Vor Bio is developing a proprietary platform built on Hematopoietic Stem Cell (HSC) biology, genome engineering, and Chimeric Antigen Receptor T (CAR-T) cells that has the potential to protect HSCs from targeted therapeutics, enabling the use of modalities such as Antibody Drug Conjugates (ADCs) and CAR-Ts without risk of graft failure or cytopenia.
Our scientists and engineers are developing a proprietary platform that genetically modifies healthy donor HSCs to remove select cell surface targets, making these HSCs and their progeny resistant to targeted therapies. This would enable targeted therapies to selectively destroy cancer cells while passing over healthy cells. Limiting on-target toxicity previously associated with targeted therapies may allow their use soon after Hematopoietic Cell Transplant (HCT), enabling new treatment opportunities for these patients. By combining our engineered HSCs with targeted therapies—including CAR-Ts, bispecific antibodies, and ADCs—we have the potential to completely replace the traditional standard of care for blood cancers such as AML.
Preclinical data supports the viability of this new approach, demonstrating cellular protection without compromising cell populations or function. We are committed to sharing with the scientific community the data that underscores our approach. In partnership with leading transplant centers across North America, we are engaging in a first in-human clinical trial for this breakthrough approach.
Trem-cel, a CRISPR/Cas9 gene-edited allograft lacking CD33, shows rapid primary engraftment with CD33-negative hematopoiesis in patients with high-risk AML and avoids hematopoietic toxicity during gemtuzumab ozogamicin (GO) post-hematopoietic cell transplant (HCT) maintenance
Novel CLL-1-directed CAR-T cells Mediate Potent Antigen-specific Cytolytic Activity in
Mouse Models of Acute Myeloid Leukemia
Clinical Holds for Cell and Gene Therapy Trials: Risks, Impact, and Lessons Learned
GUMM: A Purpose-Built Computational Workflow for Single Cell Genotyping of Gene Editing Experiments
Development of a Gene Edited Next-Generation Hematopoietic Cell Transplant to Enable Acute Myeloid Leukemia Treatment by Solving Off-Tumor Toxicity
A Single Cell DNA Sequencing Resource and Computational Approach to Quantify CRISPR-Cas9 Gene Editing Allelism
CD33-Deleted Hematopoietic Stem and Progenitor Cells Display Normal Engraftment after Hematopoietic Cell Transplant (HCT) and Tolerate Post-HCT GemtuzumabOzogamicin(GO) Without Cytopenias
Gene Editing and Immunotherapeutic Targeting of ADGRE2/EMR2 to Enable Combinatorial Targeting in Acute Myeloid Leukemia (AML)
Initial First-In-Human Results: CD33-Deleted Hematopoietic Stem and Progenitor Cells Display Normal Engraftment after Hematopoietic Cell Transplantation (HCT) and Tolerate Post-HCT Gemtuzumab Ozogamicin (GO) without Cytopenias
