Neurogene’s Gene Therapy for CLN5 Named Orphan Drug by FDA
The U.S. Food and Drug Administration (FDA) has designated Neurogene’s gene therapy, which aims to deliver a working copy of the human CLN5 gene, an orphan drug to possibly treat CLN5, a form of late-infantile Batten disease. Orphan drug status helps to encourage therapies for rare and serious diseases,…