Editas Medicine products

We are pursuing a number of diseases using an in vivo editing approach, focusing on hematopoietic stem cells and other tissue types, and we are excited about the progress we’ve made.

EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34⁺ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34⁺ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe SCD and TDT.

Genomic medicine is a developing field of medical research that harnesses recent advances in genetics to develop new medicines for diseases. In fact, we are at an inflection point where we are seeing the first real genomic medicines emerge to help patients. Advances in the scientific fields of cell therapy and gene therapy have paved the way for the success of gene editing, which in turn is now expanding and accelerating the development of a new class of genomic medicines.