huntingtin-lowering
-
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.
-
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
-
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.
-
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.
-
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
-
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
-
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.
-
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.
-
Updates from PTC Therapeutics and uniQure on their huntingtin-lowering trials
Both PTC Therapeutics and uniQure have shared updates from their respective clinical trials, testing different huntingtin-lowering approaches. We explore the data presented from both of these studies and what this means for HD family members.