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Akcea Therapeutics, Inc., a biopharmaceutical company, focuses on developing and commercializing drugs to treat patients with serious and rare diseases in the United States and internationally. The company offers TEGSEDI, which is designed to reduce the production of transthyretin protein. It develops WAYLIVRA, which has completed Phase III clinical study for the treatment of familial chylomicronemia syndrome; and that is in Phase III clinical study for the treatment of familial partial lipodystrophy. The company also develops AKCEA-APO(a)-LRx, which has completed Phase IIb clinical study for treating patients suffering with hyperlipoproteinemia; AKCEA-ANGPTL3-LRx that has completed Phase IIb clinical study for the treatment of multiple lipid disorders; and AKCEA-APOCIII-LRx, which is in Phase II study for the treatment of cardiovascular disease due to elevated triglyceride levels, as well as AKCEA-TTR-LRx to treat the broad population of patients with hereditary and wild-type forms of transthyretin amyloidosis. It has strategic collaboration with Novartis Pharma AG and PTC Therapeutics International Limited. The company was incorporated in 2014 and is headquartered in Boston, Massachusetts. Akcea Therapeutics, Inc. operates as a subsidiary of Ionis Pharmaceuticals, Inc.

Arcturus Therapeutics Holdings Inc., a late-stage clinical messenger RNA medicines and vaccine company, focuses on the development of infectious disease vaccines and other products within liver and respiratory rare diseases. Its technology platforms include LUNAR lipid-mediated delivery and STARR mRNA. The company is developing ARCT-810 (LUNAR-OTC), a mRNA-based therapeutic candidate, which is in Phase 2 clinical trial for treating ornithine transcarbamylase deficiency; and ARCT-154 (LUNAR-COV19), a mRNA vaccine candidate that is in Phase 3 arm of a Phase 1/2/3 study in Vietnam for the treatment of COVID-19, as well as ARCT-032 (LUNAR-CF), a mRNA therapeutic candidate for cystic fibrosis. Its product pipeline includes, ARCT-2301 for bivalent: ancestral/omicron which is in Phase 3; ARCT-2303 for monovalent that is in Phase 3; ARCT-2138 for quadrivalent which is in Phase 1; and LUNAR-FLU which is in pre-clinical trial. Arcturus Therapeutics Holdings Inc. was founded in 2013 and is headquartered in San Diego, California.

Kiniksa Pharmaceuticals, Ltd., a biopharmaceutical company, focuses on discovering, acquiring, developing, and commercializing therapeutic medicines for patients suffering from debilitating diseases with significant unmet medical needs worldwide. Its product candidates include ARCALYST, an interleukin-1alpha and interleukin-1beta, for the treatment of recurrent pericarditis, which is an inflammatory cardiovascular disease; Mavrilimumab, a monoclonal antibody inhibitor that completed Phase II clinical trials for the treatment of giant cell arteritis; Vixarelimab, a monoclonal antibody, that is in Phase 2b clinical trial for the treatment of prurigo nodularis, a chronic inflammatory skin condition; and KPL-404, a monoclonal antibody inhibitor of the CD40- CD154 interaction, a T-cell co-stimulatory signal critical for B-cell maturation, immunoglobulin class switching, and type 1 immune response. The company was incorporated in 2015 and is based in Hamilton, Bermuda.

Karyopharm Therapeutics Inc., a commercial-stage pharmaceutical company, discovers, develops, and commercializes drugs directed against nuclear export for the treatment of cancer and other diseases in the United States. The company discovers, develops, and commercializes novel and small molecule Selective Inhibitor of Nuclear Export (SINE) compounds that inhibit the nuclear export protein exportin 1 (XPO1). Its lead compound, include XPOVIO in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma; in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma; and for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). It also developing Selinexor for treating hematological and solid tumor malignancies, including multiple myeloma, endometrial cancer, myelofibrosis, and DLBCL; and ELTANEXOR for treating Myelodysplastic Neoplasms, as well as verdinexor, KPT-9274, and IL-12 compounds. The company has license agreement with Menarini Group to develop and commercialize selinexor for human oncology indications; license agreement with Antengene Therapeutics Limited to develop and commercialize selinexor, eltanexor, and KPT-9274 for the treatment and/or prevention of human oncology indications, as well as verdinexor for the diagnosis, treatment, and/or prevention of human non-oncology indications; and distribution agreement for the commercialization of XPOVIO with FORUS Therapeutics Inc. Further, it has a collaboration with Bristol Myers Squibb company to evaluate novel cereblon E3 ligase modulator agent mezigdomide in combination with Selinexor in patients with relapsed/refractory multiple myeloma. Karyopharm Therapeutics Inc. was incorporated in 2008 and is headquartered in Newton, Massachusetts.

Nabriva Therapeutics plc, a biopharmaceutical company, engages in the development and commercialization of novel anti-infective agents to treat serious infections. The company's product includes SIVEXTRO, an oxazolidinone-class antibacterial for the treatment of acute bacterial skin and skin structure infection (ABSSSI); and XENLETA, a semi-synthetic pleuromutilin antibiotic for oral and IV administration. It also develops XENLETA that is in Phase I clinical trial for the treatment of pediatric infections, as well as sexually transmitted infections, cystic fibrosis, ABSSSI, ventilator-associated bacterial pneumonia, complicated intra-abdominal infections, hospital-acquired bacterial pneumonia, acute bacterial skin and skin structure infections, osteomyelitis, and prosthetic joint infections. In addition, the company develops CONTEPO, an epoxide antibiotic for use in treating complicated urinary tract infections, as well as is in Phase I clinical trial for peri-operative prophylaxis. The company was formerly known as Nabriva Therapeutics Forschungs GmbH and changed its name to Nabriva Therapeutics plc in 2007. Nabriva Therapeutics plc was incorporated in 2005 and is headquartered in Dublin, Ireland.