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Nightstar receives U.S. and European Orphan Drug Designation for gene therapy to treat Choroideremia

NightstaRx Ltd ("Nightstar"), the biopharmaceutical company specialising in bringing therapies for retinal dystrophies to patients, has received both U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) Orphan Drug Designation for its lead programme, a gene therapy to treat Choroideremia, an X-linked recessive disorder that leads to progressive blindness.