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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 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

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

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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

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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.

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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.

Our clinical data has demonstrated the potential of our approach to shield patients’ healthy cells and enable targeted therapies post-transplant.

Acute Myeloid Leukemia Targeted Therapy Treatment Process

The patient is prepared to receive a hematopoietic stem cell transplant and HSCs are collected from a matched, healthy donor.

Hsc Matched Donor

HSCs collected from matched healthy donor

Genome Engineering

Genome engineering is used to remove desired surface targets

Hsc Transplant

Patient receives Vor Bio eHSC transplant

Once these eHSCs engraft, the patient may then receive a therapy that now attacks only the cancer cells that still have the target protein.

 

Healthy cells are shielded from targeted therapies

Target Attack

Learn more about our investigational treatment approach:

Our clinical trials

Vor Bio is currently conducting two clinical trials for AML and MDS.