Solid Biosciences Granted FDA ODD For SGT-003

(RTTNews) - Solid Biosciences Inc. (SLDB) on Tuesday, announced that the FDA has granted Orphan Drug Designation (ODD) for its next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate, SGT-003.

This therapy uses a novel capsid and a muscle tropic vector delivering a microdystrophin that incorporates a neuronal Nitric Oxide Synthase (nNOS) binding domain.

According to Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences, these attributes have the potential to yield more potent transduction than historical approaches and a microdystrophin that may be able to more fully address muscle resiliency.

The company stated that it is currently seeking approvals from institutional review boards (IRB) at clinical trial sites for the planned Phase 1/2 clinical trial of SGT-003, with patient screening expected to commence shortly thereafter.

Patient dosing in the trial is expected to begin in the mid-to-late first quarter of 2024.