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.
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
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.
uniQure ushered in the end of the year by releasing some promising data from their huntingtin-lowering gene therapy trials
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.
Researchers look at the cause and effect of various forms of the HTT protein. They find both expanded and unexpanded HTT contribute to brain cell communication and the brain has an amazing capacity to compensate for changes related to disease
Roche released a community letter in early 2023, to share that their Phase II clinical trial to study the huntingtin-lowering drug, tominersen, is now underway. In this article, we summarise the latest news about this huntingtin-lowering drug.