Sarepta Therapeutics Announces Progress on the MyoAAV Program and Exclusive Licensing Agreement with The Broad Institute for MyoAAV Next-generation Capsids for Rare Genetic Diseases
August 08 2022 - 8:30AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced that following
progress on its sponsored research agreement on the MyoAAV program,
it has executed a license agreement with the Broad Institute of MIT
and Harvard (Broad Institute) for MyoAAV in Duchenne muscular
dystrophy and certain other neuromuscular and cardiac indications.
The announcement follows confirmation by Sarepta’s internal
research and manufacturing teams of earlier published research from
Broad Institute, which was conducted under a research agreement
that began in 2020.
MyoAAV is a new group
of adeno-associated viruses (AAV) that use a modified outer protein
shell of AAV, known as the capsid, to deliver genetic therapies
with greater efficiency and at lower doses. Data published in the
journal Cell in 2021 found that, in mouse models of Duchenne
muscular dystrophy and X-linked myotubular myopathy, MyoAAV
demonstrated more efficient delivery of gene therapy and gene
editing payloads, resulting in complete restoration of muscle
function and improved survival. In preclinical data from non-human
primates, compared to natural AAV serotypes, MyoAAV:
- Delivered 25-50 times greater gene
expression in multiple skeletal muscles and 10-15 times greater
gene expression in cardiac muscle;
- Demonstrated reduced delivery to
the liver by 50 percent and showed lower accumulation in the
liver;
- Can be used at up to a log lower
dose than traditional AAV vectors, due to increased
efficiency.
“Research published by
Broad Institute, and so far corroborated by Sarepta’s own internal
research, reinforces the potential of MyoAAV as a breakthrough
next-generation approach in genetic medicine delivery,” said Doug
Ingram, president and chief executive officer, Sarepta. “The
significantly improved efficiency of MyoAAV may unlock the ability
to effectively deliver genetic medicine at as much as a log lower
doses when compared to current AAVs, which could substantially
reduce viral load and cost of goods in the future. As one of the
leaders in the use of AAV-mediated genetic medicine to treat rare
disease, we intend to push the science forward, and our license for
MyoAAV is a quintessential example of that effort.”
“At Sarepta, we are
committed to the future of gene therapy and gene editing, and
excited about the promise and potential of MyoAAV as a
revolutionary approach in the pursuit of novel treatments for rare
genetic disease,” said Louise Rodino-Klapac, executive vice
president, head of research and development and chief scientific
officer, Sarepta. “The MyoAAV platform is noteworthy for its broad
applicability across multiple disease states and will further
advance the science of genetic medicine allowing for swift
advancement of treatments into the clinic across a variety of
conditions, including larger rare cardiac and neuromuscular
indications.”
Under the terms of the
agreement, Sarepta will receive worldwide commercial license grants
for five neuromuscular and cardiac indications, including Duchenne
muscular dystrophy, plus exclusive options on additional targets.
In addition to an upfront payment, Broad Institute is entitled to
future royalties and milestone payments, details of which were not
disclosed.
About Sarepta
TherapeuticsSarepta is on an urgent mission: engineer
precision genetic medicine for rare diseases that devastate lives
and cut futures short. We hold leadership positions in Duchenne
muscular dystrophy (DMD) and limb-girdle muscular dystrophies
(LGMDs), and we currently have more than 40 programs in various
stages of development. Our vast pipeline is driven by our
multi-platform Precision Genetic Medicine Engine in gene therapy,
RNA and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Internet
Posting of InformationWe 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.
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 parties’ obligations and
responsibilities under the agreement; Sarepta’s right to receive
worldwide commercial license grants for five neuromuscular and
cardiac indications, plus exclusive options on additional targets;
the potential benefits of the collaboration between Sarepta and
Broad Institute; and the potential benefits of MyoAAV, including
being a breakthrough next-generation approach in genetic medicine,
and its significantly improved efficiency unlocking the ability to
effectively deliver genetic medicine at as much as a log lower
doses when compared to current AAVs, which could substantially
reduce viral load and cost of goods in the future.
These forward-looking statements involve risks
and uncertainties, many of which are beyond our control. Known risk
factors include, among others: the expected benefits and
opportunities related to the agreement 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 agreement 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; success in preclinical trials
does not ensure that later clinical trials will be successful;
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;
even if Sarepta’s programs result in new commercialized products,
Sarepta may not achieve the expected revenues from the sale of such
products; 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, 2021, 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 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,
except as required by law.
Source: Sarepta Therapeutics, Inc.
Investor Contact: Ian Estepan,
617-274-4052iestepan@sarepta.com
Media Contact: Tracy Sorrentino,
617-301-8566tsorrentino@sarepta.com
Sarepta Therapeutics (NASDAQ:SRPT)
Historical Stock Chart
From Aug 2024 to Sep 2024
Sarepta Therapeutics (NASDAQ:SRPT)
Historical Stock Chart
From Sep 2023 to Sep 2024