uniQure announces positive safety data in study of AMT-130
uniQure released a safety update this morning for the first 10 participants in the low-dose cohort of the phase I/II trial of AMT-130, the first experimental gene therapy to be tested in people with Huntington’s disease. The press release indicated that the drug has been well-tolerated with no significant safety concerns observed after the first year of follow-up. On average, mHTT levels in CSF of participants decreased by over half, and NfL levels were maintained near baseline, with decrease in levels observed in some participants. uniQure expects to release 24-month follow-up for the low-dose cohort and 12-month follow-up for the high-dose cohort in the first half of 2023.
HDBuzz on Machine Learning in HD Research
While clinicians have a strong working definition of what HD is and how symptoms will look in affected people, scientists still struggle to fully grasp the biological mechanisms behind the disease. Observational research and natural history studies of HD are helpful for better understanding symptoms and identifying biomarkers of disease progression; now, computers are using artificial intelligence and machine learning to identify new patterns from these datasets. The result of this research, completed by CHDI and IBM, is a new, 9-stage model of HD progression that could help identify candidates for clinical trials, discover new disease biomarkers, inform trial design, and more. To read more about this research and the findings that came from it, visit HDBuzz for the scoop from the editors.
Survey study for presymptomatic and at-risk individuals
Huntington’s Disease (HD) community members, Seth Rotberg and BJ Viau, are conducting a short (~10 minutes) research survey to gain a deeper understanding of the impact Huntington’s Disease has on people before they’ve been clinically diagnosed. If you’re 18+ years old, reside in the U.S.A, and are either at-risk or pre-symptomatic for HD, we encourage you to share your experience. The results will be completely anonymous and will help us show the risk presymptomatic patients are willing to take to participate in studies prior to showing symptoms. For more information or to take the survey, click here.