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APM pDNA
- Development stage
- Preclinical
- Lead developer
- Johns Hopkins University
- Modality
- Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Nanoparticles → Drug Delivery Systems
- Administration
- Intramuscular, Retro-injection Into The Salivary Duct, Intraductal (salivary Gland)
01
Overview
APM pDNA refers to plasmid DNA encoding Antigen Processing Machinery (APM) factors and transcriptional regulators. It is delivered via poly(β-amino ester) (PBAE) nanoparticles. The drug aims to restore antigen presentation in cancer cells, which often downregulate MHC-I expression to evade the immune system. By delivering genes such as NLRC5, TAP1, TAP2, Psmb9, Psmb8, IRF1, IRF2, Tapasin, Batf3, CIITA, and ERp57, APM pDNA enhances surface MHC-I and MHC-II expression on tumor cells, making them susceptible to T-cell mediated killing. This approach is being investigated as an in situ antigen-agnostic tumor therapy.
02
Targets
PDIA3 (Membrane-associated rapid response steroid-binding receptor)TAPBP (TAP-binding protein)TAP1 (Transporter 1, ATP binding cassette subfamily B member)IRF1 (Interferon regulatory factor 1)IRF2 (Interferon regulatory factor 2)CIITA (MHC class II transactivator)NLRC5PSMB9 (Immunoproteasome subunit beta type-1i)TAP2 (Transporter associated with antigen processing 2)
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