Solid Biosciences Presents Data from Duchenne Muscular Dystrophy Gene Therapy Program at ASGCT 23rd Annual Meeting
Virtual oral presentation reviews interim clinical biomarker data for SGT-001 and provides data on expression and co-localization of microdystrophin and dystrophin associated proteins in patients in the IGNITE DMD trial
Virtual poster presentation provides data characterizing novel adeno-associated virus (AAV) vectors engineered for muscle gene delivery
Abstract Number: 500
SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy. Oral presentation delivered by
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
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
Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy (Duchenne). Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.
SGT-001 is based on pioneering research in dystrophin biology by Dr.
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 IGNITE DMD clinical trial, the safety or potential efficacy of SGT-001 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. 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
Source: Solid Biosciences Inc.