Solid Biosciences Inc., a clinical-stage company focused on genetic medicines for neuromuscular and cardiac diseases, announced it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its investigational gene therapy SGT-501. This novel AAV-based therapy targets catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare and life-threatening heart rhythm disorder.
SGT-501 delivers a functional, full-length copy of the cardiac calsequestrin (CASQ2) gene directly to heart muscle cells. This approach aims to correct the calcium regulation and ryanodine receptor instability that cause the abnormal heart rhythms in CPVT, potentially restoring normal cardiac function during diastole.
The FDA’s Fast Track designation is granted to treatments addressing serious conditions with unmet medical needs, offering benefits such as increased communication with regulators and eligibility for priority review. This designation underscores the significant promise of SGT-501 as a potential first therapy for CPVT, which currently has no approved treatments.
Jessie Hanrahan, Ph.D., Chief Regulatory & Preclinical Operations Officer at Solid Biosciences, said the Fast Track status and recent FDA Investigational New Drug (IND) clearance validate the company’s efforts and highlight the urgent need for CPVT therapies. She also noted that the upcoming Phase 1b clinical trial is expected to begin in Q4 2025, marking a key milestone in bringing SGT-501 to patients.
The Phase 1b trial will be the first human study to assess the safety, tolerability, and efficacy of SGT-501 in a multicenter, open-label setting. Solid Biosciences also recently received regulatory clearance from Health Canada, signaling progress toward global development of the therapy.