NasdaqGS:QUREBiotechs
How Early AMT-191 Gene Therapy Signals Will Impact uniQure (QURE) Investors
uniQure N.V. recently reported updated preliminary safety and exploratory efficacy data from 11 patients in its ongoing Phase I/IIa trial of AMT-191, an investigational AAV gene therapy for Fabry disease, showing elevated α-galactosidase A activity across all three dose cohorts and a manageable safety profile.
An interesting early signal is that several participants have been able to discontinue enzyme replacement therapy, hinting at the potential real-world impact of a one-time gene therapy...