CSPC PHARMA (ASX: 01093) has announced that its first mRNA-LNP-based dual-target chimeric antigen receptor (CAR)-T cell injection (SYS6063 injection) has received approval from the National Medical Products Administration of China to commence clinical trials in the country.

This product is the world's first mRNA-LNP-based dual-target cell therapy to gain such approval. It works by expressing CARs that specifically recognize the CD19 and BCMA antigens, enabling the precise identification and elimination of CD19 and BCMA-positive cells in patients to achieve therapeutic effects.

The synergistic action of the dual targets aims to eradicate pathogenic cells at the source, fundamentally improving patient conditions and addressing the shortcomings of traditional treatments, which often cannot provide a cure and are prone to relapse.

Preclinical studies have demonstrated that the product can significantly kill CD19 and BCMA-positive cells while exhibiting a favorable safety and efficacy profile.

The clinical indication approved for this trial is refractory systemic lupus erythematosus.

Furthermore, the product holds potential for treating other B-cell/plasma cell-mediated autoimmune diseases, such as myasthenia gravis, rheumatoid arthritis, and ANCA-associated vasculitis. It also has applications in hematological malignancies like relapsed/refractory multiple myeloma and B-cell lymphoma, indicating substantial clinical development value.

The approval for this clinical trial represents another significant achievement for the group in the field of cell therapy, laying a solid foundation for the development of cell therapy products, including in vivo generated CAR-T therapies.