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 ↗Inovio Pharmaceuticals, Inc.
Inovio Pharmaceuticals, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of DNA medicines to treat and protect people from diseases associated with human papillomavirus (HPV), cancer, and infectious diseases. Its DNA medicines platform uses precisely designed SynCon that identify and optimize the DNA sequence of the target antigen, as well as CELLECTRA smart devices technology that facilitates delivery of the DNA plasmids. Its products in pipeline include VGX-3100 for the treatment of HPV-related cervical high-grade dysplasia; INO-3107 for HPV-related recurrent respiratory papillomatosis and is under Phase 1/2 trial; INO-3112 for the treatment of HPV-related Oropharyngeal Squamous Cell Carcinoma and is under Phase 2 trial; INO-5401 for the treatment of glioblastoma multiforme and is under Phase 2 trial; INO-4201 for Ebola Virus Disease and is under Phase 1b trial; INO-4800 for COVID-19 and is under Phase 3 trial; and INO-6160 for the treatment of human immunodeficiency virus and is under Phase 1 trial. Its partners and collaborators include Advaccine Biopharmaceuticals Suzhou Co, ApolloBio Corporation, AstraZeneca, The Bill & Melinda Gates Foundation, Coalition for Epidemic Preparedness Innovations, Defense Advanced Research Projects Agency, The U.S. Department of Defense, HIV Vaccines Trial Network, International Vaccine Institute, Kaneka Eurogentec, National Cancer Institute, National Institutes of Health, National Institute of Allergy and Infectious Diseases, the University of Pennsylvania, Plumbline Life Sciences, Regeneron Pharmaceuticals, Richter BioLogics, the Walter Reed Army Institute of Research, and The Wistar Institute. The company was incorporated in 1983 and is headquartered in Plymouth Meeting, Pennsylvania.
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