Solid Biosciences Inc. (NASDAQ:SLDB) today announced the presentation of clinical biomarker data from its SGT-001 microdystrophin gene therapy clinical trial for the treatment of Duchenne muscular dystrophy (Duchenne) on May 13 at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting, which is being held virtually. In addition, data from studies characterizing novel adeno-associated virus (AAV) vectors engineered for muscle gene delivery were presented in a poster session.
Abstract Number: 500
SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy. Oral presentation delivered by Patrick Gonzalez, PhD, Associate Director, R&D, Solid Biosciences.
Data presented from the ongoing IGNITE DMD clinical trial studying SGT-001 at a dose of 2E14 vg/kg were:
- SGT-001 administration results in dose-dependent, muscle wide microdystrophin expression in muscle biopsies collected 90 days post-SGT-001 administration.
- SGT-001-driven microdystrophin expression results in stabilization of dystrophin associated proteins.
- SGT-001-driven microdystrophin expression results in restored enzymatically active neuronal nitric oxide synthase (nNOS) at the sarcolemma.
Abstract Number: 558
Characterization of Novel AAV Vectors Engineered for Muscle Gene Delivery. Poster presentation and online Q&A delivered by Jennifer Green, PhD, Senior Scientist, R&D, Solid Biosciences.
This study utilized a rational design approach to generate a set of novel capsids predicted to have increased muscle tropism and transduction efficiency for the development of treatment for Duchenne. Key findings are:
- AAV-SLB101, a novel capsid, showed superior transduction efficiency in comparison to AAV9 in in vitro assays in both mouse and Duchenne human skeletal muscle cells.
- These in vitro results translated to increased biodistribution and microdystrophin protein expression in vivo in both quadriceps and heart, and decreased biodistribution to liver, in comparison to AAV9.
- An expanded panel of novel capsids identified two more candidates of interest, AAV-SLB102 and AAV-SLB111, that look similar to AAV-SLB101 in in vitro assays for binding, uptake and microdystrophin protein expression in C2C12 cells.
The presentation and poster are available on the Scientific Publications & Presentations page of the Solid Biosciences corporate website.