Drug intelligence / Profile preview

PGV-001

Development stage
Phase 1
Lead developer
Icahn School of Medicine
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptides, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Vaccines & Immunotherapeutics
Administration
Subcutaneous, Intradermal
01

Overview

PGV-001 is a synthetic, peptide-based, personalized cancer vaccine platform designed to stimulate the immune system to recognize and attack cancer cells by targeting patient-specific tumor neoantigens. These neoantigens are unique mutations found in an individual’s tumor but not in normal tissue. The process involves sequencing both tumor and germline DNA/RNA from each patient, computationally identifying immunogenic mutant peptides (neoantigens) using the OpenVax pipeline, and synthesizing up to 10 selected peptides per patient. The resulting multi-peptide vaccine is administered with poly-ICLC as an adjuvant to enhance immune activation. Early clinical studies have shown that PGV-001 induces robust CD4+ and CD8+ T cell responses against these neoantigens with minimal toxicity (mostly mild injection site reactions). It has been tested in patients with various cancers at high risk of recurrence after standard therapy—including non-small cell lung cancer, head and neck cancer, urothelial carcinoma, breast cancer, multiple myeloma—and is being further studied in glioblastoma and prostate cancer[1][3][4][5][6][7][8].

Brand names
PGV-001PGV001PGV 001
Other names
personalized genomic vaccine 001personalized neoantigen vaccine PGV-001
02

Targets

pMHC (Peptide-MHC complex family)

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