Sarepta Therapeutics Gets New Zealand Approval For Its Clinical Study Application For SRP-1005-101

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that Medsafe, the New Zealand Medicines and Medical Devices Safety Authority, has granted approval for its clinical trial application (CTA) for Study SRP-1005-101, also known as INSIGHTT. 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.

INSIGHTT is a Phase 1, multi-center, dose escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants. SRP-1005 leverages an advanced TfR1 (transferrin receptor protein 1) approach that uses monovalent fragment antigen binding (fAb) designed for efficient delivery to the central nervous system. Subcutaneous administration is intended to avoid saturating the transferrin receptor and achieve constant and robust penetration across the blood brain barrier. Preclinical data with SRP-1005 have demonstrated the potential for significant protein knockdown in key deep brain regions, including the putamen and caudate, as well as the temporal and frontal cortexes.