FDA Approve Gene Therapy for Blindness
After voting in favour for the blindness gene therapy, the Food and Drug Administration (FDA) have announced approval for the therapy that treats a rare form of blindness.
According to Newsweek, the drug, Luxturna has been created to treat a genetic form of blindness called retinal blindness that affects approximately 1,000 to 2,000 people in the United States.
Chief scientific officer at the Foundation Fighting Blindness, which funded some of the preliminary research on the therapy, Stephen Rose, explained, “It really is a trailblazer for using gene deliver. Now, there’s a map that shows how you do it, what needs to be done, what the FDA and other regulatory groups are going to be looking for. It provides that proof of concept and proof of principle in humans that it’s doable.”
With FDA Commissioner, Scott Gottlieb referring to this year as an “inflection point in medicine in and health,” the approval has the potential to open the door to approvals of other gene-based therapies.
The treatment is based on putting extra copies of a gene into retinal cells, which are then delivered through a virus that’s injected underneath a patient’s retina. Then, fromt hose copies of the gene, called RPE-65, cells’ own machinery can create more of the protein that they’re missing. In this particular case, that protein is vital to retinal cells’ survival and ability to send signals to the brain. Luxturna doesn’t permanently modify the DNA sequence of cells, therefore that means that if the cell does eventually break down those spare copies of the gene, then the cells will wind up right back where they started, with not enough protein being made.
Director of the FDA’s Center for Biologics Evaluation and Research, Dr. Peter Marks, believes that each approval has helped the agency understand what exactly needs to be evaluated, specifically the way treatments are manufactured and the monitoring that’s done after patients are treated.
He said, “I think this is helping the agency develop an increasing amount of knowledge that will help us. Obviously, every new product will have new aspects and new challenges to it.”
A recent press release from the University of Pennsylvania, confirmed that 41 patients in the United States have been treated with the therapy so far. One of the leaders of the research behind the therapy, Dr. Jean Bennett, added, “It has been amazing watching them grow up. It’s like they are an extended part of our family.”
Luxturna will be offered at a few centres across the country in the future.