#United States #Middleton #Angelman Syndrome #MVX-220 #MavriX Bio

MavriX Bio Launches Pioneering Gene Therapy Trial for Angelman Syndrome

MavriX Bio, a clinical-stage biotech firm, recently announced a significant milestone in genetic medicine—the commencement of patient dosing in the ASCEND-AS trial for MVX-220, a novel investigational gene therapy specifically targeting Angelman Syndrome (AS). This groundbreaking event is not only pivotal for MavriX but marks an unprecedented development in the landscape of treatments for AS, a rare neurogenetic disorder characterized by severe cognitive delays and motor difficulties.

What is Angelman Syndrome?

Angelman Syndrome affects approximately 1 in 12,000 to 20,000 individuals globally and is caused by the loss of function of the UBE3A gene, leading to debilitating symptoms that include developmental delays, speech impairment, and seizures. Currently, there are no existing therapies that modify the disease, thus creating a pressing need for innovative treatments.

First Patient Dosing in the ASCEND-AS Trial

The Phase 1/2 ASCEND-AS clinical trial, designated as NCT07181837, signifies the inaugural evaluation of a gene therapy aimed at rectifying the underlying genetic issues associated with AS. Stage one of the trial will assess the safety, tolerability, and preliminary efficacy of MVX-220 across a mixed cohort of adult and pediatric participants, representing various AS genotypes, such as deletion, uniparental disomy, and imprinting center defects.

At the core of MVX-220’s mechanism is a targeted AAV (adeno-associated virus) delivery system, which seeks to restore normal UBE3A gene expression in neuronal tissues. This innovative approach is anticipated to be a game-changer in how AS is treated, potentially improving the quality of life for those affected by the syndrome.

FDA’s Orphan Drug Designation

In conjunction with the trial's commencement, MavriX Bio has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for MVX-220 for the treatment of AS. This prestigious designation not only underlines the therapy's promise but also enhances the efficiency of its development by providing benefits including market exclusivity and potential tax incentives.

Jennifer Panagoulias, Chief Operating Officer of MavriX Bio, emphasized the importance of this milestone, stating, "Dosing our first participant in the ASCEND-AS trial is a critical step in evaluating the potential of gene-targeted therapies for AS. We owe this achievement to the relentless dedication of the Angelman community and our scientific collaborators."

Expert Insights on MVX-220

Dr. Elizabeth Berry-Kravis, a notable pediatric neurologist and the Principal Investigator for the ASCEND-AS trial, expressed optimism regarding the trial's implications. She noted, "For the first time, we are testing a one-time therapeutic approach that directly tackles the genetic cause of Angelman syndrome by restoring UBE3A expression in neurons. This could redefine standards for treating AS and serve as a blueprint for addressing other genetic neurodevelopmental disorders."

MavriX Bio and AS2Bio Collaboration

MVX-220 stems from a collaborative research effort that began at the University of Pennsylvania, backed by the Foundation for Angelman Syndrome Therapeutics (FAST). Subsequent advancements in the therapy were facilitated through AS2Bio, a drug development accelerator founded to accelerate innovation in therapies for Angelman syndrome. MavriX Bio has obtained the rights to MVX-220 from AS2Bio and is dedicated to bringing it through clinical phases, aiming for effective solutions for individuals with AS.

A Promising Future for Angelman Syndrome Treatment

With the ascent of MVX-220 through the clinical trial process, families grappling with Angelman Syndrome may finally find hope. By bridging significant knowledge gaps and pioneering gene therapy approaches, MavriX Bio is setting a new benchmark in the quest for effective treatments for this challenging disorder. As the trial progresses, the outcomes will be closely watched by both the scientific community and families affected by AS. The potential for MVX-220 to transform the treatment landscape for Angelman Syndrome is indeed significant, offering new possibilities for the future of genetic medicine.