https://www.businesswire.com/news/home/20200811005348/en/
https://www.neurogene.com/press-releases/neurogene-announces-fda-orphan-drug-designation-for-cln7-batten-disease-gene-therapy/
Neurogene Announces FDA Orphan Drug Designation for CLN7 Batten Disease Gene Therapy
FDA grants Orphan Drug Designation to Neurogene’s adeno-associated virus vector with engineered transgene encoding the human CLN7 gene
August 11, 2020 09:00 AM Eastern Daylight Time
NEW YORK–(BUSINESS WIRE)–Neurogene Inc., a company founded with a mission to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to adeno-associated virus vector with engineered transgene encoding the human CLN7 gene for patients with CLN7, a form of Batten disease. Batten disease, a common name for a rare class of diseases called neuronal ceroid lipofuscinoses (NCLs), affects an estimated 2-4 out of every 100,000 children in the United States. In July, the company announced it had received Orphan Drug Designation from the FDA for its gene therapy for the treatment of CLN5 Batten disease.
