MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome

First Patient Dosing Anticipated in Second Half of 2025 

Additional Details to be Provided by Webinar in Coming Months 

MIDDLETON, Mass., May 12, 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 U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for MVX-220, an investigational adeno-associated virus (AAV) gene therapy for the treatment of AS. MavriX Bio expects to initiate the "ASCEND-AS" first-in-human (FIH) clinical study of MVX-220 in the second half of 2025.

MVX-220 is designed to restore functional expression of the UBE3A gene in neurons, the underlying cause of AS, using targeted AAV delivery. The FIH study will evaluate the safety, tolerability, and efficacy of MVX-220 in adult and pediatric individuals living with various genotypes of AS, including some rare genotypes such as uniparental disomy and imprinting center defects.

"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."

MVX-220 was developed at the University of Pennsylvania with full support from the Foundation for Angelman Syndrome Therapeutics (FAST), which funded both the development and nonclinical research activities. The program was subsequently licensed to MavriX Bio, a portfolio company of FAST's drug development accelerator AS²Bio, to enable clinical translation in collaboration with GEMMABio, a therapeutics company focused on developing transformative gene therapies and ensuring global access to treatments.

"This collaboration represents the culmination of decades of partnership and scientific innovation aimed at addressing complex monogenic neurological disorders," said Jim Wilson, MD, PhD, President and CEO of GEMMABio. "We are excited to support MavriX Bio as they advance MVX-220 into clinical evaluation – an important milestone offering renewed hope for families affected by Angelman syndrome."

MavriX Bio will host a webinar in the coming months to provide additional details regarding the clinical trial design, eligibility criteria, and expected timelines. Registration details for the webinar will be available through the Foundation for Angelman Syndrome Therapeutics and the Angelman Syndrome Foundation closer to the time of the webinar.

About Angelman Syndrome
Angelman syndrome is a rare, non-degenerative, monogenic, neurologic disorder characterized by severe developmental delay, lack of verbal speech, sleep disturbances, seizures, motor and balance impairments, among other symptoms. It is caused by the loss of function of the UBE3A gene in neurons. Angelman syndrome is thought to affect 1:12,000-1:20:000 individuals. There are currently no approved treatments for Angelman syndrome.

About MVX-220
MVX-220 is an investigational hu68AAV gene therapy designed to deliver the human UBE3A gene to neurons of the brain with a single intra-cisterna magna (ICM) injection. It has been shown in preclinical studies to restore UBE3A protein expression and ameliorate symptoms in the AS mouse model. MVX-220 will be evaluated in the ASCEND-AS (AAV-mediated expression in neurons for Angelman syndrome) Phase 1/2 clinical trial.

About MavriX Bio
MavriX Bio is a biotechnology company dedicated to developing gene therapies for Angelman syndrome. By advancing world-class research and leveraging strategic partnerships, MavriX Bio is committed to delivering transformative treatments to patients in need.

For more information, please visit www.mvxbio.com

About AS²Bio
AS²Bio is drug development accelerator established by FAST to create an integrated approach to drug development in Angelman syndrome, leveraging collective expertise, resources, data and vital networks to provide a "bridge" for new technologies so they can move from proof-of-concept to early-stage clinical trials. MavriX Bio is one of the portfolio companies that AS²Bio supports.

For more information, please visit as2bio.com.

About GEMMABio
GEMMABio is a therapeutics company focused on advancing research and global access to life-changing advanced therapies for those living with rare diseases. The company provides research and product development functions to bring gene therapy discoveries from the bench to the bedside faster and affordably.

GEMMABio is led by gene therapy industry pioneer Jim Wilson and his team of experts, who previously conducted their work in academia. Wilson is also the Chairperson of Franklin Biolabs, a Contract Research Organization that provides a full range of services from discovery to clinical vector manufacturing to the global genetic medicines industry.

For more information, please visit gemmabiotx.com.

Media Contact:
MavriX Bio: www.mvxbio.com
[email protected]

SOURCE MavriX Bio