Breakthroughs in gene therapy don’t happen every day. @uniQure’s pivotal Phase I/II study of AMT-130 in Huntington’s Disease met its primary endpoint, showing a 75% slowing of disease progression at 36 months.
What stands out is the consistency: multiple endpoints pointing in the same direction, biomarkers reinforcing the story, and a safety profile that remains manageable. For patients and families facing Huntington’s disease (HD), this is more than data. It is hope.
It is a milestone that matters. AMT-130 is the first gene therapy designed specifically for HD that has made it into human clinical trials. It could become the first gene therapy for HD that gets approval.
I’m grateful that one of my very first projects as a patent attorney was to support this development. Success at this level is never about one element alone. It’s science, strategy, and persistence coming together. Congratulations to the entire uniQure team!