MavriX Bio Gets FDA Approval for First Human Trial of Angelman Syndrome Gene Therapy

MavriX Bio, a biotechnology firm specializing in advanced genetic therapies for Angelman syndrome (AS), has announced that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for MVX-220. This investigational gene therapy, utilizing adeno-associated virus (AAV) technology, aims to treat AS, and the company plans to start its first-in-human (FIH) clinical trial, termed "ASCEND-AS," in the latter half of 2025.

The focus of MVX-220 is to restore the functional expression of the UBE3A gene in neurons, a crucial step in addressing the root cause of Angelman syndrome. The upcoming clinical study will assess the safety, tolerability, and effectiveness of MVX-220 in both adult and pediatric patients with various AS genotypes, including rare types like uniparental disomy and imprinting center defects.

Allyson Berent, Chief Development Officer at MavriX Bio, expressed excitement over reaching this significant milestone, emphasizing the company's dedication to providing innovative therapeutic solutions and potential single-treatment options for those with Angelman syndrome. The approval of the IND marks a pivotal moment for their development program.

The creation of MVX-220 was facilitated at the University of Pennsylvania, financially backed by the Foundation for Angelman Syndrome Therapeutics (FAST). The development and foundational research were supported by FAST before the initiative was licensed to MavriX Bio. MavriX Bio is part of AS2Bio, FAST's drug development accelerator, which aids in the clinical translation in collaboration with GEMMABio, a therapeutics firm committed to advancing transformative gene therapies.

Jim Wilson, President and CEO of GEMMABio, highlighted the collaboration as the outcome of extensive partnerships and scientific advancements targeting complex monogenic neurological disorders. He emphasized the importance of MVX-220 advancing to clinical trials, signifying renewed hope for families impacted by Angelman syndrome.

In the coming months, MavriX Bio plans to host a webinar to share more information about the clinical trial's design, eligibility criteria, and expected timelines. Details on registration will be made available through FAST and the Angelman Syndrome Foundation as the webinar date approaches.

Angelman syndrome is a rare neurological disorder characterized by profound developmental delay, absence of verbal speech, sleep problems, seizures, and motor and balance difficulties, among other symptoms. The condition results from the loss of function of the UBE3A gene in neurons and affects approximately 1 in 12,000 to 20,000 individuals. Currently, there are no approved treatments for Angelman syndrome.

MVX-220 represents a novel investigational gene therapy designed to deliver the human UBE3A gene to brain neurons via a single intra-cisterna magna injection. Preclinical studies have indicated that MVX-220 can restore UBE3A protein expression and alleviate symptoms in an AS mouse model. The therapy will undergo evaluation in the ASCEND-AS Phase 1/2 clinical trial.

MavriX Bio is dedicated to developing gene therapies for Angelman syndrome, advancing top-tier research, and forging strategic alliances to offer transformative treatments to patients in need.

AS2Bio acts as a drug development accelerator, established by FAST to provide an integrated drug development approach for Angelman syndrome. It draws on collective expertise, resources, and networks to serve as a "bridge" for new technologies from proof-of-concept to early-stage clinical trials. MavriX Bio is among the portfolio companies supported by AS2Bio.

GEMMABio focuses on advancing research and ensuring global access to life-changing therapies for rare diseases, facilitating the transition of gene therapy discoveries from research to practical application.