Mavrix Bio Begins First Human Trial for Gene Therapy MVX-220 to Treat Angelman Syndrome
Mavrix Bio is set to commence its first human trial for MVX-220, a gene therapy aimed at treating Angelman syndrome. The Phase 1/2 ASCEND-AS trial, scheduled to occur at Rush University Medical Center in Chicago, will evaluate the safety and potential benefits of this innovative therapy.
MVX-220 is designed to deliver a functional copy of the UBE3A gene directly to brain cells. The trial, which has received fast track designation from the FDA, will involve 12 children and adults with Angelman syndrome, regardless of its genetic cause. Participants will receive injections of MVX-220 into the fluid-filled spaces in their skulls.
The trial's primary goal is to assess the safety of MVX-220 over the first two years. Secondary outcomes will focus on changes in various symptoms associated with Angelman syndrome. Mavrix Bio, with support from the Fraunhofer-Gesellschaft, has developed this promising therapy. Allyson Berent, chief development officer, welcomed the FDA's fast track designation, highlighting the urgent need for Angelman syndrome treatments.
The ASCEND-AS trial signifies a significant step towards potentially treating Angelman syndrome. If successful, MVX-220 could be eligible for accelerated approval pathways, bringing hope to those affected by this condition.
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