FDA approves gene therapy for inherited blindness developed by the University of Pennsylvania and Children's Hospital of Philadelphia

(University of Pennsylvania School of Medicine) In a historic move, the US Food and Drug Administration (FDA) today approved a gene therapy initially developed by researchers at the University of Pennsylvania and Children's Hospital of Philadelphia (CHOP) for the treatment of a rare, inherited form of retinal blindness. The decision marks the nation's first gene therapy approved for the treatment of a genetic disease, and the first in which a new, corrective gene is injected directly into a pati...