Solid Biosciences announced today that they are working with the FDA to resolve questions related the to clinical hold placed on the IGNITE DMD trial in Duchenne muscular dystrophy patients. The trial was placed on full clinical hold after the first patient dosed with SGT-001 experienced a serious adverse event. The company reported that the treating physician of the reported that the patient is doing well.
Solid Biosciences also reported that they have successfully resolved the partial clinical hold the FDA placed on planned high dose of SGT-001 in IGNITE DMD. The partial hold was related to manufacturing-related questions from the FDA.
SGT-001 is a microdystrophin gene transfer therapy. It is administered via adeno-associated virus and has been shown to stabilize the dystrophin glycoprotein complex and rescue muscle function.