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Our Investigational Programs

Treatment-resistant HCTs that allow new, potentially curative targeted therapy opportunities for blood cancers

By protecting healthy blood cells, our approach to Hematopoietic Cell Transplant (HCT) expands the anti-leukemia armamentarium for patients with AML and other blood cancers.

Our proprietary approach genetically modifies healthy donor HSCs to remove select cell surface targets that are shared between healthy blood cells and cancer cells. This approach aims to enable targeted therapies to destroy cancer cells selectively while preserving healthy blood cells. Protection of healthy blood cells can expand the use of different therapeutic approaches following transplant that have not been possible previously. By combining our engineered HSCs with targeted leukemia therapies—including Antibody Drug Conjugates (ADCs) and Chimeric Antigen Receptor T (CAR-T) cells—we hope to advance the standard of care for AML and other blood cancers.

Our clinical data have demonstrated that all patients transplanted thus far with trem-cel achieved primary engraftment as well as a high level of CD33-negative hematopoiesis, consistent with the dispensability of CD33. Additionally, all three patients treated with Mylotarg have demonstrated durable hematologic protection from deep cypotenias through repeat doses.

Investigational Treatment Process

The patient is prepared to receive a hematopoietic cell transplant and HSCs are collected from a fully HLA-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 trem-cel transplant

Once these eHSCs engraft, the patient may then receive a targeted therapy that selectively attacks the leukemia cells that express the target protein, as the healthy blood cells are now invisible to the targeting therapy. In VBP101, an approved antibody drug conjugate (ADC), Mylotarg™ is being administered.

Target Attack

Learn more about our investigational treatment approach

In partnership with leading transplant centers across North America, we are actively enrolling patients with high-risk AML who may benefit from this approach.

To learn more about our clinical trials, please see our clinicaltrials.gov listings:

If you have questions, please contact us.

The science behind the Vor Bio platform

We are applying proven technologies in new ways by genetically modifying healthy donor HSCs for protection and enabling use of modalities such as ADCs and CAR-Ts post-transplant.

Clinical Trials

Vor Bio is currently conducting two clinical trials for AML.