Vor Biopharma is an immuno-oncology company pioneering haematopoietic stem cell (HSC) therapies for the treatment of haematological malignancies and affiliate of PureTech Health.
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 haematologic 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 haematopoietic 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 haematological malignancies, including acute myeloid leukaemia (AML).
PureTech Health Vice President and Vor programme lead Aleks Radovic-Moreno said: “The USPTO’s allowance of these broad claims provides validation that our approach and intellectual property are novel and first-in-class.
“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, MD, 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.
About VOR33
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 leukaemia (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.