gene-therapy
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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 -
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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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.
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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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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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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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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