Solid Biosciences Announces Collaboration with Mayo Clinic to Accelerate Gene Therapy Innovation and Advance Development of Cardiac Gene Therapies
05 December 2024 - 12:00AM
Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a
life sciences company developing precision genetic medicines for
neuromuscular and cardiac diseases, today announced a collaboration
with Mayo Clinic seeking to advance a cutting-edge AAV gene therapy
platform for the development of next-generation therapies to treat
sudden cardiac death-predisposing genetic cardiomyopathies and
channelopathies with high unmet medical need and significant
patient populations.
Under the terms of the agreement, Mayo Clinic’s
Windland Smith Rice Sudden Death Genomics Laboratory will provide
Solid with an exclusive license to their Suppression and
Replacement (“Sup-Rep”) gene therapy platform. The collaboration
will also provide Solid with an exclusive license to develop and
commercialize six cardiac gene therapy programs developed using
Mayo Clinic’s “Sup-Rep” platform. These programs will use Solid’s
next-generation AAV capsids, including AAV-SLB101, and advanced
manufacturing capabilities to suppress and replace genes implicated
in potentially life-threatening genetic heart diseases. Mayo Clinic
will be responsible for research and development efforts for each
cardiac gene therapy candidate up to IND-enabling studies, at which
point, Solid will have the option to pursue continued development
and commercialization of each licensed program.
“Mayo Clinic’s suppression-replacement gene
therapy platform has the potential for a novel way of treating
patients at risk for sudden cardiac death from their genetic heart
disease,” said Michael J. Ackerman, M.D., Ph.D., Genetic
Cardiologist and Director of Mayo Clinic’s Windland Smith Rice
Sudden Death Genomics Lab in Rochester, MN. “It is gratifying to
have the opportunity to work toward developing new therapeutic
solutions for patients to help them live and thrive despite their
diagnosis.”
“Genetic cardiomyopathies and channelopathies
represent the next frontier for the gene therapy field, and our
collaboration with Dr. Ackerman and his research team places Solid
at the forefront of innovation in this space,” said Bo Cumbo,
President and CEO of Solid. “Beginning with our CPVT IND submission
expected in the first half of 2025, followed by the development of
the Sup-Rep AAV platform and multiple additional therapies
targeting high-impact indications, we have laid the foundation for
Solid to become the leader in cardiac precision genetic medicine.
We look forward to working alongside Mayo Clinic and Dr. Ackerman
to transform cardiac patient care.”
Mayo Clinic has a financial interest in the
Company and technology referenced in this press release. Mayo
Clinic will use any revenue it receives to support its
not-for-profit mission in patient care, education and research.
About Solid BiosciencesSolid
Biosciences is a precision genetic medicine company focused on
advancing a portfolio of gene therapy candidates including SGT-003
for the treatment of Duchenne muscular dystrophy (Duchenne),
SGT-501 for the treatment of catecholaminergic polymorphic
ventricular tachycardia (CPVT), SGT-601 for the treatment of
TNNT2-mediated dilated cardiomyopathy, SGT-401 for the treatment of
BAG3-mediated dilated cardiomyopathy, and additional assets for the
treatment of fatal cardiac diseases. Solid is advancing its diverse
pipeline across rare neuromuscular and cardiac diseases, bringing
together experts in science, technology, disease management, and
care. Patient-focused and founded by those directly impacted,
Solid’s mandate is to improve the daily lives of patients living
with these devastating diseases. For more information, please visit
www.solidbio.com.
Forward-Looking StatementsThis
press release contains “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995,
including statements regarding future expectations, plans and
prospects for the company; the ability to successfully achieve and
execute on our collaborations with Mayo Clinic and other partners,
the company’s goals, priorities and achieve key clinical
milestones; the company’s SGT-003 program, including expectations
for additional CTA filings, site activations, expanded clinical
development, accelerated production of multiple GMP batches of
SGT-003, initiation and enrollment in clinical trials, dosing,
availability of clinical trial data and potential accelerated
approval; the company’s expectations for submission of an IND for
SGT-501 and to submit additional INDs by the end of 2026; the cash
runway of the company and the sufficiency of the Company’s cash,
cash equivalents, and available-for-sale securities to fund its
operations; and other statements containing the words “anticipate,”
“believe,” “continue,” “could,” “estimate,” “expect,” “intend,”
“may,” “plan,” “potential,” “predict,” “project,” “should,”
“target,” “would,” “working” and similar expressions. Any
forward-looking statements are based on management’s current
expectations of future events and are subject to a number of risks
and uncertainties that could cause actual results to differ
materially and adversely from those set forth in, or implied by,
such forward-looking statements. These risks and uncertainties
include, but are not limited to, risks associated with the
company’s ability to advance SGT-003, SGT-501, SGT-601, SGT-401 and
other preclinical programs and capsid libraries on the timelines
expected or at all; obtain and maintain necessary approvals from
the FDA and other regulatory authorities; replicate in clinical
trials positive results found in preclinical studies and
early-stage clinical trials of the company’s product candidates;
obtain, maintain or protect intellectual property rights related to
its product candidates; compete successfully with other companies
that are seeking to develop Duchenne and other neuromuscular and
cardiac treatments and gene therapies; manage expenses; and raise
the substantial additional capital needed, on the timeline
necessary, to continue development of SGT-003, SGT-501, SGT-601,
SGT-401 and other candidates, achieve its other business objectives
and continue as a going concern. For a discussion of other risks
and uncertainties, and other important factors, any of which could
cause the company’s actual results to differ from those contained
in the forward-looking statements, see the “Risk Factors” section,
as well as discussions of potential risks, uncertainties and other
important factors, in the company’s most recent filings with the
Securities and Exchange Commission. In addition, the
forward-looking statements included in this press release represent
the company’s views as of the date hereof and should not be relied
upon as representing the company’s views as of any date subsequent
to the date hereof. The company anticipates that subsequent events
and developments will cause the company's views to change. However,
while the company may elect to update these forward-looking
statements at some point in the future, the company specifically
disclaims any obligation to do so.
Solid Biosciences Investor
Contact:Nicole AndersonDirector, Investor Relations and
Corporate CommunicationsSolid Biosciences
Inc.investors@solidbio.com
Media Contact:Glenn SilverFINN
Partnersglenn.silver@finnpartners.com
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