Advisers to the U.S. Food and Drug Administration will meet Thursday to consider whether to recommend approval of a gene therapy aimed at improving vision for some people with hereditary blindness.
It 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 a patient. Only one gene therapy is sold in the U.S. now, a cancer treatment approved in August.
The eye treatment, called Luxturna, is made by Philadelphia-based Spark Therapeutics. A study of 29 patients found it improved vision for nearly all of those given it and seemed safe. It's intended to be given just once, supplying a gene to make a protein needed for sight that people with the defective gene lack.
Marilynn Marchione, The Associated Press
