FDA Grants Gene Therapy Orphan Drug Status for Rare Genetic Disorder
Agilis Biotherapeutics announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational gene therapy AGIL-AS for the treatment of Angelman syndrome (AS), a rare neuro-genetic disorder characterized by severe intellectual and developmental disability.
The Company entered into an exclusive worldwide license agreement with the University of South Florida (USF) in May 2015 for the treatment of AS using gene therapy technology developed by Edwin Weeber, PhD, Professor, Molecular Pharmacology and Physiology, Director of the Neurobiology of Learning and Memory, and Chief Scientific Officer at the USF Health Byrd Alzheimer's Research Institute. Dr. Weeber and colleagues have established efficacy of UBE3A gene therapy in the central nervous system of AS models.
AGIL-AS, the first drug to be granted an Orphan Drug designation by the FDA for Angelman syndrome, delivers a corrective UBE3A gene to rescue neurological deficits in patients suffering from this rare disease.
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