The US Food and Drug Administration (FDA) has given the go-ahead to Spark Therapeutics' gene therapy for treating biallelic RPE65-mutation-associated retinal dystrophy. The December 19 approval is the first gene therapy in the US to use an adeno-associated virus for delivery and the first for treating an inherited disease. Voretigene neparvovec, branded as Luxturna, received a unanimous vote of approval from an FDA advisory committee on October 12 (Nat. Biotechnol. 35, 998, 2017). In concert with the milestone approval, the FDA announced that it is developing a policy framework for processing candidate gene therapies. “Next year, we'll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters—including new clinical measures—for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted,” FDA Commissioner Scott Gottlieb said in a statement announcing the approval of Luxturna. Biallelic RPE65-mutation-associated retinal dystrophy affects an estimated 1,000–2,000 people in the US. The condition causes progressive sight impairment that leads to near total blindness in almost all patients. Luxturna is a one-time treatment. It is injected into the subretinal space where it delivers a functional copy of RPE65 cDNA to retinal pigment epithelial (RPE) cells, restoring the cells' ability to produce the key retinoid cycle enzyme all-trans retinyl ester isomerase. Spark Therapeutics announced that Luxturna will be available in the first quarter of 2018 from retinal surgeons trained by the company. Spark Therapeutics said it will announce pricing in early January. Industry estimates of the price range from $500,000 to $1.5 million, based partly on the $475,000 price tag for Novartis' adoptive chimeric antigen receptor (CAR)-T cell leukemia therapy Kymriah (tisagenlecleucel), which is also a one-time gene therapy treatment. The European Medicines Agency is reviewing Luxturna. Spark Therapeutics has gene therapies for treating hemophilia and neurodegenerative diseases in its pipeline.