The U.S. Food and Drug Administration today approved first directly administered gene therapy, Luxturna (voretigene neparvovec-rzyl) to treat children and adult patients with an inherited form of vision loss that may result in blindness.Luxturna is an virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Treating physician must determine the presence of viable retinal cells before prescribing it.
Today’s approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb,
Approximately 600 individuals with Leber congenital amaurosis (LCA2) could be candidates for gene therapy aimed at treating biallelic RPE65 mutations.
Gene therapy is an experimental technique that uses genes to treat or prevent disease.
Approaches used for gene therapy are:
Source of image: imdb