Vor Biopharma Announces Issuance of Foundational U.S. Patent Broadly Covering Compositions and Methods to Treat Hematologic Malignancies

, 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

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

“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 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.