<unk> , 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). Leber's congenital amaurosis, or biallelic RPE65-mediated inherited retinal disease, is an inherited disorder causing progressive blindness.<unk>  is the first treatment available for this condition. The gene therapy is not a cure for the condition, but substantially improves vision in those treated. It is given as a subretinal injection.<unk>  was approved for medical use in Australia in August 2020 and in Canada, in October 2020.
Voretigene neparvovec
Vibegron
Ofranergene obadenovec
Mestranol/noretynodrel
Enadenotucirev
Seviteronel
Crofelemer
Telotristat ethyl