uniQure N.V. announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-191, the Company's gene therapy candidate for Fabry disease. AMT-191 comprises an AAV5 vector that delivers an a-galactosidase A (GLA) transgene designed to target the liver and produce the deficient GLA protein. The first-in-human Phase I/IIa clinical trial will be conducted in the United States.

The multicenter, open-label trial consists of two dose-escalating cohorts of three patients each to assess safety, tolerability, and efficacy of AMT-191 in patients with Fabry disease. About AMT-191: AMT-191 is an AAV5 gene therapy product that delivers a GLA transgene designed to targets the liver to produce GLA protein. In patients with Fabry disease, a pathogenic variant in the GLA gene leads to GLA enzyme deficiency which results in a progressive accumulation of lipids in multiple cell types creating a multi-system disorder.

AMT-191 represents a novel potential one-time administered approach to treating Fabry disease.