Two companies developing medicines for Huntington’s disease announced news about their huntingtin-lowering drugs. Let’s talk about the history and the caveats surrounding these hopeful pieces of news.
The first round of findings from the halted tominersen huntingtin lowering trial, GENERATION-HD1, run by Roche were shared this week with the HD community. HDBuzz explains what they found and what’s next.
Wave Life Sciences shared the disappointing news that their two ASOs in Phase 1/2 trials in HD patients did not successfully lower mutant huntingtin.
Disappointing news from Roche and Ionis; the phase III Tominersen huntingtin-lowering trial has been halted early
Wave Life Sciences announces that its antisense drug WVE-120102 has lowered mutant huntingtin protein in cerebrospinal fluid, but investors seem disappointed. Rather confusing – what do we know for sure?
Hot off the presses - New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
Finally, a big study that shows what childhood HD looks like. This will help us work out if new drugs work in children too
Work with genome editing techniques (zinc fingers and CRISPR) brings these tools closer to use in HD clinical trials