Capricor Therapeutics Inc
Capricor Therapeutics, Inc., a clinical-stage biotechnology company, engages in the development of transformative cell and exosome-based therapeutics for treating duchenne muscular dystrophy (DMD) and other diseases with unmet medical needs in the United States. Its lead product candidate is the Deramiocel, an allogeneic cardiosphere-derived cells, which is in phase 3 clinical trial for the treatment of DMD. The company also develops Exosome protein-based vaccine, which is in preclinical trial to treat SARS-CoV-2; StealthX Exosome Platform, and exosome platform program consists of engineered exosomes for vaccine and therapeutic development and is in preclinical trial. In addition, it engages in developing StealthX, an engineered exosome-based vaccine candidate, under phase 1 clinical study for a range of therapeutic applications, including targeted RNA, protein, and small molecule therapeutics to treat or prevent a variety of diseases; and CAP-2003, and allogeneic cardiosphere-derived cells-exosomes, which is in preclinical trial to treat DMD. It has license agreement with Johns Hopkins University, University of Rome License, and Cedars-Sinai Medical Center to develop and commercialize licensed products and licensed services under the licensed patent rights in all fields and a nonexclusive right to the know-how; Cell Line License Agreement with Life Technologies for the development of exosomes platform. The company was founded in 2005 and is headquartered in San Diego, California.
Open CAPR 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.
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