AMT-191

uniQure has completed enrollment for the first cohort in its Phase I/IIa trial of AMT-191 gene therapy for Fabry disease, with safety review showing no significant concerns.

Multiple gene therapy trials for Fabry disease demonstrated significant progress in 2024, with AMT-191, 4D-310, and ST-920 showing promising safety and efficacy data. These therapies aim to address the underlying genetic cause of Fabry disease through different approaches, potentially offering long-term treatment options for patients.

The FDA has agreed that existing Phase I/II data for uniQure's AMT-130, compared to external controls, can support a Biologics License Application for accelerated approval.

Clinical trials are actively progressing for Fabry Disease, with companies like Idorsia and Sanofi Genzyme developing novel therapies.

uniQure's AMT-130 demonstrated a statistically significant, dose-dependent slowing of Huntington's disease progression in Phase I/II trials, as measured by the cUHDRS.