-profit organizations or other non-commercial entities to use the
Licensed Technology for educational and non-commercial research purposes.
 
2.4          Government Rights.  Licensee acknowledges that the United States
government retains certain rights in intellectual property funded in whole or
part under any contract, grant, or similar agreement with a federal agency.  The
license grants hereunder are expressly subject to all applicable United States
government rights, including any applicable requirement that products resulting
from such intellectual property sold in the United States must be substantially
manufactured in the United States.
 
2.5          Sublicensing.
 
2.5.1          The license granted pursuant to Section 2.1 and, if applicable,
Section 2.2.2, is sublicensable by Licensee to any Affiliates or Third Parties
(through multiple tiers); provided that any such sublicense must comply with the
provisions of this Section 2.5 (including Section 2.5.2).
 
2.5.2          The right to sublicense granted to Licensee under this Agreement
is subject to the following conditions:
 

(a)
Licensee may only grant sublicenses pursuant to a written sublicense agreement
with the Sublicensee.  Licensor must receive written notice as soon as
practicable following execution of any such sublicenses.  Any further
sublicenses granted by any Sublicensees (to the extent permitted hereunder) must
comply with the provisions of this Section 2.5 (including Section 2.5.2) to the
same extent as if Licensee granted such sublicense directly.

 

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

 

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

 

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

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

 
2.6          Non-Exclusive License Under Licensee Inventions.  Licensee hereby
grants to Licensor a non-exclusive, worldwide, royalty-free, transferable,
sublicensable, irrevocable, perpetual license under Licensee Inventions (and any
intellectual property rights with respect thereto) to practice the Licensee
Inventions solely in connection with AAV9, (or * * * or * * * if Licensee
exercises the Commercial Option) and solely outside of the Field, including, for
avoidance of doubt, the right to conduct research and develop and commercialize
products and services outside of the Field.
 
2.7          Improvements.
 
2.7.1          Licensee hereby grants to Licensor a non-exclusive, worldwide,
royalty-free, transferable, sublicensable, irrevocable, perpetual license:
 

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

 

(b)
to practice the Licensed Back Improvements (and any intellectual property rights
with respect thereto) in connection with * * *, AAV9 and * * *, including the
right to research, develop, make, have made, use, offer for sale, and sell
products and services; provided that Licensor shall have no right, under the
license in this Section 2.7.1(b), to practice the Licensed Back Improvements in
the Field.

 
2.7.2          Licensee agrees to provide prompt notice to Licensor upon the
filing of any patent application covering any Licensee Invention and/or any
Licensed Back Improvement, together with a reasonably detailed description of,
or access to, such Licensee Inventions and/or Licensed Back Improvement to
permit the practice of any such invention or improvement.
 
2.8          Transfer of Licensed Know-How.    Following the Effective Date and
from time to time during the Term, at Licensee’s sole expense and request,
Licensor may agree at its sole discretion to disclose to Licensee within * * *
following such written request from Licensee manufacturing and development
Know-How, including through in-person or telephonic meetings at such times and
places as agreed to by the Parties, necessary or reasonably useful to make or
practice the Licensed Vectors that Licensor agrees to provide to Licensee. 
Licensee acknowledges and agrees that all Licensed Know-How disclosed pursuant
to this Section 2.8 will be deemed “Confidential Information” of Licensor,
regardless of whether such information is marked or identified as confidential
and without an obligation to summarize oral information.
 
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2.9          Section 365(n) of the Bankruptcy Code.  All rights and licenses
granted to Licensee or Licensor under or pursuant to this Agreement are and will
otherwise be deemed to be, for purposes of Section 365(n) of the United States
Bankruptcy Code (Title 11, U.S. Code), as amended (the “Bankruptcy Code”) or any
comparable law outside the United States, licenses of rights to “intellectual
property” as defined in Section 101(35A) of the Bankruptcy Code.  The Parties
will retain and may fully exercise all of their respective rights and elections
under the Bankruptcy Code and any comparable law outside the United States.
 
ARTICLE 3:  CONSIDERATION
 
3.1          Initial Fee.  In partial consideration of the rights and licenses
granted to Licensee under this Agreement, Licensee shall pay Licensor an initial
fee of $7,000,000 within * * * of the Effective Date.
 
3.2          Commercial Option Fee.  Licensee shall pay Licensor a fee of
$2,000,000 within * * * of the exercise of each Commercial Option.
 
3.3          Annual Maintenance Fee.  In partial consideration of the rights and
licenses granted to Licensee under this Agreement, Licensee shall pay Licensor
on-going annual maintenance fees of * * * per Licensed Vector on each
anniversary of the Effective Date.  For clarity, the maintenance fee for * * *
will not be due unless the respective Commercial Options have been exercised.
 
3.4          Milestone Fees.  In partial consideration of the rights and
licenses granted to Licensee under this Agreement, Licensee shall pay Licensor
the following milestone payments within * * * of each Licensed Product
containing a distinct Licensed Vector (specifically, * * *, AAV9 * * *), to
achieve such milestone event:
 
 Milestone
Milestone Payment
1.  First treatment of human subject in the first clinical trial (i.e., first
patient, first dose)
* * *
2.  First treatment of first human subject in Phase 3 Clinical Trial (i.e.,
first patient, first dose)
* * *
3.  BLA application submitted to the FDA in the United States
* * *
4.  Marketing Authorization application submitted to the EMA in the European
Union
* * *
5.  BLA approval received from the FDA in the United States
* * *
6.  Marketing Authorization approval received from the EMA in the European Union
* * *
Total (per Licensed Product):
$13,000,000

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3.4.1          For clarity, the milestone payments set forth in Section 3.4 are
payable regardless of whether the milestone is achieved by Licensee, any
Affiliate, or any Sublicensee, and regardless of whether the milestone is
achieved by more than one Licensed Product with distinct Licensed Vectors.  To
the extent that e ither of the first two development milestones in Section 3.4
(i.e., first treatment of a human subject in a clinical trial or first treatment
in Phase 3 Clinical Trial) has not been paid at the time of achievement of
either the BLA or Marketing Authorization submission milestone, then, upon the
achievement of either of such submission milestones, the preceding unpaid
development milestone payments shall be made in addition to the payment
corresponding to the applicable submission milestone that has been achieved.
 
3.5          Royalties.  In further consideration of the rights and licenses
granted to Licensee under this Agreement, Licensee shall pay to Licensor the
following royalties based upon the annual Net Sales of Licensed Products,
subject to the reductions in royalty rates set forth in Section 3.5.1:
 
Cumulative Annual Net Sales of all Licensed
Products Worldwide
Royalty Percentage
Portion of Annual Net Sales less than $* * *
* * *
Portion of Annual Net Sales greater than (and including) $* * *
* * *

For clarity, the Annual Net Sales resets to zero at the beginning of each
calendar year.

3.5.1          Third Party Royalties Stacking Provision.  If Licensee must
obtain a license from one or more Third Parties to avoid infringement of such
Third Party(ies)’s rights in order to manufacture, use, or commercialize a given
Licensed Product and if the royalties required to be paid to such Third
Party(ies) for such license, together with those royalties payable to Licensor,
in the aggregate, exceed * * *  of Net Sales for any Licensed Product, then the
royalty owed to Licensor for that Licensed Product will be reduced by an amount
calculated as follows:
 
STACKING ROYALTY CALCULATIONS
 
R = (C * (A / (A+B)))
 
Where
 
R = reduction of Licensor royalty,
A = unreduced Licensor royalty,
B = sum of all Third Party royalties,
C = increment of projected total royalty above * * * .
 
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Example Calculation:
 
Assume:
i)           all Third Party royalties = * * *
 
ii)          unreduced Licensor royalty = * * *
 
iii)         projected total royalty = * * *
    R = * * *  R = * * *  R = * * *  Licensor Stacked Royalty = * * * (but
subject to the cap described below) 

Notwithstanding the foregoing, Licensee will pay to Licensor no less than * * *
of the royalties that Licensee would otherwise pay to Licensor with respect to
Net Sales of Licensee if there were no royalties due to Third Parties.
 
3.5.2          Adjustment of Royalties For Licenses.  On a Licensed
Product-by-Licensed Product, country-by-country basis, upon the date on which
the manufacture, use, sale, offer for sale, or import of a Licensed Product does
not infringe or is not covered by a Valid Claim in such country, then the
royalty percentage applicable to Net Sales of such Licensed Product under this
Section 3.5 in such country shall be reduced by * * *.
 
3.5.3          Royalty Payment Period.  Licensee’s obligation hereunder for
payment of a royalty under this Section 3.5 on the Net Sales of Licensed
Products in a given country will end on a country-by-country, Licensed
Product-by-Licensed Product basis on the later of: (i)  expiration, lapse,
abandonment, or invalidation of the last Valid Claim of the Licensed Patents to
expire, lapse, become abandoned or become unenforceable for the applicable
Licensed Product in the applicable country, or (ii) * * * from the first
commercial sale of the applicable Licensed Product in the applicable country.
 
3.6          Sublicense Fees & Priority Review Voucher Transfer Fee.
 
3.6.1          In further consideration of the rights and licenses granted to
Licensee under this Agreement, Licensee will pay Licensor * * * of any
sublicense fees (* * *) received by Licensee or its Affiliates from a Third
Party for the Licensed Technology from any Sublicensee or from any person or
entity granted any option to obtain a sublicense (“Sublicensing Revenue”).
 
