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 ↗Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Solid Biosciences Inc.; Regeneron; Abeona; and Arcturus Therapeutics Holdings Inc. The company was incorporated in 2010 and is headquartered in Novato, California.
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