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Orchard Therapeutics plc, a gene therapy company, research, develops, and commercialize hematopoietic stem cell and gene therapies in the United Kingdom, Italy, France, and Germany. It offers OTL-200, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of patients with metachromatic leukodystrophy under the Libmeldy brand; and Strimvelis, a gammaretroviral vector-based gene therapy for the treatment of adenosine deaminase deficiency. The company's program for neurodegenerative disorders comprises clinical proof of concept-stage program, which includes OTL-203 for the treatment of mucopolysaccharidosis type I and OTL-201 for mucopolysaccharidosis type IIIA, or MPS-IIIA; and pre-clinical program, OTL-204 for the treatment of frontotemporal dementia with progranulin mutations. In addition, it develops program for immunological disorders consist of pre-clinical programs, which includes OTL-104 for Crohn's disease with mutations in the nucleotide-binding oligomerization domain-containing protein 2; and OTL-105 for the treatment of hereditary angioedema. The company was formerly known as Orchard Rx Limited. Orchard Therapeutics plc was founded in 2015 and is headquartered in London, the United Kingdom. As of January 24, 2024, Orchard Therapeutics plc operates as a subsidiary of Kyowa Kirin Co., Ltd..

Protalix BioTherapeutics, Inc., a biopharmaceutical company, engages in the development, production, and commercialization of recombinant therapeutic proteins based on its proprietary ProCellEx plant cell-based protein expression system in the United States, Australia, Canada, Israel, Brazil, Russia, Turkey, and internationally. The company offers Elelyso for the treatment of Gaucher disease; and Elfabrio for the treatment of adult patients with a confirmed diagnosis of Fabry disease. It is also developing PRX-115, a plant cell-expressed recombinant PEGylated Uricase which is in Phase I trial for the treatment of gout; and PRX-119, a plant cell-expressed PEGylated recombinant human DNase I product candidate that is in preclinical phase for the treatment of NETs-related diseases. The company has agreements and partnerships with Pfizer; Fundação Oswaldo Cruz; and Chiesi Farmaceutici S.p.A. The company was incorporated in 1993 and is headquartered in Karmiel, Israel. Protalix BioTherapeutics, Inc. is a subsidiary of Protalix Ltd.

Renovacor, Inc., a biotechnology company, focuses on delivering various precision therapies to enhance the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. It primarily focuses on the treatment of BCL2-associated athanogene 3 (BAG3) mutation-associated dilated cardiomyopathy (DCM). The company's lead product candidate is REN-001, a recombinant adeno-associated virus 9-based gene therapy designed to deliver a functional BAG3 gene to augment BAG3 protein levels in cardiomyocytes, and slow or halt progression of BAG3 DCM. It is also developing a pipeline of BAG3-associated gene therapies for diseases with high unmet medical need associated with mutations in the BAG3 gene and mechanistically linked to BAG3's expression and function. The company was founded in 2013 and is based in Greenwich, Connecticut.

Surface Oncology, Inc., a clinical-stage immuno-oncology company, engages in the development of cancer therapies in the United States. The company develops various antibodies that include NZV930, a fully human immunoglobulin isotype G4 monoclonal antibody for the production of extracellular adenosine; SRF617, a fully human IgG4 monoclonal antibody that inhibits CD39 enzymatic activity for the production of adenosine and the breakdown of adenosine triphosphate; SRF388 which is in phase II clinical trial targeting interleukin 27; GSK4381562, an antibody targeting CD112R, an inhibitory protein expressed on natural killer and T cells; and SRF114 that is in phase 1/2 clinical trials targeting the chemokine receptor CCR8. Surface Oncology, Inc. has a license agreement with GlaxoSmithKline Intellectual Property (No. 4) Limited and Vaccinex, Inc. to develop, manufacture, and commercialize antibodies, which targets SRF114. The company was incorporated in 2014 and is headquartered in Cambridge, Massachusetts. As of September 8, 2023, Surface Oncology, Inc. operates as a subsidiary of Coherus BioSciences, Inc.