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On April 29, 2026, Beijing Grit Bio-Medicine Co., Ltd. ("Grit Bio") announced that an innovative universal type, independently researched by Grit Bio and currently developed by Shanghai Anwentai Biotechnology Co., Ltd. ("Anwentai"),CAR-iNKT Cell Therapy Project GT719 Cell InjectionFollowing the receipt of its first ODD designation, it has recently been granted Orphan Drug Designation (ODD) by the US FDA.Newly added indication: Acute Lymphoblastic Leukemia (ALL). This achievement represents further recognition of the potential indications for GT719, as well as an opportunity to benefit more patients and indicates greater market potential.
GT719 is an innovative universal allogeneic CAR-iNKT cell therapy.Derived from allogeneic hematopoietic stem cells and expressing anti-CD19 chimeric antigen receptor, iTCR, and IL-15, its core design leverages the unique biological properties of iNKT cells. Through iTCR recognition of lipid antigens presented by CD1d, it achieves allogeneic application without the need for TCR knockout, avoiding graft-versus-host disease (GvHD). At the same time, it combines the triple killing mechanisms of NK, iTCR, and CAR to achieve multiple anti-tumor effects. Supported by Grail Bio's self-developed OptiDiff™ platform, GT719 enables high-purity, trillion-scale cell production while enriching memory-like iNKT cells, ensuring long-term in vivo survival, reducing treatment costs, and enhancing patient accessibility.The ODD designation for GT719 not only affirms its potential in treating rare hematologic malignancies but also secures policy support and development incentives for future clinical advancement. The ODD status will provide GT719 with significant advantages in clinical trials, development progress, and regulatory interactions, expediting its market entry and bringing new therapeutic hope to patients with ALL."The patient population with acute lymphoblastic leukemia is large, and the clinical need is urgent. GT719 is expected to bring an innovative treatment option to more patients."Dr. Yarong Liu, Founder and CEO of Gracell Biotechnologies, stated"The granting of orphan drug designation by the U.S. FDA for the newly added ALL indication marks not only a significant milestone in the company’s global R&D strategy, but also highlights our firm commitment and technical capability to expand into the acute lymphoblastic leukemia market and advance GT719 as an innovative treatment option for more patients."Currently, GT719, as the world's first stem cell-derived off-the-shelf CAR-iNKT therapy, is undergoing clinical research at the IIT stage, with preliminary safety and efficacy data already available. In the future, Grail Bio and AvantGen will rely on clinical research platforms in both China and the U.S. to further advance the development of GT719 across various tumor and non-tumor indications, providing more patients with innovative cell therapy solutions that offer breakthrough benefits. The company will continue to be driven by innovation, bringing more hope to blood cancer patients worldwide through advanced cell and gene technologies.Source: Grail Bio
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