It's been a truly historic year for modern medicine with the FDA for the first time approving several gene therapies for public use. The latest approval, this time for treating a rare form of inherited vision loss, marks 2017 as a milestone year in the field of medicine.
The new therapy is called Luxturna, and it targets a rare inherited retinal disease called Leber congenital amaurosis (LCA). Sufferers of this condition experience a progressive deterioration of vision due to a mutation in the RPE65 gene, which is responsible for producing a protein vital for eyesight.
The treatment attaches a healthy version of that gene to a genetically modified harmless virus that is injected into the patient's eye, resulting in the retinal cells successfully generating the missing protein.
The FDA tick comes after the groundbreaking approval of the first gene therapy for public use back in August. Called Kymriah, this extraordinarily expensive and controversial cancer treatment uses genetically engineered immune cells to target and kill specific cancer cells.
Luxturna, on the other hand, is the first gene therapy to be approved by the FDA that works by fixing missing or mutated genes that cause disease by simply inserting a healthy replacement.
"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," says FDA Commissioner Scott Gottlieb.
Research into a wide range of genetic diseases is surging at the moment and 2017 has been the first year these treatments have crossed that threshold moving from clinical research experiments to medicines delivered to the general public.
"I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses," says Gottlieb.
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