3.6.2          In further consideration of the rights and licenses granted to
Licensee under this Agreement, Licensee will pay Licensor twenty percent (20%)
of the fees related to the sale or other transfer or rights to a Third Party
under any priority review voucher issued in connection with or otherwise related
to a Licensed Product.   Should Licensee or its Affiliates elect to use any
priority review voucher issued in connection with or otherwise related to a
Licensed Product rather than sell or transfer the priority review voucher to a
Third Party, Licensee will pay Licensor 20% of the average value of the last
five publicly disclosed priority review voucher sales at the time of use.
 
3.6.3          With respect to the obligations under this Section 3.6, Licensee
shall not be required to submit any amounts received from a Third Party for the
following:
 
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(a)
Reimbursement or payment of Licensee’s actual costs or on an arm’s length cost
plus arrangement for research, development, and/or manufacturing activities
performed by Licensee or its Affiliates corresponding directly to the research,
development and/or manufacturing of Licensed Products pursuant to a specific
agreement;

 

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

 

(c)
Consideration received for the purchase of an equity interest in Licensee or its
Affiliates at fair market value or in the form of loans at arm’s length rates of
interest.

 
3.6.4          If Licensee or its Affiliates receives sublicense fees from
Sublicensees or from any person or entity granted any option to obtain a
sublicense under this Agreement in the form of non-cash consideration, then
Licensee shall pay Licensor a cash payment as required under Section 3.6
determined based on the fair market value of such non-cash consideration.  If
Licensee or its Affiliate enters into any sublicense that is not an arm’s length
transaction, fees due under this Section 3.6 will be calculated based on the
fair market value of such transaction, at the time of the transaction, assuming
an arm’s length transaction made in the ordinary course of business, as
determined jointly by Licensor and Licensee based on transactions of a similar
type and standard industry practice, if any.
 
3.6.5          To the extent Licensee or its Affiliates receives payment from a
Third Party relating to one or more of the milestone events set forth in the
table in Section 3.4, then the amount of the payment made to Licensor under such
Section 3.4 with respect to such milestone event shall not be deemed sublicense
fees under this Section 3.6; instead, the amounts due under this Section 3.6
shall be calculated by applying the applicable sublicense fee rate set forth in
Section 3.6 above to the sublicense fees received by Licensee or its Affiliates
from such Third Party after deducting the amount of the payment under Section
3.4.
 
3.7          Reports and Records.
 
3.7.1          Licensee must deliver to Licensor within * * * after the end of
each Calendar Quarter beginning after the first commercial sale of a Licensed
Product a report setting forth the calculation of the royalties due to Licensor
for such Calendar Quarter, including:
 

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

 

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

 

(c)
Qualifying costs to be excluded from the gross consideration, as described in
Section 1.25, listed by category of cost and by country;

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

 

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

 

(f)
Royalties owed to Licensor; and

 

(g)
The computations for any applicable currency conversions.

 
3.7.2          Licensee shall pay the royalties due under Section 3.5 within * *
* following the last day of the Calendar Quarter in which the royalties accrue. 
Licensee shall send the royalty payments along with the report described in
Section 3.7.1.
 
3.7.3          Within * * * after the occurrence of a milestone event described
in Section 3.4, Licensee must deliver to Licensor a report describing the
milestone event that occurred, together with a payment of the applicable amount
due to Licensor pursuant to Section 3.4.
 
3.7.4          Within * * * after the receipt of any fees from any Third Party
as described in Section 3.6, Licensee must deliver to Licensor a report
describing the fees received, together with a payment of the applicable amount
due to Licensor pursuant to Section 3.6.
 
3.7.5          All financial reports under this Section 3.7 will be certified by
the chief financial officer of Licensee or Licensee’s qualified financial
representative.
 
3.7.6          Licensee shall maintain and require its Affiliates and all
Sublicensees to maintain, complete, and accurate books and records that enable
the royalties, fees, and payments payable under this Agreement to be verified. 
The records must be maintained for * * * after the submission of each report
under Article 3.  Upon reasonable prior written notice to Licensee, Licensee and
its Affiliates and all Sublicensees will provide Licensor and/or the REGENXBIO
Licensors (and their respective accountants) with access to all of the relevant
books, records, and related background information required to conduct a review
or audit of the royalties, fees, and payments payable to Licensor under this
Agreement to be verified.  Access will be made available: (a) during normal
business hours; (b) in a manner reasonably designed to facilitate the auditing
party’s review or audit without unreasonable disruption to Licensee’s business;
and (c) no more than once each calendar year during the term of this Agreement
and for a period of five years thereafter.  Licensee will promptly pay to
Licensor the amount of any underpayment determined by the review or audit, plus
accrued interest.  If the review or audit determines that Licensee has underpaid
any payment by * * * or more, then Licensee will also promptly pay the costs and
expenses of Licensor and the REGENXBIO Licensors and their respective
accountants in connection with the review or audit.  If the review or audit
determines that Licensee has overpaid any payment, then Licensor shall refund
the overpayment to Licensee.
 
3.8          Currency, Interest.
 
3.8.1          All dollar amounts referred to in this Agreement are expressed in
United States dollars.  All payments to Licensor under this Agreement must be
made in United States dollars.
 
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3.8.2          If Licensee receives payment in a currency other than United
States dollars for which a royalty or fee or other payment is owed under this
Agreement, then (a) the payment will be converted into United States dollars at
the conversion rate for the foreign currency as published in the eastern edition
of the Wall Street Journal, N.Y. edition, as of the last business day of the
Calendar Quarter in which the payment was received by Licensee; and (b) the
conversion computation will be documented by Licensee in the applicable report
delivered to Licensor under Section 3.7.
 
3.8.3          All amounts that are not paid by Licensee when due will accrue
interest from the date due until paid at a rate equal to 1.5% per month (or the
maximum allowed by law, if less).
 
3.9          Taxes and Withholding.
 
3.9.1          All payments hereunder will be made free and clear of, and
without deduction or deferment in respect of, and Licensee shall pay and be
responsible for, and shall hold Licensor harmless from and against, any taxes,
duties, levies, fees, or charges, including sales, use, transfer, excise,
import, and value added taxes (including any interest, penalties, or additional
amounts imposed with respect thereto) but excluding withholding taxes to the
extent provided in Section 3.9.2.  At the request of Licensee, Licensor will
give Licensee such reasonable assistance, which will include the provision of
documentation as may be required by the relevant tax authority, to enable
Licensee to pay and report and, as applicable, claim exemption from or reduction
of, such tax, duty, levy, fee, or charge.
 
3.9.2          If any payment made by Licensee hereunder becomes subject to
withholding taxes with respect to Licensor’s gross or net income under the laws
of any jurisdiction, then Licensee will deduct and withhold the amount of such
taxes for the account of Licensor to the extent required by law and will pay the
amounts of such taxes to the proper governmental authority in a timely manner
and promptly transmit to Licensor appropriate proof of payment of such
withholding taxes.  At the request of Licensor, Licensee will give Licensor such
reasonable assistance, which will include the provision of appropriate
certificates of such deductions made together with other supporting
documentation as may be required by the relevant tax authority, to enable
Licensor to claim exemption from or reduction of, or otherwise obtain repayment
of, such withholding taxes, and will upon request provide such additional
documentation from time to time as is reasonably required to confirm the payment
of withholding tax.
 
ARTICLE 4:  DILIGENCE
 
4.1          Diligence Obligations.  Licensee will use commercially reasonable
efforts to develop, commercialize, market, promote, and sell Licensed Products
in the Field.  Commercially reasonable efforts means efforts equivalent to those
utilized by * * *.  Without limiting the foregoing, Licensee will  meet the
following: acceptance by the FDA or foreign equivalent of an investigational new
drug application, for a Licensed Product by no later than * * * after the
Effective Date; provided, however, that, if Licensee expects not to achieve the
milestone on or before the specified deadline, Licensee may pay Licensor an
extension fee of * * * on or before such deadline and the deadline shall then be
extended by an * * *.  Licensee will provide Licensor written notice within * *
* of achieving the milestone.
 
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4.2          Development Plans.
 
4.2.1          For each Licensed Product in the Field, Licensee will prepare and
deliver to Licensor a development plan and budget (each a “Development Plan”). 
The initial Development Plans for the Licensed Product in the Field will be
delivered within * * * after the Effective Date.
 
4.2.2          Each Development Plan will cover the next 2 years, and will
include future development activities that Licensee plans to undertake (either
directly or through its Affiliates, or any Sublicensees) during those 2 years
that relate directly to the Licensed Product, Licensee’s strategy to bring the
Licensed Product to commercialization, and projected timeline for completing the
necessary tasks to accomplish the goals of the strategy.
 
4.2.3          Following receipt by Licensor of each Development Plan, Licensor
will promptly notify Licensee of any comments or suggested revisions, and
Licensee shall incorporate any appropriate comments and suggested revisions in
good faith.
 
