MavriX Bio Announces FDA Rare Pediatric Disease Designation for Investigational MVX-220 for the Treatment of Angelman Syndrome
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MavriX Bio Announces First Patient Dosed in ASCEND-AS Trial of MVX-220, Investigational Gene Therapy for Angelman Syndrome
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MavriX Bio Receives FDA Fast Track Designation for MVX-220 for Treatment of Angelman Syndrome
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Jim Wilson move from UPENN to Biotech and what this means for Angelman syndrome.
“This is a great decision by Dr. Wilson and that of The University of Pennsylvania to get the development of these gene therapies out of academia and into a well-resourced biotech environment that can execute more efficiently. This is exciting news for rare disease and for other companies that can now leverage the expertise of […]
FAST to Advance Gene Therapy Candidate through IND-Enabling Studies Conducted in Partnership with the University of Pennsylvania
The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome (AS). Angelman syndrome is a rare neurogenetic disorder that is estimated to impact approximately […]