huntingtin-lowering
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The Other Shoe Drops: uniQure Shares Plans To Submit Licensing Application With The FDA for AMT-130
8 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
9 min read
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
8 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.
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A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
5 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.
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Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
9 min read
PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to 52% slowing of disease progression. Here’s what the data show, what’s still missing, and the roll out of the Phase 3 trial.
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The Number on the Scale: What the TFC Score Measures, and Why It Matters Right Now
9 min read
The Total Functional Capacity score has been used in Huntington’s disease research for decades. Here’s what it measures, what it misses, & why it’s at the center of an anticipated clinical trial.
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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
7 min read
An oral Huntington’s disease drug is eligible for accelerated approval in Australia. This isn’t full approval yet, but it opens a faster path to potentially getting this once-daily pill to people with HD sooner.
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The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
10 min read
The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.
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UniQure Secures Type A Meeting with FDA: What This Means for AMT-130
6 min read
UniQure secured a Type A FDA meeting, a high-priority discussion for urgent issues. Within 30 days, both sides will discuss what kind of data package might support the advancement of AMT-130 in the US.
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First participants dosed in new POINT-HD huntingtin-lowering trial
6 min read
POINT-HD has begun dosing its first participants with the drug RG6496, marking an early but important step for a new selective huntingtin-lowering approach.