Sept. 24, 2025 – uniQure has an ongoing clinical trial in the United States and the United Kingdom testing gene therapy AMT-130, which is not currently available in Canada. They have released topline results from their phase I/II trial showing for the first time that a drug can slow the progression of Huntington disease (HD) in people. This update is very encouraging, but it is still early on in the process. These results are from fewer than 30 early manifest participants who have received the treatment so far, so they need to be taken with caution.
uniQure will meet the FDA later in 2025, aiming to file for approval in early 2026, while scaling up manufacturing and surgical capacity. The findings further support huntingtin-lowering as a therapeutic strategy, and boost prospects for other treatments in development, all made possible by the extraordinary commitment of trial participants and the broader HD community.
The Huntington Society of Canada is following the research closely, sharing updates with the community, and talking with researchers and companies about bringing trials and new treatments to Canada. While we cannot speed up approvals, we are committed to keeping Canadian families informed and advocating for access.
Resources
To read uniQure’s press release, click here: https://uniqure.gcs-web.com/node/12261/pdf
To read the HDBuzz article, click here: https://en.hdbuzz.net/the-first-domino-falls-amt-130-gene-therapy-slows-huntingtons-in-landmark-trial/
If you would like to connect with a member of the Family Services team, please reach out to your RCD or, if you’re new to HSC, please fill out our online form: https://contactme.cloud/form/huntingtonsociety
Media
Ron Culp – Hanomansing Tonight:
Sébastien Heins – Global News:
Prof. Ed Wild and Fergus Walsh – BBC News:
Prof. Ed Wild – CBC Radio (1:44 to 9:13):
Vern Barrett – 680 CJOB The Jim Toth Show (20:10 to 30:57):
Dr. Justyna Sarna – The Calgary Eyeopener: www.cbc.ca/listen/live-radio/1-5-calgary-eyeopener/clip/16171706-gene-therapy-help-treat-huntingtons-disease
Alexander Maxan – CBC’s Daybreak South with Chris Walker: www.cbc.ca/listen/live-radio/1-110-daybreak-south/clip/16173162-new-research-showing-potential-treat-huntingtons-disease
Rachel Harding – CBC News: www.cbc.ca/news/health/huntington-s-disease-gene-therapy-1.7644129
Rachel Harding – Scientific American:www.scientificamerican.com/article/first-treatment-that-slows-huntingtons-disease-comes-after-years-of/
Staying Connected
Staying connected with HSC is the best way to make sure you hear about new research opportunities and updates. Please sign up for our e-newsletters here: www.huntingtonsociety.ca/enewsletters/. You can also follow HSC on our social media: Facebook, Instagram, and LinkedIn.