huntingtin-lowering
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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
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Going boldly: First person treated in Phase 1 clinical trial by Alnylam Pharmaceuticals
A new Phase 1 clinical trial for the huntingtin-lowering drug ALN-HTT02 was initiated this week with the first dose given. Read on to learn details about the trial and how it compares to other ongoing huntingtin-lowering trials in the clinic.
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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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Two birds, one stone: HTT-lowering drugs also target CAG expansions
In a surprising twist, oral HTT-lowering drugs also slow somatic expansion in the HTT gene. A new study that used cells in a dish for this fortuitous discovery identified the gene PMS1 as a key player in the slowing of CAG expansions.
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Steady progress from uniQure – promising data to end the year
uniQure ushered in the end of the year by releasing some promising data from their huntingtin-lowering gene therapy trials
By Dr Leora Fox -
Could halting CAG expansions be a new treatment for HD?
The gene MSH3 is getting a lot of attention in HD research lately. New findings suggest MSH3 lowering could halt CAG repeat expansions, offering a new therapeutic avenue.