Crispr Therapeutics

Crispr Therapeutics

Crispr Therapeutics AG is a company with the main part of its operations in Boston, MA. The company focuses on the development of transformative genetic medicines for serious diseases using its Clustered Regularly Interspaced Short Palindromic Re-peats (CRISPR)/Cas9 gene-editing platform. CRISPR/Cas9 can be programmed to cut, edit and correct disease-associated deoxyribonucleic acid (DNA) in a patient’s cell. Among the CRISPR companies, Crispr Therapeutics is unique in being the first to have entered the clinic (in late 2018), initially focusing on ex vivo applications. CTX-001 is in the clinic in Europe for transfusion-dependent beta-thalassemia and in the US for sickle cell disease (profit sharing for both programs with Vertex), promising data from first patients showing successful editing, engraftment and clinical improvement have been presented. Crispr Therapeutics has retained full rights for its allogenic CAR-T programs, they are specifically pursuing CD19, BCMA and CD70 as initial targets and have entered the clinic in late 2019. Crispr further pursues in vivo programs in partnership with Vertex (DMD, DM-1, CF), on its own through the former Bayer joint venture named Casebia as well as a regenerative medicines pact with Viacyte to de-immunize a syn-thetic pancreas device with gene editing technology.