Solid Bio First Gene Therapy for Fatal Heart Rhythm Disorder Advances to Clinical Trials

Solid Biosciences (NASDAQ:SLDB) has received both FDA IND and Health Canada CTA approvals for SGT-501, their first-in-class cardiac gene therapy designed to treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). The therapy has secured Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.

CPVT is a rare, life-threatening genetic heart condition with no currently approved therapies. The company plans to initiate a Phase 1b clinical trial in Q4 2025 to evaluate SGT-501's safety, tolerability, and efficacy. The therapy targets the underlying disease mechanism by addressing abnormal calcium releases from the sarcoplasmic reticulum in the heart.

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