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De novo-designed pMHC binders facilitate T cell–mediated cytotoxicity toward cancer cells

K. Johansen, Darian S. Wolff, Beatrice Scapolo, Monica L. Fernández‐Quintero, Charlotte Risager Christensen, Johannes R. Loeffler, Esperanza Rivera‐de‐Torre, Max D. Overath, Kamilla Kjærgaard Munk, Oliver Morell, Marie Christine Viuff, Iñigo Lacunza, A Englund, Mathilde Due, Anant Gharpure, Stefano Forli, Carlos Rodriguez Pardo, Tripti Tamhane, Emma Qingjie Andersen, Kasper H. Björnsson, J Fernandes, Lasse Frank Voss, Suthimon Thumtecho, Andrew B. Ward, Maria Ormhøj, Sine Reker Hadrup, Timothy P. Jenkins

2025Science50 citationsDOI

Abstract

The recognition of intracellular antigens by CD8 + T cells through T cell receptors (TCRs) is central for adaptive immunity against infections and cancer. However, the identification of TCRs from patient material remains complex. We present a rapid de novo minibinder (miBd) design platform leveraging state-of-the-art generative models to engineer miBds targeting the cancer-associated peptide-bound major histocompatibility complex (pMHC) SLLMWITQC/HLA-A*02:01 (NY-ESO-1). Incorporating in silico cross-panning enabled computational prescreening of specificity, and molecular dynamics simulations allowed for improved predictability of in vitro success. We identified a high-affinity NY-ESO-1 binder and confirmed its structure using cryo–electron microscopy, which, when incorporated in a chimeric antigen receptor, induced killing of NY-ESO-1 + melanoma cells. We further designed and validated binders to a neoantigen pMHC complex, RVTDESILSY/HLA-A*01:01, with unknown structure, demonstrating the potential for precision immunotherapy.

Topics & Concepts

Cancer immunotherapyIn silicoComputational biologyCytotoxicityMajor histocompatibility complexCD8StreptamerAntigenHuman leukocyte antigenCytotoxic T cellBiologyPanning (audio)T-cell receptorImmunotherapyT cellCell biologyChemistryIn vitroImmune systemImmunologyBiochemistryGenePaleontologyZoomLens (geology)CAR-T cell therapy researchImmunotherapy and Immune ResponsesT-cell and B-cell Immunology
De novo-designed pMHC binders facilitate T cell–mediated cytotoxicity toward cancer cells | Litcius