4.3          Reporting.  Within * * * after the Effective Date and within * * *
thereafter, Licensee shall provide Licensor with written progress reports,
setting forth in such detail as Licensor may reasonably request, a summary of
the progress of the development, evaluation, testing and commercialization of
each Licensed Product.  Licensee will also notify Licensor within * * * of the
first commercial sale by Licensee, its Affiliates, or any Sublicensees of each
Licensed Product.  Such a report (“Development Progress Report”), setting forth
the current stage of development of Licensed Products, shall include:
 
4.3.1          Date of Development Progress Report and time covered by such
report;
 
4.3.2          Major activities and accomplishments completed by Licensee, its
Affiliates, and any Sublicensees relating directly to the Licensed Products
since the last Development Progress Report;
 
4.3.3          Significant research and development projects relating directly
to the Licensed Products currently being performed by Licensee, its Affiliates,
and any Sublicensees and good faith, but non-binding, projected dates of
completion;
 
4.3.4          A summary update to the Development Plan, which will include a
summary of anticipated future development activities to be undertaken by
Licensee, its Affiliates, or any Sublicensees during the next reporting period
relating directly to the Licensed Products, Licensee’s strategy to bring the
Licensed Products to commercialization, and projected timeline for completing
the necessary tasks to accomplish the goals of the strategy;
 
4.3.5          Projected total development remaining before product launch of
each Licensed Product; and
 
4.3.6          Summary of significant development efforts using the Licensed
Technology being performed by Third Parties, including the nature of the
relationship between Licensee and such Third Parties.
 
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4.4          Confidential Information.  The Parties agree that Development
Progress Reports shall be deemed Licensee’s Confidential Information; provided
that Licensor may share a copy of such reports with the REGENXBIO Licensors.
 
4.5          Improvements.  Simultaneously with the Development Progress Report,
Licensee shall deliver a detailed description of any Licensee Inventions and any
Licensed Back Improvements, if not previously provided pursuant to Section
2.7.3.
 
ARTICLE 5:  CONFIDENTIALITY
 
5.1          Treatment of Confidential Information.  Each Party, as a receiving
party (a “Receiving Party”), agrees that it will (a) treat Confidential
Information of the other Party (the “Disclosing Party”) as strictly
confidential; (b) protect the Confidential Information of the Disclosing Party
with at least the same degree of care as it protects its own confidential and
proprietary information, and in any event with not less than a reasonable degree
of care; (c) not disclose such Confidential Information to Third Parties without
the prior written consent of the Disclosing Party, except as may be permitted in
this Agreement; provided that any disclosure permitted hereunder shall be under
confidentiality agreements with provisions at least as stringent as those
contained in this Agreement; and (d) not use such Confidential Information for
purposes other than those authorized expressly in this Agreement.  The Receiving
Party agrees to ensure that its employees who have access to Confidential
Information are obligated in writing to abide by confidentiality obligations at
least as stringent as those contained under this Agreement.
 
5.2          Public Announcements.
 
5.2.1          The Parties may agree to issue a joint press release.  Except as
provided in Section 5.2.2, any press releases by either Party with respect to
the other Party or any other public disclosures concerning the existence of or
terms of this Agreement shall be subject to review and approval by the other
Party.  Once a joint press release or any other written statement is approved
for disclosure by both Parties, either Party may make subsequent public
disclosure of the contents of such statement without the further approval of the
other Party.
 
5.2.2          Notwithstanding Section 5.2.1, Licensor has the right to publish
(through press releases, scientific journals, or otherwise) and refer to any
clinical, regulatory, or research results related to Licensee’s Licensed Product
or Licensed Vector program that have been publicly disclosed by Licensee,
including referring to Licensee by name as a licensee of Licensor, which
publication or referral by Licensor shall not require the prior consent of
Licensee.
 
5.3          Authorized Disclosure.  Notwithstanding the provisions of Section
5.1 or 5.2, either Party may disclose the other’s Confidential Information or
make such a disclosure of the existence of and/or terms of this Agreement to any
* * *; provided that, in each case, such recipient of Confidential Information
is obligated to keep such information confidential on terms no less stringent
than those set forth in this Agreement.  Furthermore, Licensee agrees that
Licensor may share a copy of this Agreement, reports and notices provided by
Licensee to Licensor pursuant to the terms of this Agreement, and copies of
sublicense agreements provided to Licensor hereunder, with the REGENXBIO
Licensors to the extent required by the GSK Agreement and the Penn Agreement,
under confidentiality.  In the event that the Receiving Party receives service
of legal process that purports to compel disclosure of the Disclosing Party’s
Confidential Information or becomes obligated by law, rule, regulation or rules
of a security exchange, to disclose the Confidential Information of the
Disclosing Party or the existence of or terms of this Agreement to any
governmental authority, then, to the extent legally permitted, the Receiving
Party shall promptly notify the Disclosing Party, so that the Disclosing Party
may seek an appropriate protective order or other remedy with respect to
narrowing the scope of such requirement and/or waive compliance by the Receiving
Party with the provisions of this Agreement.  The Receiving Party will, at the
Disclosing Party’s request and expense, provide the Disclosing Party with
reasonable assistance in obtaining such protective order or other remedy.  If,
in the absence of such protective order or other remedy, the Receiving Party is
nonetheless required by law, rule, regulation or rules of a security exchange,
to disclose the existence of or terms of this Agreement or other Confidential
Information of the Disclosing Party, then the Receiving Party may disclose such
Confidential Information without liability hereunder; provided that the
Receiving Party shall furnish only such portion of the Confidential Information
that is legally required to be disclosed and only to the extent required by law.
 
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5.4          Term of Confidentiality.  The obligations of this Article 5 shall
continue for a period of * * * following the expiration or termination of this
Agreement.
 
ARTICLE 6: TERM AND TERMINATION
 
6.1          Term of Agreement.  This Agreement will commence on the Effective
Date and continue in effect on a country-by-country, Licensed
Product-by-Licensed Product basis until the later of: (i) the expiration, lapse,
abandonment, or invalidation of the last Valid Claim of the Licensed Patents to
expire, lapse, become abandoned, become unenforceable for the applicable
Licensed Product, or (ii) 10 years from the first commercial sale of a Licensed
Product, unless sooner terminated as provided in this Agreement.  Upon
expiration of the Agreement, the license grant to Licensee pursuant to Section
2.1 shall become irrevocable, perpetual, royalty-free and fully paid-up.
 
6.2          Termination for Failure to Exercise Commercial Option(s).  Each
Commercial Option will terminate automatically if such Commercial Option has not
been exercised at the end of the Option Term; provided that such termination
will not affect Licensee’s rights under this Agreement with respect to the
license granted under Section 2.1.
 
6.3          Licensee’s Right to Terminate.  Licensee may, upon * * * prior
written notice to Licensor, terminate this Agreement for any reason, with or
without cause.  In exercising such termination right, Licensee may terminate the
Agreement in its entirety or, if desired, Licensee may specify in the written
notice that this Agreement is terminating only with respect to one or more
Licensed Vectors in the Field.
 
6.4          Termination for Breach.
 
6.4.1          Licensor may terminate this Agreement, if Licensee is late in
paying to Licensor royalties, fees, or any other monies due under this
Agreement, and if Licensee does not pay Licensor in full within * * * upon
written demand from Licensor, which termination shall be effective immediately
upon the expiration of such * * * cure period.
 
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6.4.2          Either Party may terminate this Agreement, if the other Party
materially breaches this Agreement and does not cure such material breach within
* * * after written notice of the breach, which termination shall be effective
immediately upon the expiration of such * * * cure period. Notwithstanding the
above, if Licensee disputes in good faith that such material breach exists, and
gives Licensor written notice of such dispute within * * * following Licensee’s
receipt of Licensor’s notice of default, then, Licensor may not terminate this
agreement until the dispute is resolved in accordance with Section 10.6;
provided that Licensor shall be entitled to terminate this Agreement at the end
of the original * * * cure period, without waiting for resolution of the dispute
in accordance with Section 10.6, if the breach by Licensee of this Agreement
would cause Licensor to be in breach of the GSK Agreement or the Penn Agreement.
 
6.5          Termination for Insolvency. Licensor shall have the right to
terminate this Agreement, upon notice to the Licensee, in the event that:
 
(a)     Licensee shall have: (i) voluntarily commenced any proceeding or filed
any petition seeking relief under the bankruptcy, insolvency or other similar
laws of any jurisdiction, (ii) applied for, or consented to, the appointment of
a receiver, trustee, custodian, sequestrator, conciliator, administrator or
similar official for it or for all or substantially all of its property, (iii)
filed an answer admitting the material allegations of a petition filed against
or in respect of it in any such proceeding, (iv) made a general assignment for
the benefit of creditors of all or substantially all of its assets, (v) admitted
in writing its inability to pay all or substantially all of its debts as they
become due, or (vi) taken corporate action for the purpose of effecting any of
the foregoing; or
 
(b)     An involuntary proceeding shall have been commenced, or any involuntary
petition shall have been filed, in a court of competent jurisdiction seeking:
(i) relief in respect of Licensee, or of its property, under the bankruptcy,
insolvency or similar laws of any jurisdiction, (ii) the appointment of a
receiver, trustee, custodian, sequestrator, conciliator, administrator or
similar official for the Licensee or for all or substantially all of its
property, or (iii) the winding-up or liquidation of the Licensee; and, in each
case, such proceeding or petition shall have continued undismissed for 60 days,
or an order or decree approving or ordering any of the foregoing shall have
continued unstayed, unappealed and in effect for 30 days.
 
