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.
In this Monday, Oct. 2, 2017, photo, Beth Guardino, left, and her son, Christian, talk about the 17-year-old’s life before and after gene therapy treatment for his hereditary blindness during an interview at their home in Patchogue, N.Y. Christian was diagnosed with hereditary blindness and received gene therapy as part of a study. On Thursday, Oct. 12, U.S. Food and Drug Administration advisers will consider whether to recommend approval of the gene therapy. (AP Photo/Julie Jacobson)
In this Oct. 4, 2017 photo, Dr. Albert Maguire checks the eyes of Misa Kaabali, 8, at the Children’s Hospital of Philadelphia. Misa was 4 years old when he received his gene therapy treatment. On Thursday, Oct. 12, U.S. Food and Drug Administration advisers will consider whether to recommend approval of the gene therapy.(AP Photo/Bill West)
In this Monday, Oct. 2, 2017, photo, sunlight reflected off a window lights up Christian Guardino’s face, as he stands for a portrait outside his home in Patchogue, N.Y. Christian was diagnosed with hereditary blindness and received gene therapy as part of a study. On Thursday, Oct. 12, U.S. Food and Drug Administration advisers will consider whether to recommend approval of the gene therapy. Before treatment, “It was dark, life without light,” Christian said. “I found a way to work through it, to cope with it, and that was music.” (AP Photo/Julie Jacobson)
In this Monday, Oct. 2, 2017, photo, Beth Guardino, left, and her son, Christian, talk about the 17-year-old’s life before and after gene therapy for his hereditary blindness during an interview at their home in Patchogue, N.Y. Christian was diagnosed with hereditary blindness and received gene therapy as part of a study. On Thursday, Oct. 12, U.S. Food and Drug Administration advisers will consider whether to recommend approval of the gene therapy. (AP Photo/Julie Jacobson)
In this Monday, Oct. 2, 2017, photo, Beth Guardino, left, and her son, Christian, look through old family photos during an interview at their home in Patchogue, N.Y. Christian was diagnosed with hereditary blindness and received gene therapy as part of a study. On Thursday, Oct. 12, U.S. Food and Drug Administration advisers will consider whether to recommend approval of the gene therapy. (AP Photo/Julie Jacobson)
In this Monday, Oct. 2, 2017, photo, Beth Guardino, left, hugs her son, Christian, as they talk about the 17-year-old’s life before and after gene therapy for his hereditary blindness during an interview at their home in Patchogue, N.Y. Christian was diagnosed with hereditary blindness and received gene therapy as part of a study. On Thursday, Oct. 12, U.S. Food and Drug Administration advisers will consider whether to recommend approval of the gene therapy. (AP Photo/Julie Jacobson)
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