CRISPR Therapeutics AG

CRISPR Therapeutics AG is a biopharmaceutical company focused on developing gene-based medicines using CRISPR/Cas9 technology. This technology allows for the precise alteration of specific genomic DNA sequences. The company achieved the first approval of a CRISPR-based therapy, CASGEVY (exagamglogene autotemcel [exa-cel]), in 2023, in collaboration with Vertex Pharmaceuticals Incorporated. CRISPR Therapeutics AG's therapeutic programs are organized into four core franchises: hemoglobinopathies, in vivo approaches, CAR T approaches, and regenerative medicine. The company employs either an ex vivo approach, editing cells outside the body before administration, or an in vivo editing approach, delivering the therapeutic directly to target cells within the body. In the hemoglobinopathies franchise, CASGEVY has received approval in the United States and other countries for treating eligible patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). Further research in this area, also with Vertex, includes targeted conditioning and in vivo editing of hematopoietic stem cells. The in vivo approaches franchise is developing a portfolio of programs for common and rare diseases, utilizing both in vivo editing and siRNA approaches. The CAR T franchise is advancing next-generation gene-edited cell therapy programs, including allogeneic chimeric antigen receptor T cell (CAR T) candidates for autoimmune indications and oncology. Lastly, the regenerative medicine franchise is developing a deviceless beta cell replacement product candidate, consisting of unencapsulated precursor islet cells derived from induced pluripotent stem cells, for the treatment of Type 1 diabetes (T1D).