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SYS6020
- Development stage
- Phase 2
- Lead developer
- CSPC Pharmaceutical Group
- Modality
- CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Gene Silencing → Gene Therapies, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
SYS6020 is an autologous, mRNA-transfected, anti-BCMA chimeric antigen receptor (CAR) T cell therapy developed using synthetic mRNA and lipid nanoparticle (LNP) technology. The therapy involves collecting a patient's own T cells, transfecting them ex vivo with synthetic mRNA encoding a CAR targeting B-cell maturation antigen (BCMA), and reinfusing the modified cells back into the patient. Upon administration, these engineered CAR-T cells specifically recognize and induce cytotoxicity in BCMA-expressing cells. This approach aims to provide targeted treatment for diseases characterized by pathogenic BCMA-positive plasma or B-cells, such as multiple myeloma and certain autoimmune disorders. Notably, SYS6020 is the first reported mRNA-LNP-based CAR-T cell therapy to enter clinical trials globally[7][8][10]. It is being investigated for its safety and preliminary efficacy in refractory systemic lupus erythematosus, generalized myasthenia gravis, and multiple myeloma[1][3][4][6].
- Brand names
- SYS6020SYS-6020SYS 6020
- Other names
- autologous mRNA-transfected anti-BCMA-CAR T lymphocytes SYS6020
02
Targets
BCMA (B-cell maturation antigen)
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