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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Sana Biotechnology, Inc., a biotechnology company, focuses on utilizing engineered cells as medicines in the United States. It develops ex vivo and in vivo cell engineering platforms for various therapeutic areas with unmet treatment needs, including type 1 diabetes, B cell mediated autoimmune diseases, and oncology. The company's product pipeline consist of UP421, a HIP-modified allogeneic primary islet cell product, which is in phase 1 clinical trial to treat type 1 diabetes; SC451, a HIP-modified, stem cell derived pancreatic islet cell therapy, which is in preclinical trial for the treatment of type 1 diabetes; SC291, a HIP-modified allogeneic CD19-dirtected allogeneic CAR T cell product candidate which is in phase 1 clinical trial to treat systemic lupus erythematosus and ANCA-associated vasculitis, as well as in preclinical trial to treat other indications and b-cell mediated autoimmune disease; and SG299, a CD8-targeted fusosome, which is in preclinical trial to treat patients with hematologic malignancies and B cell mediated autoimmune diseases. It also develops SC262, a hypoimmune-modified CD22-directed allogeneic CAR T program, which is in phase 1 to treat non-Hodgkin's lymphoma; and SG299, a CD8-targeted fusosome, which is in preclinical trial to treat patients with hematologic malignancies and B cell mediated autoimmune diseases. The company has an option and license agreement with Beam Therapeutics Inc. for use of Beam's proprietary CRISPR Cas12b nuclease editing technology to research, develop, and commercialize engineered cell therapy products; and a license agreement with Harvard College to access certain intellectual property for the development of hypoimmune-modified cells. The company was formerly known as FD Therapeutics, Inc. and changed its name to Sana Biotechnology, Inc. in September 2018. Sana Biotechnology, Inc. was incorporated in 2018 and is headquartered in Seattle, Washington.
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