MagicRNA Announces Clinical Data for In Vivo CAR-T Therapy in Lupus

SHENZHEN, China—Sep 15 , 2025—MagicRNA, a clinical-stage biotechnology company, has announced the publication of the world’s first clinical data for an mRNA-lipid nanoparticle (mRNA-LNP) based in vivo CAR-T candidate, HN2301, for the treatment of refractory systemic lupus erythematosus (SLE). The study, published in The New England Journal of Medicine, represents the first demonstration of in vivo CAR-T generation and activity in SLE patients.

The therapy was administered intravenously without prior lymphodepletion, with the goal of reprogramming CD8+ T cells directly in the patient’s body to become CD19-targeted CAR-T cells. While other gene therapies, such as those using viral vectors like AAV, deliver a gene to a target cell, this approach uses mRNA to transiently create the therapeutic cells in vivo.

Key Clinical Findings

The clinical trial enrolled five patients with long-standing, treatment-refractory SLE. Data from the study showed:

• At a low dose, HN2301 reprogrammed up to 60% of CD8+ T-cells in the peripheral blood.
• This reprogramming resulted in complete depletion of circulating B cells and a reduction of up to 20 points in the patients’ SLEDAI scores within three months.
• The treatment demonstrated a favorable safety profile, with no severe adverse events or neurotoxic effects observed.

“For the first time, we see functional CAR-T cells generated directly in patients’ bodies, achieving rapid B-cell clearance and clinical improvement of autoimmune disease,” said Prof. Georg Schett, a pioneer in the field.

Advancing Next-Generation Immunotherapy

Dr. Gavin Zha, CEO of MagicRNA, stated that this study provides the first clinical proof-of-concept for the company’s EnC-LNP platform in autoimmune disease. He added that the technology overcomes major limitations of traditional ex vivo CAR-T therapies, including complex manufacturing, high costs, and the need for lymphodepletion.

Based on these encouraging results, MagicRNA plans to continue with dose-escalation studies to further evaluate the therapy’s potential to achieve long-term, drug-free remission for patients worldwide. The company’s pipeline includes additional in vivo cell reprogramming therapeutics and cell-targeted LNP based medicines for other immune-related diseases.