The first two participants have been treated in a 26-subject Phase 1/2 clinical trial evaluating uniQure’s (NASDAQ:QURE) AMT-130 in patients with Huntington’s disease (HD),
an inherited neurodegenerative disorder characterized by the
deterioration of physical and mental abilities caused by the progressive
breakdown of nerve cells in the brain.
The first two patients will be observed for an
initial 90-day period followed by a review of the results by the Data
Safety Monitoring Board before additional patients are dosed.
The primary endpoint is safety. Secondary endpoints include efficacy measures.
AMT-130
is a one-time administered AAV5 gene therapy that carries an artificial
microRNA designed to silence the huntingtin gene, mutations in which
cause HD.
https://seekingalpha.com/news/3584467-treatment-underway-in-study-of-uniqure-gene-therapy-in-huntingtons-disease
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