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May 2025: This Month in Huntington’s Disease Research
Latest News
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Mini brains grown in a dish shed light on Huntington’s disease and how we might treat it
Exciting new findings using 3D human lab-grown mini brains inform ongoing HTT-lowering trials and suggest that stem cell transplants for HD may improve cell-to-cell communication and reduce disease features.
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Hereditary Disease Foundation (HDF) conference 2024 – Day 4
Read our live tweet compilation from clinical trials and scientific research on Huntington’s disease from Day 4 of the 2024 HDF Milton Wexler Biennial Symposium #HD2024
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Hereditary Disease Foundation (HDF) conference 2024 – Day 3
Read our live tweet compilation from clinical trials and scientific research on Huntington’s disease from Day 3 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024
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Hereditary Disease Foundation (HDF) conference 2024 – Day 2
Read updates from clinical trials and scientific research on Huntington’s disease from Day 2 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024
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Hereditary Disease Foundation (HDF) conference 2024 – Day 1
Read updates from clinical trials and scientific research on Huntington’s disease from Day 1 of the 2024 HDF Milton Wexler Biennial Symposium #HDF2024
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Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515
There’s more good news in the forecast in the Huntington’s disease therapeutic space as we receive positive results from Skyhawk Therapeutics about their small molecule SKY-0515 that lowers huntingtin and targets somatic expansion.
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Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntington’s disease in Phase I/II clinical trial
More good news for the HD community from uniQure, whose one-and-done gene therapy appears safe and shows hints of slowing down signs and symptoms of the disease
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Positive news from Wave Life Sciences SELECT-HD trial
The outcomes of the SELECT-HD clinical trial are out, with encouraging news for this allele-selective huntingtin lowering therapy
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No pivot needed for PTC-518
PTC Therapeutics shared 12 month data from the PIVOT-HD trial, testing the oral HTT lowering drug PTC-518. While designed to assess safety, they shared encouraging results that the drug showed promising signs for biomarkers and some clinical metrics.
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SURVEYOR opens the door for drugs that treat cognition
Sage Therapeutics announced results from SURVEYOR, a trial that looked at cognitive changes in people with HD, and tested short-term safety of dalzanemdor. The study was small but met key goals, and additional trials are in progress.