Vor Biopharma Announces Issuance of Foundational U.S. Patent Broadly Covering Compositions and Methods to Treat Hematologic Malignancies
Vor Biopharma, an immuno-oncology company pioneering hematopoietic stem cell (HSC) therapies for the treatment of hematological malignancies and affiliate of PureTech Health plc (LSE: PRTC), today announced that the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 10,137,155 related to the Company’s technology platform. This foundational patent is the first of its kind in the immuno-oncology field and broadly covers compositions and therapeutic methods related to using novel modified HSCs to enable targeted immunotherapies. The platform technology underlies Vor’s pipeline of immuno-oncology candidates, including lead candidate VOR33.
The now patented technology is designed to address the fundamental limitations of traditional targeted therapies, including antibody-drug conjugates, bispecific antibodies, and chimeric antigen receptor (CAR) T cells, for the treatment of hematologic malignancies. These existing therapies target antigens on malignant cells that also appear on healthy cells, which can result in mass depletion of critical life-sustaining cells. Vor’s approach employs the use of antigen-modified hematopoietic stem cells (amHSCs), which are designed to repopulate a patient’s blood with cells that have a benign genetic modification to a specific surface antigen that escapes targeting and protects the cells from depletion by targeted immunotherapies. By sparing healthy blood cells, this approach may potentially improve patient safety, enable maximal dosing of cancer-targeted therapies, increase the number of potential patient populations that could benefit from these therapies, and expand the reach of targeted immunotherapies beyond B-cell cancers to a broad range of hematological malignancies, including acute myeloid leukemia (AML).
“The USPTO’s allowance of these broad claims provides validation that our approach and intellectual property are novel and first-in-class,” said Aleks Radovic-Moreno, Ph.D., PureTech Health Vice President and Vor program lead. “The compositions and methods covered in the patent protect our lead product candidate as we continue development of our technology towards the clinic. This technology has the potential to provide a novel therapeutic approach for patients with aggressive blood cancers that otherwise have very few treatment options and poor prognoses.”
The relevant intellectual property is exclusively licensed to Vor Biopharma and is based on technology developed by Siddhartha Mukherjee, M.D., D. Phil, Associate Professor of Medicine at Columbia University and a Staff Physician at Columbia University Medical Center, and his colleagues.
“This approach has the potential to broaden the use of targeted immunotherapies beyond B-cell cancers, and to help patients who have very limited treatment options,” said Dr. Mukherjee.
Vor’s lead product candidate, VOR33, is designed to enable maximal CD33-targeted immunotherapy. CD33, a target that is present in the vast majority of acute myeloid leukemia (AML) patients, is also expressed in normal myeloid progenitor cells. Depletion of normal myeloid progenitor cells prevents the beneficial use of several CD33-targeted therapies at important stages throughout the treatment process, at higher doses, and for longer periods of time. By enabling new CD33-targeted therapies, VOR33 has the potential to overcome these challenges and improve treatment for AML. Vor anticipates initiating IND-enabling studies for VOR33 in early 2019.
About Vor Biopharma
Vor Biopharma is an immuno-oncology company developing novel antibody and 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 to 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.
Vor’s platform is broad and can potentially be used to vastly improve the therapeutic window of several CAR-modified cells (such as CAR T cells, CAR NK cells, and others), expanding the reach of CAR-modified cells 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 and solid tumors. When combined with targeted therapies, this technology could potentially enable transformative outcomes in patients with otherwise grim prognoses.
This technology platform is covered by broad intellectual property exclusively licensed from Columbia University, as well as Vor-owned intellectual property. Co-founded by PureTech Health (LSE: PRTC), Vor is working with some of the world’s leading oncologists and immunologists to develop a pipeline of potentially life-altering immunotherapies that extend beyond what is possible with current treatment.
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