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Our Investigational Treatment Approach

A new and transformative approach targets Acute Myeloid Leukemia (AML) and other blood cancers

For some types of Acute Myeloid Leukemia (AML), a Hematopoietic Cell Transplant (HCT) is the only potentially curative treatment option.

However, in many cases, AML can relapse even after HCT. A major challenge for AML treatment after Hematopoeitic Cell Transplant (HCT) is that many of the current AML therapies that are intended to kill the cancer cells can also target and destroy healthy blood cells.

Our AML targeted therapy approach is based on an elegant idea: a shielded transplant that enables cancer cells to be directly targeted for destruction.

Current Challenge with Standard of Care Transplants for AML

Patient receives unmodified stem cells (from healthy donor) that engraft and rebuild the immune system.

Watchful waiting is the only option. In half of patients, the cancer returns, and the 2 year survival rate is <20%.

How the Trem-cel Treatment System Works

Vor Bio's Shielded Stem Cells (trem-cel)

Patient receives stem cells (from healthy donor) that have been modified using gene editing to remove CD33 (trem-cel). The shielded stem cells engraft and rebuild the immune system.

ADC and CAR-T targeting cell and Vor Bio's shielded stem cells

A targeted therapy, such as an ADC or CAR-T, can now be used to kill any residual cancer cells. The shielded stem cells persist and remain unaffected.