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 ↗CRISPR Therapeutics AG
CRISPR Therapeutics AG, a gene editing company, focuses on developing gene-based medicines for serious human diseases using its Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) platform. The company's CRISPR/Cas9 is a revolutionary technology for gene editing which is the process of precisely altering specific sequences of genomic DNA. It has a portfolio of therapeutic programs across a range of disease areas, including hemoglobinopathies, CAR T cell therapies, in vivo, and type 1 diabetes, as well as develops investigational CAR T programs, including an autologous, gene-edited CAR T program targeting glypican-3 (GPC3) for the potential treatment of solid tumors. The company's lead product candidate is CASGEVY, an ex vivo CRISPR/Cas9 gene-edited cell therapy for treating patients suffering from transfusion-dependent beta-thalassemia, severe sickle cell disease (SCD), and hemoglobinopathies in which a patient's hematopoietic stem and progenitor cells are edited to produce high levels of fetal hemoglobin in red blood cells. It also develops CAR T cell therapies, including CTX112 targeting cluster of differentiation 19 (CD19) and CTX131 targeting CD70 for oncology and autoimmune indications; CTX310 and CTX320, in vivo gene editing to address the cardiovascular disease by disrupting the validated targets angiopoietin-like protein 3 and lipoprotein; and CTX211, an allogeneic, gene-edited, hypoimmune stem cell-derived product candidate for the treatment of T1D. It has strategic partnerships with Vertex Pharmaceuticals Incorporated, Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was incorporated in 2013 and is headquartered in Zug, Switzerland.
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