Solid Biosciences Highlights Expertise in AAV Gene Therapy with Multiple Abstracts to be Presented at the American Society of...
May 04 2021 - 8:00AM
Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company
focused on advancing meaningful therapies for Duchenne muscular
dystrophy (Duchenne), today announced the presentation of three
abstracts at the upcoming American Society of Gene & Cell
Therapy (ASGCT) 24th Annual Meeting. The abstracts report data from
research-stage studies of novel adeno-associated viral (AAV)
vectors for muscle gene delivery and mechanisms of immune responses
to AAV9, as well as 12-month data from the ongoing IGNITE DMD trial
Phase I/II study of SGT-001 microdystrophin gene therapy in
patients with Duchenne, which were previously presented at the
Muscular Dystrophy Association Virtual Clinical & Scientific
Conference in March 2021. The ASGCT Annual Meeting is being held
virtually May 11-14, 2021.
Presentation
DetailsTitle: AAV9 Capsid-Anti-AAV9
Antibody Immune Complexes Promote Complement Activation and
Cytokine Release In VitroPresenter: Qian Chen,
Senior Director, R&D, Solid BiosciencesPresentation
Type: Digital posterAbstract Number:
747
Title: Continued In Vitro and
In Vivo Characterization of Novel AAV Vectors Engineered for Muscle
Gene DeliveryPresenter: Jennifer Green, PhD,
Principal Scientist, R&D, Solid BiosciencesPresentation
Type: Digital posterAbstract Number:
319
Title: IGNITE-DMD: Phase I/II
Study of Single SGT-001 Microdystrophin Gene Therapy for
DMDPresenter: Carl Morris, PhD, Chief Scientific
Officer, Solid BiosciencesPresentation Date &
Time: Friday, May 14, 2021, 1:45-2:00 PM
ETPresentation Type: OralAbstract
Number: 263
Abstracts for the presentations can be viewed
online at: https://annualmeeting.asgct.org/
About SGT-001Solid’s SGT-001 is
a novel adeno-associated viral (AAV) vector-mediated gene transfer
therapy designed to address the underlying genetic cause of
Duchenne. Duchenne is caused by mutations in the dystrophin gene
that result in the absence or near absence of dystrophin protein.
SGT-001 is a systemically administered candidate that delivers a
synthetic dystrophin gene, called microdystrophin, to the body.
This microdystrophin encodes for a functional protein surrogate
that is expressed in muscles and stabilizes essential associated
proteins, including neuronal nitric oxide synthase (nNOS). Data
from Solid’s clinical program suggests that SGT-001 has the
potential to slow or stop the progression of Duchenne, regardless
of genetic mutation or disease stage.
SGT-001 is based on pioneering research in
dystrophin biology by Dr. Jeffrey Chamberlain of
the University of Washington and Dr. Dongsheng
Duan of the University of Missouri. SGT-001 has been
granted Rare Pediatric Disease Designation, or RPDD, and Fast Track
Designation in the United States and Orphan Drug
Designations in both the United States and European
Union.
About Solid BiosciencesSolid
Biosciences is a life sciences company focused on advancing
transformative treatments to improve the lives of patients living
with Duchenne. Disease-focused and founded by a family directly
impacted by Duchenne, our mandate is simple yet comprehensive –
work to address the disease at its core by correcting the
underlying mutation that causes Duchenne with our lead gene therapy
candidate, SGT-001. 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 the ability of the Company to
continue dosing patients in the IGNITE DMD trial, the implication
of interim clinical data, the safety or potential treatment
benefits of SGT-001 in patients with DMD, the Company’s
expectations for reporting future data from the IGNITE DMD trial,
the Company’s regulatory plans and timelines 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 or continue IGNITE DMD on the
timeline expected or at all; obtain and maintain necessary
approvals from the FDA and other regulatory authorities; obtain and
maintain the necessary approvals from investigational review boards
at IGNITE DMD clinical trial sites and the IGNITE DMD independent
data safety monitoring board; enroll patients in IGNITE DMD on the
timeline expected; the Company’s dosing strategy; replicate in
clinical trials positive results found in preclinical studies and
earlier stages of clinical development; whether the interim data
presented in this release will be predicative of the final results
of the trial or will demonstrate a safe or effective treatment
benefit of SGT-001; whether the methodologies, assumptions and
applications we utilize to assess particular safety or efficacy
parameters will yield meaningful statistical results; advance the
development of its product candidates under the timelines it
anticipates in current and future clinical trials; successfully
optimize and scale its manufacturing process; obtain, maintain or
protect intellectual property rights related to its product
candidates; compete successfully with other companies that are
seeking to develop Duchenne treatments and gene therapies; manage
expenses; and raise the substantial additional capital needed, on
the timeline necessary, to continue development of SGT-001, 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.
Investor Contact:David CareyFINN
Partners212-867-1768David.Carey@finnpartners.com
Media Contact:Erich SandovalFINN
Partners917-497-2867Erich.Sandoval@finnpartners.com
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