ET-01 refers to autologous CD34+ hematopoietic stem/progenitor cells with the elytroid-specific enhancer of the BCL11A gene modified by CRISPR/Cas9.

Investigational New Drug (IND) application for ET-01, an investigational CRISPR/Cas 9 gene-editing therapy for patients with transfusion dependent β-thalassemia, has been approved by the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA). 

Using gene-editing technology to edit BCL11A erythroid enhancer in hematopoietic stem cells (HSC), ET-01 is an autologous cell therapy being developed to increase the fetal hemoglobin level in red blood cells for severe β-thalassemia patients.

EdiGene presented the manufacturing scale-up and preclincial development data of ET-01 for the β-thalassemia gene editing treatment at the 61st ASH annual meeting in 2019.

EdiGene presented preliminary safety and efficacy results of ET-01 at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition in 2022

Edit immuno-rejection molecules in  T cells from healthy donors, leveraging partner’s proprietary CAR-T to create best-in-class U CART process and U CAR-T product.