voretigene neparvovec (Luxturna) Report issue

Gene therapy Orphan Drug FDA Approved FDA Breakthrough Therapy FDA

Active Ingredient History

  • Now
Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis. It was developed by Spark Therapeutics and Children's Hospital of Philadelphia. It is the first in vivo gene therapy approved by the US Food and Drug Administration (FDA).   Wikipedia

More Chemistry


Data collection and curation is an ongoing process for CDEK - if you notice any information here to be missing or incorrect, please let us know! When possible, please include a source URL (we verify all data prior to inclusion).

Report issue