349 Vor Bbiopharma 2022.10.05 Genevieve De Manio Photography Scaled Aspect Ratio 1200 384

Our Investigational Program

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

By protecting healthy blood cells, our approach to Hematopoietic Stem Cell Transplant (HSCT) expands the anti-leukemia armamentarium for patients with AML and other blood cancers who have a poor prognosis with a high risk of relapse.

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 would enable targeted therapies to destroy cancer cells selectively while preserving healthy blood cells. Protection of healthy blood cells could 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.

Robust preclinical data supports the promise of this novel approach. This data demonstrates that our engineered HSCs retain healthy cell function as demonstrated by the production of downstream progenitor cells with normal function. Furthermore, in preclinical models, transplant with our engineered HSCs enables the successful clearing of AML cells with targeted therapies while preserving the normal blood cells.

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 diseased cells that still have the target protein.

Healthy cells are invisible to targeted therapies

Target Attack

Learn more about our investigational treatment approach

We are now working to bring this therapy and the possibility of a cure to patients with high-risk AML, a disease that carries a poor prognosis even with aggressive therapies including traditional HSCT, which has been the standard of care for decades. In partnership with leading transplant centers across North America, we are actively enrolling patients with high-risk AML who are first-time HSCT candidates to participate in our first in-human Phase 1/2a clinical trial (VBP101).

To learn more about our clinical trial, VBP101, please see our clinicaltrials.gov listing:

Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML

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

We have initiated VBP101, our first-in-human Phase 1/2a trial of trem-cel (formerly VOR33) in combination with Mylotarg™.