uniQure N.V. announced on February 3, 2025, the completion of enrollment in the first cohort of its Phase I/IIa trial of AMT-191, an investigational gene therapy for Fabry disease. This milestone signifies progress in the clinical evaluation of the AAV5-based therapy.
The Independent Data Monitoring Committee (IDMC) reviewed safety data from the initial two patients in the first cohort and found no significant safety concerns. Consequently, the IDMC recommended proceeding with enrollment in the second cohort, which uniQure expects to initiate in the first quarter of 2025.
This positive safety outcome and progression to the next dose cohort are encouraging for AMT-191, which aims to address the α-galactosidase A enzyme deficiency in Fabry disease patients. The trial will continue to explore the safety, tolerability, and early signs of efficacy by measuring the expression of lysosomal enzyme α-Gal A.
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