Drug intelligence / Profile preview

PNT01

Development stage
Preclinical
Lead developer
Zhongshan Hospital, Fudan University
Modality
mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Vaccines & Immunotherapeutics
Administration
Injection
01

Overview

PNT01 is an AI-guided personalized mRNA cancer vaccine designed for the treatment of intrahepatic cholangiocarcinoma (iCCA). Developed using the EchoNeo 1.0 deep learning-based antigen discovery platform, the vaccine consists of codon-optimized mRNA sequences encoding tumor-specific neoantigens, which are encapsulated in lipid nanoparticles (LNPs). PNT01 is engineered to elicit robust tumor-specific T-cell responses, specifically increasing the proportion of intratumoral CD8+ T cells to suppress tumor growth. Preclinical studies in murine models have demonstrated that PNT01 provides dose-dependent tumor control and exhibits synergistic antitumor activity when combined with immune checkpoint inhibitors, such as anti-PD-L1 antibodies.

02

Targets

pMHC (Peptide-MHC complex family)

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