Exploratory Study on mRNA Therapy Targeting CD19 for the Treatment of Refractory Autoimmune Diseases
Status:
NOT_YET_RECRUITING
Trial end date:
2028-12-31
Target enrollment:
Participant gender:
Summary
Autoimmune diseases, such as immune thrombocytopenia (ITP), immune hemolytic anemia (AIHA), systemic lupus erythematosus (SLE), lupus nephritis (LN), idiopathic inflammatory myopathy (IIM), ulcerative colitis (UC), and systemic sclerosis (SSc), are a type of chronic disabling disease characterized by the immune system mistakenly attacking the body itself, leading to tissue damage and organ dysfunction.Autoimmune hematological diseases, especially difficult to treat autoimmune diseases, are a type of disease that is difficult to treat and has a significant impact on patients' lives. Although there are various treatment methods currently available, there are still many limitations to autoimmune sexually transmitted diseases that aim for long-term remission, and further research and breakthroughs are urgently needed.
The advent of COVID-19 vaccine has brought LNP mRNA technology into the public's view. After years of development, it not only shines brilliantly in COVID-19 vaccine, but also is widely used in the treatment and exploration of cancer, rare diseases and other fields. Lipid nanoparticles (LNP) are currently the most mature non viral delivery platform, capable of protecting mRNA from nuclease degradation, promoting intracellular uptake, and achieving efficient translation in vivo.
The core of LNP-mRNA technology targeting CD19 is to encapsulate the mRNA encoding specific proteins (such as anti-CD19 related proteins) in lipid nanoparticles and deliver them to the body through intravenous or intramuscular injection.