Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, and Genevant Sciences, a
leading nucleic acid delivery company with world-class platforms
and the industry’s most robust and expansive lipid nanoparticle
(LNP) patent estate, today announced a research collaboration
and option agreement for the delivery of LNP-gene editing
therapeutics in Sarepta’s pipeline for neuromuscular diseases. LNPs
offer the potential for a non-viral approach to gene editing and
can provide both optimal uptake into desired cells and efficient
release, resulting in functional delivery of gene editing cargo,
such as CRISPR-Cas, to target tissues.
Gene editing has the potential to revolutionize
the treatment of diseases caused by genetic mutations - including
rare neuromuscular diseases - by permanently altering genes that
lead to disease. Sarepta is pursuing a variety of approaches to
genetic medicine including exon skipping, gene therapies and gene
editing in pursuit of cures for rare diseases.
Under the terms of the agreement, Genevant will
design and collaborate with Sarepta in the development of muscle
targeted LNPs to be applied to gene editing targets in early stage
development. Sarepta will have rights to an exclusive license
to Genevant’s LNP technology for up to four neuromuscular
indications, including Duchenne muscular dystrophy. Genevant may
receive approximately $50 million in near-term payments and is also
eligible for significant future development, regulatory and
commercial milestones and tiered royalties ranging from the
mid-single to low-double digits on future product sales.
“As Sarepta works to advance precision genetic
medicine across multiple modalities, we’ve invested in partnering
and research efforts focused on improving the utility and benefit
of gene-based medicines and providing the greatest possible outcome
to patients. This includes advancing our pre-clinical gene editing
program, looking at both viral and non-viral methods to produce a
functional gene in order to treat a broad range of neuromuscular
diseases,” said Doug Ingram, president and chief executive officer,
Sarepta Therapeutics. “Genevant’s established leadership and
proven LNP technology offers the potential to deliver gene editing
machinery to targeted tissue through a non-viral delivery approach.
Applying this science to neuromuscular diseases fits squarely
within our mission to translate scientific breakthroughs into
meaningful advances for patients whose lives have been impacted by
rare disease.”
“Genevant scientists have been at the forefront
of LNP delivery of nucleic acids for over 20 years. Our platform is
the most clinically validated in the space and is the delivery
technology behind the first nucleic acid-LNP product to have
achieved FDA approval,” said Pete Lutwyche, Ph.D., president and
chief executive officer, Genevant Sciences Corporation. “Efficient,
optimized delivery is often the difference between successful and
unsuccessful nucleic acid drug development, and we are excited to
bring our experience to Sarepta’s gene editing programs in
neuromuscular disease where new options – and new approaches – are
desperately needed.”
About Genevant SciencesGenevant
Sciences is a leading nucleic acid delivery company with
world-class platforms, the industry’s most robust and expansive
lipid nanoparticle (LNP) patent estate, and decades of experience
and expertise in nucleic acid drug delivery and development.
The Company’s scientists have pioneered LNP delivery of
nucleic acids for over 20 years, and the Company’s LNP platform,
which has been studied across more than a dozen discrete product
candidates and is the delivery technology behind the first and only
approved RNAi-LNP (patisiran), enables a wide array of RNA-based
applications, including vaccines, therapeutic protein production,
and gene editing. Genevant Sciences is committed to
transforming the future of human health. For more information,
please visit www.genevant.com.
About Sarepta
TherapeuticsAt Sarepta, we are leading a revolution in
precision genetic medicine and every day is an opportunity to
change the lives of people living with rare disease. The Company
has built an impressive position in Duchenne muscular dystrophy
(DMD) and in gene therapies for limb-girdle muscular dystrophies
(LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth
(CMT), and other CNS-related disorders, with more than 40 programs
in various stages of development. The Company’s programs and
research focus span several therapeutic modalities, including RNA,
gene therapy and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Forward-Looking StatementsThis
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
parties’ obligations and responsibilities under the agreement,
potential payments and fees and Sarepta’s right to an exclusive
license to Genevant’s LNP technology for up to four neuromuscular
indications; the potential benefits of LNPs, including offering a
non-viral approach to gene editing that can provide both optimal
uptake into desired cells and efficient release, resulting in
functional delivery of gene editing cargo, such as CRISPR-Cas, to
target tissues; the potential for gene editing to revolutionize the
treatment of diseases caused by genetic mutations – including rare
neuromuscular diseases – by permanently altering genes that lead to
disease; the goal of Genevant to design and collaborate with
Sarepta in the development of muscle-targeted LNPs that can be
applied to gene editing targets in early stage development;
and Sarepta’s goal to advance its pre-clinical gene editing
program, looking at both viral and non-viral methods to produce a
functional gene in order to treat a broad range of
neuromuscular 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
Genevant 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 the discovery of any new therapeutic compounds or 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; Sarepta may not be
able to execute on its business plans and goals, including meeting
its expected or planned regulatory milestones and timelines,
clinical development plans, and bringing its product candidates to
market, due to a variety of reasons, many of which may be outside
of Sarepta’s control, including possible limitations of company
financial and other resources, manufacturing limitations that may
not be anticipated or resolved for in a timely manner, 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 the COVID-19 pandemic; 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,
2019, 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 Sarepta which you are encouraged to review.
Any of the foregoing risks could materially and
adversely affect Sarepta’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 the 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.
Internet Posting of Information by
Sarepta
We routinely post information that may be
important to investors in the 'For Investors' section of our
website at www.sarepta.com. We encourage investors and
potential investors to consult our website regularly for important
information about us.
Source: Sarepta Therapeutics, Inc.
Contacts:
Sarepta Therapeutics, Inc.Investors: Ian Estepan, 617-274-4052,
iestepan@sarepta.comMedia: Tracy Sorrentino, 617-301-8566,
tsorrentino@sarepta.com
Genevant SciencesPete Zorn, pete.zorn@genevant.com
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