Solid Biosciences Inc.

This early-stage trial tests a new gene therapy called SGT-212 in 10 people with Friedreich's ataxia, a rare genetic disorder that affects movement and coordination. The treatment is given both as an injection into the brain and through an IV. The main goal is to see if it is saf…

This study tests a single intravenous dose of SGT-003, a gene therapy designed to help boys with Duchenne muscular dystrophy produce a shortened version of the dystrophin protein. About 60 males from infancy to under 18 years old will take part. Researchers will monitor safety an…

This study tests a single intravenous dose of SGT-003, a gene therapy, in 80 boys with Duchenne muscular dystrophy who can still walk. The goal is to see if it improves muscle function, like how fast they can stand up or climb stairs. Participants will receive either the therapy …

This early-stage study tests the safety of a single dose of SGT-501, a gene therapy for people with catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare inherited heart condition that can cause dangerous heart rhythms during exercise or stress. Up to 18 adults and…