The US Food and Drug Administration is set to decide whether Luxturna, a gene therapy developed by Spark Therapeutics for people with retinal disorders, should be granted approval. The majority of these people would eventually lose their current level of vision entirely without treatment, and there are currently no drugs designed for people with an RPE65 mutation that are approved by the FDA. Dispensed via injection, the Luxturna therapy corrects the RPE65 gene, enabling the creation of a protein essential to eyesight. Spark said that more than two dozen people who have undergone the treatment experienced an improvement in their vision. In another development, researchers at Stanford University announced on Monday they had discovered how to use CRISPR gene-editing technology to edit genes inside human cells.
The US Food and Drug Administration is set to decide whether Luxturna, a gene therapy developed by Spark Therapeutics for people with retinal disorders, should be granted approval. Dispensed via injection, the Luxturna therapy corrects the RPE65 gene, enabling the creation of a protein essential to eyesight. Spark said that more than two dozen people who have undergone the treatment experienced an improvement in their vision. In another development, researchers at Stanford University announced on Monday they had discovered how to use CRISPR gene-editing technology to edit genes inside human cells.