Puretech Ownership
Product Candidate
Initial Indication(s)
Stage of Development
11.8% Equity
VOR33
Acute myeloid leukemia
Preclinical
Engineered hematopoietic stem cells designed to limit on-target toxicities associated with targeted therapies

Founded by PureTech, Vor is a cell therapy company that combines a novel patient engineering approach with targeted therapies to provide a single company solution for patients suffering from hematological malignancies.

Vor’s proprietary platform leverages its expertise in hematopoietic stem cell (HSC) biology and genome engineering to remove surface targets expressed by cancer cells by genetically modifying HSCs. By removing these targets, Vor makes these HSCs and their progeny unrecognizable by targeted therapies and enables these treatments to selectively destroy cancerous cells while sparing healthy cells. As a result, Vor’s engineered HSCs (eHSCs) are designed to limit the on-target toxicities associated with these targeted therapies, or companion therapeutics, thereby enhancing their utility and broadening their applicability.

  • Program Discovery Process by the PureTech Team
    • PureTech was interested in approaches to treat hematological malignancies that currently have poor response rates or poor adverse event profiles despite recent advances in cell therapies and targeted therapies. PureTech engaged leading hematological cancer specialists and became aware of work from the laboratory of Vor Scientific Board chair Siddhartha Mukherjee, MD, PhD, assistant professor of Medicine at Columbia University and Pulitzer Prize-winning author of The Emperor of All Maladies: A Biography of Cancer. Dr. Mukherjee pioneered the idea of genetically engineering stem cells to eliminate a particular target such that healthy stem cells and progeny cells would be spared from targeted cancer therapy. PureTech worked with Dr. Mukherjee on this IP, which Vor exclusively in-licensed from Columbia in April 2016, and on advancing this concept through critical POC experiments. With our support, Vor secured additional intellectual property rights (both in-licensed from Columbia and owned by Vor), assembled an excellent research team and completed a round of fundraising.
    • In July 2019, Bill Lundberg, MD, was appointed to Vor’s board of directors. In August 2019, Robert Ang, MBBS, MBA, was appointed president and chief executive officer of Vor. In May 2020, Vor announced the appointment of Nathan Jorgensen, PhD, as chief financial officer, in July 2020, Vor announced the closing of a $110M Series B financing and the appointments of Daniella Beckman and David Lubner to its board of directors and Christopher Slapak, MD, as chief medical officer, in August 2020, Vor announced the appointment of John King as chief commercial officer, and in October 2020 Vor announced the appointment of Matthew Patterson to its board of directors.
  • Patient Need & Market Potential
    • The prognosis for relapsed and refractory blood-borne malignancies is very poor and can be measured in a few months, depending on patient-specific risk factors. For example, for acute myeloid leukemia (AML), which affects approximately 60,000 patients at any one time in the United States, only about 30 percent of patients with active disease following a bone marrow transplant survive past 12 months.
    • Targeted therapies, such as CAR-T cells and bispecific antibodies, antibody-drug conjugates, and conventional mAbs, have shown excellent outcomes, particularly in patients with certain hematologic malignancies expressing B cell markers. However, these targeted therapies frequently target both cancer and normal cells, causing substantial toxicities and limiting their potential. There is a need for new strategies that can enable selectively targeting cancer cells without impacting a patient’s normal cells.
  • Innovative Approach to Solving the Problem
    • Vor’s proprietary platform leverages its expertise in HSC biology and genome engineering to remove surface targets expressed by cancer cells by genetically modifying HSCs. By removing these targets, Vor makes these HSCs and their progeny unrecognizable by targeted therapies and enables these treatments to selectively destroy cancerous cells while sparing healthy cells. As a result, Vor’s eHSCs are designed to limit the on-target toxicities associated with these targeted therapies, or companion therapeutics, thereby enhancing their utility and broadening their applicability.
    • Vor’s platform and expertise allow it to advance its goal of replacing the patient’s HSCs with next-generation, treatment-resistant eHSCs that unlock the potential of highly-potent target therapies.
    • VOR33 is Vor’s eHSC product candidate designed to transform the standard of care in AML and potentially other myeloid malignancies. To create VOR33, Vor genetically modifies donor HSCs in order to remove the CD33 surface target that is highly expressed in most AML cells.
  • Milestones Achieved
    • In May 2019, preclinical research was published in the scientific journal Proceedings of the National Academy of Sciences supporting Vor’s novel approach to treating cancer via eHSCs.
    • Vor has achieved ex vivo POC for its technology and received validation of its technology in engineered humanized mouse models.
    • Vor held a pre-IND meeting with the FDA in January 2020 to gather important feedback to assemble the data package necessary for a potential IND filing.
  • Expected Milestones
    • Vor expects to initiate a Phase 1 study of VOR33 in acute myeloid leukemia in 2021.

Vor’s platform is designed to optimize targeted therapies such as CAR-T cells and bispecific antibodies, antibody-drug conjugates and conventional mAbs. Vor intends to develop VOR33 as an HSC transplant product candidate to replace the standard of care in transplant settings. Once the VOR33 cells have engrafted, patients can potentially be treated with anti-CD33 therapies, such as Mylotarg or a CAR-T therapy product candidate, with limited on-target toxicity. The combination of VOR33 and CD33-directed therapies has the potential to lead to durable antitumor activity.