24/09/2025
Ignacio Muñoz-Sanjuan, neuroscientist and founder of the non-profit Factor-H, has expressed optimism that an experimental gene therapy for Huntington’s disease, developed by uniQure and called AMT-130, could receive regulatory approval in the first half of 2026, in what is a very significant milestone for patients and families affected by the disorder. “It is a day for hope -he said-, while the therapy will at first be limited, access will increase in the years ahead and it can open a new path for treatments”
The 36-month clinical trial results reported by uniQure showed that the therapy, which is administered directly into the brain via a neurosurgical procedure, has a profound effect in slowing the decline normally observed in patients with Huntington’s disease. The trial is still ongoing, and the beneficial results communicated by uniQure include information about the disease trajectory for only 12 patients in early stages of the disease that received the high dose of the therapy. Despite the low number of participants, the results were very significant. However, Muñoz-Sanjuán cautioned that the reported results have not yet been independently analyzed and that the program has not been reviewed by the regulatory authorities. Therefore, the therapy is not yet available for other patients anywhere in the world. Munoz-Sanjuán stated that we need to wait for regulatory feedback early in 2026 to understand when the therapy might be available more broadly.
Muñoz-Sanjuan stated in a recent interview:
“while the therapy will at first be limited, access will increase in the years ahead.”
Muñoz-Sanjuan served in a leadership role at the CHDI Foundation, where he led a team of scientists working directly to develop therapeutics for Huntington’s disease.
CHDI Foundation is a nonprofit biomedical research organization focused on accelerating therapeutic development for Huntington’s disease. During his tenure there, Muñoz-Sanjuan was part of the scientific leadership driving preclinical research and drug discovery efforts directed at silencing the Huntingtin gene, and worked closely with the team at uniQure.
While such a timeline is hopeful, experts caution that an approval is only one step; the challenges of manufacturing, regulatory adoption, cost, and distribution remain significant.
But “For families living with Huntington’s disease, a window has opened that marks a turning point in their hope. He also admitted the difficulty for this gene therapy to be broadly adopted, since it requires a long neurosurgical procedure; but he added that “a new paradigm has been established with the results of the AMT-130 uniQure trial, showing disease amelioration over a 3-year period, and showing broad impact in motor, functional and cognitive endpoints, for the first time in the history of the disease”. He also states that “these results will undoubtedly encourage other companies to persist in the development of HTT-silencing therapies with different modalities.”
Muñoz-Sanjuan wanted to send a warm message immediately to the families the Factor-H team works with day to day in Venezuela, Colombia, Perú and other Latin American countries.
BBC News:
Huntington’s disease successfully treated for first time
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