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www.fdanews.com/articles/173997-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.