Thomas Marron, MD, PhD, on the research design for the neoantigen peptide vaccine, PGV-001

Marron detailed the research process for a Phase 1 trial of PGV-001 presented virtually at the 2021 annual meeting of the American Association for Cancer Research.

Thomas Marron, MD, PhD, Icahn School of Medicine at Mount Sinai, spoke with CancerNetwork® at the American Association for Cancer Research (AACR) 2021 Virtual Annual Meeting on Research Methods to Study the Neoantigen Peptide Vaccine, PGV-001.


The PGV-001 neoantigen peptide vaccine uses the Open Vax platform, which is a computer pipeline that essentially takes whole exome sequencing and bulk RNA sequencing from tumors that have typically been excised. for the majority of patients. We sequence tumor DNA and RNA, we also perform germline DNA sequencing, and identify tumor-specific mutations. We then confirm expression using bulk RNA sequencing. And then once we have a list of potential neo-antigens in the tumor, we use the patient-specific HLA type to predict which of those neo-antigens might be the most immunogenic. [or] most likely for the immune system to be able to recognize based on this patient’s unique immune characteristics. And then once we identified the top 10 neo-antigens initially – we actually identified the first 20, then based on the synthetic capabilities of our partner who synthesized the peptides in Germany – we vaccinated patients with these peptides.


Brown TU, Saxena M, Bhardwaj N, et al. Adjuvant personalized neoantigen peptide vaccine for the treatment of malignant tumors (PGV-001). Presented at the 2021 AACR Annual Meeting; April 10-15, 2021; virtual. Abstract LB048.

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