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Epizyme, Inc., a commercial-stage biopharmaceutical company, discovers, develops, and commercializes novel epigenetic medicines for patients with cancer and other diseases in the United States. The company offers Tazemetostat for the treatment of metastatic or locally advanced epithelioid sarcoma for adults and pediatric patients. It also develops R-CHOP in front-line patients with high risk diffuse large B-cell lymphoma; and PARP inhibitor in patients with PARPi-resistant solid tumors, such as castration-resistant prostate cancer, small cell lung cancer, and others. In addition, the company develops Tazemetostat in patients with INI1-negative tumors in adults and pediatrics; PRMT5 inhibitor for patients with solid tumors; and PRMT1 inhibitor. It has collaboration agreements with Roche Molecular Systems, Inc.; Lymphoma Academic Research Organization; Eisai Co. Ltd.; HUTCHMED (China) Limited; and Roche Sequencing Solutions, Inc. The company was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Intercept Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics to treat progressive non-viral liver diseases in the United States, Europe, and Canada. The company markets Ocaliva, a farnesoid X receptor agonist used for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid in adults. It is also developing Ocaliva for various indications, including nonalcoholic steatohepatitis and NASH; and other product candidates in various stages of clinical and preclinical development. The company has a license agreement with Aralez Pharmaceuticals Canada Inc. to develop and commercialize bezafibrate in the United States. It markets its products through an internal commercial organization and third-party distributors. The company was incorporated in 2002 and is headquartered in Morristown, New Jersey. As of November 8, 2023, Intercept Pharmaceuticals, Inc. operates as a subsidiary of Alfasigma S.p.A..

Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.

Trillium Therapeutics, Inc. is a clinical stage immuno-oncology company, which engages in the research and development of therapies for the treatment of cancer. It offers clinical programs, TTI-621 and TTI-622, target CD47, a signal that cancer cells frequently use to evade the immune system. The company was founded on March 31, 2004 and is headquartered in Mississauga, Canada.