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Agios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes for the treatment of hemolytic anemias. The company develops AG-946, a PK activator for treating lower-risk myelodysplastic syndrome and hemolytic anemias; and AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria. Its preclinical product is siRNA for the treatment of polycythemia vera, a rare blood disorder. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Amicus Therapeutics, Inc., a biotechnology company, focuses on discovering, developing, and delivering medicines for rare diseases. Its commercial product and product candidates include Galafold, an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant; and Pombiliti + Opfolda, for the treatment of late onset. It has collaboration and license agreements with the University of Pennsylvania to research and develop parvovirus gene therapy products; and GlaxoSmithKline. Amicus Therapeutics, Inc. was incorporated in 2002 and is headquartered in Princeton, New Jersey.

MorphoSys AG, together with its subsidiaries, engages in the development and commercialization of therapeutics for patients suffering from various cancers in Europe, Asia, and the United States. The company's product pipeline includes Pelabresib that is in Phase 3 trials to treat myelofibrosis and thrombocythemia; Tulmimetostat, a product candidate in Phase 1/2 trials for the treatment of solid tumors and lymphomas; Felzartamab, an antibody directed against CD38 for renal autoimmune diseases and relapsed/refractory multiple myeloma; Ianalumab, a candidate in Phase 3 clinical trials for Sjögren's disease, lupus nephritis, and other autoimmune diseases; Abelacimab that is in Phase 3 trials for venous thromboembolism prevention and cancer-associated thrombosis; Setrusumab, which is in Phase 2/3 trials for osteogenesis imperfecta; and Bimagrumab, a product candidate in Phase 2b trials for adult obesity. It also develops MOR210/TJ210/HIB210 that is in Phase 1 clinical trials for relapsed or refractory advanced solid tumors; and NOV-8, a candidate in Phase 2 trials for the treatment of pulmonary sarcoidosis and dermatitis. The company has collaboration and licensing agreements with I-Mab Biopharma, Novartis, Anthos Therapeutics, Ultragenyx, Mereo BioPharma, Lilly, Human Immunology Biosciences, Inc. Incyte Corporation, and Xencor, Inc. MorphoSys AG was founded in 1992 and is headquartered in Planegg, Germany.

Pacira BioSciences, Inc. engages in the development, manufacture, marketing, distribution, and sale of non-opioid pain management and regenerative health solutions to healthcare practitioners in the United States. The company offers EXPAREL, a bupivacaine liposome injectable suspension; ZILRETTA, a triamcinolone acetonide extended-release injectable suspension; and iovera system, a non-opioid handheld cryoanalgesia device used to produce controlled doses of cold temperature to targeted nerves. It has a development and commercialization, and supply agreement with Aratana Therapeutics, Inc. for NOCITA, a bupivacaine liposome injectable suspension product. The company was formerly known as Pacira Pharmaceuticals, Inc. and changed its name to Pacira BioSciences, Inc. in April 2019. Pacira BioSciences, Inc. was incorporated in 2006 and is headquartered in Tampa, Florida.

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.