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FDA gives orphan drug designation for retinitis pigmentosa gene therapy

17:06 EDT 3 Aug 2017 | Healio

The FDA has given orphan drug designation to a gene therapy product intended for treatment of X-linked retinitis pigmentosa, Applied Genetic Technologies Corp. announced in a press release.The drug candidate, which is intended specifically to treat X-linked retinitis pigmentosa caused by mutations in the RPGR gene, was granted orphan medicinal product designation for the same indication by the European Commission in June 2016, according to the release.

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