The first gene therapy for an inherited disease approved by the Food and Drug Administration was Luxturna, a treatment for a form of blindness caused by genetic mutations.
The landmark clearance, coupled with the advantages of targeting the eye rather than other, harder-to-reach organs, helped spur development of many other gene therapies for ocular disorders. A number of those treatments are now advancing into later stages of clinical testing, including J&J and MeiraGTx’s.
