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Calliditas Therapeutics AB (publ), a commercial-stage specialty pharmaceutical company, focused on identifying, developing, and commercializing pharmaceuticals products for treatments in orphan indications with initial focus on renal and hepatic diseases. It offers Nefecon, an oral formulation of budesonide that is an immunosuppressant for the treatment of the autoimmune renal disease immunoglobulin A nephropathy. The company's lead product candidate is Setanaxib, a NOX inhibitor that is in Phase 2b/3 clinical trial for the treatment of primary biliary cholangitis; and in Phase 2 clinical trial for the treatment of squamous cell carcinoma of the head and neck cancer and idiopathic pulmonary fibrosis, as well as for type 1 diabetic kidney disease. It also develops Budenofalk 3 mg oral capsules for the treatment of autoimmune hepatitis. The company was founded in 2004 and is headquartered in Stockholm, Sweden.

Chinook Therapeutics, Inc., a clinical-stage biotechnology company, focuses on the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead clinical program is atrasentan, a Phase III endothelin receptor antagonist for the treatment of IgA nephropathy and other proteinuric glomerular diseases. Its product candidates also include BION-1301, an anti-APRIL monoclonal antibody is being evaluated in a Phase I/II trial for IgA nephropathy; and CHK-336, an oral small molecule LDHA inhibitor for the treatment of primary hyperoxaluria, as well as research programs for other rare and severe chronic kidney diseases. Chinook Therapeutics, Inc. is headquartered in Seattle, Washington. As of August 11, 2023, Chinook Therapeutics, Inc. operates as a subsidiary of Novartis AG.

Pharming Group N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company's commercialized product is RUCONEST, a plasma-free rhC1INH protein replacement therapy for the treatment acute hereditary angioedema (HAE) attacks. It also engages in the development of leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome; OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE; and alpha-glucosidase therapy for the treatment of Pompe and fabry diseases. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. is headquartered in Leiden, the Netherlands.

Silence Therapeutics plc, a biotechnology company, focuses on the discovery and development novel molecules incorporating short interfering ribonucleic acid (siRNA) to inhibit the expression of specific target genes in hematology, cardiovascular, and rare diseases. The company's mRNAi GalNAc Oligonucleotide Discovery platform consists of precision engineered product candidates designed to target specific disease-associated genes in the liver. The company develops Zerlasiran (SLN360), which is in phase 2 clinical trial for cardiovascular disease associated with elevated lipoprotein(a); and Divesiran (SLN124), an siRNA molecule that is in Phase I clinical trials for the treatment of genetic hematological conditions, including polycythemia vera. It is also developing SLN-COMP-1 and SLN-COMP-2 for complement-mediated diseases; and SLN-HAN-1 and SLN-HAN-2. It has collaboration agreements with AstraZeneca PLC to discover, develop, and commercialize siRNA therapeutics for the treatment of cardiovascular, renal, metabolic, and respiratory diseases; Hansoh Pharmaceutical Group Company Limited to develop siRNAs; and Mallinckrodt Pharma IP Trading DAC to develop and commercialize RNAi drug targets designed to silence the complement cascade in complement-mediated disorders. The company was formerly known as SR Pharma plc and changed its name to Silence Therapeutics plc in May 2007. Silence Therapeutics plc was incorporated in 1994 and is headquartered in London, the United Kingdom.

Xenon Pharmaceuticals Inc., a clinical-stage biopharmaceutical company, engages in developing therapeutics to treat patients with neurological disorders in Canada. Its clinical development pipeline includes XEN496, a Kv7 potassium channel opener that is Phase III clinical trials for the treatment of KCNQ2 developmental and epileptic encephalopathy; and XEN1101, a Kv7 potassium channel opener, which is in Phase II clinical trial for the treatment of epilepsy and other neurological disorders. The company's product candidates also comprise NBI-921352, a selective Nav1.6 sodium channel inhibitor that is in Phase II clinical trials for the treatment of SCN8A developmental and epileptic encephalopathy, and other indications, including adult focal epilepsy. It has a license and collaboration agreement with the Neurocrine Biosciences, Inc. to develop treatments for epilepsy. The company was incorporated in 1996 and is headquartered in Burnaby, Canada.