SGT-501 / Solid BioSci - LARVOL DELTA

A novel AAV gene therapy strategy to correct calcium dysregulation in catecholaminergic polymorphic ventricular tachycardia (ASGCT 2026) - "Upon beta-adrenergic stimulation (2mg/kg epinephrine and 120mg/kg caffeine), a significant decrease in ventricular tachycardia (VT) incidence was observed at the mid dose (4/25, 16%) and high dose (1/28, 4%) compared to vehicle (13/25, 52%), with cardiac CASQ2 protein levels measured at 1.6- fold and 6-fold above vehicle levels, respectively. Conclusion A single dose of SGT-501 effectively corrected electrophysiological abnormalities in a CPVT mouse model and was well-tolerated at all doses in NHPs. These encouraging results provide the foundation for the ARTEMIS Phase 1b clinical trial, which will further evaluate the therapy’s safety and potential for patients with CPVT."

Gene therapy • Cardiovascular • Gene Therapies • Ventricular Tachycardia • RYR2

ARTEMIS: A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Solid Biosciences Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiovascular • Gene Therapies • Ventricular Tachycardia