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Orchard Therapeutics is a commercial-stage biotechnology company specializing in the development of potentially curative gene therapies for rare genetic diseases. The company utilizes an ex vivo autologous hematopoietic stem cell (HSC) gene therapy approach, which involves genetically modifying a patient's own stem cells outside the body using lentiviral vectors before reintroducing them. Their lead product, Lenmeldy (atidarsagene autotemcel), is the first therapy approved for the treatment of metachromatic leukodystrophy (MLD). Orchard's pipeline also includes advanced programs for primary immune deficiencies and other neurometabolic disorders. In early 2024, the company was acquired by Kyowa Kirin to serve as a global hub for its genetic medicine business.
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