Spark Therapeutics (NASDAQ:ONCE) may be a household name in gene therapy if the Food and Drug Administration’s (FDA) approves its new therapy for an inherited retinal disease that causes blindness.
Spark has successfully completed clinical trials for the treatment called voretigene neparvovec, which cures a mutation in the RPE65 gene. This ground-breaking therapy is a one time injection of an Adeno-Associated Viral Vector into the mutated RPE65 gene.
