Solid Biosciences Inc.

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 their ability to stand up, walk, and climb stairs compared to a placebo. All participants will receive the ther…

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

This study tests a single intravenous dose of SGT-003, a gene therapy designed to help boys with Duchenne muscular dystrophy produce a shortened but functional version of the dystrophin protein. About 60 boys aged 0 to 18 will receive the treatment and be followed for 5 years. Th…

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. The trial will enro…