In vivo delivery of a novel CD3-targeted Lentiviral Vector generates CD19 CAR-T cells in two different humanized mouse models and results in complete B cell depletion

Frederic Vigant, Anirban Kundu, Dongming Zhang, Wei Zhang, Ewa Jaruga-Killeen, Michelle Andraza, Gregory Schreiber, Alissa R Kerner, Junyi Zhang, John Henkelman, Renata Soares, Ramya Yarlagadda, Gregory I Frost & Sid P Kerkar

Abstract #3294

Key Messages:

  • In vivo CD3-targeted Lentivectors (LV) delivery of CAR-T transgenes represents a promising CAR-T gene therapy approach (GCAR)
  • CD3-directed CAR LV generate CD19 CAR-T cells in vivo, resulting in a complete depletion of hCD20+ B cells in circulation and in lymphoid organs
  • This novel approach for the rapid and direct formation of CAR-T cells in vivo was demonstrated in two different humanized mouse models
  • B-Cell aplasia was obtained in lymphoreplete animal models without the use of a conditioning chemotherapy regimen, consistent with our results from studies of ex vivo transduced PBMC with CD3-directed LV