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Orchard Therapeutics Announces FDA Granted Orphan Drug Designation For OTL-102 For Treatment Of X-linked Chronic Granulomatous Disease

Orchard Therapeutics (NASDAQ:ORTX), a global gene therapy leader, today announced that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for OTL-102, the company's ex

Benzinga · 01/29/2020 12:10

Orchard Therapeutics (NASDAQ:ORTX), a global gene therapy leader, today announced that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for OTL-102, the company's ex vivo autologous hematopoietic stem cell (HSC) gene therapy being investigated for the treatment of X-linked chronic granulomatous disease (X-CGD). The FDA may grant orphan designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S.