VOR33

BIOLOGICAL2 trials

Sponsors

Vor Biopharma

Conditions

Leukemia, Myeloid, AcuteMyelodysplastic Syndromes

Phase 1

Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS

TerminatedNCT04849910

Vor BiopharmaLeukemia, Myeloid, Acute, Myelodysplastic Syndromes

Start: 2021-12-16End: 2025-05-28Updated: 2025-07-29

Unknown Phase

A Long-term Follow-up Study of Patients Who Received VOR33

TerminatedNCT05309733

Vor BiopharmaLeukemia, Myeloid, Acute

Start: 2022-04-15End: 2025-05-28Updated: 2025-07-29

Related Papers

CRISPR−Cas9 CD33-deleted allogeneic hematopoietic cell transplantation with gemtuzumab ozogamicin maintenance in AML: a phase 1/2 trial

Nature Medicine2026-05-12

A CD33-Deleted Allograft (Trem-cel) Enables Post-Hematopoietic Cell Transplant (HCT) Maintenance Dosing of Gemtuzumab Ozogamicin (GO) with Therapeutic Levels of Drug Exposure and Low Hematologic and Hepatic Toxicity in Patients with High-Risk Acute Myeloid Leukemia (AML)

Blood2024-11-052 citations

Genome editing in clinical practice: A model study for next-gen hematopoietic cell transplants in hematologic malignancies

Molecular Therapy — Methods & Clinical Development2024-02-26

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)

Blood2023-11-029 citations

CD33-Deleted Hematopoietic Cells (Trem-Cel) Are Protected from CD33xCD3 Bispecific Antibody Treatment and Produce Significantly Reduced Levels of Inflammatory Cytokines in Preclinical Studies

Blood2023-11-02

Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity

Molecular Therapy — Methods & Clinical Development2023-10-1315 citations

P1396: CD33-DELETED HEMATOPOIETIC STEM AND PROGENITOR CELLS DISPLAY NORMAL ENGRAFTMENT AFTER HEMATOPOIETIC CELL TRANSPLANT (HCT) AND TOLERATE POST-HCT GEMTUZUMAB OZOGAMICIN (GO) WITHOUT CYTOPENIAS

HemaSphere2023-08-013 citations

Trial in Progress: A First-in-Human Phase 1/2 Study of Hematopoietic Stem Cell Transplantation (HCT) with VOR33 (CD33-Deleted Allograft) Followed By Treatment with Gemtuzumab Ozogamicin (GO) in Patients with CD33+ Acute Myeloid Leukemia (AML) Who Are at High-Risk of Post-HCT Relapse

Blood2022-11-154 citations