uniQure has announced that their experimental gene therapy, AMT-130, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA).
AMT-130 is a gene therapy that aims to slow the progression of HD by targeting and reducing the production of the harmful huntingtin protein. The RMAT designation is given to therapies that show promising results in early studies and treat serious or life-threatening conditions. In other words, the FDA sees potential in the early clinical data from AMT-130 and wants to support its development through closer guidance and a faster path to review in the future.
This doesn’t mean the therapy is approved yet, but it’s an important recognition that the treatment is on a promising path. The designation will allow uniQure to work more closely with the FDA as they continue gathering data and potentially move toward seeking approval.
uniQure continues to gather data from its Phase I/II trials in the U.S. and Europe, where AMT-130 is being studied in people with early manifest HD. Full results are expected later this year and into 2026.
The HD community’s involvement in these trials is key to advancing research. We’re deeply grateful to the participants, their families, and the research teams working to bring new hope to HD families around the world.