VOR

(VOR33)

 

TRIAL PHASE
Product Candidate
PureTech Ownership
Initial Indications
Preclinical
Phase 1
Phase 2
Phase 3
VOR33
30.2% (Vor)
AML

Initial Indication(s):

Acute Myeloid Leukemia (AML)

Patient Population:

~60K

Collaborators:

novartisOUP

Key Differentiation:

Advancing a new approach to selectively protect healthy normal cells from targeted therapies being used to treat hematologic malignancies 



VOR33
AML
30.2% (Vor)
Preclinical

Initial Indication(s):

Acute Myeloid Leukemia (AML)

Patient Population:

~60K

Collaborators:

novartisOUP

Key Differentiation:

Advancing a new approach to selectively protect healthy normal cells from targeted therapies being used to treat hematologic malignancies 



Selectively targeting cancer cells while sparing normal cells using modified HSCs

Founded by PureTech, Vor is developing cell therapies with broad potential for treating cancer. Vor’s key differentiation is a focus on technologies that can selectively target cancer cells without impacting normal cells. Engineered cells, such as chimeric antigen receptor (CAR) T cells, are now FDA-approved drugs for treating hematologic malignancies. However, these and similar technologies target both cancer and normal cells, causing substantial toxicities and limiting their potential. Vor is taking a fundamentally novel approach for targeting cancer selectively by developing engineered hematopoietic stem cells (HSCs). Vor’s differentiated approach is designed to enable broad targeting of lineage antigens, which are attractive targets but face serious limitations, since they are expressed on both healthy cells and cancerous cells. Vor’s eHSCs do not display a particular antigen, therefore making these antigens tumor-specific and potentially safer to target while protecting the healthy blood cells from depletion.

Our platform is broad, and can be used to enhance the therapeutic window of several CAR-modified cells (such as CAR T-cells, CAR NK cells, and others), as well as other therapies such as antibody-drug conjugates or conventional antibodies. When combined with targeted therapies, this technology could enable transformative outcomes in patients with otherwise grim prognoses.

  • Patient Need & Market Potential
    • The prognosis for relapsed and refractory blood-borne malignancies is very poor and can be measured in a few short months, depending on patient-specific risk factors. Specifically for 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 β 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.
    • Vor’s technology may also be used to substantially improve the safety profile of existing targeted immunotherapies (including CAR T technology) for several blood-borne malignancies.
  • Our Approach to Solving the Problem
    • Current CAR T therapies are limited primarily to B-cell malignancies, where patients can apparently tolerate loss of healthy B-cells along with the cancerous tissue.
    • We are advancing a new approach to selectively protect healthy cells from targeted therapies against B-cell as well as other hematologic malignancies.
    • This approach consists of a targeted CAR T therapy, which is used to eliminate cells expressing certain antigen types that appear on cancerous tissue but may also appear on healthy tissue.
    • To address the potential toxic effects and loss of healthy tissue, a hematopoietic cell transplantation (HCT) with antigen-modified hematopoietic stem cells (amHSCs) is performed.
    • These amHSCs generate healthy, functional hematopoietic cells that are protected from depletion by cancer-targeted therapies.
    • HCT, which is a standard procedure for many patients, can be performed prior to the targeted therapy, or the targeted therapy can be used as a preconditioning regimen to the HCT.
    • In this way, the population of potential target antigens can expand beyond tumor-specific antigens or B-cell antigens.
  • Intellectual Property
    • Vor has broad intellectual property coverage worldwide relating to compositions of matter and methods of using modified hematopoietic stem cells to broaden the number of potential antigens that can be targeted safely by engineered cell therapies.
    • Vor’s IP portfolio currently consists of seventeen (17) patent applications in four (4) families, including a patent that issued in 2018. This includes IP licensed exclusively from Columbia University as well as IP owned by Vor.
  • Team
    • In February 2019, Dr Kush Parmar (5AM Ventures) joined the Board of Directors as Executive Chairman. Additional board members include Dr Bill Lundberg (previously Chief Scientific Officer of CRISPR Therapeutics), Dr Bharatt Chowrira (PureTech) and Dr Josh Resnick (RA Capital Management). Dr Aleks Radovic-Moreno (PureTech) serves as operations lead.

    • Advisors include Dr Siddhartha Mukherjee (Columbia University and Pulitzer Prize Winning Author, The Emperor of All Maladies), Dr Joseph Bolen (PureTech, previously President and Chief Scientific Officer at Moderna and Chief Scientific Officer at Millennium), Dr Hans-Peter Kiem (Fred Hutchinson Cancer Research Center), Dr Dan Littman (NYU School of Medicine, Howard Hughes Medical Institute; member of the Board of Pfizer), Dr Derrick Rossi (Harvard Medical School; Founding CEO of Convelo Therapeutics; Co-founder of Moderna Therapeutics, Intellia Therapeutics, Magenta Therapeutics, and Stelexis Therapeutics), and Dr Justin Stebbing (Imperial College London; published over 600 peer-reviewed papers). 

  • Milestones Achieved
    • Vor has achieved ex vivo proof-of-concept for its technology.
    • Vor received validation of its technology in engineered humanized mouse models.
    • Vor has been granted foundational intellectual property which covers its therapeutic approach.
    • 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 engineered hematopoietic stem cells.

       

  • Collaborations
    • In February 2019, Vor completed a $42 million financing round led by 5AM Ventures and RA Capital Management, with participation from Johnson & Johnson Innovation – JJDC, Novartis Institutes for BioMedical Research, Osage University Partners, and PureTech

Vor’s platform is broad and can potentially be used to vastly improve the therapeutic window of several targeted immunotherapies, such as T cell engagers, antibody drug conjugates, and CAR T cells and others, expanding the reach beyond B-cell malignancies to other myeloid leukemias, such as acute myeloid leukemia, as well as enhancing the effectiveness of other therapies such as antibody-drug conjugates or conventional antibodies targeted against leukemias. When combined with targeted therapies, this technology could potentially enable transformative outcomes in patients with otherwise grim prognoses.