Acute myeloid leukemia (AML) remains an aggressive malignancy with poor outcomes for patients ineligible for intensive chemotherapy or stem cell transplantation, highlighting the need for novel and less toxic treatment strategies.
Immune targeting of AML is challenging due to the lack of tumor-specific antigens, as most known targets are also expressed on healthy hematopoietic cells, increasing the risk of off-tumor toxicity and prolonged myeloablation.
Endogenous retroviruses (ERVs) represent a novel class of tumor antigens from the dark genome. While largely epigenetically silenced in healthy tissues, ERVs become aberrantly expressed in AML, enabling selective targeting of malignant cells.
EVX-04 is an AI-designed off-the-shelf DNA vaccine encoding 16 ERV-derived antigenic fragments, developed to induce broad antigen-specific T-cell responses across diverse HLA types and enable T cell-mediated targeting of AML blasts expressing ERV antigens.
