GPI-80-ADC

GPI-80-ADC is a novel antibody-drug conjugate (ADC) developed for use in in-utero hematopoietic stem cell (HSC) transplantation (IUTx). This ADC is designed to selectively deplete highly proliferative fetal HSCs by targeting those expressing GPI-80. Its payload is saporin, a ribosomal toxin, which leads to the killing of the targeted cells. Preclinical studies in fetal sheep models have demonstrated its ability to enhance engraftment of human donor cells in both bone marrow and peripheral blood, with a tendency to skew engraftment towards the myeloid lineage. The drug has shown minimal off-target effects and a favorable safety profile in these experimental settings, positioning it as a potential candidate for achieving therapeutic levels of HSC engraftment after IUTx for genetic disorders.