Solid Biosciences Receives FDA and Health Canada Approval for First-in-Class Gene Therapy Targeting Fatal Heart Rhythm Disorder
- Solid Biosciences has received FDA IND and Health Canada CTA approval for SGT-501, a novel gene therapy designed to treat catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare genetic heart condition that can cause sudden death.
- SGT-501 represents the first potential therapy to address the underlying mechanisms of CPVT by delivering a functional copy of the CASQ2 gene to stabilize calcium regulation in heart muscle cells.
- The company plans to initiate a Phase 1b clinical trial in the fourth quarter of 2025, marking a significant milestone for patients with this life-threatening condition that currently has no approved treatments.
- CPVT affects approximately 1 in 10,000 individuals globally and is often triggered by physical activity or emotional stress, leading to dangerous arrhythmias in otherwise healthy hearts.
Solid Biosciences Inc.
Posted 5/6/2024