Newly FDA-approved Luxturna is expected to be available in selected treatment centers in the first quarter of 2018, according to a press release from Spark Therapeutics.
Luxturna (voretigene neparvovec) is the first directly administered gene therapy approved in the United States to target a disease caused by mutations in a specific gene, in this case biallelic RPE65 mutation-associated retinal dystrophy.
Jeffrey D. Marrazzo, CEO at Spark Therapeutics, called the approval of the one-time gene therapy a “landmark” moment. According to the release, the therapy represents a trio of
Uncategorized