Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences
company developing genetic medicines for neuromuscular and cardiac
diseases, will present positive preclinical data from studies of
AVB-202, the company’s gene therapy candidate for the treatment of
Friedreich’s Ataxia (FA). Grace Pavlath, Ph.D., Vice President,
Research at Solid, will present the data in an oral session at 4:45
pm PDT on May 18 at the American Society of Gene and Cell Therapy
(ASGCT) 2023 Annual Meeting, Los Angeles, May 16-20.
Key highlights from the AVB-202 presentation at
ASGCT include:
- AVB-202 rescued cardiac function
and extended survival in a mouse model of FA. Data from studies in
this model demonstrated dose-dependent increases in FXN protein
expression.
- Results of a six-month safety study
in non-human primates (NHPs) using dual routes of administration
(intravenous [IV] injection to target cardiac muscle and
intrathecal [IT] injection to target cells in the CNS) demonstrated
a favorable safety profile.
- NHP studies also showed that dual
IV/IT administration of AVB-202 led to robust FXN protein
expression in target tissues, including the heart and CNS.
In addition to Dr. Pavlath’s oral presentation
on AVB-202, Solid will present five abstracts relating to the
company’s AAV manufacturing methods and quality assurance assays.
Brian Collins, Ph.D., Senior Vice President at Solid, also will
deliver an invited talk in a session on Comparability Challenges
for Chemistry, Manufacturing and Controls (CMC).
ASGCT Presentation
Information
Oral PresentationSession Title: Neurological
Gene Therapies in Advanced Stages of Clinical Translation
Abstract Title: Efficacy and Safety of a Novel
FXN Gene Therapy (AVB-202-TT) for the Treatment of Friedreich's
Ataxia (Abstract #169)
Presentation Information: May 18, 4:45pm PDT,
Petree Hall D
Invited TalkSession Title: Comparability
Challenges for CMC
Presentation Title: Case Study on Comparability
Issues for AAV Products: Solid Biosciences’ Experience
Presentation Information: May 16, 1:30pm-3:15pm
PDT, Petree Hall D
- Dr. Collins’ presentation — 2:30pm
– 2:50pm PDT
- Panel Q&A — 2:50 pm – 3:15pm
PDT
Poster PresentationsAbstract Title: Development
and Qualification of a Multiplexed ddPCR Assay to Evaluate DNA
Integrity (Abstract #430)
Presentation Information: May 17, 12:00pm –
2:00pm PDT
Abstract Title: Genomic Characterization of AAV
Products Using Multiplex ddPCR and Nanopore Sequencing (Abstract
#826)
Presentation Information: May 18, 12:00pm –
2:00pm PDT
Abstract Title: Characterization of Factors That
Influence the Yield and Quality of AAV Produced Using HSV
Co-Infection (Abstract #1375)
Presentation Information: May 19, 12:00pm –
2:00pm PDT
Abstract Title: Novel AAV Capsid Identification
and Characterization for Neuromuscular and Cardiac Indications
(Abstract #1549)
Presentation Information: May 19, 12:00pm –
2:00pm PDT
Abstract Title: Characterization of Genomic
Heterogeneity in rAAV Preparations Using Short- and Long-Read Next
Generation Sequencing (Abstract #1287)
Presentation Information: May 19, 12:00pm –
2:00pm PDT
About Solid Biosciences
Solid Biosciences is a life science company
focused on advancing a portfolio of neuromuscular and cardiac
programs, including SGT-003, a differentiated gene transfer
candidate for the treatment of Duchenne, AVB-202-TT, a gene
transfer candidate for the treatment of Friedreich’s Ataxia,
AVB-401 for BAG3 mediated dilated cardiomyopathy, and additional
assets for the treatment of undisclosed cardiac diseases. Solid
aims to be the center of excellence across a given disease spectrum
bringing together those with expertise 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 Statements
This press release contains “forward-looking
statements” within the meaning of the Private Securities Litigation
Reform Act of 1995, including statements regarding the company’s
plans to present positive preclinical data from studies of AVB-202
and additional abstracts relating to the company’s adeno-associated
vector manufacturing methods and quality assurance assays; 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 ability to recognize the anticipated benefits
of Solid’s acquisition of AavantiBio; the company’s ability to
advance SGT-003, AVB-202-TT, AVB-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 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, AVB-202-TT, AVB-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:Leah
MonteiroVP, Investor Relations &
Communications617-821-4427lmonteiro@solidbio.com
Solid Biosciences Media Contact:Tim
PalmerAssociate Director, Corporate
Communications607-760-4223Tim@solidbio.com
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