huntingtin-lowering
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Skyhawk’s drug trial takes flight in the US
4 min read
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.
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FDA sets May 2027 decision date on AMT-130 gene therapy for HD
4 min read
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.
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Turning down huntingtin: Alnylam’s brezosiran shows promising early result
6 min read
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.
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AMT-130 at Four Years: Hopeful Signs, Complex Controls
12 min read
uniQure reports that after 4 years, people given high-dose AMT-130 continue to show signs of slower HD progression, providing hope for their recent regulatory submission. But the new analyses are nuanced. We unpack the hopeful, but complex results.
By Dr Leora Fox -
Taking A Short Cut: New Routes to Huntingtin-Lowering Therapies
6 min read
New research points to HTT1a, a toxic shortened form of huntingtin, as a key driver of disease and a promising therapeutic target in HD.
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A shot in the arm for HTT lowering: INSIGHTT trial begins testing SRP-1005
6 min read
A new Phase 1 study is testing SRP-1005, a huntingtin-lowering therapy given by injection under the skin. It’s early days, but this approach could add an important new arrow to the HD drug development quiver.
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Looking back and moving forward: Questions and lessons 4 weeks out from Roche’s disappointing news
10 min read
Roche’s decision to stop development of 2 HTT-lowering programmes is disappointing, but the story doesn’t end there. Years of research answered some key questions, raised many new ones, and will inform the next generation of HD trials.
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Roche ends two Huntington’s disease drug programmes after disappointing results
10 min read
Roche announced it is stopping development of two of its huntingtin-lowering ASO drugs, tominersen and RG6496. This marks a disappointing end to an important chapter in HD clinical research.
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Skyhawk Shares A Closer Look at SKY-0515 Data, Plus A Peek At Expanding US Trial Access
6 min read
New 12-month data from Skyhawk’s SKY-0515 is in and all four measures that make up the cUHDRS are at or above baseline, beating expected natural decline. Plus, a first look at expanding US trial sites.
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Precision Pruning with PROTACs: Selective Targeting of Toxic Huntingtin
6 min read
PROTACs act like a precision pruning tool, trimming away toxic huntingtin protein while leaving the healthy form intact. In cells and HD mice, this targeted clean-up improved health and slowed disease features.