Solid Biosciences Receives FDA Fast Track for CPVT Gene Therapy

Solid Biosciences' investigational gene therapy SGT-501 has received FDA Fast Track designation for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare and life-threatening genetic heart condition affecting approximately 1 in 10,000 individuals globally. SGT-501 is an AAV-based therapy designed to deliver a functional CASQ2 gene to cardiac cells, aiming to correct the underlying calcium dysregulation that causes abnormal heart rhythms in CPVT patients. This designation, along with prior orphan drug and rare pediatric disease designations, accelerates development timelines and facilitates more frequent interactions with the FDA, potentially expediting approval and market entry. SGT-501 is the only gene therapy in clinical development targeting CPVT, offering a first-in-class approach compared to current symptom-managing treatments such as beta-blockers and implantable defibrillators. Solid Biosciences plans to initiate a Phase 1b clinical trial in the fourth quarter of 2025 to test the safety and efficacy of the therapy. The regulatory momentum and unique therapeutic potential position SGT-501 as a promising breakthrough for patients with this severe unmet medical need.