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Applied Genetic's gene therapy for eye disease gets US FDA orphan designation

The U.S. Food and Drug Administration granted orphan drug designation for Applied Genetic Technologies Corp.'s gene therapy product candidate to treat X-linked retinitis pigmentosa caused by mutations in the RPGR gene.

X-linked retinitis pigmentosa is an inherited condition that causes progressive vision loss, beginning with night blindness in young boys followed by progressive constriction of the field of vision.

In June 2016, the European Commission granted the product an orphan designation for the same indication.

Applied Genetic Technologies has also been granted orphan drug designation from the FDA and European Commission for its gene therapy product candidates to treat X-linked retinoschisis and achromatopsia caused by mutations in the CNGA3 and CNGB3 genes.