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 shield healthy cells from targeted therapeutics, enabling the use of modalities such as Antibody Drug Conjugates (ADCs) and CAR-Ts which by themselves are highly toxic.
Vor Bio scientists and engineers are developing a proprietary platform for the treatment of blood cancers 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 and ADCs—we have the potential to completely replace the traditional standard of care for blood cancers such as AML and MDS.
In partnership with leading transplant centers across North America, we are conducting two clinical trials to evaluate this breakthrough approach. Our clinical data has demonstrated that our approach has the potential to shield patients’ healthy cells and enable targeted therapies post-transplant.
Trem-cel, a CRISPR/Cas9 Gene-Edited Allograft Lacking CD33, Shows Rapid Primary Engraftment with CD33-Negative Hematopoiesis in Patients with High-Risk Acute Myeloid Leukemia (AML) and Avoids Hematopoietic Toxicity During Gemtuzumab Ozogamicin (GO) Maintenance Post-Hematopoietic Cell Transplant (HCT)
Phase 1/2 Study of Donor-Derived Anti-CD33 Chimeric Antigen Receptor Expressing T Cells (VCAR33ALLO) in Patients with Relapsed or Refractory Acute Myeloid Leukemia After Allogeneic Hematopoietic Cell Transplantation (Trial in Progress)
Leveraging CRISPR/Cas9 and HDR to create an engineered CD33 CAR-T to target AML
Phase 1/2 Study of Donor-Derived Anti-CD33 Chimeric Antigen Receptor Expressing T Cells (VCAR33) in Patients with Relapsed or Refractory Acute Myeloid Leukemia after Allogeneic Hematopoietic Cell Transplantation
Non-human primate models have been crucial for preclinical development of hematopoietic stem and progenitor cell (HSPC) therapies, since they share many properties with humans.
Trem-Cel, a CRISPR/Cas9 Gene-Edited Allograft Lacking CD33, Shows Rapid Primary Engraftment with CD33-Negative Hematopoiesis in Patients with High-Risk Acute Myeloid Leukemia (AML) and Avoids Hematopoietic Toxicity during Gemtuzumab Ozogamicin (GO) Maintenance Post-Hematopoietic Cell Transplant (HCT)
CD33-Deleted Hematopoietic Cells (Trem-Cel) Are Protected from CD33xCD3 Bispecific Antibody Treatment and Produce Significantly Reduced Levels of Inflammatory Cytokines in Preclinical Studies
Multimodal Atlas of Paired Diagnosis and Relapse AML Samples Enables Novel Therapeutic Targeting of Surface Antigens
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
