MagicRNA Announces Groundbreaking Achievement in Autoimmune Disease Treatment
In-vivo application of genetically engineered CAR T cells for managing Lupus system in 2025, published in The New England Journal of Medicine.
MagicRNA, a clinical-stage biotechnology company, has made a significant breakthrough in the field of cell therapy. The organization has announced the publication of the world's first clinical data of an mRNA-lipid nanoparticle (mRNA-LNP) based in vivo CAR T-cell therapy candidate, HN2301, in The New England Journal of Medicine.
The study, titled "In vivo CD19-CAR T-Cell Therapy for Refractory Systemic Lupus Erythematosus", is the first-ever demonstration of in vivo CAR T generation and activity in SLE patients.
The study enrolled five patients with long-standing, treatment-refractory SLE, four of whom also had lupus nephritis. HN2301 was administered intravenously without prior lymphodepletion to deliver CAR-encoding mRNA to CD8+ T cells.
The generated CAR T cells achieved rapid B-cell clearance and clinical improvement of autoimmune disease. Complete depletion of circulating B cells persisted for 7-10 days following the administration of HN2301. Functional CAR T cells were generated directly in patients' bodies for the first time, according to the study.
Dr. Gavin Zha, CEO of MagicRNA, stated that the study provides the first clinical proof-of-concept of the cell targeted-LNP based in vivo CAR T-cell in autoimmune disease. Prof. Georg Schett, a pioneer in the use of CD19 CAR T cell therapy in SLE, has achieved a revolutionary breakthrough.
The treatment was generally well tolerated, with no patients experiencing grade ≥3 CRS or neurotoxic effects. The study reported a favorable safety profile for HN2301. Anti-nucleosome and anti-dsDNA antibodies significantly decreased, and low complement levels in some patients normalized at the last visit.
No severe adverse events were observed during or after treatment. Disease Activity Index (SLEDAI-2000) scores significantly decreased by as much as 20 points in all 5 patients 3 months after the infusion of HN2301.
MagicRNA will continue dose-escalation studies to evaluate HN2301's ability to achieve immune reset and long-term drug-free remission. The goal is to accelerate clinical development and ultimately bring this therapy to patients worldwide.
This breakthrough represents an important milestone for the entire field of cell therapy, according to Prof. Georg Schett. The organization responsible for the clinical-stage biotechnology company that first published clinical data on the mRNA lipid nanoparticle-based in vivo CAR T-cell therapy named HN2301 is MagicRNA.
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