Articles with the topic: huntingtin-lowering

Interim update from Vico Therapeutics on their CAG-targeting drug, VO659

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

Dr Rachel HardingDecember 10, 2024

Going boldly: First person treated in Phase 1 clinical trial by Alnylam Pharmaceuticals

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.

Dr Sarah HernandezDecember 08, 2024

Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntington’s disease in Phase I/II clinical trial

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

Dr Rachel HardingAugust 16, 2024

Steady progress from uniQure - promising data to end the year

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

Dr Leora FoxAugust 16, 2024

Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515

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.

Dr Sarah HernandezJuly 11, 2024

Positive news from Wave Life Sciences SELECT-HD trial

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

Dr Rachel HardingJune 28, 2024

Two birds, one stone: HTT-lowering drugs also target CAG expansions

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.

Dr Sarah HernandezMay 21, 2024

Could halting CAG expansions be a new treatment for HD?

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.

Dr Sarah HernandezOctober 05, 2023

Updates from PTC Therapeutics and uniQure on their huntingtin-lowering trials

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.

Dr Leora Fox and Dr Rachel HardingJune 21, 2023

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