Last month, there was exciting news for people with Huntington’s disease. A new gene therapy called AMT-130, made by uniQure, showed that it can slow down the progress of this serious brain disorder. This news has given hope not only to those with Huntington’s but also to researchers working on other treatments. The main idea behind this research is that reducing the amount of a harmful protein in the brain can help control the symptoms of Huntington’s disease. Amy Gray, the president and CEO of the Huntington’s Disease Society of America, said that there are other promising treatments in the works that could help control the disease, not just manage its symptoms. Huntington’s disease research has had many setbacks in the past. There have been several failed drug trials, and at one point, it seemed like uniQure’s gene therapy might also fail. In 2022, the trial was paused when three patients who received a high dose of the therapy had serious side effects related to the surgery needed to deliver the therapy to the brain. However, these patients recovered, and the trial continued. The uniQure gene therapy uses a virus to deliver a gene that produces a small molecule designed to reduce the levels of a protein called huntingtin, which is important for proper nerve function. Huntington’s disease is caused by a mutation in the huntingtin gene, leading to the production of a harmful form of the protein that forms damaging clumps in the brain. Unlike other treatments, AMT-130 does not distinguish between healthy and mutated forms of the protein. Instead, it aims to reduce both. It is not yet clear if reducing the levels of the healthy protein will have any negative effects. The recent trial met its main goal by reducing the progress of the disease by 75% over 30 months, compared to a group of similar patients from a Huntington’s disease registry. Dr. Sarah Tabrizi, a professor of clinical neurology, said that these results are the most convincing so far and show that the treatment could potentially modify the course of the disease. UniQure’s drug is one of several treatments targeting the huntingtin protein, but each works in different ways. PTC Therapeutics is developing a small-molecule drug called PTC-518, which is now in phase 2 of testing and has shown some success in reducing levels of the huntingtin protein in the blood. Roche and Ionis Pharmaceuticals are also developing a huntingtin-lowering drug called tominersen. They are continuing its development, looking for the best way to use it after a phase 3 trial failure in 2021. The drug originally seemed to worsen symptoms at the most frequent doses, but later analysis showed potential benefits for younger, earlier-stage patients. A targeted trial with this group is ongoing. Going forward, uniQure needs to show that AMT-130 can deliver results for more than just the small group of 29 patients in its recent trial. Questions also remain about whether the benefits will last over time and if new safety issues might arise. Dr. Walid Abi-Saab, the chief medical officer of uniQure, believes the drug has the potential to transform treatment for people with Huntington’s. The company plans to apply for FDA approval in the first quarter of 2026, which could lead to its launch later that year. However, making the drug available to more patients will be a challenge, and like many one-time gene therapies, AMT-130 is expected to be very expensive, which could raise affordability issues. This is not uniQure’s first gene therapy. In 2022, the company gained approval for the world’s first gene therapy for hemophilia B, called Hemgenix, which was commercialized by CSL Behring. If AMT-130’s results hold up over time, people with Huntington’s disease could gain years of independence and better function than they would normally expect. However, Gray noted that there are still more steps ahead, and these results are from a trial, not yet an approved therapy.