huntingtin-lowering
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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
-
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.
-
A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
⏱️ 6 min read | While the US regulatory path for AMT-130 remains complicated, uniQure announced plans to seek approval for its HD gene therapy in the UK. Here’s what that means, and what might be to come.