The first two novel gene therapies for cancer treatment passed through FDA approval earlier this year, first B-cell acute lymphoblastic leukemia (ALL) followed quickly by Yescarta for large B-cell lymphoma – a type of non-Hodgkin lymphoma.
One more gene therapy squeaked in before year’s end, not for cancer treatment, but an inherited form of vision loss that can lead to blindness called biallelic RPE65 mutation-associated retinal dystrophy. The treatment is called Luxturna.
The RPE65 gene, named for it's location of expression in the eye, the retinal pigment epithelium or RPE, is responsible for making the enzyme isomerohydrolase which is responsible for making rhodopsin, a molecule necessary for normal vision. Mutations in RPE65 block this process and result in impaired vision.
Luxterna delivers a normal, non-mutated, copy of the RPE65 gene directly to the cells of the retina. This allows the enzyme to be made and restores vision. In the Phase 3 study, 31 participants were asked to navigate an obstacle course at various light levels. Navigating in the low light settings was significantly improved in the patients who received Luxterna.
With two cancer therapies approved last year, and now a third therapy for vision, 2017 has paved the way for many more that will most certainly be coming down the pike in 2018.
Read full, original post: One More Gene Therapy Approved Before Year’s End