Account Info
Log Out
English
Back
Log in to access Online Inquiry
Back to the Top

NEWS

uniQure Announces Dosing of First Patient in Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease


uniQure N.V. (NASDAQ: QURE) has initiated dosing in a Phase I/IIa clinical trial of AMT-191 for Fabry diseasetreatment. The multi-center, open-label trial in the US will assess safety, tolerability, and early efficacy signs in two dose-escalating cohorts. AMT-191 is an AAV5-based gene therapy delivering a galactosidase alpha (GLA) transgene to the liver. It aims to address the α-galactosidase A enzyme deficiency in Fabry patients.

The trial includes two cohorts of up to six adult male patients each, with low (6x10^13 gc/kg) and high (3x10^14 gc/kg) doses administered intravenously. Patients will be followed for 24 months. This milestone aligns with uniQure's goal to advance three new gene therapies into clinical studies this year, alongside programs in Huntington's disease, temporal lobe epilepsy, and SOD1-ALS.
Disclaimer: Community is offered by Moomoo Technologies Inc. and is for educational purposes only. Read more
Translate
Report
4901 Views
Comment
Sign in to post a comment
    Spread kindness and love. Life is short. Don’t let greed eat you.
    2311Followers
    105Following
    24KVisitors
    Follow