Update (October 2026): Axovant, later renamed Sio Gene Therapies, no longer exists. The intravenous AAV9 trial continues as an investigator-led study at the National Human Genome Research Institute (NIH). For current status, see our AAV gene therapy for GM1 page and our GM1 clinical trials guide.
The first gene therapy clinical trial for GM1 gangliosidosis is a beacon of hope for children and patients suffering from this truly devastating and fatal neurodegenerative disease. We are extremely thankful to all those who have contributed to reach this historic milestone. We look forward to continued progress and advances so that some day there will be a treatment to reach all the patients suffering from GM1.
The Cure GM1 Foundation contributed significant funding to the preclinical work for this program. Substantial funding was also provided by NIH, UMass, Auburn.
For more information on the clinical trial, please see this page and enrollment information, inclusion, and exclusion criteria are specified on clinicaltrials.gov.
Please read the Press Release from Axovant on dosing the first patient.