Arrowhead Research Corporation
Arrowhead Pharmaceuticals, Inc. develops medicines for the treatment of intractable diseases in the United States. Its pipeline includes Plozasiran to reduce production of apolipoprotein C-III in Phase 3 studies; Zodasiran to reduce production of angiopoietin-like protein 3 in Phase 3 clinical trials; ARO-DIMER-PA, a dual functional RNAi molecule in a Phase 1/2a clinical trials; and ARO-PNPLA3, an investigational RNAi therapeutic in Phase 1 clinical trials. The company also develops ARO-INHBE, to reduce the hepatic expression of the INHBE gene and its secreted gene product, Activin E in Phase 1/2a clinical trials; ARO-ALK7 to silence adipocyte expression of the ACVR1C gene in Phase 1/2a clinical trials; ARO-RAGE to reduce production of the receptor for advanced glycation end products in Phase 1/2a clinical trials; and ARO-MAPT, an investigational RNAi-based therapy. In addition, it is developing ARO-C3 to reduce production of C3 in Phase 1/2a clinical trials; and ARO-CFB to reduce hepatic expression of CFB in a Phase 1/2a clinical trials. The company has collaboration and license Agreements with Glaxosmithkline Intellectual Property (No. 3) Limited; Takeda Pharmaceutical Company Limited; Amgen Inc.; and Sarepta Therapeutics, Inc. Arrowhead Pharmaceuticals, Inc. was founded in 2001 and is headquartered in Pasadena, California.
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Beam Therapeutics Inc., a biotechnology company, engages in the development of precision genetic medicines for patients suffering from serious diseases in the United States. It develops BEAM-101 for the treatment of sickle cell disease; BEAM-302, a liver-targeting LNP formulation to treat severe alpha-1 antitrypsin deficiency; and BEAM-301, a liver-targeting LNP formulation for the treatment of glycogen storage disease 1a. It also intends to develop BEAM-103, an anti-CD117 monoclonal antibody, and BEAM-104, a cell therapy for development in sickle cell disease and beta-thalassemia; and BEAM-201 and other potential ex vivo CAR-T therapies. The company has research collaboration agreement with Pfizer Inc., focus on in vivo base editing programs for targets rare genetic diseases of the liver, muscle, and central nervous system; Apellis Pharmaceuticals to conduct preclinical research on target specific genes within the complement system in various organs, including the eye, liver, and brain; Verve Therapeutics, Inc., for cardiovascular disease treatments; Sana Biotechnology, Inc., to research, develop, and commercialize rights to CRISPR Cas12b for cell therapy programs; and Orbital Therapeutics design RNA for the prevention, treatment or diagnosis of human disease. Beam Therapeutics Inc. was incorporated in 2017 and is based in Cambridge, Massachusetts.
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