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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.

Apellis Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the discovery, development, and commercialization of therapeutic compounds through the inhibition of the complement system for autoimmune and inflammatory diseases. It offers EMPAVELI for the treatment of paroxysmal nocturnal hemoglobinuria, C3 glomerulopathy and immune complex membranoproliferative glomerulonephritis, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy; and SYFOVRE for treating geographic atrophy secondary to age-related macular degeneration and geographic atrophy (GA). The company also develops APL-3007, a small interfering RNA, or siRNA, which is in a Phase 1 clinical trial, as well as an oral complement inhibitor that is in preclinical development. It has a collaboration and license agreement with Swedish Orphan Biovitrum AB (publ) for development and commercialization of pegcetacoplan; and a collaboration with Beam Therapeutics Inc. focused on the use of Beam's base editing technology to discover new treatments for complement-driven diseases. The company was incorporated in 2009 and is based in Waltham, Massachusetts.

Aurinia Pharmaceuticals Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies to treat various diseases with unmet medical need in the United States. It offers LUPKYNIS for the treatment of adult patients with active lupus nephritis. It has a collaboration and license agreement with Otsuka Pharmaceutical Co., Ltd. The company was incorporated in 1993 and is headquartered in Edmonton, Canada.

Dicerna Pharmaceuticals, Inc. is a biopharmaceutical company, which engages in discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to selectively silence genes that cause or contribute to disease. The firm uses its proprietary GalXC RNAi technology platform to build a pipeline in these therapeutic areas. The company was founded by Douglas M. Fambrough III, Mark A. Behlke, Roberto Guerciolini, James Craig Jenson, and John J. Rossi in October 2006 and is headquartered in Lexington, MA.

Karuna Therapeutics, Inc., a clinical-stage biopharmaceutical company, creates and delivers transformative medicines for people living with psychiatric and neurological conditions. Its lead product candidate includes KarXT, an oral modulator of muscarinic receptors for the treatment of acute psychosis in patients with schizophrenia; and for the treatment of central nervous system disorders, such as negative and cognitive symptoms of schizophrenia and psychosis, as well as for the treatment of various peripheral tissues and dementia-related psychosis, including Alzheimer's disease. The company also focuses on developing other muscarinic-targeted drug candidates; and intends to develop lead candidature TRPC4/5 and KAR-2618 for the treatment of depressive disorder. Karuna Therapeutics, Inc. has a license agreement with Eli Lilly and Company, and Zai Lab (Shanghai) Co., Ltd; patent license agreement with PureTech Health LLC; and drug discovery partnership with Charles River Laboratories, as well as drug discovery collaboration with PsychoGenics, Inc. The company was formerly known as Karuna Pharmaceuticals, Inc. and changed its name to Karuna Therapeutics, Inc. in March 2019. Karuna Therapeutics, Inc. was incorporated in 2009 and is headquartered in Boston, Massachusetts.