CRISPR Therapeutics AG

CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for serious human diseases. It develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), a gene editing technology that allows for precise directed changes to genomic DNA. It has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The company's lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-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. It is also developing CTX110, a donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19 positive malignancies. In addition, the company is devel