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What if potent targeted therapies could be used following HCT?

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

Following HCT, patients are typically managed via watchful waiting, essentially waiting for the cancer to recur. This is because therapies are often highly toxic to the new bone marrow, which could result in aplasia and put the patient at significant risk. But what if potent targeted therapies could be used safely following HCT? Vor Bio is developing treatment-resistant hematopoietic stem cells (HSCs), potentially allowing the use of targeted modalities such as Antibody Drug Conjugates (ADCs) and Chimeric Antigen Receptor T (CAR-T) therapies in the post-transplant setting without concerns for graft failure or prolonged cytopenia, and could be used prophylactically or therapeutically following detection of marrow blasts.

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 proteins 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

VBP101, our first-in-human Phase 1/2a trial of trem-cel (VOR33) in combination with Mylotarg™ (gemtuzumab ozogamicin), is actively enrolling patients.

The FDA has also cleared our Investigational New Drug (IND) application for VCAR33ALLO, a CAR-T cell therapy drug candidate derived from allogeneic healthy donors that targets CD33. The safety and efficacy of VCAR33ALLO is being studied in the VBP301 trial.

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.