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

2024-05-07

基因疗法临床研究细胞疗法ASH会议

— Presentations to highlight AAV manufacturing and purification improvements, vector biology updates,

and a comprehensive non-clinical data overview of the company’s lead SGT-003 program —

CHARLESTOWN, Mass., May 07, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present an oral presentation and six posters and at the American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting, Baltimore, May 7-11.

The data presentations will focus on Solid Biosciences’ research and manufacturing capabilities aimed at advancing the field of gene therapy. They will cover various aspects of adeno-associated vector (AAV) manufacturing, novel capsid development, and non-clinical studies with a particular emphasis on the company's lead SGT-003 program.

"We are excited to share our latest scientific findings and advancements at the ASGCT Annual Meeting," said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences. "Our presentations underscore our commitment to developing innovative therapies that address unmet medical needs in neuromuscular and cardiac diseases. We look forward to engaging with the gene therapy community and advancing the field together."

The presentations will feature advancements in gene therapy manufacturing, featuring novel mechanisms to enhance AAV production and refine purification methods. Additionally, innovative approaches in vector biology will be presented, leveraging in silico modeling for AAV vector design and showcasing capsid modifications for improved cardiac tissue targeting. Highlights from non-clinical studies of SGT-003 also will be shared, including findings on microdystrophin expression and functional efficacy in mouse models.

The presentations will cover key areas of Solid Biosciences' research and development initiatives, including:

Oral Presentation

A6 - AAV Vectors - Product Development Manufacturing and Approval Considerations

Novel Mechanism to Increase AAV Yield through Blocking AAV Transduction of Manufacturing HEK293 Cells During AAV Production

Lead Author: Xiaofei E

Abstract #28

May 7, 2:15-2:30pm ET

Poster Presentations

Process Development & Manufacturing

High-Throughput Workflows to Accelerate Development of Chromatographic Purification of rAAV Viral Vectors

Lead Author: Ryan DeGroot

Abstract 1543

May 10, 12:00-1:30pm ET

Identification of an AAV Affinity Chromatography Elution Buffer that Maximizes Product Recovery and Minimizes Product Degradation

Lead Author: Sierra VanSuch

Abstract 1517

May 10, 12:00-1:30pm ET

Increasing Quality and Productivity with Dual Transfection (DT) for AAV Production

Lead Author: Sarath Mandava

Abstract 1541

May 10, 12:00-1:30pm ET

Vector Biology

Engineered Cardioskeletal-Directed AAV Capsids That Detarget the Liver

Lead Author: Widler Casy

Abstract 480

May 8, 12:00-1:30pm ET

Designing Therapeutic Recombinant AAV Vectors Using In Silico Vector Modeling

Lead Author: Ethan Waple

Abstract 466

May 8, 12:00-1:30pm ET

Non-Clinical Studies

Systemic Delivery of SGT-003 Microdystrophin Gene Therapy Using the Novel Capsid AAV-SLB101 Ameliorates Muscle Pathology and Rescues Muscle Function in the mdx Mouse Model of Duchenne Muscular Dystrophy

Lead Author: Jamie Marshall

Abstract 1391

May 9, 5:30-7pm ET

About Solid Biosciences

Solid Biosciences is a life sciences 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), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy 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 Statements

This 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 company’s planned oral and poster presentations; 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, SGT-501, 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, SGT-501, 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.

Media Contact:

Glenn Silver

FINN Partners

glenn.silver@finnpartners.com