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Achillion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, discovers, develops, and commercializes small molecule drug therapies for immune system disorders. Its lead drug candidate is ACH-4471, an inhibitor of factor D that is Phase II clinical trials for patients with paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy/immune complex membranoproliferative glomerulonephritis. The company is also developing ACH-5228, a factor D inhibitor that is in Phase I clinical trials; and ACH-5548, a factor D inhibitor, which is in Phase I clinical trials for the treatment of PNH and other complement mediated diseases. It has license agreements with Ora, Inc. for the development and commercialization of ACH-702; and GCA Therapeutics, Ltd for elvucitabine, a nucleoside reverse transcriptase inhibitor for the treatment of hepatitis B infection and human immunodeficiency virus infection. The company was founded in 1998 and is based in New Haven, Connecticut.

Aprea Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing and commercializing synthetic lethality-based cancer therapeutics that targets DNA damage response pathways. Its lead product candidate is the ATRN-119 that is in Phase I clinical trials for treating advanced solid tumors, ovarian, breast, and prostate cancers. The company is also developing ATRN-1051, ATRN-354, and APRE-DDRi for the treatment of advanced solid tumors. The company is headquartered in Doylestown, Pennsylvania.

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.

Rhythm Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on the rare neuroendocrine diseases. The company's lead product candidate is IMCIVREE (setmelanotide), a rare melanocortin-4 receptor for the treatment of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1, leptin receptor (LEPR) deficiency obesity, and Bardet-Biedl and Alström syndrome. It is in Phase 3 clinical trials for treating POMC or LEPR heterozygous deficiency obesities, steroid receptor coactivator 1 deficiency obesity, SH2B1 deficiency obesity, MC4 receptor deficiency obesity, and other MC4R disorders. The company has licensing agreements with LG Chem, Ltd; Ipsen Pharma S.A.S; Camurus; RareStone Group Ltd.; and LG Chem, Ltd. The company was formerly known as Rhythm Metabolic, Inc. and changed its name to Rhythm Pharmaceuticals, Inc. in October 2015. Rhythm Pharmaceuticals, Inc. was founded in 2008 and is headquartered in Boston, Massachusetts.

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary targeted augmentation of nuclear gene output to develop antisense oligonucleotides to selectively restore protein levels. Its lead clinical candidate is STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy. The company also develops STK-001, which is in phase I/II clinical trial to treat Dravet syndrome; and programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye. The company has a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.