ORCHARD THERAPEUTICS PLC
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About ORTX
Orchard Therapeutics plc is a gene therapy company. The Company is engaged in transforming the lives of people affected by rare diseases through the development of curative gene therapies. Its ex vivo autologous hematopoietic stem cell (HSC), gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. Its ex vivo autologous HSC gene therapy approach seeks to transform a patient’s autologous HSCs into a gene-modified cellular drug product to treat the patient’s disease. Its lead program is OTL-200 operates under the brand name Libmeldy for eligible patients with early-onset metachromatic leukodystrophy (MLD). Its programs in immunological disorders consist of two pre-clinical programs, OTL-104 for Crohn’s disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2 (NOD2-CD), and OTL-105 for hereditary angioedema (HAE).
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