6.6          Patent Challenge.
 
6.6.1          Licensor may terminate this Agreement, effective immediately upon
written notice to Licensee, upon the commencement by Licensee or any of its
Affiliates of a Patent Challenge.
 
6.6.2          Licensee shall include in each sublicense agreement entered into
with a Sublicensee a right of Licensee to terminate such sublicense agreement if
such Sublicensee commences a Patent Challenge; and Licensee shall terminate the
sublicense agreement, effective immediately upon written notice to the
Sublicensee, if the Sublicensee commences a Patent Challenge.  If a Sublicensee
commences a Patent Challenge and Licensee fails to terminate the applicable
sublicense agreement, then Licensor may terminate this Agreement, effective
immediately upon written notice to the Licensee.
 
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6.6.3          For purposes of this Section 6.6, “Patent Challenge” means any
action against Licensor or the REGENXBIO Licensors, including an action for
declaratory judgment, to declare or render invalid or unenforceable the Licensed
Patents, or any claim thereof.
 
6.7          Effects of Termination.  The effects of termination by Licensee
pursuant to Section 6.3, by either Party, as applicable, pursuant to Section
6.4, or by Licensor pursuant to Section 6.5 or 6.6 shall be as follows:
 
6.7.1          The applicable licenses granted by Licensor hereunder shall
terminate, and Licensee, its Affiliates, and (unless the sublicense agreement is
assigned pursuant to Section 6.7.2) all Sublicensees shall cease to make, have
made, use, import, sell, and offer for sale all Licensed Products and shall
cease to otherwise practice the Licensed Technology; provided that Licensee, its
Affiliates, and its Sublicensees shall have the right to continue to sell their
existing inventories of Licensed Products under the terminated licenses for a
period not to exceed * * * after the effective date of such termination;
 
6.7.2          If termination is by Licensor pursuant to Sections 6.4, 6.5 or
6.6, then, at Licensor’s request, Licensee shall assign to Licensor any or all
sublicenses granted to Third Parties to the extent of the rights licensed to
Licensee hereunder and sublicensed to the Sublicensee; provided that (i) prior
to such assignment, Licensee shall advise Licensor whether such Sublicensee is
then in full compliance with all terms and conditions of its sublicense and
continues to perform thereunder, and, if such Sublicensee is not in full
compliance or is not continuing to perform, Licensor may elect not to have such
sublicense assigned; and (ii) following such assignment, Licensor shall not be
liable to such Sublicensee with respect to any obligations of Licensee to the
Sublicensee that are not consistent with, or not required by, Licensor’s
obligations to Licensee under this Agreement; and all sublicenses not requested
to be assigned to Licensor shall terminate.  If termination is for any other
reason, then all sublicenses shall terminate;
 
6.7.3          If termination is by Licensee pursuant to Section 6.3 or by
Licensor pursuant to Section 6.4, 6.5, or 6.6, then Licensee shall grant, and
hereby grants, to Licensor a non-exclusive, perpetual, irrevocable, worldwide,
royalty-free, transferable, sublicensable license under any Licensed Back
Improvements, for use by Licensor for the research, development, and
commercialization of products in any therapeutic indication;
 
6.7.4          Licensee shall pay all monies then-owed to Licensor under this
Agreement;
 
6.7.5          Each Receiving Party shall, at the Disclosing Party’s request,
return all Confidential Information of the Disclosing Party.  Notwithstanding
the foregoing, one copy may be kept by either Party for a record of that Party’s
obligations; and
 
6.7.6          If termination is only with respect to one or more Licensed
Vectors within the Field, but not all Licensed Vectors, then the provisions of
this Section 6.7 shall only apply with respect to the terminated Licensed
Vectors, and this Agreement shall continue with respect to the non-terminated
Licensed Vectors.
 
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6.8          Survival.  Licensee’s obligation to pay all monies due and owed to
Licensor under this Agreement that have matured as of the effective date of
termination or expiration shall survive the termination or expiration of this
Agreement.  In addition, the provisions of Section 2.3, (Retained Rights),
Section 2.4 (Government Rights), Section 2.6 (Non-Exclusive License Under
Licensee Inventions), Section 2.7 (Improvements), Article 3 (Consideration)
(with respect to any final reports or to the extent any amounts are due but
unpaid), Section 3.7 (Reports and Records), Section 4.4 (Confidential
Information), Article 5 (Confidentiality), Article 6 (Term and Termination),
Section 8.3 (Disclaimer of Warranties, Damages), Section 8.4 (Indemnification),
Section 8.5 (Insurance), Article 9 (Use of Name), and Article 10 (Additional
Provisions) shall survive such termination or expiration of this Agreement in
accordance with their respective terms.
 
ARTICLE 7:  PATENT MAINTENANCE; PATENT INFRINGEMENT
 
7.1          Prosecution of Licensed Patents.  As between Licensor and Licensee,
the Parties agree as follows:
 
7.1.1          Licensor shall have the sole right, but not the obligation, to
Prosecute patent applications and issued patents within Licensed Patents, in
Licensor’s sole discretion.  Subject to Section 7.1.3, Licensor shall provide
Licensee with a reasonable opportunity to review and provide comments in
connection with the Prosecution of the Licensed Patents; and Licensor shall keep
Licensee reasonably informed as to all material developments with respect to
such Licensed Patents and shall supply to Licensee copies of material
communications received and filed in connection with the Prosecution of such
Licensed Patents.
 
7.1.2          Nothing in this Agreement obligates Licensor to continue to
Prosecute any patent applications or issued patents, and Licensee acknowledges
that Licensor shall have no obligation to undertake any inter-party proceedings,
such as oppositions, inter partes review, or interferences, or to undertake any
re-examination or re-issue proceedings, in either case, with respect to the
Licensed Patents.
 
7.1.3          Licensee acknowledges that The Trustees of the University of
Pennsylvania control Prosecution of the Licensed Patents, with Licensor having
certain rights to review.  Licensee acknowledges and agrees that (a) the rights
and obligations under this Section 7.1 are subject to the rights of the
REGENXBIO Licensors set forth in the GSK Agreement and Penn Agreement with
respect to the Licensed Patents, and (b) Licensor’s obligations under this
Agreement only apply to the extent of Licensor’s rights with respect to
participation in Prosecuting the Licensed Patents under the GSK Agreement and
the Penn Agreement.  Licensor shall have the sole right, but not the obligation,
to Prosecute patent applications and issued patents within Licensed Patents, in
Licensor’s sole discretion.
 
7.2          Infringement Actions Against Third Parties.
 
7.2.1          Licensee is responsible for notifying Licensor promptly of any
infringement of Licensed Patents (other than Retained Rights) that may come to
Licensee’s attention, including any “patent certification” filed in the United
States under 21 U.S.C. § 355(b)(2) or 21 U.S.C. § 355(j)(2) or similar
provisions in other jurisdictions alleging the invalidity, unenforceability or
non-infringement of any Licensed Patents, and any notification received pursuant
to subsection (k) of 42 U.S.C § 262 for any Licensed Product that becomes a
“reference product.”  However, Licensee is under no obligation to search for
potential infringers.
 
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7.2.2          As between Licensor and Licensee, but subject to any obligations
of Licensor to the REGENXBIO Licensors, Licensor shall have the sole right, but
not the obligation, to prosecute any such infringement * * *.  In any action to
enforce any of the Licensed Patents, Licensee, at the request and expense of
Licensor, shall cooperate to the fullest extent reasonably possible, including
in the event that, if Licensor is unable to initiate or prosecute such action
solely in its own name, Licensee shall join such action voluntarily and shall
execute all documents necessary to initiate litigation to prosecute, maintain,
and settle such action.  Nothing in this Agreement obligates Licensor to bring
or prosecute lawsuits against Third Parties for infringement of any Licensed
Patents.
 
7.2.3          Licensee shall have no right to undertake prosecution of any such
infringement.
 
7.3          Defense of Infringement Claims.  In the event Licensee or Licensor
becomes aware that Licensee’s or any of its Affiliates’ or any Sublicensees’
practice of the Licensed Patents is the subject of a claim for patent
infringement by a Third Party, that Party shall promptly notify the other, and
the Parties shall consider the claim and the most appropriate action to take. 
Licensee shall cause each of its Affiliates and each Sublicensee to notify
Licensee promptly in the event such entity becomes aware that its practice of
the Licensed Patents is the subject of a claim of patent infringement by
another.  To the extent Licensor takes any action, Licensor (or the REGENXBIO
Licensors) shall have the right to require Licensee’s reasonable cooperation in
any such suit, upon written notice to Licensee; and Licensee shall have the
obligation to participate upon Licensor’s request, in which event, Licensor
shall bear the cost of Licensee’s participation.  Without Licensor’s prior
written permission, not to be unreasonably withheld, Licensee must not settle or
compromise any such suit in a manner that imposes any material obligations or
restrictions on Licensor or the REGENXBIO Licensors or grants any rights to the
Licensed Patents other than rights that Licensee has the right to grant under
this Agreement.
 
