Solid Biosciences Inc. (Nasdaq: NASDAQ:SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular dystrophy (Duchenne) gene therapy candidate.
“Obtaining ODD status for SGT-003, along with Fast Track Designation granted last month, furthers our efforts to meet the ongoing challenge of treating this devastating disease as expeditiously as possible,” said Bo Cumbo, President and Chief Executive Officer at Solid Biosciences. “These designations are important milestones for Solid, supporting the continued development of next-generation therapies for Duchenne.”
The company is currently in the process of securing approvals from the institutional review boards (IRB) at the clinical trial sites for the planned Phase 1/2 clinical trial of SGT-003 and expects to commence patient screening shortly thereafter. Patient dosing in the trial is expected to commence in mid-to-late first quarter of 2024.
“SGT-003 therapy stands out among other Duchenne gene therapy candidates by leveraging a novel capsid and a muscle tropic vector delivering a microdystrophin that incorporates a neuronal Nitric Oxide Synthase (nNOS) binding domain. These attributes among others, have the potential to yield both more potent transduction than historical approaches, and a microdystrophin that may be able to more fully address muscle resiliency,” said Gabriel Brooks, M.D., Chief Medical Officer at Solid Biosciences. “Obtaining ODD status is an exciting development that we believe will aid our efforts to bring advanced treatment options to those patients affected by Duchenne.”