Quest and Sarepta Therapeutics Expand
Collaboration to Develop AAV Companion Diagnostics (CDx) for
Sarepta's Gene Therapies
SECAUCUS, N.J. and CAMBRIDGE, Mass., Aug. 30,
2023 /PRNewswire/ -- Quest Diagnostics (NYSE: DGX),
the nation's leading provider of diagnostic information services,
today announced that its AAVrh74 ELISA assay (CDx) has been granted
Breakthrough Device Designation from the U.S. Food and Drug
Administration (FDA). Breakthrough Device Designation is an FDA
process designed to expedite the development and review of certain
medical devices and device-led combination products that provide
for more effective treatment or diagnosis of life-threatening or
irreversibly debilitating diseases or conditions.
The enzyme-linked immunosorbent in vitro diagnostic assay
is for the semi-quantitative detection of antibodies (IgG) to
AAVrh74 capsid in human serum. The test is intended to be used in
conjunction with other available clinical information as an aid to
identify patients eligible for treatment with Sarepta Therapeutics,
Inc.'s (NASDAQ: SRPT) ELEVIDYS (delandistrogene moxeparvovec-rokl),
gene therapy for certain individuals with Duchenne muscular
dystrophy.
In addition, Quest and Sarepta, the leader in precision genetic
medicine for rare diseases, today announced an expanded
collaboration under which Quest will develop one or more companion
or complementary diagnostics in connection with Sarepta's portfolio
of investigational and on-market gene therapies. The collaboration
may encompass screening assays for antibodies to Sarepta's
AAV-vector based gene therapies for muscular dystrophies, including
Duchenne muscular dystrophy (DMD) and limb girdle muscular
dystrophies (LGMD). The first companion diagnostic will
be for Sarepta's first gene therapy, ELEVIDYS (delandistrogene
moxeparvovec-rokl), intended to treat the underlying genetic cause
of DMD, which received U.S. Food and Drug Administration approval
in June 2023. Quest will also provide
clinical lab testing for Sarepta.
"Achieving breakthrough designation for our AAVrh74 ELISA, and
collaboration with AAV-gene therapy pioneer Sarepta, positions
Quest at the forefront of companion diagnostics innovation in the
growing field of gene therapies," said Bill
Finger, General Manager, Pharma Services, Quest Diagnostics.
"Our AAV testing expertise has the potential to empower many life
science collaborators to develop, and ultimately bring to market,
companion diagnostics for their gene therapies."
"Sarepta is pleased to expand its collaboration with Quest
beyond our investigational therapies and to partner to develop
essential diagnostic tools for our approved gene therapies that
help streamline/match gene therapy treatment to eligible patients.
Quest's diagnostic R&D capabilities and laboratory capacity
play a critical role in identifying the patients who may benefit
from these treatments," said Louise
Rodino-Klapac, executive vice president, head of R&D and
chief scientific officer, Sarepta Therapeutics.
About AAV-Based Gene Therapies
Defective genes account for approximately 80% of more than 7,000
rare diseases. The goal of gene therapy is to deliver a functional
copy of a missing or malfunctioning gene to the targeted cells.
Current gene therapies are one-time treatments.
Adeno-associated virus (AAV) vectors may be engineered to act as
therapeutic delivery vehicles to transfer coding changes into the
cell. AAV vectors are being explored for numerous therapeutic
applications, and they are the most commonly used viral gene
delivery system in clinical trialsi. As pre-existing
antibodies to the AAV can pose a potential risk to patient safety
as well as limit the therapeutic potential, patients must be
screened for antibodies to determine patient
eligibility.ii Testing to identify the quantity of total
binding antibodies (TABs) can help identify eligible
patients.iii
About Quest Diagnostics
Quest Diagnostics empowers
people to take action to improve health outcomes. Derived from the
world's largest database of clinical lab results, our diagnostic
insights reveal new avenues to identify and treat disease, inspire
healthy behaviors and improve health care management. Quest
annually serves one in three adult Americans and half the
physicians and hospitals in the United
States, and our approximately 50,000 employees understand
that, in the right hands and with the right context, our diagnostic
insights can inspire actions that transform lives.
www.QuestDiagnostics.com. For more on the company's life sciences
solutions, visit www.QuestDiagnostics.com/LifeSciences.
About Sarepta Therapeutics
Sarepta 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.
Sarepta 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 related to the test
and the expanded collaboration between Sarepta and Quest, including
the potential to develop one or more complementary
diagnostics.
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 and the companion
diagnostics may not be realized or may take longer to realize than
expected due to challenges and uncertainties inherent in product
research and development; the results of future research may not be
consistent with past positive results or may fail to meet
regulatory approval requirements for safety and efficacy; any
potential future inability of the parties to fulfill their
commitments and obligations under the collaboration; and those
risks identified under the heading "Risk Factors" in Sarepta's most
recent Annual Report on Form 10-K 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, except as required by
law.
i AAV vectors: The Rubik's cube of human gene
therapy: Molecular Therapy (cell.com)
ii Human Gene Therapy for Rare Diseases; Guidance for
Industry (fda.gov)
iii Bioengineering a better adeno-associated virus
vector for gene therapy (medicalxpress.com)
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SOURCE Quest Diagnostics