uniQure
NEWS
Last month, “historic positive results” from uniQure’s gene therapy snapped the Huntington’s community out of years of failure. As the biotech prepares to submit for FDA approval, BioSpace looks at four more candidates on the near horizon.
Pivotal results from uniQure’s gene therapy for Huntington’s disease have brought new light to patients who have known only disappointment in recent years—but one expert worries that communication of the results is creating “false expectations.”
If approved, uniQure’s gene therapy AMT-130—which slowed disease progression by 75%—would be the first genetic treatment for Huntington’s disease. A BLA submission is planned for the first quarter of 2026.
Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on target for a Q1 2026 submission for its one-time gene therapy for Huntington’s disease—but patients have been here before.
In a Type B meeting, the FDA signified that it will allow uniQure to use a natural history control, the composite Unified Huntington’s Disease Rating Scale, and neurofilament light chain levels to support the accelerated approval of its gene therapy AMT-130.
Following a disappointing readout last year, uniQure on Tuesday posted promising Phase I/II data for its investigational gene therapy AMT-130 and nabbed the first-ever Regenerative Medicine Advanced Therapy designation from the FDA in Huntington’s disease.
Gene therapy company uniQure announced Tuesday “ongoing evidence” of a clinical benefit for its Huntington’s disease treatment. However, investors were not impressed as the stock dropped nearly 20%.
The Huntington’s disease space saw a flurry of activity Wednesday as PTC and uniQure released data from their respective mid-phase trials.
Clinical results for experimental treatments in Alzheimer’s, ALS and more are expected over the coming months. BioSpace highlights a few of the more highly anticipated datasets.
JOBS
IN THE PRESS