CSPC PHARMA (01093) has announced that the Phase I clinical trial of its investigational therapy, SYS 6063, for refractory systemic lupus erythematosus (SLE) has officially commenced at the first Chinese study center.
This product is currently the world's first CAR-T cell therapy to receive approval for SLE clinical trials, utilizing LNP-mRNA transfection technology to target CD19 and BCMA. It employs lipid nanoparticles (LNP) to transfect T cells with mRNA encoding CARs for CD19 and BCMA, enabling simultaneous expression of two complete CAR proteins. This approach effectively kills CD19 and BCMA-positive cells that secrete autoantibodies, offering a potentially novel, safe, and effective treatment option for SLE patients.
Currently, no CAR-T therapy has been approved globally for SLE. Compared to traditional CAR-T products, SYS 6063 boasts higher cell viability and CAR positivity rates, no risk of tumorigenesis from genomic integration, and a lower incidence of adverse reactions such as cytokine release syndrome (CRS). Preclinical studies indicate the product can significantly kill tumor cells positive for CD19 and BCMA antigens while demonstrating a favorable safety profile.
In terms of cost, using LNP to transfect T cells reduces the high expenses associated with lentiviral vectors, potentially easing the financial burden on patients. The company previously received a Drug Clinical Trial Approval Notice from China's National Medical Products Administration in June 2026, and after approval, initiated this Phase I trial for SLE.
This Phase I study is a single-arm, open-label, two-stage (dose escalation and dose expansion), multi-center clinical trial in patients with refractory SLE. It aims to evaluate the safety, tolerability, efficacy, pharmacokinetics, and immunogenicity of SYS 6063. Recruitment and screening of subjects are currently progressing actively.
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