Solid Biosciences’ AAV Gene Therapy SGT-501 Granted FDA Fast Track for CPVT

CHARLESTOWN, Mass., July 23, 2025 /GLOBE NEWSWIRE/ – Solid Biosciences announced yesterday that its investigational AAV-based gene therapy, SGT-501, received Fast Track designation from the FDA for catecholaminergic polymorphic ventricular tachycardia (CPVT).

CPVT is a rare, severe heart condition affecting approximately 1 in 10,000, caused by RYR2 and CASQ2 gene mutations disrupting calcium regulation in heart muscle cells. SGT-501 is designed to deliver a functional CASQ2 gene to stabilize RYR2 and reduce calcium leak, addressing the disease’s underlying cause.

This Fast Track status follows FDA IND approval (July 8, 2025) and previous Orphan Drug and Rare Pediatric Disease designations. SGT-501 is poised to be a first-in-class therapy for CPVT, a condition with no FDA-approved treatments.

“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,” said Jessie Hanrahan, PhD, Preclinical Operations Officer at Solid Biosciences.

Gabriel Brooks, MD, Chief Medical Officer, added, “SGT-501 offers a precision genetic approach targeting the underlying pathophysiology of this underserved, often fatal, cardiac disease.”