FDA Clears Mavrix Bio's MVX-220 for Angelman Syndrome Trial
Mavrix Bio's MVX-220, an investigational gene therapy for Angelman syndrome, has received the FDA's green light to start the ASCEND-AS trial. This rare disorder affects around 1 in 15,000 people, causing severe neurological symptoms. The trial aims to treat the root cause by delivering a functional UBE3A gene directly to the brain.
MVX-220 will be administered via intrathecal injection, targeting the fluid-filled spaces in the skull for direct brain delivery. The Phase 1/2 ASCEND-AS trial, set to take place at Rush University Medical Center in Chicago, will enrol 12 children and adults with Angelman syndrome. Participants will be monitored for five years.
The FDA has granted fast track designation to MVX-220, recognizing the unmet medical need for Angelman syndrome. This designation aims to expedite the clinical development and regulatory review process. James Wilson, founder of Gemmabio, is involved in the MVX-220 development program, likely in a leadership or scientific role.
A webinar hosted by the Foundation for Angelman Syndrome Therapeutics (FAST) and the Angelman Syndrome Foundation on Oct. 9 at 1 p.m. EST will discuss the study's design, enrollment criteria, and protocol. FAST played a crucial role in the early development of MVX-220 and launched Mavrix Bio to advance the treatment into clinical testing.
The ASCEND-AS trial marks a significant step towards potentially treating Angelman syndrome at its genetic root. With the FDA's fast track designation and the support of FAST and Gemmabio, Mavrix Bio is poised to make progress in this rare disease area.
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