CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for the treatment of serious human diseases using its regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9) gene-editing platform in Switzerland. Its lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. The company is also developing CTX110, a donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19 positive malignancies. In addition, it is developing allogeneic CAR-T programs targeting B-Cell maturation antigen and CD70; CTX120, a CAR-T cell product candidate for the treatment of multiple myeloma; CTX130 for the treatment of solid tumors and hematologic malignancies; programs to treat Hurler Syndrome and severe combined immunodeficiency disease, as well as glycogen storage disease Ia; and programs targeting diseases, such as Duchenne muscular dystrophy and cystic fibrosis. It has a collaboration agreements with Vertex Pharmaceuticals, Incorporated and Vertex Pharmaceuticals (Europe) Limited to develop, manufacture, commercialize, sell, and use various therapeutics; and StrideBio LLC to develop adeno-associated viral capsids. The company also has research collaboration agreements with Neon Therapeutics for developing neoantigen-based therapeutic vaccines and T cell therapies; Massachusetts General Hospital Cancer Center to develop T cell therapies for cancer; ViaCyte, Inc. for designing, developing, and commercializing gene-edited allogeneic stem cell therapies for the treatment of diabetes; and ProBioGen AG to develop novel in vivo delivery modalities for CRISPR/Cas9. CRISPR Therapeutics AG is headquartered in Zug, Switzerland.
CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Beta Thalassemia
Prime Editing by Harvard scientists unveiled; makes CRISPR look like a child's scissors https://www.cnet.com/news/breakthro...-can-find-and-replace-dna-better-than-crispr/ "prime editors aren't designed to take over from CRISPR-Cas9 or base editors in any way -- and in fact, they should work together." "Each have complementary strengths and weaknesses," says Liu. "We anticipate all three classes [CRISPR, base editor, Prime Editor] of editing agents in mammalian cells have, or will have, roles in basic research and in applications such as human therapeutics and agriculture."