luxturna
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FDA OKs first gene therapy for hereditary disease
The FDA has approved a new gene therapy that proves the technique can also be used to treat a variety of diseases other than cancer. According to the agency's announcement, the therapy called Luxturna can treat biallelic RPE65 mutation-associated retinal dystrophy, a rare eye disease that leads to blindness, in both adults and children. This is the first time the agency approved a gene therapy designed to treat a hereditary illness and the first to target a disease caused by mutations in a specific gene -- it's a major milestone in the field and a huge victory for its proponents.
