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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.

BELLUS Health, Inc. is a biopharmaceutical development company, which engages in the research and development of products that provide health solutions and address critical unmet medical needs. Its products include BLU-5937, KIACTA, AMO-01, and ALZ-801. The company was founded on June 17, 1993 and is headquartered in Laval, Canada.

Syndax Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, develops therapies for the treatment of cancer. Its lead product candidates are revumenib, a potent, selective, small molecule inhibitor of the menin-MLL binding interaction for the treatment of KMT2A rearranged, acute leukemias, and solid tumor; and SNDX-6352 or axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1, or CSF-1 receptor for the treatment of patients with chronic graft versus host disease (cGVHD) and idiopathic pulmonary fibrosis (IPF). The company is also developing Entinostat. It has an agreement with Eddingpharm International Company Limited for licensing, development, and commercialization of Entinostat. Syndax Pharmaceuticals, Inc. was incorporated in 2005 and is headquartered in Waltham, 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.

Vanda Pharmaceuticals Inc., a biopharmaceutical company, focuses on the development and commercialization of therapies to address high unmet medical needs worldwide. The company's marketed products include HETLIOZ to treat non-24-hour sleep-wake disorders; and Fanapt oral tablets for the treatment of schizophrenia. Its pipeline products include HETLIOZ (tasimelteon) to treat jet lag disorder, insomnia, delayed sleep phase disorder, sleep disturbances in autism spectrum disorder, and pediatric Non-24; Fanapt (iloperidone) for the treatment of bipolar I disorder and Parkinson's disease psychosis, as well as a long acting injectable (LAI) formulation to treat schizophrenia; and Tradipitant (VLY-686), a small molecule neurokinin-1 receptor (NK-1R) antagonist to treat gastroparesis, motion sickness, atopic dermatitis, and COVID-19 pneumonia. The company's pipeline products also include VTR-297, a small molecule histone deacetylase inhibitor to treat hematologic malignancies and with potential use as a treatment for various oncology indications; VQW-765, a small molecule nicotinic acetylcholine receptor partial agonist to treat performance anxiety and psychiatric disorders; VHX-896, an active metabolite of iloperidone; and antisense oligonucleotide molecules. In addition, it offers a portfolio of cystic fibrosis transmembrane conductance regulator activators and inhibitors that include VSJ-110 for the treatment of dry eye and ocular inflammation; and VPO-227 for the treatment of secretory diarrhea disorders comprising cholera. Vanda Pharmaceuticals Inc. was incorporated in 2002 and is headquartered in Washington, the District of Columbia.