Solid Biosciences Inc.

This study is testing a single intravenous dose of an experimental gene therapy called SGT-003 in boys with Duchenne muscular dystrophy (DMD). The main goals are to see if the treatment is safe and if it helps produce a needed muscle protein (microdystrophin) and improves muscle …

This is the first human study testing SGT-212, an experimental gene therapy for Friedreich's ataxia, a rare inherited neurological condition. The trial will enroll 10 participants to determine safe dosing levels and monitor side effects when the therapy is delivered both into a s…

This study is testing whether a single intravenous dose of an experimental gene therapy called SGT-003 can help boys with Duchenne muscular dystrophy (DMD) walk and move better. It will involve 80 boys who can still walk, comparing those who get the therapy to those who get a pla…

This is an early safety study testing a single dose of an experimental gene therapy called SGT-501 in people with CPVT, a rare inherited condition that causes dangerous fast heart rhythms during exercise or stress. The main goal is to see if the treatment is safe and tolerable in…