Urgent Needs in Cancer

Acute Myeloid Leukemia

Acute myeloid leukemia (AML) is a cancer of the bone marrow that needs more effective therapies. According to the National Cancer Institute, more than 60,000 people in the U.S. have AML, and less than 30% of patients survive five years following diagnosis.

AML cancer cells frequently express a protein called CD33. Currently, treating AML with a therapy that targets CD33 can be effective, but the therapy may be limited in utility due to toxicity to the normal blood and bone marrow.

Lead Candidate: VOR33

Our lead engineered hematopoietic stem cell (eHSC) product candidate, VOR33, is in preclinical development to help treat AML.

VOR33 eHSCs lack the protein CD33. Once these cells are transplanted into a cancer patient, CD33 becomes a far more cancer-specific target, potentially avoiding toxicity to the normal blood and bone marrow associated with CD33-targeted therapies. In doing so, we aim for VOR33 to improve the therapeutic window and effectiveness of CD33-targeted therapies, as well as create new treatment opportunities for these patients, thereby potentially broadening the clinical benefit to patients suffering from AML.

Hematopoietic stem cells treated with VOR33 produce appropriate cell lineages

Diagram

Leveraging the Vor Platform Beyond CD33

We are actively screening a range of different molecular targets that fulfill two simple criteria: first, that they are targetable on cancer cells, and second, that these proteins display biological redundancy.

Based on these two elegant criteria, we are discovering, identifying and validating targets that are potentially suitable for future medicines that treat blood cancers and beyond. Targets may include those which are already an area of focus in drug development or may be part of a wholly novel approach to cancer targets made possible only through our platform.

Each additional target beyond CD33 can lead to a standalone product. More importantly, edits to these targets can be multiplexed (that is, deletion of multiple targets in the same eHSCs), which could allow patients to benefit from a far broader range of treatment options such as multiple complementary targeted therapies given in sequence or as treatment combinations.