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CD19 CAR mRNA lipid nanoparticle

Development stage: Preclinical
Lead developer: University of Pennsylvania
Modality: mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration: Intravenous

Overview

CD19 CAR mRNA lipid nanoparticle is a non-viral gene delivery platform designed to transiently engineer cells to express chimeric antigen receptors (CARs) targeting the CD19 antigen. This approach utilizes lipid nanoparticles (LNPs) to encapsulate and deliver messenger RNA (mRNA) encoding a CD19-specific CAR construct, typically comprising an anti-CD19 single-chain variable fragment (scFv), costimulatory domains (e.g., 4-1BB or CD28), and a CD3ζ signaling domain. Unlike traditional viral vector-based CAR-T cell therapies, this technology enables transient CAR expression, which may reduce the risk of long-term toxicities and insertional mutagenesis while simplifying manufacturing. It is being investigated for both ex vivo and in vivo generation of CAR-T cells to treat B-cell malignancies, including B-cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma.

Other names: CD19 CAR mRNA LNPCD-19 CAR mRNA LNPCD 19 CAR mRNA LNPCD19-targeted CAR mRNA lipid nanoparticlesCD-19-targeted CAR mRNA lipid nanoparticlesCD 19-targeted CAR mRNA lipid nanoparticles

Targets

CD19 (B lymphocyte antigen CD19)

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