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AMT-130 at Four Years: Hopeful Signs, Complex Controls
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September 2026: This month in Huntington’s disease research
September brought progress across the HD research landscape. From brain waves and visual thinking to DNA repeats, nutrition, inclusive care, new technologies, and encouraging SKY-0515 data, we round up a busy month in Huntington’s disease research.
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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
⏱️ 8 min read | An oral Huntington’s disease drug is eligible for accelerated approval in Australia. This isn’t full approval yet, but it opens a faster path to potentially getting this once-daily pill to people with HD sooner.
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The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
⏱️ 10 min read | The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.
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Vico’s Trial Adjusts With Twice-Yearly Dosing, And The U.S. Is Next
⏱️ 6 min read | Vico Therapeutics’ Huntington’s disease drug VO659 is now being tested just twice a year, and the FDA has cleared the way for US trials to begin later this year.
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February 2026: This Month in Huntington’s Disease Research
⏱️ 8 min read | February 2026 HD research recap: published trial data from branaplam trial; new insights into the genetics of the HD “grey zone”; plus worms, brain circuits, eye tracking, and the hidden psychological weight of living at risk.
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A Window into the Eyes: Using Ocular Biomarkers to Track Progression in Huntington’s Disease
A new review from the University of Cambridge explores ocular biomarkers in HD, including retinal scans and eye movement tracking.
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A Fault In The Supercomputer
⏱️ 8 min read | Your brain is an advanced supercomputer. But in HD, a core processor starts to malfunction. A research group at UCLA has taken a close look to visualize the specific circuits going haywire – and how they look different in HD.
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Split Between Two Worlds: The Psychological Challenges for People at Risk of Huntington’s Disease
⏱️ 7 min read | Living at risk for Huntington’s disease imposes a massive psychological burden. A small interview-style study of at risk 12 people dove into how these people cope day-to-day.
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Putting it in print: oral drug branaplam lowered huntingtin, but safety concerns halted development
⏱️10 min read | Results from the VIBRANT-HD trial are now published in a peer reviewed journal. This trial tested the oral drug, branaplam, which lowered HTT but had serious safety problems, ultimately halting the trial.
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A hitch in the stitch reveals why DNA sequence patterns matter in Huntington’s disease
Two individuals with the same CAG repeat count can have vastly different HD journeys. A new study looking at individuals with 36-42 repeats shows that DNA sequence patterns play a major role in symptom onset and progression.
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Worms to the Rescue: Separating the Good, the Bad, and the Clumpy Huntingtin
⏱️ 7 min read | A team of researchers developed a simple platform to test how toxic different types of HTT protein clumps are using a laboratory worm model. They showed that certain types of clumps are harmful to the worms, while others are not.
HDBuzz Trial Tracker
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Sites for Phase 3 trial for votoplam, INVEST-HD, now open. Updates on recruitment and first dosing imminent.
REGEN4HD, Phase 1 stem cell implantation study commences
Progress update for NCT06585449 – A Study to Evaluate ALN-HTT02
