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Vor Bio Successfully Demonstrates Multiplex Editing of Hematopoietic Stem Cells for Next-generation AML Treatment Presented at EHA
Multiplex deletion of myeloid antigens CD33 and CLL-1 in human hematopoietic stem cells demonstrates potential of next-generation HSC transplants for
About this update from Vor Biopharma Inc.
[{"type":"text","content":"Multiplex deletion of myeloid antigens CD33 and CLL-1 in human hematopoietic stem cells demonstrates potential of next-generation HSC transplants for treatment of acute myeloid leukemia Dual edited hematopoietic stem and progenitor cells persisted long-term post engraftment, with minimum translocation risk CAMBRIDGE, Mass., June 10, 2022 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today announced successful first of its kind dual editing of CD33 and CLL-1 in human hematopoietic stem cells (HSCs) demonstrating continued progress on its novel approach for the treatment of acute myeloid leukemia (AML). The data is being presented at the European Hematology Association Congress in Vienna, Austria. The pre-clinical data demonstrates that multiplex deletion by CRISPR/Cas9 of CD33 and CLL-1 from human CD34+ hematopoietic stem and progenitor cells (HSPCs) maintained cell function and persisted long-term post engraftment in vivo, with a high-level of editing, no counterselection, and minimum translocation risk when compared to unedited control cells. In addition, genetically modifying HSPCs to remove select cell surface targets does not impair their function and these dual engineered cells showed significant protection from targeted immunotherapy in vitro. “As most tumor antigens are also expressed on normal blood cells or bone marrow, traditional targeted immunotherapy increases the risk of severe cytopenia,” explained Tirtha Chakraborty, Ph.D., Vor Bio’s Chief Scientific Officer. “Our pre-clinical proof-of-concept data shows that the knockout of both CD33 and CLL-1 from allogeneic HSC grafts can restrict these antigens to only the patients’ AML cells, thereby protecting the healthy HSCs and making them resistant to the toxic effects of targeted therapies. These data validate that CD33 and CLL-1 may both be independently biologically dispensable, where we envision our multiplex treatment system has the potential to avoid concerns regarding tumor heterogeneity or escape mechanisms.” AML is the most common type of acute leukemia in adults and is characterized by excessive proliferation of immature myeloid progenitor cells and their failure to properly differentiate into mature blood cells. Healthy donor HSC transplantation is the standard of care and currently around 40% of patients wit...