Solid Biosciences’ SGT-501 AAV Gene Therapy Cleared for Clinical Trials!

CHARLESTOWN, Mass., July 08, 2025 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (Nasdaq: SLDB) announced a significant advancement for its lead cardiac program! The U.S. Food and Drug Administration (FDA) has approved Solid’s Investigational New Drug (IND) application, alongside a Clinical Trial Application (CTA) approval from Health Canada for SGT-501.

SGT-501 is a novel, AAV-based gene therapy candidate aimed at treating Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). CPVT is a highly malignant, arrhythmogenic channelopathy caused by genetic mutations impacting the ryanodine receptor (RYR2) in cardiac muscle. This underserved and often fatal cardiac disease currently lacks FDA-approved therapies, making SGT-501 a critical development.

SGT-501 is designed to deliver a functional, full-length, codon-optimized copy of the human cardiac calsequestrin (CASQ2) gene directly to heart muscle cells. This precision genetic approach works by increasing CASQ2 protein levels, which enhances the buffering of free calcium in the sarcoplasmic reticulum. This action stabilizes the RYR2, reducing diastolic calcium leak into the cytosol and supporting the maintenance of normal cardiac rhythm to protect against ventricular tachycardia.

“Despite being identified nearly 50 years ago, CPVT still lacks FDA-approved therapies – this announcement reflects a critical development in the treatment of this underserved, often fatal, cardiac disease,” said Gabriel Brooks, M.D., Chief Medical Officer of Solid. “SGT-501 offers a precision genetic approach targeting the underlying pathophysiology. We believe SGT-501 has the unique potential to provide durable protection and may be capable of liberating patients from the ever-present threat of lethal arrhythmias and life-limiting prohibitions on exercise.”

SGT-501 previously received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. FDA, underscoring its potential as a first-in-class therapy. The AAV-CASQ2 gene therapy approach was pioneered by Dr. Silvia Priori and her lab in Italy, with Solid Biosciences in-licensing this innovative therapeutic in 2023.

Solid Biosciences expects to initiate a Phase 1b clinical trial to evaluate the safety, tolerability, and efficacy of SGT-501 in the fourth quarter of 2025. This represents a pivotal step towards bringing a potentially life-changing medicine to individuals living with CPVT.