Phase |/||

Therapeutic Approach

Restoring or Replacing Dystrophin

SGT-001 is an adeno-associated virus (AAV) mediated gene transfer that contains an engineered version of the dystrophin gene (microdystrophin). The microdystrophin gene in SGT-001 has been designed to produce a functional form of dystrophin protein in skeletal and cardiac muscles.


A Phase 1/2 trial is actively recruiting.


This program is sponsored by Solid Biosciences.

Related Studies

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Media Library

MAR 2021

Webinar: Solid Biosciences Presents Safety & Efficacy Update

On March 16, 2021, Solid Biosciences joined PPMD for a webinar to provide a safety and efficacy update on the ongoing IGNITE DMD phase I/II clinical study of SGT-001 microdystrophin gene therapy in patients with Duchenne muscular dystrophy.
JUNE 2019

Solid BioSciences Presents at the PPMD 2019 Annual Conference

JUNE 2018

Solid BioSciences Presents at the PPMD 2018 Annual Conference

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