mRNA Delivery to Hematopoietic Stem and Progenitor Cells (HSPC)

eAwaz Medicine

New York – Of the nearly 30 trillion cells in the body, approximately 90% are of hematopoietic origin. Hematopoietic lineages originating in the bone marrow are intimately involved in maintaining homeostasis and human health. Nonetheless, there are hundreds of hematologic or immune-related disorders caused or exacerbated by cells of the hematopoietic lineage. In a disease setting, cells of hematopoietic origin interact with host tissues to drive chronic inflammatory and immune disorders. Because some hematopoietic stem and progenitor cells (HSPC) have self-renewal and pluripotent properties, targeting HSPC has the potential to modulate underlying chronic inflammation and immune-related disorders. Advances in lipid nanoparticle (LNP) technology has allowed for delivery to the bone marrow following systemic LNP administration in vivo. This achievement has enabled Moderna scientists to deliver mRNA directly to bone marrow HSPC in vivo, leading to HSPC transfection and long-term modulation of all hematopoietic lineages. This represents a major milestone in impacting chronic inflammatory and immune related disease.