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Akebia Therapeutics, Inc., a biopharmaceutical company, focuses on the development and commercialization of therapeutics for patients with kidney diseases. The company's lead product investigational product candidate is Vafseo (vadadustat), an oral hypoxia-inducible factor prolyl hydroxylase, which is in Phase III development for the treatment of anemia due to chronic kidney disease (CKD) in dialysis-dependent and non-dialysis dependent patients. It offers Auryxia, a ferric citrate that is used to control the serum phosphorus levels in adult patients with DD-CKD on dialysis; and the treatment of iron deficiency anemia in adult patients with CKD not on dialysis. The company's product pipeline includes AKB-9090, a drug targeting critical-care indications; and AKB-10108, a drug targeting conditions related to premature birth. It has collaboration agreements with Mitsubishi Tanabe Pharma Corporation for the development and commercialization of vadadustat in Japan and other Asian countries, as well as research and license agreement with Janssen Pharmaceutica NV for the development and commercialization of hypoxia-inducible factor prolyl hydroxylase targeted compounds worldwide. Akebia Therapeutics, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.

Catalyst Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company, focuses on developing and commercializing therapies for people with rare debilitating, chronic neuromuscular, and neurological diseases in the United States. It offers Firdapse, an amifampridine phosphate tablets for the treatment of patients with lambert-eaton myasthenic syndrome (LEMS); and Ruzurgi for the treatment of pediatric LEMS patients. The company develops Firdapse for the treatment of MuSK antibody positive myasthenia gravis and spinal muscular atrophy type. It has license agreements with BioMarin Pharmaceutical Inc.; and collaboration and license agreement with Endo Ventures Limited for the development and commercialization of generic Sabril tablets. The company was founded in 2002 and is based in Coral Gables, Florida.

NextCure, Inc., a clinical-stage biopharmaceutical company, engages in discovering and developing novel immunomedicines to treat cancer and other immune-related diseases by restoring normal immune function. The company is developing NC410, an immunomedicine designed to block immune suppression mediated by LAIR-1; NC762, a monoclonal antibody that binds specifically to B7-H4, a protein expressed on multiple tumor types; and NC525, a monoclonal antibody that binds to LAIR-1 to functionally kill AML blast cells and leukemic stem cells while preserving normal blood cells, including hematopoietic stem cells. Its discovery and research programs include an antibody in preclinical evaluation of other potential novel immunomodulatory molecules. It has a license agreement with Yale University. The company was incorporated in 2015 and is headquartered in Beltsville, Maryland.

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.