Solid Biosciences to Present at the American Society of Gene and Cell Therapy Annual Meeting

2023-05-16

基因疗法临床研究

— Oral presentation highlighting data from multiple animal models demonstrated administration of AVB-202 resulted in frataxin (FXN) expression in cardiac and central nervous system (CNS) tissues with a favorable safety pro may improve cardiac function and extend survival — — Five additional abstracts relating to the company’s next-generation capsid activities, adeno-associated vector manufacturing methods and quality assurance assays to be presented as posters — CHARLESTOWN, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- 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 Presentation Session 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 Talk Session 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 Presentations Abstract 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 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 . 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 Monteiro VP, Investor Relations & Communications 617-821-4427 lmonteiro@solidbio.com Solid Biosciences Media Contact: Tim Palmer Associate Director, Corporate Communications 607-760-4223 Tim@solidbio.com