MavriX Bio Announces First Patient Dosed in ASCEND-AS Trial of MVX-220, Investigational Gene Therapy for Angelman Syndrome

First Gene Therapy Trial for Angelman Syndrome

-Orphan Drug Designation Granted for MVX-220

Excerpt from the Press Release:

MIDDLETON, Mass., Nov. 6, 2025 /PRNewswire/ — MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the first patient has been dosed in the Phase 1/2 ASCEND-AS clinical trial of MVX-220, an investigational gene therapy for AS.

The ASCEND-AS trial (NCT07181837) represents the first clinical evaluation of a gene therapy for AS. Supported by the Foundation for Angelman Syndrome Therapeutics (FAST) and AS²Bio, MavriX Bio developed MVX-220 to restore functional expression of the UBE3A gene in neurons using targeted AAV delivery to the central nervous system. The trial will evaluate safety, tolerability, and efficacy of MVX-220 in both adult and pediatric participants with different AS genotypes, including deletion, uniparental disomy, and imprinting center defects.

In addition to this important clinical milestone, MavriX Bio announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for MVX-220 for treatment of AS. Orphan Drug Designation is a status granted to support the development of investigational therapies intended for the treatment of rare diseases that affect fewer than 200,000 people in the US. This designation confers various benefits related to market exclusivity, waiver or reduction of application fees, and certain tax credits.

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