UniQure Says FDA Clears IND Application For AMT-191 Gene Therapy For Fabry Disease

(RTTNews) - Gene therapy company uniQure N.V. (QURE) announced Wednesday 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 is an AAV5 gene therapy product that delivers an a-galactosidase A (GLA) transgene designed to target the liver and produce the deficient GLA protein.

The company said it is looking forward to enrolling its first patient in the first half of 2024.

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.