huntingtin-lowering
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AMT-130 at Four Years: Hopeful Signs, Complex Controls
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
⏱️5 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
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
⏱️10min 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.
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The Other Shoe Drops: uniQure Shares Plans To Submit Licensing Application With The FDA for AMT-130
⏱️ 9 min read | After a turbulent past year, uniQure have shared encouraging news that the FDA has agreed their Phase I/II study data can serve as the basis for an application that could lead to the approval of AMT-130 in the US.
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Skyhawk reports encouraging 12-month results for oral Huntington’s disease drug SKY-0515
One year into treatment, SKY-0515 continues to lower huntingtin and shows encouraging trends with signs and symptoms of HD. A larger trial is enrolling to determine whether the drug truly slows HD progression
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Fixing the Recipe: Lowering a Slice of Huntingtin
⏱️7 min read | In a recent paper, scientists targeted a small, harmful piece of the huntingtin message (HTT1a) in HD mice. This reduced toxic protein clumps and delayed genetic changes, more than targeting full length HTT.