huntingtin-lowering
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2025 HDBuzz Prize: When Good Huntingtin Goes Missing: A Step Toward Designing Safe HD Treatments
We’re proud to announce Gravity Guignard as a 2025 HDBuzz Prize winner! A new study points to one of the reasons why past HD trials might have failed. The good news? This shows the path to safer drugs, getting us closer to effective therapies.
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The First Domino Falls: AMT-130 Gene Therapy Slows Huntington’s in Landmark Trial
In an update from uniQure, they report that their experimental gene therapy, AMT-130, has the potential to slow Huntington’s disease progression in key clinical study.
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SKY-0515 Lowers Huntingtin In People With Huntington’s Disease In Trial Update
SKY-0515, an oral drug, safely lowers huntingtin in people with HD and may also reduce PMS1. This could offer a possible two-pronged approach to treat HD. SKY-0515 is now being tested in a larger Phase 2/3 trial.
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Spark ignited: first HD patient dosed in new Roche gene therapy trial
Spark and Roche issued a joint community letter to share that the first patient has been dosed in a new HD gene therapy trial.
By Dr Leora Fox -
Full Steam Ahead: uniQure’s On Track With Hope for Accelerated Approval of Huntington’s Disease Drug
uniQure has aligned with the US FDA on next steps to achieve accelerated approval of AMT-130. If data remain positive, they will be on track to market the first HD gene therapy.
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Roche provides an update on tominersen: What’s next for this huntingtin-lowering drug?
Roche gave an update this week about GENERATION HD2, testing the HTT-lowering drug tominersen in people with HD. The trial is continuing, but only the higher dose will move forward. What does this update mean for the HD community and this trial?
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Molecular Surgeons for Huntington’s Disease Catch a RIDE with CRISPR Advancements
A new CRISPR-based technology, called RIDE, is a leap forward for this trail-blazing technology. With the precision of a scalpel sharp enough to rewrite the very code of life, researchers have used it with the hope of treating Huntington’s disease.
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Huntington’s Disease Clinical Expert Prof. Ed Wild Shares Hopeful View For 2025
The HDBuzz team caught up with editor emeritus and Huntington's disease specialist, Prof Ed Wild to hear his thoughts on what 2025 has in store for the Huntington's disease community
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2024: Year in Review
As we begin 2025, we look back on all of the Huntington’s disease research news and progress the field has made in the last 12 months.
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Interim update from Vico Therapeutics on their CAG-targeting drug, VO659
Vico Therapeutics have shared interim data about their drug, VO659, which targets the CAG expansion that causes several genetic diseases, including #Huntingtonsdisease and #spinocerebellarataxia