Solid Biosciences Secures FDA Fast Track Designation for Gene Therapy Candidate SGT-501 Targeting CPVT
Thursday, July 24, 2025
Solid Biosciences has received Fast Track designation from the US Food and Drug Administration (FDA) for SGT-501, its investigational gene therapy for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT). This rare genetic heart condition is characterised by irregular heart rhythms triggered by physical activity or emotional stress, which may lead to fainting, seizures, or sudden cardiac death.
SGT-501 is an adeno-associated virus (AAV)-based therapy designed to deliver a functional, full-length, codon-optimised copy of the human cardiac calsequestrin (CASQ2) gene directly to cardiac muscle. This approach aims to correct the underlying causes of CPVT by stabilising the ryanodine receptor (RYR2) and improving calcium regulation in heart cells—key factors in maintaining a normal heartbeat.
The therapy has already received FDA clearance for an Investigational New Drug (IND) application and Health Canada approval for a Clinical Trial Application (CTA). A Phase 1b clinical trial is scheduled to begin in the fourth quarter of 2025. This first-in-human, open-label, multicentre study will assess the safety, tolerability and preliminary efficacy of SGT-501.
The FDA’s Fast Track designation is intended to accelerate the development of new treatments for serious or life-threatening conditions with unmet medical needs. SGT-501 will benefit from enhanced regulatory communication and may also be eligible for priority review in future submissions.
SGT-501 is the third investigational therapy from Solid Biosciences to gain IND clearance in the past two years. It has also received Orphan Drug and Rare Paediatric Disease designations from the FDA, further highlighting its potential as a first-in-class therapy for CPVT.
The AAV-CASQ2 approach used in SGT-501 was originally developed by Dr Silvia Priori and her team at IRCCS Maugeri Pavia in Italy. Solid Biosciences acquired the rights to the therapy in 2023 and progressed it through preclinical development.
CPVT is estimated to affect around 1 in 10,000 people globally and is often diagnosed in children and young adults. The condition is most commonly linked to mutations in the RYR2 and CASQ2 genes, which lead to impaired calcium handling in the heart. There are currently no approved therapies that address the root cause of the disease.
Source: globenewswire.com
