AMT‑130 Slows Huntington’s 75%; uniQure Shares Surge

University College London and uniQure reported AMT‑130, a one‑time gene therapy delivered via stereotactic brain surgery, slowed Huntington’s disease clinical progression by about 75% over 36 months in the high‑dose group of a 29‑patient trial. UniQure said AMT‑130 was well tolerated with no adverse effects observed since 2022 and that investigators observed a sustained, statistically significant slowing of progression, though the data are preliminary and not yet fully peer‑reviewed. AMT‑130 has been granted FDA Breakthrough Therapy and RMAT designations, and uniQure said it plans to pursue accelerated approval and is planning a U.S. filing in the coming years. The announcement triggered a dramatic market reaction—uniQure shares jumped roughly 248% in a single session and several analysts raised price targets. Clinicians and patient advocates called the result potentially transformative, but researchers and outside neurologists emphasized that the small trial size, use of external controls, the invasive surgery, manufacturing scale‑up, regulatory review and equitable rollout require larger, randomized studies before broad clinical availability.