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.
Open ARWR dashboard ↗Rocket Pharmaceuticals, Inc. Common Stock
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 in the United States. It develops vivo adeno-associated viral (AAV) programs, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure (RP-A501), which is in Phase 2 trial; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), which is in phase 1; and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is a form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart, which is in preclinical stage. It also develops vivo lentiviral (LV) programs, which include programs for Leukocyte Adhesion Deficiency-I (LAD-I), a genetic disorder that causes the immune system to malfunction (RP-L201); Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102); and Pyruvate Kinase Deficiency (PKD), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301). 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; REGENXBIO, Inc.; and Temple University. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.
Open RCKT dashboard ↗