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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.

Constellation Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, discovers and develops therapeutics to address serious unmet medical needs in patients with cancers associated with abnormal gene expression or drug resistance. The company's lead product candidates include Pelabresib that is in Phase 3 clinical trials for the treatment of patients with myelofibrosis in combination with ruxolitinib. It is also developing CPI-0209 that is in Phase 1 dose escalation portion of a Phase 1/2 clinical trial to treat solid tumors; and CPI-482, which is in preclinical stage for cancer patients. The company was formerly known as Epigenetix, Inc. and changed its name to Constellation Pharmaceuticals, Inc. in March 2008. Constellation Pharmaceuticals, Inc. was incorporated in 2008 and is headquartered in Cambridge, Massachusetts.

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.

Kura Oncology, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer. The company's pipeline consists of small molecule product candidates that target cancer. Its lead product candidates are ziftomenib, an orally bioavailable small molecule inhibitor of the menin-KMT2A interaction for the treatment of genetically defined subsets of acute leukemias, including acute myeloid leukemia and acute lymphoblastic leukemia; tipifarnib, an orally bioavailable farnesyl transferase inhibitor combination with alpelisib for patients with PIK3CA-dependent HNSCC; and KO-2806, a farnesyl transferase inhibitor for the treatment of solid tumors. It has a clinical collaboration with Novartis Pharma AG to evaluate the combination of tipifarnib and alpelisib in patients with head and neck squamous cell carcinoma whose tumors have HRAS overexpression and/or PIK3CA mutation and/or amplification. The company was founded in 2014 and is headquartered in San Diego, California.