huntingtin-lowering
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First participants dosed in new POINT-HD huntingtin-lowering trial
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.
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A road less traveled: how making less huntingtin can alter somatic instability and may delay symptoms
CAG repeats can get longer over time as the HD gene is used, like the growing potholes and cracks in an old road. New research finds that blocking cells from using their HTT gene slows this wear and tear, which might slow the onset of symptoms in HD.
By AJ Keefe -
UniQure Receives FDA Meeting Minutes on AMT-130 While Community Support Remains Strong
UniQure received FDA’s meeting minutes on AMT-130. While there were no new updates, the community’s response has been powerful, with 41K+ petition signatures & unified advocacy from major HD orgs.
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UniQure and FDA No Longer in Alignment on Approval Pathway for AMT-130
In a new press release, uniQure shared that the FDA no longer agrees with their approach for advancing AMT-130, specifically the use of an external control group. The path forward is uncertain, but uniQure remains committed to the HD community.
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Additional Clarity: What We Know 4 Weeks After the uniQure News
We’re 4 weeks out from uniQure’s positive update about AMT-130 that took the world by storm. Does the data match the media hype? Where do uncertainties remain? And what are some of the key questions from the Huntington’s disease community?
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2025 HDBuzz Prize: When Good Huntingtin Goes Missing: A Step Toward Designing Safe HD Treatments
We’re proud to announce Gravity Guignard as a 2025 HDBuzz Prize winner! A new study points to one of the reasons why past HD trials might have failed. The good news? This shows the path to safer drugs, getting us closer to effective therapies.
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The First Domino Falls: AMT-130 Gene Therapy Slows Huntington’s in Landmark Trial
In an update from uniQure, they report that their experimental gene therapy, AMT-130, has the potential to slow Huntington’s disease progression in key clinical study.
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SKY-0515 Lowers Huntingtin In People With Huntington’s Disease In Trial Update
SKY-0515, an oral drug, safely lowers huntingtin in people with HD and may also reduce PMS1. This could offer a possible two-pronged approach to treat HD. SKY-0515 is now being tested in a larger Phase 2/3 trial.
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Spark ignited: first HD patient dosed in new Roche gene therapy trial
Spark and Roche issued a joint community letter to share that the first patient has been dosed in a new HD gene therapy trial.
By Dr Leora Fox -
Full Steam Ahead: uniQure’s On Track With Hope for Accelerated Approval of Huntington’s Disease Drug
uniQure has aligned with the US FDA on next steps to achieve accelerated approval of AMT-130. If data remain positive, they will be on track to market the first HD gene therapy.