Solid Biosciences Inc. a clinical stage life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for SGT-501, the Company’s novel, AAV-based investigational gene therapy for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT). SGT-501 will deliver a functional, full-length, codon-optimized copy of the human cardiac calsequestrin (CASQ2) gene to cardiac muscle cells, an approach intended to address the underlying ryanodine receptor (RYR2) instability and calcium dysregulation seen in CPVT, thereby normalizing cardiac rhythm in diastole.

Fast Track designation is granted to products that are developed to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. This designation is intended to facilitate development and expedite review of qualifying drugs. SGT-501 will benefit from this designation through more frequent interactions with the FDA along with the potential to be eligible for priority review.

Jessie Hanrahan, Ph.D., Chief Regulatory & Preclinical Operations Officer of Solid Biosciences, commented: “FDA IND clearance and Fast Track designation provide important scientific validation of SGT-501 and recognition of the continuing and severe unmet needs posed by CPVT. We believe SGT-501’s robust preclinical and CMC data package supports the potential for a durable and transformational treatment for a disease that has no FDA-approved therapies. SGT-501 is our third IND clearance in the past two years, demonstrating Solid’s deep regulatory expertise in the genetic medicines space. We want to thank the FDA for their support and partnership and look forward to continued collaboration with regulators globally as we advance our cardiac and neuromuscular candidates.”

FDA Investigational New Drug (IND) clearance and Health Canada Clinical Trial Application (CTA) approval for SGT-501 were announced on July 8, 2025. The planned Phase 1b trial will be a first-in-human, open-label, multicenter study to evaluate the safety, tolerability and efficacy of SGT-501 and is expected to initiate in the fourth quarter of 2025.

The FDA’s Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.

SGT-501 is an AAV-based gene therapy candidate designed to deliver a functional, full length, codon-optimized copy of the human cardiac calsequestrin (CASQ2) gene to heart muscle cells. In the context of RYR2 variants, increasing CASQ2 protein levels enhances buffering of free calcium in the sarcoplasmic reticulum, stabilizing the RYR2, which results in reduced diastolic calcium leak into the cytosol. Stabilization of the RYR2 in its closed conformation supports the maintenance of normal cardiac rhythm with the potential to protect against ventricular tachycardia. SGT-501 received Fast Track, Orphan Drug and Rare Pediatric Disease designations from the U.S. FDA and has potential as a first-in-class therapy to correct the underlying RYR2 instability and calcium dysregulation causes of CPVT.

Source: globenewswire.com