uniQure's Fabry Disease Gene Therapy Shows Sustained Enzyme Activity in Phase I/IIa Trial
Event summary
- All 11 patients in uniQure's Phase I/IIa trial of AMT-191 showed elevated α-Gal A activity as of January 8, 2026.
- Six of 11 patients discontinued enzyme replacement therapy (ERT) due to elevated α-Gal A activity.
- Two patients in the mid-dose cohort experienced Grade 3 liver enzyme elevations, pausing further dosing in that group.
- AMT-191 maintained stable lyso-Gb3 levels post-dosing across all cohorts, regardless of ERT status.
The big picture
uniQure's updated Phase I/IIa data for AMT-191 reinforces the potential of gene therapy to provide a one-time treatment for Fabry disease, a condition currently managed with bi-weekly enzyme replacement infusions. The sustained increases in α-Gal A activity and stable lyso-Gb3 levels suggest AMT-191 could address limitations of existing therapies, positioning uniQure as a leader in genetic lysosomal storage disorder treatments.
What we're watching
- Regulatory Pathway
- Whether the sustained enzyme activity and manageable safety profile will support accelerated regulatory approval for AMT-191.
- Dosing Strategy
- How uniQure will address the dose-limiting toxicity observed in the mid-dose cohort and its impact on future trial design.
- Therapeutic Potential
- The pace at which AMT-191 could displace enzyme replacement therapy as the standard of care for Fabry disease.
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