ARTICLE 8:  WARRANTIES; INDEMNIFICATION
 
8.1          Representations and Warranties by Licensor.  Licensor represents
and warrants to Licensee as of the Effective Date:
 
8.1.1          Licensor has the right, power, and authority to enter into this
Agreement and to grant to Licensee the rights specified in this Agreement;
 
8.1.2          This Agreement when executed shall become the legal, valid, and
binding obligation of it, enforceable against it, in accordance with its terms;
 
8.1.3          There are no actions, suits, proceedings, or arbitrations pending
or, to Licensor’s knowledge, threatened against Licensor relating to the
Licensed Patents that would be inconsistent with the rights granted to Licensee
under this Agreement;
 
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8.1.4          To Licensor’s knowledge, (a) the Licensed Patents are solely
owned by The Trustees of the University of Pennsylvania, and (b) no Third Party
(other than the REGENXBIO Licensors) has any right, interest, or claim in or to
such Licensed Patents in the Field that are inconsistent with those granted to
Licensee in the Field under this Agreement;
 
8.1.5          Licensor has not received any written notice from any Third Party
patentee alleging infringement of such Third Party’s patents by the practice of
the Licensed Patents in the Field; and
 
8.1.6          To Licensor’s knowledge, Licensor does not Control as of the
Effective Date any patent or patent application (other than the Licensed Patents
(as defined in Section 1.17) that would necessarily be infringed by the use or
sale of Licensed Vectors in the Field.  If it is determined, in accordance with
the procedure of this Section 8.1.6, that Licensor Controls as of the Effective
Date a patent or patent application (other than the Licensed Patents) that would
necessarily be infringed by the use or sale of Licensed Vectors in the Field,
then Licensee’s sole remedy shall be the inclusion of the applicable patent or
patent application as a “Licensed Patent” hereunder but solely to the extent of
the claim(s) that would necessarily be infringed by the use or sale of Licensed
Vectors.  At any time during the term of this Agreement, Licensee may notify
Licensor in writing of any such patent or patent application that Licensee
believes should be included as a “Licensed Patent” pursuant to this Section
8.1.6.  Such written notice shall identify the relevant patent or patent
application and relevant claim(s) and shall explain briefly why Licensee, in
good faith, believes it should be included as a “Licensed Patent.”  Licensor has
* * * following Licensor’s receipt of Licensee’s written notice to dispute the
inclusion of such patent or patent application or the scope of the remedy; in
which event, such dispute will be resolved in accordance with Section 10.6. 
Upon the Parties’ agreement (or a resolution, in favor of Licensee, of the
dispute pursuant to Section 10.6), the applicable claim(s) of the applicable
patent or patent application will be deemed a “Licensed Patent” hereunder. For
the avoidance of doubt, Licensor makes no representation or warranty under this
Section 8.1.6 as to any claim of a patent or patent application covering the
manufacture of Licensed Patents, and Licensee acknowledges that manufacturing
claims of any patents or patent applications will not be added as “Licensed
Patents” pursuant to the procedure set forth in this Section 8.1.6.  For the
purpose of this Section 8.1.6, “Control” means the possession by Licensor
(whether by ownership or license, other than pursuant to this Agreement) of the
ability to grant to Licensee access, a license, or a sublicense (as applicable)
to the applicable patent or patent application on the terms and conditions set
forth herein without violating the terms of any agreement or other arrangement
with any Third Party. 
 
8.2          Representations and Warranties by Licensee.  Licensee represents
and warrants to Licensor as of the Effective Date that:
 
8.2.1          Licensee has the right, power, and authority to enter into this
Agreement and to grant the rights granted by it hereunder;
 
8.2.2          This Agreement when executed shall become the legal, valid, and
binding obligation of it, enforceable against it, in accordance with its terms;
 
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8.2.3          Licensee has the ability and the resources, including financial
resources, necessary to carry out its obligations under this Agreement; and
 
8.2.4          There are no actions, suits, proceedings, or arbitrations pending
or, to Licensee’s knowledge, threatened against Licensee that would impact
Licensee’s activities under this Agreement.
 
8.3          Disclaimer of Warranties, Damages.  EXCEPT AS SET FORTH IN SECTION
8.1, THE LICENSED TECHNOLOGY, LICENSED PRODUCTS, AND ALL RIGHTS LICENSED UNDER
THIS AGREEMENT ARE PROVIDED ON AN “AS IS” BASIS, AND LICENSOR MAKES NO
REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, WITH RESPECT THERETO.  BY WAY
OF EXAMPLE BUT NOT OF LIMITATION, EXCEPT AS SET FORTH IN SECTION 8.1, LICENSOR
MAKES NO REPRESENTATIONS OR WARRANTIES, AND HEREBY DISCLAIMS ALL EXPRESS AND
IMPLIED REPRESENTATIONS AND WARRANTIES, (i) OF COMMERCIAL UTILITY, ACCURACY,
COMPLETENESS, PERFORMANCE, TITLE, MERCHANTABILITY, FITNESS FOR A PARTICULAR
PURPOSE, VALIDITY OR ENFORCEABILITY OF THE LICENSED TECHNOLOGY, AND
PROFITABILITY; OR (ii) THAT THE USE OF THE LICENSED TECHNOLOGY, OR LICENSED
PRODUCTS WILL NOT INFRINGE ANY TECHNOLOGY, COPYRIGHT, TRADEMARK, OR OTHER
PROPRIETARY RIGHTS OF THIRD PARTIES.  EXCEPT AS SET FORTH HEREIN, NONE OF
LICENSOR AND  THE REGENXBIO LICENSORS SHALL BE LIABLE TO LICENSEE, LICENSEE’S
SUCCESSORS OR ASSIGNS, ANY SUBLICENSEES, OR ANY THIRD PARTY WITH RESPECT TO: 
(a) ANY CLAIM ARISING FROM USE OF THE LICENSED TECHNOLOGY, LICENSED PRODUCTS,
AND ANY OR ALL RIGHTS LICENSED UNDER THIS AGREEMENT OR FROM THE DEVELOPMENT,
TESTING, MANUFACTURE, USE, OR SALE OF LICENSED PRODUCTS; OR (b) ANY CLAIM FOR
LOSS OF PROFITS, LOSS OR INTERRUPTION OF BUSINESS, OR FOR INDIRECT, SPECIAL,
INCIDENTAL, EXEMPLARY, PUNITIVE, OR CONSEQUENTIAL DAMAGES OF ANY KIND, INCLUDING
ANY ARISING FROM OR RELATING TO ANY BREACH OF THIS AGREEMENT OR THE EXERCISE OF
RIGHTS HEREUNDER, REGARDLESS OF ANY NOTICE OF SUCH DAMAGES.  NOTHING IN THIS
SECTION 8.3 IS INTENDED TO LIMIT OR RESTRICT THE INDEMNIFICATION RIGHTS OR
OBLIGATIONS OF EITHER PARTY UNDER SECTION 8.4 OR TO LIMIT A PARTY’S LIABILITY
FOR BREACHES OF ITS OBLIGATION REGARDING CONFIDENTIALITY UNDER ARTICLE 5.
 
8.4          Indemnification.
 
8.4.1          By Licensee.  Licensee shall defend, indemnify, and hold harmless
Licensor, the REGENXBIO Licensors, and their respective shareholders, members,
officers, trustees, faculty, students, contractors, agents, and employees
(individually, a “Licensor Indemnified Party” and, collectively, the “Licensor
Indemnified Parties”) from and against any and all Third Party liability, loss,
damage, action, claim, fee, cost, or expense (including attorneys’ fees)
(individually, a “Third Party Liability” and, collectively, the “Third Party
Liabilities”) suffered or incurred by the Licensor Indemnified Parties from
claims of such Third Parties that result from or arise out of:  * * *; provided,
however, that Licensee shall not be liable for claims to the extent based on any
breach by Licensor of the representations, warranties, or obligations of this
Agreement or the gross negligence or intentional misconduct of any of the
Licensor Indemnified Parties.  Without limiting the foregoing, Licensee must
defend, indemnify, and hold harmless the Licensor Indemnified Parties from and
against any Third Party Liabilities resulting from:
 
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(a)
any * * * or other claim of any kind related to the * * * by a Third Party of a
Licensed Product that * * * by Licensee, its Affiliates, any Sublicensees, their
respective assignees, or vendors;

 

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

 

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

 
8.4.2          By Licensor.  Licensor shall defend, indemnify, and hold harmless
Licensee, its shareholders, members, officers, contractors, agents, and
employees (individually, a “Licensee Indemnified Party” and, collectively, the
“Licensee Indemnified Parties”) from and against any and all Third Party
Liabilities suffered or incurred by the Licensee Indemnified Parties from claims
of such Third Parties that result from or arise out of:  (i) any breach by
Licensor of the representations, warranties, or obligations of this Agreement;
and (ii) Licensor’s or its Affiliates’ gross negligence or intentional
misconduct; provided, however, that Licensor shall not be liable for claims
based on any breach by Licensee of the representations, warranties, or
obligations of this Agreement or the gross negligence or intentional misconduct
of any of the Licensee Indemnified Parties.
 
