Thomas Marron, MD, PhD, on 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 American Association for Cancer Research Annual Meeting 2021.

Thomas Marron, MD, PhD, of the Icahn School of Medicine at Mount Sinai, spoke with CancerNetwork® at the virtual American Association for Cancer Research (AACR) Annual Meeting 2021 about research methods for investigating the neoantigen peptide vaccine, PGV-001.


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


Marron TU, Saxena M, Bhardwaj N, et al. An adjuvant personalized neoantigen peptide vaccine for the treatment of malignancies (PGV-001). Presented at: AACR Annual Meeting 2021; April 10-15, 2021; virtual. Abstract LB048.