Drug intelligence / Profile preview

circFAM53B mRNA vaccine

Development stage
Phase 2
Lead developer
Sun Yat-Sen Memorial Hospital
Modality
mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Vaccines & Immunotherapeutics
Administration
Intramuscular
01

Overview

The **circFAM53B mRNA vaccine** is an investigational cancer vaccine designed to elicit an immune response against tumor cells overexpressing a specific circular RNA-derived peptide. Preclinical research identified **circFAM53B**, a circular RNA specifically overexpressed in certain tumor cells, as containing a hidden open reading frame that encodes a cryptic peptide known as **FAM53B-219aa**. This peptide serves as a tumor-specific antigen. In this vaccine formulation, the sequence encoding the FAM53B-219aa peptide is linearized and encapsulated within **lipid nanoparticles (LNPs)** for delivery. Once administered, the mRNA instructs the patient's cells to produce the cryptic peptide, thereby priming the immune system to recognize and attack malignant cells presenting this antigen. It is currently being evaluated in clinical trials for advanced solid tumors, often in combination with immune checkpoint inhibitors like toripalimab.

Other names
circFAM53B-derived mRNA vaccinecircFAM-53B-derived mRNA vaccinecircFAM 53B-derived mRNA vaccineFAM53B-219aa mRNA vaccineFAM-53B-219aa mRNA vaccineFAM 53B-219aa mRNA vaccine
02

Targets

CircFAM53B-219aa pMHC-I (CircFAM53B-219aa peptide-HLA class I complex)

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