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"A global gene therapy leader developing potential cures for severe genetic diseases."
Orchard Therapeutics develops hematopoietic stem cell gene therapies for patients with severe genetic diseases where treatment options are limited or do not exist. The company harnesses the power of a patient's own genetically modified blood stem cells to potentially correct the underlying cause of disease using a single administration. Orchard builds on decades of clinical research to advance ex vivo autologous gene therapies that aim to transform the lives of individuals and families affected by conditions like metachromatic leukodystrophy, Wiskott Aldrich syndrome, and mucopolysaccharidosis. More than 160 people have been treated in clinical trials with the company's therapies, with up to 10 years of follow up in some conditions.
193
Team Members
2015
Founded
Biotechnology
Sector
Hybrid
Work Environment
A world where genetic diseases no longer devastate lives, and every patient has access to potentially curative therapies that restore health and transform futures.
Orchard's mission is to fully unlock the curative potential of hematopoietic stem cell gene therapy for the benefit of patients, families, and communities affected by severe genetic diseases.