Vico Therapeutics have shared interim data about their drug, VO659, which targets the CAG expansion that causes several genetic diseases, including #Huntingtonsdisease and #spinocerebellarataxia
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.
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
uniQure ushered in the end of the year by releasing some promising data from their huntingtin-lowering gene therapy trials
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.
The outcomes of the SELECT-HD clinical trial are out, with encouraging news for this allele-selective huntingtin lowering therapy
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.
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.
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.