Solid Biosciences Announces FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy To Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)

Solid Biosciences Inc., which acquired UF startup AavantiBio, announced approval of its Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) and clinical trial application (CTA) by Health Canada for SGT-501, a novel gene therapy approach for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a highly malignant, arrhythmogenic channelopathy caused by genetic mutations that impact the ryanodine receptor (RYR2) in cardiac muscle. The Company expects to initiate a Phase 1b clinical trial to evaluate the safety, tolerability, and efficacy of SGT-501 in the fourth quarter of 2025.

Gabriel Brooks, M.D., Chief Medical Officer of Solid, said: “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. SGT-501 offers a precision genetic approach targeting the underlying pathophysiology of the disease: abnormal calcium releases from the sarcoplasmic reticulum in an otherwise structurally sound heart. 

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