Vor Biopharma’s mission is to develop transformative treatments for patients suffering from hematological malignancies. We are a clinical-stage cell therapy company combining a novel patient engineering approach with targeted therapies to provide a single company solution for patients suffering from hematological malignancies. For many patients, the only way to achieve durable remission or a cure is through hematopoietic stem cell transplant (HSCT). Despite undergoing HSCT, about 40% of acute myeloid leukemia (AML) patients relapse and face an extremely poor prognosis, with two-year survival rates of less than 20%.

We are developing our lead eHSC product candidate, VOR33, and our companion therapeutic, VCAR33, which together, we believe, have the potential to transform the treatment paradigm for AML and other hematological malignancies. The U.S. Food and Drug Administration (FDA) approved our Investigational New Drug (IND) application for VOR33 in January 2021, and we intend to initiate a first-in-human Phase 1/2a trial of VOR33 in AML patients in combination with Mylotarg, an FDA-approved CD33-directed therapy owned by Pfizer, in the first half of 2021. We expect initial data from this trial to be reported in late 2021 or in the first half of 2022.

We use gene editing to remove cancer drug targets from healthy cells to protect the patient’s bone marrow from the cancer treatment’s toxicity. VOR33 consists of hematopoietic stem cells engineered to lack the CD33 protein; this is a cell therapy candidate intended to replace the HSCT standard of care for AML patients at high risk for relapse. (eHSC stands for engineered hematopoietic stem cell.)

(Note: Vor Biopharma said its IPO was upsized at its Feb. 4, 2021, pricing: 9.83 million shares at $18 – up 8.82 million shares at a $16-to-$18 price range – to raise 196.6 million.)