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.
Open CRSP dashboard ↗Theriva Biologics, Inc.
Theriva Biologics, Inc., a clinical-stage company, develops therapeutics to treat cancer and related diseases in areas of high unmet need in the United States. The company's lead product candidate is VCN-01, a clinical stage oncolytic human adenovirus that is in a Phase 2 clinical study for the treatment of pancreatic cancer; a Phase 1 clinical study for the treatment of retinalblastoma; a Phase 1 clinical study for the treatment of head and neck squamous cell carcinoma, ovarian cancer, colorectal cancer; and a Phase 1 clinical study for the treatment of solid tumors. It also develops VCN-11 for treating solid tumors; SYN-004 designed to degrade various commonly used intravenous beta-lactam antibiotics in gastrointestinal (GI) tract for the prevention of microbiome damage to prevent overgrowth and infection by pathogenic organisms, such Clostridioides difficile infection and vancomycin resistant Enterococci, and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant recipients; and SYN-020, a recombinant oral formulation for the enzyme intestinal alkaline phosphatase to treat both local GI and systemic diseases. In addition, the company develops clinical stage products, such as SYN-006 to prevent aGVHD and infection by carbapenem resistant enterococci; and SYN-007 for preventing antibiotic associated diarrhea with oral ß-lactam antibiotics. It has collaboration agreements with The University of Texas at Austin, Catalan Institute of Oncology, Fundació Privada Institut d'Investigacio Biomedica de Bellvitge, Saint Joan De Déu, and Massachusetts General Hospital, as well as a clinical trial agreement with Washington University School of Medicine in St. Louis to conduct a Phase 1b/2a clinical trial of SYN-004. Theriva Biologics, Inc. is headquartered in Rockville, Maryland.
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