8.4.3          Indemnification Procedure.  Each Party, as an indemnifying party
(an “Indemnifying Party”), shall not be permitted to settle or compromise any
claim or action giving rise to Third Party Liabilities in a manner that imposes
any restrictions or obligations on any indemnified party (an “Indemnified
Party”) without the other Party’s prior written consent or, if Licensee is the
Indemnifying Party, that grants any rights to the Licensed Technology or
Licensed Products other than those Licensee has the right to grant under this
Agreement without Licensor’s prior written consent.  The Indemnifying Party
shall be permitted to control any litigation or potential litigation involving
the defense of any claim subject to indemnification pursuant to this Section
8.4, including the selection of counsel, with the reasonable approval of the
Indemnified Party.  Upon the Indemnifying Party’s reasonable request, the
Indemnified Parties will reasonably cooperate with the Indemnifying Party in the
defense and settlement of any such claim, at the Indemnifying Party’s cost and
expense.  If an Indemnifying Party fails or declines to assume the defense of
any such claim or action within * * * after notice thereof, then the Indemnified
Party may assume the defense of such claim or action at the cost and risk of the
Indemnifying Party, and any Third Party Liabilities related thereto shall be
conclusively deemed a Third Party Liability of the Indemnifying Party.  The
indemnification rights of an Indemnified Party contained in this Agreement are
in addition to all other rights that such Indemnified Party may have at law or
in equity or otherwise.  The Indemnifying Party will pay directly all Third
Party Liabilities incurred for defense or negotiation of any claim hereunder or
will reimburse the Indemnified Party for all documented Third Party Liabilities
incident to the defense or negotiation of any such claim within * * * after the
Indemnifying Party’s receipt of invoices for such fees, expenses, and charges.
 
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8.5          Insurance.  Licensee will procure and maintain insurance policies
for the following coverages with respect to product liability, personal injury,
bodily injury, and property damage arising out of Licensee’s (and its
Affiliates’ and any Sublicensees’) performance under this Agreement:  (a) during
the term of this Agreement, comprehensive general liability, including broad
form and contractual liability, in a minimum amount of * * * combined single
limit per occurrence (or claim) and in the aggregate annually; (b) prior to the
commencement of clinical trials involving Licensed Products and thereafter for a
period of not less than * * * (or such longer period as Licensee is required by
applicable law to continue to monitor the participants in the clinical trial),
clinical trials coverage in amounts that are reasonable and customary in the
U.S. pharmaceutical industry, subject always to a minimum limit of * * *
combined single limit per occurrence (or claim) and in the aggregate annually;
and (c) from prior to the first commercial sale of a Licensed Product until * *
* after the last sale of a Licensed Product, product liability coverage, in
amounts that are reasonable and customary in the U.S. pharmaceutical industry,
subject always to a minimum limit of * * * combined single limit per occurrence
(or claim) and in the aggregate annually.  Licensor may review periodically the
adequacy of the minimum amounts of insurance for each coverage required by this
Section 8.5, and Licensor reserves the right to require Licensee to adjust the
limits accordingly.  The required minimum amounts of insurance do not constitute
a limitation on Licensee’s liability or indemnification obligations to the
Licensor Indemnified Parties under this Agreement.  The policies of insurance
required by this Section 8.5 will be issued by an insurance carrier with an A.M.
best rating of * * * or better and will name Licensor as an additional insured
with respect to Licensee’s performance (and its Affiliates’ and any
Sublicensees’) under this Agreement.  Licensee will provide Licensor with
insurance certificates evidencing the required coverage within * * * after the
Effective Date and the commencement of each policy period and any renewal
periods.  Each certificate will provide that the insurance carrier will notify
Licensor in writing at least * * * prior to the cancellation or material change
in coverage.  Licensee will cause all Sublicensees to comply with the terms of
this Section 8.5 to the same extent as Licensee.
 
ARTICLE 9:  USE OF NAME
 
9.1          Licensee, its Affiliates, any Sublicensees, and all of its and
their employees and agents must not use Licensor’s, the University of
Pennsylvania’s, or SmithKline Beecham Corporation’s name, seal, logo, trademark,
or service mark (or any adaptation thereof) or the name, seal, logo, trademark,
or service mark (or any adaptation thereof) of any of such entities’
representative, school, organization, employee, or student in any way without
the prior written consent of Licensor or such entity, as applicable, unless
required to do so pursuant to applicable law, rule, regulation or rules of a
securities exchange; provided, however that Licensee may acknowledge the
existence and general nature of this Agreement, subject to Section 5.2 or 5.3,
as applicable.
 
9.2          Licensor and all of its employees and agents must not use
Licensee’s name, seal, logo, trademark, or service mark (or any adaptation
thereof) in any way without the prior written consent of Licensee; provided,
however that Licensor may acknowledge the existence and general nature of this
Agreement, subject to Section 5.2 or 5.3, as applicable, and refer to Licensee
as a licensee of Licensor.
 
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ARTICLE 10:  ADDITIONAL PROVISIONS
 
10.1        Relationship.  Nothing in this Agreement shall be deemed to
establish a relationship of principal and agent between Licensee and Licensor,
nor any of their agents or employees for any purpose whatsoever, nor shall this
Agreement be construed as creating any other form of legal association or
arrangement which would impose liability upon one Party for the act or failure
to act of the other Party.
 
10.2        Assignment.  The rights and obligations of Licensee and Licensor
hereunder shall inure to the benefit of, and shall be binding upon, their
respective permitted successors and assigns.  Licensee may not assign or
otherwise transfer (by operation of law or otherwise) this Agreement or any of
its rights or obligations under this Agreement without the prior written consent
of Licensor, which consent is in the absolute discretion of Licensor but shall
not be unreasonably withheld (except Licensee shall have the right to assign
this Agreement without Licensor’s consent to a wholly owned Affiliate, in which
case Licensee shall remain responsible for the performance of this Agreement by
such Affiliate); provided, however, Licensee shall be permitted to transfer (by
operation of law or otherwise) this Agreement without Licensor’s consent in
connection with a Change of Control; provided that, Licensee: (i) requires any
transferee or successor to agree in writing to be legally bound by this
Agreement to the same extent as Licensee and provides Licensor with a copy of
such undertaking;  (ii) provides Licensor with written notice of the Change of
Control to Licensor within * * * of the consummation of the transaction
resulting in a Change of Control of Licensee; and (iii) provides Licensor with a
copy of the definitive agreement for the Change of Control of Licensee within *
* * of the consummation of the transaction (provided, that Licensee shall be
entitled to include customary redactions in such copy provided to Licensor, to
the extent such redacted information is not necessary to verify compliance with
the terms of this Agreement or otherwise required by the Penn Agreement and/or
GSK Agreement).  Notwithstanding anything to the contrary in this Agreement, for
clarity, in case of a Licensee Change of Control, in no event shall any
intellectual property rights owned or controlled by the acquirer or its
Affiliates immediately prior to such Licensee Change of Control be included in
any of the licenses granted to Licensor under this Agreement.  Licensor may
assign this Agreement and its rights and obligations without the consent of
Licensee.  No assignment shall relieve the assigning Party of responsibility for
the performance of any accrued obligations that it has prior to such
assignment.  Any attempted assignment by Licensee in violation of this Section
10.2 shall be null and void and of no legal effect.
 
10.3        Waiver.  A waiver by either Party of a breach of any provision of
this Agreement will not constitute a waiver of any subsequent breach of that
provision or a waiver of any breach of any other provision of this Agreement.
 
10.4        Notices.  Notices, payments, statements, reports, and other
communications under this Agreement shall be in writing and shall be deemed to
have been received as of the date received if sent by public courier (e.g.,
Federal Express), by Express Mail, receipt requested, by facsimile, or by
electronic mail (with a copy of such facsimile or electronic mail also sent by
one of the other methods of delivery) and addressed as follows:
 
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If for Licensor:
with a copy to:
   
REGENXBIO Inc.
REGENXBIO Inc.
9600 Blackwell Road
9600 Blackwell Road
Suite 210
Suite 210
Rockville, MD 20850
Rockville, MD 20850
USA
USA
Attn:  Chief Executive Officer
Attn:  General Counsel
Telephone:
240-552-8181
Telephone:

240-552-8181
Facsimile:

240-652-9692
Facsimile:
240-652-9692    
If for Licensee:
with a copy to:
   
Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc.
350 Fifth Avenue
350 Fifth Avenue
Suite 7530
Suite 7530
New York, NY 10118
New York, NY 10118
USA
USA
Attn: Chief Executive Officer
Attn: Director of Legal & Corporate Development
Telephone:

646-440-9100
Telephone:

646-440-9100
Facsimile:
 
Facsimile:
 

Either Party may change its official address upon written notice to the other
Party in accordance with this Section 10.4.
 
10.5        Applicable Law.  This Agreement shall be construed and governed in
accordance with the laws of the State of New York, without giving effect to
conflict of law provisions that may require the application of the laws of
another jurisdiction.  Subject to Section 10.6, the Parties hereby submit to the
exclusive jurisdiction of and venue in the courts located in the State of New
York with respect to any and all disputes concerning the subject of this
Agreement.
 
10.6        Dispute Resolution.  In the event of any controversy or claim
arising out of or relating to this Agreement, the Parties shall first attempt to
resolve such controversy or claim through good faith negotiations for a period
of not less than * * * following notification of such controversy or claim to
the other Party.  If such controversy or claim cannot be resolved by means of
such negotiations during such period, then such controversy or claim shall be
resolved by binding arbitration administered by the American Arbitration
Association (“AAA”) in accordance with the Commercial Arbitration Rules of the
AAA in effect on the date of commencement of the arbitration, subject to the
provisions of this Section 10.6.  The arbitration shall be conducted as follows:
 
10.6.1        The arbitration shall be conducted by three arbitrators, each of
whom by training, education, or experience has knowledge of the research,
development, and commercialization of biological therapeutic products in the
United States.  The arbitration shall be conducted in English and held in New
York, New York.
 
