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Solid Biosciences Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Gene Therapy SGT-003
– Next-generation gene transfer therapy to treat Duchenne receives FDA Fast Track Designation – CHARLESTOWN, Mass., Dec. 07, 2023 (GLOBE NEWSWIRE) -- Solid
About this update from Solid Biosciences Inc.
[{"type":"text","content":"– Next-generation gene transfer therapy to treat Duchenne receives FDA Fast Track Designation – CHARLESTOWN, Mass., Dec. 07, 2023 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it 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 “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.” About SGT-003SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding a shortened form of the dystrophin protein (microdystrophin), containing the R16-R17 nNOS binding domain. Preclinical data suggests this may be important for both muscular function and durability of benefit in patients. About Fast Track DesignationThe Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate ...