CRISPR Therapeutics’ mission is to develop transformative gene-based medicines for patients with serious diseases. Our therapeutic approach is to cure diseases at the molecular level using the breakthrough gene editing technology called CRISPR/Cas9. We are focused on the treatment of somatic (non-germline) cells, analogous to the approach taken by more traditional and established gene therapy methods. We are not using human germline modifications and we support the current recommendations of the International Society for Stem Cell Research in this regard. Together with other Gene Editing Companies, we have issued a Joint Statement - Position Regarding Human Germline Gene Editing. Our development of transformative new medicines will involve working closely with patients and families, healthcare professionals, regulatory agencies and other groups dedicated to improving healthcare.
We are building a portfolio of therapeutic candidates based on several important criteria that include among others the seriousness of the condition, the unmet medical need, and a clear link between the underlying genetic abnormality and the respective disease. We are evaluating both in vivo treatments, administering CRISPR/Cas9 technology in cells inside the human body, and ex vivo treatments, removing specific cells from the body, editing the gene causing the disease(s) and delivering the modified cells back to the patient. Therapeutic indications with potential for ex vivo treatment include hemoglobinopathies, such as sickle cell disease and beta thalassemia, certain types of immunodeficiencies, and specific approaches to immune therapies for cancer. In vivo treatment approaches using CRISPR/Cas9 gene editing may be used for diseases of the liver, eye and lung among others, for which delivery mechanisms have been established.