uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today 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 α-galactosidase A (GLA) transgene designed to target the liver and produce the deficient GLA protein.
“The clearance of the IND for AMT-191 represents a key milestone for the company, with four programs now in clinical phase,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “AMT-191 has the potential to be a differentiated gene therapy for the one-time treatment of Fabry disease, incorporating a proprietary promoter and leveraging our validated AAV5 technology comprised within HEMGENIX®, an approved liver-directed gene therapy for the treatment of hemophilia B developed by uniQure. We have designed the Phase I/II study to provide dose-ranging biomarker data as rapidly and cost-effectively as possible, and we look forward to enrolling our 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.
https://finance.yahoo.com/news/uniqure-announces-fda-clearance-investigational-120500847.html
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.