Clinical trial of gene therapy for Angelman syndrome set to commence in 2022

In a significant development for families affected by Angelman syndrome, Mavrix Bio's experimental gene therapy, MVX-220, has received approval from the U.S. Food and Drug Administration (FDA) to start a first-in-human clinical trial.

The approval marks a key transition point for Mavrix Bio's development program for Angelman syndrome. Mavrix Bio, launched by the Foundation for Angelman Syndrome Therapeutics, was developed at the University of Pennsylvania with funding from the same foundation.

Mavrix will be leading clinical development of MVX-220 in collaboration with Gemmabio, a company focusing on rare diseases. Jim Wilson, MD, PhD, president and CEO of Gemmabio, expressed excitement about supporting Mavrix Bio as they advance MVX-220 into clinical evaluation.

MVX-220 is designed to deliver a healthy copy of the UBE3A gene to the body's cells using an AAV vector, a harmless, engineered virus. The clinical trial will evaluate the safety, tolerability, and efficacy of MVX-220 in children and adults with Angelman syndrome who have various genetic anomalies.

The chief development officer of Mavrix Bio, Allyson Berent, expressed excitement about moving closer to delivering the first gene therapy for Angelman syndrome. Mavrix Bio plans to initiate the clinical trial later this year, with the study design and expected timeline for the trial to be announced in the coming months.

Mavrix Bio is committed to advancing novel therapeutic options and a potential single treatment therapy for the Angelman community. The collaboration between Mavrix and Gemmabio is a culmination of decades of partnership and scientific innovation aimed at addressing complex monogenic neurological disorders.

This development offers renewed hope for families affected by Angelman syndrome, a rare genetic disorder characterised by severe intellectual disability, speech impairment, and frequent seizures. More updates on the clinical trial are expected in the near future.