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SOLID Biosciences: Letter to the Duchenne Community

January 13, 2022

Defeat Duchenne Canada is pleased to share an update regarding the IGNITE-DMD Phase I/II clinical trial of SGT-001 from SOLID Biosciences Inc., as well as the progress in expanding their pipeline of treatments for Duchenne muscular dystrophy.

Earlier this week SOLID provided an update on its 2022 strategic priorities and other business initiatives at the Annual JP Morgan Healthcare Conference and has summarized the content of these updates into a letter for our Canadian Duchenne community:

About SGT-001 (Phase I/II)

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. SGT-001 is a systemically administered candidate that delivers microdystrophin to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including nNOS. Data from Solid’s clinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne.

SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, and Fast Track Designation in the United States and Orphan Drug Designations in both the United States and European Union.

The safety and efficacy of SGT-001 are currently being evaluated in IGNITE-DMD, a Phase I/II clinical trial. For more information on this clinical trial, please visit and look for the study ID number: NCT03368742.

About SGT-003 (Preclinical)

Solid’s SGT-003 is a rationally designed next generation adeno-associated virus (AAV) gene transfer therapy leveraging a novel, internally developed capsid with improved muscle-tropic properties. In preclinical studies, SGT-003 shows enhanced muscle tropism and microdystrophin expression. Biodistribution data show increased vector genomes in muscle and heart as well as decreased vector genomes in liver with SGT-003 compared to AAV9 in various in vitro and in vivo models. This biodistribution profile has the potential to increase efficiency and specifically target muscle cells, which could potentially allow for a reduced total viral load.