Spark Therapeutics

FDA approval of retinal dystrophy drug launches era of ocular gene therapyVoretigene neparvovec-rzyl (Luxturna, Spark Therapeutics) was approved in December 2017 for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Clinical trial results and patient selection issues for this gene therapy are discussed.
Blindness gene therapy price tag stirs controversyA patient advocacy group is criticizing the hefty price tag of a new treatment for blindness.
Gene therapy benefits for retinal dystrophies persist through trialResults from 3 years of follow-up in the phase III trial investigating treatment with adeno-associated viral vector delivery of human RPE65 (voretigene neparvovec, Spark Therapeutics) show this gene therapy has an acceptable safety profile.