Core Insights - Solid Biosciences has announced the approval of its Investigational New Drug (IND) application for SGT-501, a novel gene therapy for catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare and life-threatening cardiac condition [1][2][3] - SGT-501 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, indicating its potential as a first-in-class therapy [1][3] - The company plans to initiate a Phase 1b clinical trial for SGT-501 in the fourth quarter of 2025, expanding its clinical pipeline to address urgent unmet medical needs in cardiac diseases [1][2] Company Overview - Solid Biosciences is focused on developing precision genetic medicines for neuromuscular and cardiac diseases, including Duchenne muscular dystrophy and CPVT [10] - The company aims to improve the lives of patients with rare diseases through innovative gene therapy approaches [10] Scientific Background - SGT-501 is designed to deliver a functional copy of the human cardiac calsequestrin (CASQ2) gene to heart muscle cells, which may stabilize the ryanodine receptor (RYR2) and reduce the risk of ventricular tachycardia [3][4] - The therapy is based on research conducted by Dr. Silvia Priori and the IRCCS ICS Maugeri lab, which demonstrated the therapeutic potential of calsequestrin overexpression in CPVT models [4][5] Disease Context - CPVT is characterized by abnormal heart rhythms triggered by stress or physical activity, leading to severe outcomes such as fainting, seizures, and sudden death [5][6] - It is estimated to affect approximately 1 in 10,000 individuals globally, often misdiagnosed, and currently lacks approved treatments that address its underlying mechanisms [5][6]
Solid Biosciences Announces FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy to Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)