FDA Advisers Endorse Gene Therapy To Treat A Form Of Blindness
A potentially groundbreaking treatment for a rare form of blindness moved one step closer to U.S. approval, as federal health advisers endorsed the experimental gene therapy for patients with an inherited condition that gradually destroys eyesight.
The panel experts to the FDA voted unanimously in favor of Spark Therapeutics’ injectable therapy, which aims to improve vision by replacing a defective gene needed to process light.
Inherited retinal diseases (IRDs) are a group of rare blinding conditions caused by one of more than 220 different genes. Some people living with IRDs experience a gradual loss of vision, eventually leading to complete blindness. Others may be born with or experience vision loss in infancy or early childhood.
If approved, Luxturna would be the first gene therapy in the U.S. for an inherited disease and the first in which a corrective gene is given directly to patients
Doctors deliver the therapy with an injection in each eye that inserts a replacement gene into the retina via a modified virus.
In their experimental genetic study, one year after treatment…