October 22, 2024
ASF Funded Research leads to a collaboration for gene therapy to correct deficiency in UBE3A gene
Asklepios BioPharmaceutical, Inc. (AskBio), a leading clinical-stage adeno-associated virus (AAV) gene therapy company, entered into a research collaboration and licensing agreement with the University of North Carolina at Chapel Hill (UNC) for the development and commercialization of gene therapy for Angelman syndrome.
The UNC School of Medicine team, led by Ben Philpot, PhD, and Mark Zylka, PhD, has found preclinical evidence that gene therapy may help individuals with AS by dramatically improving seizure and motor outcomes.
The ASF is proud of its history funding the work on Dr. Philpot and Dr. Zylka that has lead to this collaboration.
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The mission of Angelman Syndrome Foundation is to advance the awareness and treatment of Angelman syndrome through education and information, research and support for individuals with Angelman syndrome, their families and other concerned parties. We exist to give all of them a reason to smile, with the ultimate goal of finding a cure.