UniQure’s Huntington’s gene therapy shows reduced benefit in fourth year, but patient remains optimistic

UniQure reported new data on its experimental Huntington’s disease gene therapy, AMT-130, in a four‑year follow‑up of 12 patients. The update showed the treatment slowed disease progression by 44%, a decline from the 75% slowdown reported after three years.

The reduced effect suggests the therapy’s benefit may wane over time, a point that analysts and investors are watching closely. The durability of the response is now a key question for the company’s future prospects.

Lauren Holder, a Huntington’s disease advocate who is also living with early‑stage symptoms, said she is not worried about the waning effect. Her reaction highlights a contrast between patient sentiment and market concerns.

Wall Street analysts have flagged the durability issue as a potential risk factor for UniQure’s valuation, even as patients remain hopeful about the therapy’s overall impact.