Orchard Therapeutics plc
Načítám obsah…
Orchard Therapeutics plc, a biopharmaceutical company, develops gene therapies for serious and life-threatening rare diseases in the United Kingdom, European Union, and the United States. The company's gene therapy approach seeks to transform a patient's hematopoietic stem cells into a gene-modified cellular drug product to treat the patient's disease through a single administration.
It provides Strimvelis, a gammaretroviral-based product for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company's clinical development products comprise OTL-101 for the treatment of ADA-SCID; OTL-200 to treat metachromatic leukodystrophy; OTL-103 for the treatment of Wiskott-Aldrich syndrome; OTL-102 for X-linked chronic granulomatous disease; and OTL-300 for transfusion-dependent beta-thalassemia.
Its preclinical programs include OTL-203 for mucopolysaccharidosis type I, OTL-201 for mucopolysaccharidosis type MPS-IIIA, and OTL-202 for mucopolysaccharidosis type IIIB. It has a strategic collaboration agreement with Pharming Group N.V. for research, development, manufacturing, and commercialization of OTL-105, an investigational ex vivo autologous hematopoietic stem cell gene therapy for the treatment of hereditary angioedema.
