uniQure announces FDA clearance of IND application for AMT-191 gene therapy

uniQure announced that the U.S. Food and Drug Administration has cleared the Investigational New Drug application for AMT-191, the Company’s gene therapy candidate for Fabry disease. AMT-191 comprises an AAV5 vector that delivers an alpha-galactosidase A 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.