ORTX Profile
Orchard Therapeutics plc (ORTX) is a global gene therapy company that develops transformative treatments for patients with severe genetic diseases. The company's portfolio includes clinical-stage gene therapies for primary immune deficiencies, neurometabolic disorders, and hemoglobinopathies.
ORTX's lead gene therapy candidate, OTL-101, is a treatment for adenosine deaminase severe combined immunodeficiency (ADA-SCID), a rare, life-threatening inherited disorder that affects the immune system. The therapy uses a patient's own stem cells that have been genetically modified to correct the mutation that causes ADA-SCID.
Other gene therapy candidates in the company's pipeline include treatments for metachromatic leukodystrophy (MLD), Wiskott-Aldrich syndrome (WAS), transfusion-dependent beta-thalassemia, and sickle cell disease.
Orchard Therapeutics is headquartered in London, UK, with additional offices in Boston, Massachusetts, and Foster City, California.
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