We are rapidly translating our specific, efficient and versatile CRISPR/Cas9 gene-editing platform into therapies to treat hemoglobinopathies, cancer, diabetes and other diseases

Our multi-disciplinary team of world-class researchers and drug developers works every day to translate our CRISPR/Cas9 technology into breakthrough human therapeutics. Our lead program targeting the blood diseases β-thalassemia and sickle cell disease has entered clinical testing, as has our first allogeneic CAR-T program targeting B-cell malignancies. We are also advancing additional blood stem cell, immuno-oncology, regenerative medicine and in vivo programs towards the clinic.