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

Summary

This is a Phase 1/2a, multicenter, open-label, first-in-human (FIH) study of VOR33 in participants with AML or MDS who are undergoing human leukocyte antigen (HLA)-matched allogeneic hematopoietic cell transplant (HCT).

Timeline

Start

2021-12-16

Primary completion

2025-05-28

Completion

2025-05-28

Outcome

Met primary endpoint

Stopped (Business): “Lack of Funding”

paper All 30 patients receiving trem-cel achieved the primary safety endpoint of neutrophil engraftment by day 28 PMID 42120728 ↗

Publications

• DiPersio JF, Koehne G, Shah NN, Bernard L, Suh HC, Koura D, Tamari R, Mushtaq MU, Maakaron J, Rimando J, Kennedy VE, Patel SS, Hudson C, Loken MR, Slapak CA, Lloyd DM, Stanizzi DA, Lee-Sundlov MM, Thosar S, Mundelboim G, Guo G, Ge HG, Li BE, Xavier-Ferrucio J, Hyzy SL, Lin MI, Raffel GD, Cooper BW. CRISPR-Cas9 CD33-deleted allogeneic hematopoietic cell transplantation with gemtuzumab ozogamicin maintenance in AML: a phase 1/2 trial. Nat Med. 2026 May;32(5):1763-1772. doi: 10.1038/s41591-026-04362-1. Epub 2026 May 12.
• Lydeard JR, Lin MI, Ge HG, Halfond A, Wang S, Jones MB, Etchin J, Angelini G, Xavier-Ferrucio J, Lisle J, Salvadore K, Keschner Y, Mager H, Scherer J, Hu J, Mukherjee S, Chakraborty T. Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity. Mol Ther Methods Clin Dev. 2023 Oct 13;31:101135. doi: 10.1016/j.omtm.2023.101135. eCollection 2023 Dec 14.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Gemtuzumab Ozogamicin	Monoclonal antibody	—	—
Subject	tremtelectogene empogeditemcel	Cell therapy	—	—

Indications