Drug intelligence / Profile preview

CPTX2309

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

Overview

CPTX2309 is an investigational in vivo CAR-T cell therapy developed by Capstan Therapeutics for the treatment of B cell-mediated autoimmune diseases. Unlike conventional ex vivo CAR-T therapies, which require removal and genetic modification of a patient's T cells outside the body, CPTX2309 uses targeted lipid nanoparticles (tLNPs) to deliver messenger RNA encoding an anti-CD19 chimeric antigen receptor (CAR) directly to CD8+ cytotoxic T cells within the patient. This approach enables rapid and transient engineering of functional anti-CD19 CAR T cells in vivo without lymphodepleting chemotherapy or viral vectors. Preclinical studies have shown that this method leads to robust depletion of B cells in blood and tissues, followed by repopulation with predominantly naïve B cells—suggesting potential for immune system reset. The therapy is currently being evaluated in a Phase 1 clinical trial for safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers[2][5][6][7][8].

Other names
CPTX2309CPTX-2309CPTX 2309
02

Targets

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