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10.6.2        In its demand for arbitration, the Party initiating the
arbitration shall provide a statement setting forth the nature of the dispute,
the names and addresses of all other parties, an estimate of the amount involved
(if any), the remedy sought, otherwise specifying the issue to be resolved, and
appointing one neutral arbitrator.  In an answering statement to be filed by the
responding Party within * * * after confirmation of the notice of filing of the
demand is sent by the AAA, the responding Party shall appoint one neutral
arbitrator.  Within * * * from the date on which the responding Party appoints
its neutral arbitrator, the first two arbitrators shall appoint a chairperson.
 
10.6.3        If a Party fails to make the appointment of an arbitrator as
provided in Section 10.6.2, the AAA shall make the appointment.  If the
appointed arbitrators fail to appoint a chairperson within the time specified in
Section 10.6.2 and there is no agreed extension of time, the AAA shall appoint
the chairperson.
 
10.6.4        The arbitrators will render their award in writing and, unless all
Parties agree otherwise, will include an explanation in reasonable detail of the
reasons for their award.  Judgment upon the award rendered by the arbitrators
may be entered in any court having jurisdiction thereof, including in the courts
described in Section 10.5.  The arbitrators will have the authority to grant
injunctive relief and other specific performance; provided that the arbitrators
will have no authority to award damages in contravention of this Agreement, and
each Party irrevocably waives any claim to such damages in contravention of this
Agreement.  The arbitrators will, in rendering their decision, apply the
substantive law of the State of New York, without giving effect to conflict of
law provisions that may require the application of the laws of another
jurisdiction.  The decision and award rendered by the arbitrators will be final
and non-appealable (except for an alleged act of corruption or fraud on the part
of the arbitrator).
 
10.6.5        The Parties shall use their reasonable efforts to conduct all
dispute resolution procedures under this Agreement as expeditiously,
efficiently, and cost-effectively as possible.
 
10.6.6        All expenses and fees of the arbitrators and expenses for hearing
facilities and other expenses of the arbitration will be borne equally by the
Parties unless the Parties agree otherwise or unless the arbitrators in the
award assess such expenses against one of the Parties or allocate such expenses
other than equally between the Parties.  Each of the Parties will bear its own
counsel fees and the expenses of its witnesses except to the extent otherwise
provided in this Agreement or by applicable law.
 
10.6.7        Compliance with this Section 10.6 is a condition precedent to
seeking relief in any court or tribunal in respect of a dispute, but nothing in
this Section 10.6 will prevent a Party from seeking equitable or other
interlocutory relief in the courts of appropriate jurisdiction, pending the
arbitrators’ determination of the merits of the controversy, if applicable to
protect the confidential information, property, or other rights of that Party or
to otherwise prevent irreparable harm that may be caused by the other Party’s
actual or threatened breach of this Agreement.
 
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10.7        No Discrimination.  Licensee, its Affiliates, and Licensee shall use
reasonable efforts to require that any Sublicensees, in their respective
activities under this Agreement, shall not discriminate against any employee or
applicant for employment because of race, color, sex, sexual, or affectional
preference, age, religion, national, or ethnic origin, handicap, or because he
or she is a disabled veteran or a veteran (including a veteran of the Vietnam
Era).
 
10.8        Compliance with Law.  Licensee (and its Affiliates’ and any
Sublicensees’) must comply with all prevailing laws, rules, and regulations that
apply to its activities or obligations under this Agreement.  Without limiting
the foregoing, it is understood that this Agreement may be subject to United
States laws and regulations controlling the export of technical data, computer
software, laboratory prototypes, and other commodities, articles, and
information, including the Arms Export Control Act as amended in the Export
Administration Act of 1979 and that Licensee’s obligations are contingent upon
compliance with applicable United States export laws and regulations.  The
transfer of certain technical data and commodities may require a license from
the cognizant agency of the United States Government and/or written assurances
by Licensee that Licensee shall not export data or commodities to certain
foreign countries without prior approval of such agency.  Licensor neither
represents that a license is not required nor that, if required, it will issue.
 
10.9        Entire Agreement.  This Agreement embodies the entire understanding
between the Parties relating to the subject matter hereof and supersedes all
prior understandings and agreements, whether written or oral, including that
certain Mutual Non-Disclosure Agreement dated * * * between the Parties.  All
“Confidential Information” (as defined in such Mutual Non-Disclosure Agreement)
disclosed by one Party to the other Party pursuant to such Mutual Non-Disclosure
Agreement shall be deemed “Confidential Information” of such disclosing Party
under this Agreement (unless and until it falls within one of the exclusions set
forth in Section 1.9).  This Agreement may not be varied except by a written
document signed by duly authorized representatives of both Parties.
 
10.10      Marking.  Licensee, its Affiliates, and any Sublicensees shall mark
any Licensed Product (or their containers or labels) made, sold, or otherwise
distributed by it or them with any notice of patent rights necessary or
desirable under applicable law to enable the Licensed Patents to be enforced to
their full extent in any country where Licensed Products are made, used, sold,
offered for sale, or imported.
 
10.11      Severability and Reformation.  If any provision of this Agreement is
held to be invalid or unenforceable by a court of competent jurisdiction, then
such invalid or unenforceable provision will be automatically revised to be a
valid or enforceable provision that comes as close as permitted by law to the
Parties’ original intent; provided that, if the Parties cannot agree upon such
valid or enforceable provision, then the remaining provisions of this Agreement
will remain in full force and effect, unless the invalid or unenforceable
provisions are of such essential importance to this Agreement that it is to be
reasonably assumed that the Parties would not have entered into this Agreement
without the invalid or unenforceable provisions.
 
10.12      Further Assurances.  Each Party hereto agrees to execute,
acknowledge, and deliver such further instruments, and to do all other
reasonable acts, as may be necessary or appropriate in order to carry out the
purposes and intent of this Agreement.
 
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10.13      Interpretation; Construction.  The captions to the several Articles
and Sections of this Agreement are included only for convenience of reference
and shall not in any way affect the construction of, or be taken into
consideration in interpreting, this Agreement.  In this Agreement, unless the
context requires otherwise, (a) the word “including” shall be deemed to be
followed by the phrase “without limitation” or like expression; (b) references
to the singular shall include the plural and vice versa; (c) references to
masculine, feminine, and neuter pronouns and expressions shall be
interchangeable; (d) the words “herein” or “hereunder” relate to this Agreement;
(e) “or” is disjunctive but not necessarily exclusive; (f) the word “will” shall
be construed to have the same meaning and effect as the word “shall”; (g) all
references to “dollars” or “$” herein shall mean U.S. Dollars; (h) unless
otherwise provided, all reference to Sections, Articles, and exhibits in this
Agreement are to Sections, Articles, and exhibits of and in this Agreement; and
(i) whenever this Agreement refers to a number of days, such number shall refer
to calendar days unless business days are specified.  Business days shall mean a
day on which banking institutions in Washington, D.C. are open for business. 
Each Party represents that it has been represented by legal counsel in
connection with this Agreement and acknowledges that it has participated in the
drafting hereof.  In interpreting and applying the terms and provisions of this
Agreement, the Parties agree that no presumption will apply against the Party
which drafted such terms and provisions.
 
10.14      Cumulative Rights and Remedies.  The rights and remedies provided in
this Agreement and all other rights and remedies available to either Party at
law or in equity are, to the extent permitted by law, cumulative and not
exclusive of any other right or remedy now or hereafter available at law or in
equity.  Neither asserting a right nor employing a remedy shall preclude the
concurrent assertion of any other right or employment of any other remedy, nor
shall the failure to assert any right or remedy constitute a waiver of that
right or remedy.
 
10.15      Counterparts.  This Agreement may be executed in one or more
counterparts, each of which will be deemed an original, but all of which
together will constitute one and the same instrument.

[REMAINDER OF PAGE INTENTIONALLY LEFT BLANK]

31

--------------------------------------------------------------------------------

IN WITNESS WHEREOF, the Parties, intending to be legally bound, have caused this
License Agreement to be executed by their duly authorized representatives.
 
REGENXBIO INC.
 
ROCKET PHARMACEUTICALS, LTD.
     
By:
/s/ Kenneth Mills   
By:

/s/ Gaurav D. Shah 

Name:
Kenneth Mills   
Name:

Gaurav D. Shah 
Title:
President and CEO  
Title:

CEO and President

--------------------------------------------------------------------------------

Exhibit A-1

Licensed Patents (AAV9)
 
Application #
Patent #
Filing Date
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Status
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--------------------------------------------------------------------------------

Exhibit A-2
 
Licensed Patents * * *
 
Application #
Patent #
Filing Date
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Status
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--------------------------------------------------------------------------------

Exhibit A-3
 
Licensed Patents * * *
 
Application #
Patent #
Filing Date
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Status
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--------------------------------------------------------------------------------

Exhibit B
Press Release

[image1.jpg]
[image00002.jpg]

Rocket Pharmaceuticals and REGENXBIO Announce New License Agreement for the
Treatment of Danon Disease Using NAV AAV9 Vector

- REGENXBIO grants Rocket exclusive, worldwide rights to NAV AAV9 for the
development and commercialization of treatments for Danon Disease -

 - Investigational New Drug Application Expected to be Filed in 2019; Clinical
Trial to Follow -

NEW YORK and ROCKVILLE, Md.– November 26, 2018 - Rocket Pharmaceuticals, Inc.
(Nasdaq: RCKT), a leading U.S.-based multi-platform gene therapy company, and
REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company
seeking to improve lives through the curative potential of gene therapy based on
its proprietary NAV® Technology Platform, today announced an exclusive,
worldwide license agreement for Rocket to develop and commercialize gene therapy
treatments for Danon disease using REGENXBIO’s NAV AAV9 vector, as well as
exclusive options for two additional NAV AAV vectors for the treatment of Danon
disease.

