In 2017, we witnessed gene therapy breakthroughs up to the very end. In the final weeks of the year, the first in vivo gene therapy, Luxturna, was approved by the FDA. Luxturna is intended to improve sight for patients with rare forms of inherited vision loss.
Luxturna is administered through a one-time injection into a patient’s eye that replaces the missing genetic material with a synthetic version. This treatment delivers a functioning copy of the RPE65 gene which enables cells to produce a protein necessary for eyes to process light.
This monumental treatment is the first gene therapy approved in the U.S. that alters genes in the patient’s own body rather than in the lab.
This technology holds tremendous potential for the entire medical field. To learn more about this approval, view the FDA News Release.