FDA Awards Orphan Therapy Designation to Agilis’ Angelman Syndrome Therapy
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.