FDA Approves First-Ever Gene Therapy For Treating An Inherited Form Of Blindness


The Food and Drug Administration approved on Tuesday the first-ever gene therapy for treating an inherited disease known as retinal dystrophy, which is a type of blindness. This potential medical breakthrough could pave way for patients to see things around them, including their parents’ faces.

The new treatment is called Luxturna, which is a drug that tells the cells to generate an enzyme needed for normal vision. Retinal dystrophy is a rare disease that damages cells in the retina, causing blindness, according to NPR.

The new drug carries a healthy gene that is applied to eyes of those with retinal dystrophy. It is reported that the said form of blindness affects between 1,000 and 2,000 people in the United States and probably a number of people around the world, as reported by Reuters.

FDA Commissioner Scott Gottlieb said that approval signifies another first in the field of gene therapy. He further said that this milestone strengthens the potential of this discovery in healing many challenging and rare diseases.

This inherited disease is caused by defects in a gene called RPE65. Luxturna then delivers about 150 billion viral vector particles that have a correct copy of the RPE65 gene to retinal cells. This could restore their capacity to generate the needed enzyme. It could be applied to both children and adults with a damaged RPE65 gene. The new drug must be given only once. However, it is not clear how long it will ultimately last, according to Reuters.


There are clinical trials conducted in examining the proficiency of Luxturna. The results indicated that about 93 percent of the participants had improvement in their visions.

The developer of Luxturna is Spark Therapeutics, which has yet to reveal the cost it will charge. However, it is expected that it might cost at least $1 million to treat a patient, according to NPR.

Gene therapy has been making breakthrough lately in the field of medicine. It is reported that FDA just recently approved Kymriah, the first gene-therapy for treating leukemia. And in October, it also certified the Yescarta, a second gene-based treatment that could treat a form of lymphoma.