uniQure, a gene therapy company based in the Netherlands, has asked regulators in the United States and the United Kingdom to approve AMT-130, a one-time gene therapy meant to slow Huntington’s disease1. The company announced on 2 September 2026 that it had filed with the US Food and Drug Administration (FDA) and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA)2. In the US, it is asking for accelerated approval, a faster route that lets a drug reach the market on early evidence that it is likely to help patients1.
Huntington’s is an inherited brain disease that causes movement problems, memory and thinking decline, and changes in behavior1. About 70,000 people in the US and Europe have a diagnosis, and no approved therapy delays or slows the disease3. The filing is not an approval. The International Huntington Association stressed that AMT-130 is still experimental and that both agencies still have to review it and decide2.
What happened
The treatment is given once, by surgery directly into the brain1. It is not a cure. It is designed to slow the disease by switching off production of huntingtin, the protein whose faulty form is thought to drive Huntington’s1. A harmless virus shell, known as an AAV vector, carries the genetic instructions into brain cells4. Surgeons use MRI scans to guide delivery into the striatum, a deep brain region3.
“The submission of licensing applications for [AMT-130] represents an important milestone for the Huntington’s disease community,” uniQure CEO Matt Kapusta said in a company press release1.
A standard FDA review takes about 10 months after the agency accepts an application. uniQure has asked for priority review, which would shorten that to about six months1. If the therapy gets accelerated approval, the company will have to run more studies to confirm that it helps patients, and keeping the approval will depend on those results. uniQure said it is already planning that confirmatory trial1. The company has not announced filings or timelines for any other regions2.
The evidence so far
The filings rest on three-year results from two early-stage trials, one in the US and one in Europe1. These are Phase 1/2 trials, the first tests in people, built mainly to check safety and look for early signs of benefit56. According to uniQure, people who received a high dose declined 75% more slowly than a comparison group of untreated patients1.
Results the company announced in September 2025 give more detail. After 36 months, scores on a combined Huntington’s rating scale, the composite Unified Huntington’s Disease Rating Scale (cUHDRS), fell by 0.38 points in treated patients and by 1.52 points in the comparison group4. A second measure, Total Functional Capacity, a scale that rates everyday functioning, showed 60% slower decline4. The company also reported that levels of neurofilament light, a protein in the fluid around the brain and spine, stayed below where they started on average. The FDA had agreed this marker could count as supportive evidence4.
The sources describe the data a little differently. Huntington’s Disease News says the 75% figure comes from both trials1. CGTlive’s report on the 2025 results describes it as coming from one of them4.
What it does not show yet
The key comparison group did not come from inside the trials. Treated patients were compared with untreated people from natural history studies, which follow how the disease progresses without treatment1. The comparison group was drawn from a large Huntington’s database called Enroll-HD and statistically matched to resemble the treated patients32. That is weaker than a trial where people are randomly assigned to treatment or a placebo (a dummy treatment), because the groups can differ in ways matching does not fix.
The trials are small. The European study enrolled 14 people6. The US study lists an estimated 43, and some of its groups received imitation (sham) surgery for comparison, while its newest group gets the high dose openly, with everyone knowing what they received5. In one European group, participants also received drugs that suppress the immune system: dexamethasone, sirolimus and rituximab6.
Neither trial has posted results on the public trial registry, and both list main completion dates in 202956. Both trials’ main goals are safety measures, such as side effects56. The sources reviewed for this article do not give detailed safety results. uniQure said it will present four-year data in the coming months1.
A bumpy road with the FDA
The filing follows several changes of direction at the FDA. In late 2024, uniQure said the agency had agreed that the early trials, compared with outside data, could be the main basis for an application, so no new trial was needed first4. In June 2025, the company said the FDA had accepted the combined rating scale as a measure for accelerated approval, and it expected to file in early 20263.
Then in November 2025, uniQure said that at a meeting held before filing, the FDA no longer appeared to agree that the early trial data were enough. The company said the timing of its application was unclear7. Kapusta called it “a drastic change from the guidance the FDA provided in November 2024”7. At the time, uniQure said it would also talk with regulators in the European Union and the UK7.
According to Huntington’s Disease News, the FDA had asked for an additional trial before an application. After a meeting with uniQure this summer, the agency dropped that request and agreed the existing trials could support an application1.
Who is behind it
uniQure sponsors both trials56. The company trades on the Nasdaq exchange and already has an approved gene therapy for hemophilia B, a bleeding disorder7. That product is called HEMGENIX, and the FDA agreed uniQure could draw on its experience making it when validating how AMT-130 is manufactured3. The FDA has also given AMT-130 several designations meant to speed up drugs for serious unmet needs, including fast track, breakthrough therapy and regenerative medicine advanced therapy status1.
Most of the results described here come from company announcements. Sarah Tabrizi, director of the University College London Huntington’s Disease Centre, praised the 2025 results in a uniQure press release, calling them “the most convincing in the field to date”4.
What to watch next
The next steps are whether the FDA formally accepts the application, whether it grants priority review, and what the four-year data show1. Each agency will reach its own decision2.
Patient groups are also looking past approval. The International Huntington Association said access could depend on cost, on whether health systems are ready, and on the specialist expertise needed to deliver a treatment that requires brain surgery2. Its president, Svein Olaf Olsen, called the filings “an important milestone for the entire Huntington’s disease community”2.
