Drug intelligence / Profile preview

in vivo CD19-targeted CAR-T mRNA-LNP therapy

Development stage
Phase 1
Lead developer
Xinqiao Hospital
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous, Intramuscular
01

Overview

This experimental therapy represents an in vivo approach to chimeric antigen receptor (CAR) T-cell therapy, developed by Xinqiao Hospital of Chongqing. It utilizes lipid nanoparticle (LNP) technology to deliver mRNA encoding an anti-CD19 CAR directly into the patient's body via intravenous or intramuscular injection. Unlike conventional CAR-T therapies that require complex ex vivo cell engineering (harvesting, modifying, and re-infusing a patient's cells), this method aims to reprogram the patient's own T cells within the body to transiently express the CAR. The resulting CAR-T cells target and deplete CD19-positive B cells, which are central to the pathogenesis of various refractory autoimmune diseases. This 'off-the-shelf' approach potentially simplifies the manufacturing process and reduces the time to treatment compared to traditional cell therapies.

Other names
in vivo CAR-T drug based on LNP-mRNAmRNA Therapy Targeting CD19
02

Targets

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