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Home » FDA Awards Orphan Therapy Designation to Agilis’ Angelman Syndrome Therapy
FDA Awards Orphan Therapy Designation to Agilis’ Angelman Syndrome Therapy
November 10, 2015
The FDA has granted orphan status to Agilis Biotherapeutics’ AGIL-AS, a gene therapy candidate being developed as a treatment for the neuro-genetic disorder Angelman syndrome.
The disorder is characterized by severe intellectual and developmental disability, and the therapy delivers a UBE3A gene to correct neurological deficits in patients with the disease.
The product is the first to be granted orphan status for the syndrome, according to the Cambridge, Mass.-based company.
Angelman syndrome affects roughly 1 in 15,000 live births, according to the Foundation for Angelman Syndrome Therapeutics.
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