Skyhawk’s drug trial takes flight in the US
Skyhawk’s FALCON-HD trial has dosed the first participants in the US with the drug, SKY-0515. The once-daily pill targets two drivers of HD, but does it actually slow disease? That’s the big question this study hopes to answer.
FDA sets May 2027 decision date on AMT-130 gene therapy for HD
The FDA has accepted uniQure’s application for AMT-130, an experimental gene therapy for Huntington’s disease, and set a May 2027 decision date. Here’s what this milestone means, and what still needs to happen.
October 2026: This Month in Huntington’s Disease Research
From sticky thoughts and pigs that model HD to DNA repair and promising drug trial results, October brought fresh insights into HD. Here’s what you might have missed!
Turning down huntingtin: Alnylam’s brezosiran shows promising early result
An experimental treatment for HD developed by Alnylam has shown long-lasting huntingtin lowering after just one dose, with encouraging early safety data. A pivotal trial is now planned for 2027 to figure out if this drug, brezosiran, can slow HD.
Picking MSH3’s Pocket: New Insights into CAG Repeat Expansion
Scientists discovered a pocket on a protein called MSH3 that helps it grab DNA containing CAG repeats. By blocking this interaction, they slowed the growth of CAG repeats in cells, without removing the protein entirely.
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