Solid Biosciences to Present at ASGCT Annual Meeting

Solid Biosciences Inc., a biotech firm committed to developing precision genetic medicines for neuromuscular and cardiac diseases, is set to present significant findings at the upcoming American Society of Gene and Cell Therapy (ASGCT) 2024 Annual Meeting in Baltimore from May 7-11. The company will share insights through an oral presentation and six posters, focusing on advancements in adeno-associated vector (AAV) manufacturing, capsid development, and non-clinical data relating to the leading SGT-003 program.

Bo Cumbo, President and CEO of Solid Biosciences, expressed enthusiasm about presenting their latest research. Cumbo highlighted that these presentations demonstrate the company's dedication to innovative therapies addressing unmet medical needs in neuromuscular and cardiac diseases. He emphasized the importance of engaging with the gene therapy community to advance the field collectively.

The presentations will cover developments in gene therapy manufacturing, including new methods to enhance AAV production and refine purification processes. There will also be discussions on innovative approaches in vector biology, such as in silico modeling for AAV vector design and capsid modifications aimed at improving targeting of cardiac tissue. Key findings from non-clinical studies of SGT-003 will be shared, showcasing microdystrophin expression and functional efficacy in mouse models.

Key areas of Solid Biosciences' research and development initiatives will be featured in the following presentations:

Oral Presentation:
- A6 - AAV Vectors - Product Development Manufacturing and Approval Considerations
- Topic: Novel Mechanism to Increase AAV Yield Through Blocking AAV Transduction of Manufacturing HEK293 Cells During AAV Production
- Lead Author: Xiaofei E
- Abstract #28
- Scheduled: May 7, 2:15-2:30pm ET

Poster Presentations:
1. Process Development & Manufacturing:
- High-Throughput Workflows to Accelerate Development of Chromatographic Purification of rAAV Viral Vectors
- Lead Author: Ryan DeGroot
- Abstract 1543
- Scheduled: 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
- Scheduled: May 10, 12:00-1:30pm ET

- Increasing Quality and Productivity with Dual Transfection (DT) for AAV Production
- Lead Author: Sarath Mandava
- Abstract 1541
- Scheduled: May 10, 12:00-1:30pm ET

2. Vector Biology:
- Engineered Cardioskeletal-Directed AAV Capsids That Detarget the Liver
- Lead Author: Widler Casy
- Abstract 480
- Scheduled: May 8, 12:00-1:30pm ET

- Designing Therapeutic Recombinant AAV Vectors Using In Silico Vector Modeling
- Lead Author: Ethan Waple
- Abstract 466
- Scheduled: May 8, 12:00-1:30pm ET

3. 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
- Scheduled: May 9, 5:30-7pm ET

Solid Biosciences is a life sciences company focused on developing gene therapy candidates to treat severe neuromuscular and cardiac diseases. Their pipeline includes SGT-003 for Duchenne muscular dystrophy, SGT-501 for catecholaminergic polymorphic ventricular tachycardia, AVB-401 for BAG3-mediated dilated cardiomyopathy, and other assets targeting fatal cardiac conditions. The company aims to improve patients' daily lives by bringing together expertise in science, technology, disease management, and care, driven by a patient-focused mission initiated by those directly affected by these diseases.