Targeted Therapies in Cancer

(VOR101)

Immune System

TRIAL PHASE
MECHANISMINDICATION(S)PRODUCT NAMEPreclinicalPhase 1Phase 2Phase 3
Targeted Therapies in Cancer
Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML)
Vor
(81.3%*)

Immune System

Targeted Therapies in Cancer
INDICATION(S):Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML)
NAME:Vor (81.3%*)
STAGE:Preclinical
Targeted Therapies in Cancer

We are developing novel cell therapies with broad potential for treating cancer. Our 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 B cell malignancies. However, these and similar technologies target both cancer and normal cells, causing substantial toxicities and limiting their potential. We are taking a fundamentally novel approach for targeting cancer selectively by developing antigen-modified hematopoietic stem cells (amHSCs). These amHSCs generate healthy, functional cells that are protected from depletion by cancer-targeted therapies.

  • Patient Need & Market Potential
    • The prognosis for relapsed and refractory blood-borne malignancies remains bleak, despite significant progress in recent years.
    • Engineered cell therapies, particularly CAR T-cells, have been successfully applied to treat B-cell malignancies. However, these therapies cause substantial toxicities and are not effective with non B-cell malignancies. Extending the applicability of CAR T-cells beyond B-cell malignancies has been difficult due to challenges in selectively targeting cancer cells without affecting healthy cells.
    • There is a need for new approaches that could enable successful treatment of not only B-cell malignancies with a far superior safety profile, but also target non-B-cell malignancies – our approach has the potential to address this need.
    • Our technology may also be used to improve the safety profile of existing 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
    • We have 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.
    • Our IP portfolio currently consists of 5 patent applications in 2 families and includes IP licensed exclusively from Columbia University as well as IP owned by Vor.
  • Team
    • Advisors include Dr. Siddhartha Mukherjee (Columbia University), Dr. Sanjiv Sam Gambhir (Stanford University), Dr. Dan Littman (NYU School of Medicine, Howard Hughes Medical Institute; member of the Board of Pfizer), Dr. Crystal Mackall (Stanford University), Dr. Derrick Rossi (Harvard), and Dr. Justin Stebbing (Imperial College London).
    • The team includes Mr. David Steinberg (PureTech Health), Dr. Joseph Bolen (PureTech Health), and Dr. Aleks Radovic-Moreno (PureTech Health), as acting Chief Executive Officer, acting Chief Scientific Officer, and operations lead, respectively.
  • Milestones Achieved
    • We established lab operations in 2018.
    • We expanded our scientific team with the addition of key hires.
  • Expected Milestones
    • Results are anticipated from our proof-of-concept study in preclinical models in 2018.
    • We plan to initiate GMP process development in by 2019.
    • First-in-human clinical studies are expected to begin in 2020 or 2021.

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.