CSPC PHARMA: First CAR-T cell injection based on mRNA-LNP (SYS 6020) approved for new indication clinical trial

According to the news from Zhitong Finance and Economics APP, CSPC PHARMA (01093) announced that the first chimeric antigen receptor T-cell infusion (SYS6020) targeting B-cell maturation antigen (BCMA) based on mRNA-LNP technology developed by the group has been approved by the China National Medical Products Administration for conducting clinical trials for the indication of systemic lupus erythematosus (SLE) in China. Previously, this product had obtained clinical trial approval for the indication of multiple myeloma (MM) in China. This product is currently the world's first mRNA-LNP-based cell therapy product approved for SLE clinical trials. By expressing a CAR that specifically recognizes the BCMA antigen, it targets and kills immune cells that bind to BCMA on the surface of mature B lymphocytes and plasma cells, eliminating elevated autoantibodies. It is a new, safe, and effective potential treatment option for SLE patients. Currently, there is no approved CAR-T therapy for treating SLE globally. Compared to traditional CAR-T products, this product has the advantages of high cell viability, high CAR positivity rate, low risk of tumorigenesis caused by genomic integration, and lower side effects such as cytokine release syndrome (CRS). Preclinical studies have shown that this product can significantly kill BCMA-positive myeloma cells and has good safety. In terms of cost, using LNP to transfect T cells can reduce the high cost of using lentiviral vectors, easing the burden on patients. SLE is a typical systemic autoimmune disease characterized by abnormal activation of the immune system leading to the production of large amounts of autoantibodies, causing acute or chronic inflammation and functional damage in multiple organs (such as kidneys, heart, lungs, and skin). Most patients need lifelong treatment. Treatment with steroids combined with immunosuppressants has improved the long-term survival of SLE patients, but inevitable disease relapses and irreversible organ damage remain important causes of patient death. Therefore, there is an urgent need for new treatment methods to achieve sustained relief of symptoms, control organ damage, improve long-term survival of patients, and even achieve complete cure. The approval of the SLE indication clinical trial for this product is another important achievement in the group's layout in the field of cell therapy, laying a good foundation for the development of other cell therapy products, such as in vivo generated CAR-T cells