Vor Biopharma Inc. announced VBP101, its Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (formerly VOR33) in patients with acute myeloid leukemia (AML). These data were presented by Guenther Koehne, MD, PhD, Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida in a poster presentation at the European Hematology Association (EHA) 2023 Congress in Frankfurt, Germany. As previously reported, neutrophil and platelet cell counts were maintained in patient 1 who received multiple Mylotarg doses at 0.5 mg/m2, suggesting protection from Mylotarg-induced hematotoxicity.

The Company also announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for VCAR33ALLO, a T-cell therapy derived from allogeneic healthy donors using a chimeric antigen receptor (CAR) specifically binding to CD33. Trem-cel is planned to be studied in the VBP301 clinical trial, which will focus on patients who have relapsed following allogeneic stem cell transplant where T cells harvested from the original donor are used as starting material for the drug product. VBP101 is a Phase 1/2a, multicenter, open- label, first-in-human trial of trem-cel (VOR33) in participants with AML who are undergoing human leukocyte antigen (HLA)-matched allogeneic HCT.

Trem-cel is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell (HSPC) therapy product, lacking the CD33 protein.