Solid Biosciences, a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.
“Receipt of FDA Fast Track Designation underscores the importance of rapidly developing SGT-003 to potentially aid the unmet needs of the Duchenne community,” said Bo Cumbo, President and CEO at Solid Biosciences. “Having received IND clearance for SGT-003 last month, we are pleased to be expediting the development of a potentially life-changing therapy and look forward to continuing to work closely with the FDA.”
IND clearance by the FDA for SGT-003 was received in November 2023. The planned Phase 1/2 trial, SGT-003-101, is a first-in-human, open-label, multicenter trial to determine the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. SGT-003 will be administered as a one-time intravenous infusion to patients in two cohorts with a minimum of three patients each, with the potential for cohort expansion. Cohort 1 will study patients aged 4 to < 6 years of age with DMD. Long-term safety and efficacy will be evaluated for a total of 5 years following treatment.
“We appreciate the FDA’s partnership and their recognition of the continuing unmet need in the DMD community,” said Jessie Hanrahan, Ph.D., Chief Regulatory Officer at Solid Biosciences. “We look forward to frequent engagements with the Agency to discuss development of SGT-003 and ensure that our clinical development program will appropriately collect the data needed to support review of SGT-003 for future marketing authorization.”