Edit Code of Life,Creating Transformative Solutions for Serious Human Diseases

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ET-01

  • About ET-01

    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). 

  • Therapeutic Approach

    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.

  • Poster Presentation at the 61st ASH Annual Meeting

    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.

ET-02

  • Therapeutic Approach


    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.