Sarepta Therapeutics, Inc. announced the submission of its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT, to Medsafe, the New Zealand Medicines and Medical Devices Safety Authority. Pending approval, Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026. SRP-1005 is an investigational small interfering RNA (siRNA) therapeutic for the treatment of Huntington's Disease.
Symptoms typically appear between ages 30 and 50 and worsen over time, with every child of an affected parent having a 50% chance of inheriting the condition. There is currently no known cure or approved disease-modifying treatments that address the underlying cause. Sarepta's next-generation siRNA platform is focused on chronically administered therapies for neurodegenerative and pulmonary diseases and includes investigational treatments for: Facioscapulohumeral muscular dystrophy (FSHD); Myotonic dystrophy type 1 (DM1); Spinocerebellar ataxia type 2 (SCA2); Idiopathic Pulmonary Fibrosis (IPF); Huntington's disease (HD).
Sarepta is also pursuing preclinical programs for Spinocerebellar Ataxia type 1 (SCA1) and Spinocerebellarataxia type 3 (SCA3) and has an exclusive collaboration with Arrowhead Pharmaceuticals to develop therapies forletal muscle diseases, with plans to pursue up to six discovery targets in muscle or central nervous system disorders.
