Last updated: October 2026

SRP-1005 is an investigational small-interfering RNA treatment being developed by Sarepta Therapeutics to lower huntingtin expression.

Current status

Its first-in-human clinical program is focused on safety, tolerability, and evidence of target engagement. This candidate is investigational and is not FDA approved. Timing, eligibility, safety, effectiveness, availability, and cost remain uncertain.

For the newest coverage across the HD treatment pipeline, visit What’s Happening in HD Treatment.