Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision
genetic medicine for rare diseases, and StrideBio, Inc., a leading
developer of novel adeno-associated viral (AAV) based gene
therapies, today announced the signing of a collaboration and
license agreement to develop in vivo AAV-based therapies for up to
eight central nervous system (CNS) and neuromuscular targets.
Pursuant to the agreement, Sarepta is granted an exclusive license
on selected targets to leverage StrideBio’s novel, structure-driven
capsid technology, intended to enhance specific tropism to tissues
of interest and evade neutralizing antibodies. The parties also
plan to focus on strategies intended to address re-dosing
challenges in patients who have received AAV-delivered gene
therapy. StrideBio will conduct all investigational new drug (IND)
enabling research, development and manufacturing for the first four
CNS targets, which are MECP2 (Rett syndrome), SCN1A (Dravet
syndrome), UBE3A (Angelman syndrome), and NPC1 (Niemann-Pick).
Additionally, Sarepta will have an exclusive option to four
additional targets based on StrideBio’s capsid technology.
StrideBio possesses an innovative and proprietary platform that
is enabled by a deep knowledge of AAV structure and a unique
approach to engineering capsids with novel functions, and was
co-founded by Mavis Agbandje-McKenna, Ph.D., and Aravind Asokan,
Ph.D., leading scientists in the field of AAV biology and gene
therapy. StrideBio has created a growing portfolio of novel
AAV capsids evolved in non-human primates that show reduced
seroprevalence and potential for improved tropism to targeted
tissues.
“With our partnership with StrideBio, Sarepta continues to build
on its leadership position in gene therapies to treat rare
diseases. We are excited to work with StrideBio and access its
innovative AAV platform for next-generation capsids,” said Doug
Ingram, Sarepta’s President and Chief Executive Officer. “Our
partnership with StrideBio expands our research portfolio by up to
eight new targets and, through our strategic partnering approach
that has our collaborator lead all IND-enabling research and
development, ensures that we gain access to new technology and
targets while not distracting Sarepta from its near-term
priorities.”
“We are very excited to initiate this multi-target collaboration
with Sarepta, an established leader in the development and
commercialization of genetic medicines,” stated Sapan Shah, Ph.D.,
Chief Executive Officer, StrideBio. “This partnership will provide
significant resources and expertise to enable StrideBio’s continued
rapid expansion of our research and manufacturing platform, as well
as accelerate the development of AAV gene therapies for multiple
rare disease targets. We are looking forward to working together
with Sarepta to bring novel treatments to patients as quickly as
possible.”
Terms of Agreement
Under the terms of the agreement, StrideBio will be responsible
for AAV capsid development, non-clinical development and
manufacturing of preclinical candidates to be selected for
advancement into clinical studies. The parties will also share
early clinical development activities for selected targets, with
Sarepta responsible for late stage development and
commercialization. A total of four initial targets are specified
under the collaboration. StrideBio will receive a $48 million
upfront payment in the form of cash and Sarepta stock, in addition
to significant future development, regulatory and commercial
milestones for the four programs. StrideBio will also receive
royalties on worldwide net sales of any commercial products
developed through the collaboration. Sarepta has also obtained an
exclusive option to expand the collaboration to include up to an
additional four targets with an upfront payment of up to $42.5
million along with future downstream milestone payments, while
StrideBio has an option to obtain co-development and co-commercial
rights to one of the collaboration targets. In addition, Sarepta
has made a commitment to invest in StrideBio’s next financing
round. Further financial terms were not disclosed.
About Sarepta Therapeutics
Sarepta is at the forefront of precision genetic medicine,
having built an impressive and competitive position in Duchenne
muscular dystrophy (DMD) and more recently in gene therapies for
Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA and other
CNS-related disorders, totaling over 20 therapies in various stages
of development. The Company’s programs and research focus span
several therapeutic modalities, including RNA, gene therapy and
gene editing. Sarepta is fueled by an audacious but important
mission: to profoundly improve and extend the lives of patients
with rare genetic-based diseases. For more information, please
visit www.sarepta.com.
