Solid Biosciences Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

– Patient dosing expected to begin mid-to-late Q1 2024 –

CHARLESTOWN, Mass., Jan. 16, 2024 (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 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.”

About SGT-003

SGT-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 Orphan Drug Designation

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended to treat a rare disease or condition that affects fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including specified financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

About DMD

Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in the United States alone.

About Solid Biosciences

Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates and neuromuscular and cardiac programs, including SGT-003, for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, AVB-202-TT for the treatment of Friedreich’s ataxia, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. 

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