Mavrix Bio Begins First-in-Human Trial for Angelman Syndrome Gene Therapy
Mavrix Bio is set to commence its first-in-human clinical trial, the Phase 1/2 ASCEND-AS trial (NCT07181837), for MVX-220, a sustainable gene therapy targeting Angelman syndrome. The trial, supported by the FDA's fast track designation, will take place at Rush University Medical Center in Chicago.
MVX-220, developed by Gemmabio Therapeutics and now supported by Mavrix Bio, aims to deliver a functional copy of the UBE3A gene to brain cells. The trial will enroll 12 children and adults with Angelman syndrome, caused by various genetic factors such as deletions, uniparental disomy, and imprinting center defects. Participants will receive a single dose of MVX-220, injected directly into the fluid-filled spaces in the skull. The FDA's fast track designation may pave the way for accelerated approval and priority review.
The development of MVX-220 was initially supported by FAST, which later launched Mavrix Bio to facilitate its clinical testing. James M. Wilson, MD, PhD, who initiated the development of MVX-220 at the University of Pennsylvania, now serves as CEO of Gemmabio Therapeutics, overseeing its clinical development in partnership with Mavrix Bio.
The ASCEND-AS trial will evaluate the safety and efficacy of MVX-220, bringing hope to individuals with Angelman syndrome and their families. If successful, MVX-220 could offer a transformative treatment for this serious neurological condition.
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