About StrideBio, Inc.
StrideBio, Inc is a gene therapy company focused on creating and
developing novel adeno-associated viral (AAV) therapies for rare
diseases. Our STRucture
Inspired DEsign
approach holds the potential to generate unique AAV capsids with
improved characteristics including potency, tissue tropism, and
ability to evade pre-existing antibodies. This powerful new
approach has broad application, enabling gene addition, gene
silencing and gene editing modalities for a wide range of diseases,
including rare genetic diseases. StrideBio is headquartered in
Research Triangle Park, NC. Current investors include
Hatteras Venture Partners, Takeda Ventures, UCB Ventures and
Alexandria Real Estate Equities, Inc. For more information,
please visit www.stridebio.com.
Forward-Looking Statements
This press release contains "forward-looking statements." Any
statements contained in this press release that are not statements
of historical fact may be deemed to be forward-looking statements.
Words such as "believes," "anticipates," "plans," "expects,"
"will," "intends," "potential," "possible" and similar expressions
are intended to identify forward-looking statements. These
forward-looking statements include statements regarding the
potential benefits of the collaboration between Sarepta and
StrideBio, including enhancing specific tropism to tissues of
interest, evading neutralizing antibodies, granting Sarepta access
to new technology and targets, providing significant resources and
expertise to enable StrideBio’s continued rapid expansion of its
research and manufacturing platform, and accelerating the
development of AAV gene therapies for multiple rare disease
targets; the potential of StrideBio’s portfolio to improve tropism
to targeted tissues; the parties’ plan to focus on strategies
intended to address re-dosing challenges in patients who have
received AAV-delivered gene therapy; the terms of the agreement
between Sarepta and StrideBio, including expected and future
payments, future development, regulatory and commercial milestones,
future royalties, options and Sarepta’s commitment to invest in
StrideBio’s next financing round; and Sarepta’s mission to
profoundly improve and extend the lives of patients with rare
genetic-based diseases.
These forward-looking statements involve risks and
uncertainties, many of which are beyond Sarepta’s control. Known
risk factors include, among others: the expected benefits and
opportunities related to the collaboration between Sarepta and
StrideBio may not be realized or may take longer to realize than
expected due to challenges and uncertainties inherent in product
research and development; in particular, the collaboration may not
result in any viable treatments suitable for commercialization due
to a variety of reasons, including any inability of the parties to
perform their commitments and obligations under the agreement, the
results of research may not be consistent with past results or may
not be positive or may otherwise fail to meet regulatory approval
requirements for the safety and efficacy of product candidates,
possible limitations of company financial and other resources,
manufacturing limitations that may not be anticipated or resolved
for in a timely manner, and regulatory, court or agency decisions,
such as decisions by the United States Patent and Trademark Office
with respect to patents that cover Sarepta’s product candidates;
and those risks identified under the heading “Risk Factors” in
Sarepta’s most recent Annual Report on Form 10-K for the year ended
December 31, 2018 and most recent Quarterly Report on Form 10-Q
filed with the Securities and Exchange Commission (SEC) as well as
other SEC filings made by the Company which you are encouraged to
review.
Any of the foregoing risks could materially and adversely affect
the Company’s business, results of operations and the trading price
of Sarepta’s common stock. For a detailed description of risks and
uncertainties Sarepta faces, you are encouraged to review Sarepta's
2018 Annual Report on Form 10-K and most recent Quarterly Report on
Form 10-Q filed with the SEC as well as other SEC filings made by
Sarepta. We caution investors not to place considerable reliance on
the forward-looking statements contained in this press release.
Sarepta does not undertake any obligation to publicly update its
forward-looking statements based on events or circumstances after
the date hereof.
Contacts:
Sarepta Therapeutics, Inc.Investors: Ian
Estepan, 617-274-4052, iestepan@sarepta.comMedia: Tracy Sorrentino,
617-301-8566, tsorrentino@sarepta.com
StrideBio Inc.Robert Hughes, 984-213-7301,
r.hughes@stridebio.com
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