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.
PTC Therapeutics shared 12 month data from the PIVOT-HD trial, testing the oral HTT lowering drug PTC-518. While designed to assess safety, they shared encouraging results that the drug showed promising signs for biomarkers and some clinical metrics.
Sage Therapeutics announced results from SURVEYOR, a trial that looked at cognitive changes in people with HD, and tested short-term safety of dalzanemdor. The study was small but met key goals, and additional trials are in progress.
A new form of the chorea drug valbenazine (INGREZZA) has been approved by the United States Food and Drug Administration (FDA) for those who have difficulty swallowing pills.
Check out research updates from Day 1 of the 2024 HD Therapeutics Conference #HDTC2024
Casgevy is the first CRISPR-based drug to make its way through the approval process, all but curing Sickle Cell Disease and it’s paving the way for similar drugs targeting other diseases. Is Huntington’s disease next?
Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?
The HD pipeline is rich and varied. Let's talk about some out-of-the-box approaches for developing drugs for HD that don't involve huntingtin lowering.
