Voretigene neparvovec ( Luxturna ) is a novel gene therapy for the treatment of Leber’s 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 FDA. 
Leber’s congenital amaurosis, or biallelic RPE65- mediated inherited retinal disease, is an inherited progressive progressive blindness. Voretigene is the first treatment for this condition. The gene therapy is not a cure for the condition, but rather improves vision in those treated.  It is given as an subretinal injection.
The price of the treatment has not been announced, but it is expected to cost around $ 1 million.  
Voretigene neparvovec is an AAV2 vector containing human RPE65 cDNA with a modified Kozak sequence . The virus is grown in HEK 293 cells and purified for administration. 
It was granted orphan drug status for Leber congenital amaurosis and retinitis pigmentosa .  [ better source needed ] A biologics license application was submitted to the FDA in July 2017 with Priority Review .  Phase III clinical trial results were published in August 2017.  On October 12, 2017, a key advisory panel to the Food and Drug Administration (FDA) , composed of 16 experts, unanimously recommended approval of the treatment.  The US FDA approved the drug on December 19, 2017. With the approval, Spark Therapeutics received apediatric disease priority review voucher . 
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