Gene Therapy for Angelman Syndrome Cleared by FDA for First-in-Human Trial

MavriX Bio announced that it has received Food and Drug Administration (FDA) clearance for its Investigational New Drug (IND) application for MVX-220 (MavriX Bio, Middleton, MA), an adeno-associated virus (AAV) gene therapy for Angelman syndrome. MVX-220 is intended to restore neuronal UBE3A gene expression; the development of Angelman syndrome is associated with loss of function of the maternal UBE3A gene. In a press release, MavriX Bio stated that following the FDA’s decision to clear the therapy, the company intends to initiate a fist-in-human, Phase 1/2 clinical trial called ASCEND-AS in the second half of 2025.

The planned ASCEND-AS clinical trial will assess the safety, tolerability, and preliminary efficacy of MVX-220 in both adult and pediatric individuals with Angelman syndrome. Importantly, it will include individuals with rare genotypes such as uniparental disomy and imprinting center defects, expanding beyond the typical maternal deletion cohort.

"We are thrilled to achieve this critical milestone and move one step closer to delivering the first gene therapy to individuals living with Angelman syndrome," said Allyson Berent, DVM, DACVIM, Chief Development Officer of MavriX Bio. "The clearance of our IND signals a key transition point for our development program and our unwavering commitment to advance novel therapeutic options and a potential single treatment therapy for the Angelman community."