Medical Professionals Aspect Ratio 1200 384

For Medical Professionals

What if potent targeted therapies could be used following HSCT?

Hematopoietic Stem Cell Transplant (HSCT) remains the only potentially curative option for patients with high-risk hematologic malignancies. However, many patients do not achieve long-term remission as around 40% of AML patients will relapse after HSCT. The prognosis for these patients is dismal since many, particularly those with early relapse, cannot tolerate the intensive or targeted chemotherapy that would be indicated in this setting.

What if potent targeted therapies could be used following HSCT? We are developing treatment-resistant HSCs, potentially allowing use of targeted modalities such as ADCs and CAR-Ts in the post-transplant setting without the concerns for graft failure or prolonged cytopenia that currently eliminate many post-transplant treatment options.

By protecting healthy blood cells, our approach to HSC transplant expands the anti-leukemia armamentarium for patients with Acute Myeloid Leukemia (AML) and other blood cancers who have a poor prognosis with a high risk of relapse.

Our Investigational Program

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.

Clinical Trials

We have initiated VBP101, our first-in-human Phase 1/2a trial of trem-cel (formerly VOR33) in combination with Mylotarg, to provide important validating evidence to support our approach.

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