YouSino Bio's UC101 Becomes World’s First Umbilical Cord Blood-Derived Allogeneic CAR-T Therapy Approved for IND by U.S. FDA

Recently, UCELLO announced that its self-developed CD19-targeted chimeric antigen receptor (CAR) allogeneic universal T-cell injection (UC101) received approval from the U.S. Food and Drug Administration (FDA) for a new drug clinical trial application (IND) on January 11, 2025.

UC101 is the world's first umbilical cord blood-derived allogeneic universal CAR-T product to receive FDA IND approval. T cells derived from umbilical cord blood are the youngest T cells, with the natural advantages of low immunogenicity and being in an early differentiation state. The low immunogenicity of umbilical cord blood T cells can reduce host-versus-graft (HvGR) reactions, allowing allogeneic universal CAR-T cells to expand more easily and effectively within patients, significantly prolonging the median in vivo persistence time of allogeneic CAR-T cells. More importantly, 90% of umbilical cord blood T cells are naïve T cells, and after extensive in vitro expansion, 80% remain as Tscm and Tcm cells. Tscm and Tcm cells, which are in the early stages of differentiation, exhibit stronger in vivo expansion capabilities and persistence, making the product more therapeutically effective.

UC101 is also the world's first CAR-T product using a stable cell line to produce lentiviral vectors and has been approved for IND. The production of lentiviral vectors using a stable cell line does not require plasmids as raw materials, making the production process simpler. Lentiviral vectors produced under this process have the advantages of high batch-to-batch consistency, high viral vector titer, low rate of empty viral shells, high transduction efficiency, and more controllable quality. Compared with lentiviral vectors produced by traditional transient transfection processes, this method significantly reduces costs while improving quality.

UC101 employs a unique gene-editing combination to reduce immunogenicity and enhance efficacy while better controlling adverse reactions. In this FDA IND application, the company successfully utilized imported umbilical cord blood from the U.S. to complete multiple batches of large-scale production under GMP conditions. A single batch can produce enough for over 100 patients, significantly reducing the cost of CAR-T cell therapy and improving medical accessibility.

UC101 is the first allogeneic universal CAR-T product in China to receive FDA IND approval, marking the official entry of China's allogeneic universal CAR-T products onto the international stage. The FDA IND approval of UC101 signifies that UCELLO's umbilical cord blood allogeneic universal CAR-T cell technology platform has gained recognition from top international regulatory authorities for its numerous innovations. It also marks the completion of the validation of the first product from the company’s technology platform, laying a solid industrial foundation for the subsequent development of new indications and new target products based on this platform, showcasing the company's immense potential in its globalization strategy.

UCELLO's early platform technology has demonstrated significant efficacy and good safety in the IIT clinical trial for acute B lymphoblastic leukemia (B-ALL). The latest platform technology has expanded the scope of IIT clinical trials to include multiple indications such as multiple myeloma (MM), systemic lupus erythematosus (SLE), and Sjögren's syndrome (SjD). In the IIT trial targeting systemic lupus erythematosus, significant CAR-T cell expansion has been achieved in consecutive patients, along with successful B-cell reconstitution. Patients' autoreactive antibodies have significantly decreased, and drug-free remission is anticipated, showing comparable results to international industry data on autologous CAR-T. UCELLO is expected to provide more cost-effective treatment options for a broader patient population in the future, improving quality of life and restoring confidence in life.