Mito
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Gene Therapy for Blindness Appears Initially Effective, Says U.S. FDA
Spark Therapeutics Inc’s experimental gene therapy for a rare inherited form of blindness is effective, though it is unclear whether the benefit lasts over time, according to a preliminary review by the U.S. Food and Drug Administration.
The therapy, Luxturna, or voretigene neparvovec, would be the first-ever gene therapy for any inherited disease to be approved in the United States.
Inherited retinal diseases are a group of rare conditions caused by mutations in one or more than 220 different genes, including one known as RPE65, which tells cells how to produce a crucial enzyme needed for normal vision
https://gizmodo.com/an-fda-panel-just-greenlit-a-breakthrough-gene-therapy-1819412787
A panel of U.S. health advisers has endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine.
A panel of experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics' injectable therapy, which aims to improve vision in patients with a rare mutation that gradually destroys normal vision. The vote amounts to a recommendation to approve the therapy.
If approved, it would be the first gene therapy in the U.S. for an inherited disease, offering hope to patients with a variety of related conditions.
Spark Therapeutics Inc’s experimental gene therapy for a rare inherited form of blindness is effective, though it is unclear whether the benefit lasts over time, according to a preliminary review by the U.S. Food and Drug Administration.
The therapy, Luxturna, or voretigene neparvovec, would be the first-ever gene therapy for any inherited disease to be approved in the United States.
Inherited retinal diseases are a group of rare conditions caused by mutations in one or more than 220 different genes, including one known as RPE65, which tells cells how to produce a crucial enzyme needed for normal vision
https://gizmodo.com/an-fda-panel-just-greenlit-a-breakthrough-gene-therapy-1819412787
A panel of U.S. health advisers has endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine.
A panel of experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics' injectable therapy, which aims to improve vision in patients with a rare mutation that gradually destroys normal vision. The vote amounts to a recommendation to approve the therapy.
If approved, it would be the first gene therapy in the U.S. for an inherited disease, offering hope to patients with a variety of related conditions.
