FDA Grants Fast Track to Mavrix Bio's Gene Therapy for Angelman Syndrome
Mavrix Bio's investigational gene therapy, MVX-220, has received fast track designation from the FDA for treating Angelman syndrome. The therapy aims to deliver a healthy copy of the UBE3A gene to brain cells using a viral carrier. The first-in-human clinical trial, ASCEND-AS, is set to begin at Rush University Medical Center in Chicago, supported by Gemmabio and FAST.
MVX-220 is intended for Angelman syndrome, a rare genetic disorder caused by the absence or dysfunction of the UBE3A gene. The ASCEND-AS trial will evaluate the safety and efficacy of MVX-220 in 12 participants over five years. It will include individuals with different genetic causes of Angelman syndrome, involving a single dose injected into the fluid-filled spaces in the skull.
The trial's launch was greenlit by the FDA in May. Marian, Inc. is developing the therapy, with FAST supporting its early development and launching Mavrix Bio to move it into clinical testing.
The fast track designation for MVX-220 aims to speed up its clinical development and regulatory review. If successful, the ASCEND-AS trial could pave the way for a new treatment option for people living with Angelman syndrome.