Under the terms of the agreement, REGENXBIO has granted Rocket an exclusive,
worldwide license, with rights to sublicense, to REGENXBIO’s NAV AAV9 vector for
the development and commercialization of gene therapy treatments for Danon
disease. REGENXBIO has also granted Rocket exclusive options to two additional
undisclosed NAV AAV vectors for the treatment of Danon disease for up to four
years. In return for these rights, REGENXBIO will receive a $7 million upfront
payment, ongoing fees, milestone payments and high-single to low-double digit
royalties on net sales of products incorporating the licensed intellectual
property.

“Rocket is delighted to partner with REGENXBIO to advance our first gene therapy
product derived from the NAV Technology Platform, reinforcing the potential of
our disease-focused, multi-platform development approach allowing for the
potential first-mover advantage without being limited by vector type,” said
Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “Danon
disease represents a significant area of unmet medical need as no specific
therapies are available for the patients and families suffering from the
disease. We believe the well-understood disease biology and severe patient need
makes Danon disease an ideal target for gene therapy and we expect to enter the
clinic next year."

“This license agreement provides further validation of the potential for the
diverse application of our NAV Technology Platform for the treatment of severe
diseases with high unmet medical,” said Kenneth T. Mills, President and Chief
Executive Officer of REGENXBIO. ““We are pleased to initiate our partnership
with Rocket, an emerging leader in gene therapy for rare diseases, via
development of a gene therapy treatment for Danon disease.”

--------------------------------------------------------------------------------

[image1.jpg]
[image00002.jpg]

About Danon Disease

Danon disease is a rare neuromuscular and cardiovascular disease characterized
by profound cardiomyopathy, skeletal myopathies, and mild cognitive impairment.
It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S.
and the European Union. Danon disease is caused by mutations in the gene
encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator
of autophagy. The LAMP-2 protein has three distinct variants generated by
alternative splicing: LAMP-2A, LAMP 2B and LAMP-2C. Mutations resulting in
LAMP-2B dysfunction are associated with severe cardiac disease features. The
disease is often fatal in patients in the second or third decade of life due to
progressive heart failure unless treated with a cardiac transplantation, which
is nonetheless not considered curative. There are no specific therapies
available for the treatment of Danon disease.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an emerging,
clinical-stage biotechnology company focused on developing first-in-class gene
therapy treatment options for rare, devastating diseases. Rocket’s
multi-platform development approach applies the well-established lentiviral
vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms.
Rocket's lead clinical program is a LVV-based gene therapy for the treatment of
Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone
marrow failure and potentially cancer. Preclinical studies of additional bone
marrow-derived disorders are ongoing and target Pyruvate Kinase Deficiency
(PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant
Osteopetrosis (IMO). Rocket’s first AAV-based gene therapy program targets Danon
disease, a rare neuromuscular and cardiovascular disease. For more information
about Rocket, please visit www.rocketpharma.com.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve
lives through the curative potential of gene therapy. REGENXBIO's NAV Technology
Platform, a proprietary adeno-associated virus (AAV) gene delivery platform,
consists of exclusive rights to more than 100 novel AAV vectors, including AAV7,
AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform
Licensees are applying the NAV Technology Platform in the development of a broad
pipeline of candidates in multiple therapeutic areas.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations,
plans and prospects, including without limitation, Rocket's expectations
regarding the safety, effectiveness and timing of product candidates that Rocket
may develop, including in collaboration with academic partners, to treat Fanconi
Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency
(PKD) and Infantile Malignant Osteopetrosis (IMO), and the safety, effectiveness
and timing of related pre-clinical studies and clinical trials, may constitute
forward-looking statements for the purposes of the safe harbor provisions under
the Private Securities Litigation Reform Act of 1995 and other federal
securities laws and are subject to substantial risks, uncertainties and
assumptions. You should not place reliance on these forward-looking statements,
which often include words such as "believe", "expect", "anticipate", "intend",
"plan", "will give", "estimate", "seek", "will", "may", "suggest" or similar
terms, variations of such terms or the negative of those terms. Although Rocket
believes that the expectations reflected in the forward-looking statements are
reasonable, Rocket cannot guarantee such outcomes. Actual results may differ
materially from those indicated by these forward-looking statements as a result
of various important factors, including, without limitation, Rocket's ability to
successfully demonstrate the efficacy and safety of such products and
pre-clinical studies and clinical trials, its gene therapy programs, the
preclinical and clinical results for its product candidates, which may not
support further development and marketing approval, Rocket's ability to commence
a registrational study in FA within the projected time periods, the potential
advantages of Rocket's product candidates, actions of regulatory agencies, which
may affect the initiation, timing and progress of pre-clinical studies and
clinical trials of its product candidates, Rocket's and its licensors ability to
obtain, maintain and protect its and their respective intellectual property, the
timing, cost or other aspects of a potential commercial launch of Rocket's
product candidates, Rocket's ability to manage operating expenses, Rocket's
ability to obtain additional funding to support its business activities and
establish and maintain strategic business alliances and new business
initiatives, Rocket's dependence on third parties for development, manufacture,
marketing, sales and distribution of product candidates, the outcome of
litigation, and unexpected expenditures, as well as those risks more fully
discussed in the section entitled "Risk Factors" in Rocket's Annual Report on
Form 10-K for the year ended December 31, 2017. Accordingly, you should not
place undue reliance on these forward-looking statements. All such statements
speak only as of the date made, and Rocket undertakes no obligation to update or
revise publicly any forward-looking statements, whether as a result of new
information, future events or otherwise.

--------------------------------------------------------------------------------

[image1.jpg]
[image00002.jpg]

REGENXBIO Forward-Looking Statements

This press release includes "forward-looking statements," within the meaning of
Section 27A of the Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended. These statements express a belief,
expectation or intention and are generally accompanied by words that convey
projected future events or outcomes such as "believe," "may," "will,"
"estimate," "continue," "anticipate," "design," "intend," "expect," "could,"
"plan," "potential," "predict," "seek," "should," "would" or by variations of
such words or by similar expressions. The forward-looking statements include
statements relating to, among other things, REGENXBIO's future operations and
clinical trials. REGENXBIO has based these forward-looking statements on its
current expectations and assumptions and analyses made by REGENXBIO in light of
its experience and its perception of historical trends, current conditions and
expected future developments, as well as other factors REGENXBIO believes are
appropriate under the circumstances. However, whether actual results and
developments will conform with REGENXBIO's expectations and predictions is
subject to a number of risks and uncertainties, including the timing of
enrollment, commencement and completion and the success of clinical trials
conducted by REGENXBIO, its licensees and its partners, the timing of
commencement and completion and the success of preclinical studies conducted by
REGENXBIO and its development partners, the timely development and launch of new
products, the ability to obtain and maintain regulatory approval of product
candidates, the ability to obtain and maintain intellectual property protection
for product candidates and technology, trends and challenges in the business and
markets in which REGENXBIO operates, the size and growth of potential markets
for product candidates and the ability to serve those markets, the rate and
degree of acceptance of product candidates, and other factors, many of which are
beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's
Discussion and Analysis of Financial Condition and Results of Operations"
sections of REGENXBIO's Annual Report on Form 10-K for the year ended December
31, 2017 and comparable "risk factors" sections of REGENXBIO's Quarterly Reports
on Form 10-Q and other filings, which have been filed with the U.S. Securities
and Exchange Commission (SEC) and are available on the SEC's website at
www.sec.gov. All of the forward-looking statements made in this press release
are expressly qualified by the cautionary statements contained or referred to
herein. The actual results or developments anticipated may not be realized or,
even if substantially realized, they may not have the expected consequences to
or effects on REGENXBIO or its businesses or operations. Such statements are not
guarantees of future performance and actual results or developments may differ
materially from those projected in the forward-looking statements. Readers are
cautioned not to rely too heavily on the forward-looking statements contained in
this press release. These forward-looking statements speak only as of the date
of this press release. REGENXBIO does not undertake any obligation, and
specifically declines any obligation, to update or revise any forward-looking
statements, whether as a result of new information, future events or otherwise.

Rocket Contact:

Claudine Prowse, Ph.D.
SVP, Corporate Strategy and IRO
Rocket Pharma, Inc.
The Empire State Building, Suite 7530
New York, NY 10118
cp@rocketpharma.com
www.rocketpharma.com
investors@rocketpharma.com

REGENXBIO Contacts:

Investors
Natalie Wildenradt, 646-681-8192
natalie@argotpartners.com

Media
Adam Pawluk, 202-591-4063
apawluk@jpa.com

--------------------------------------------------------------------------------