Our Platform Aspect Ratio 1200 384

Our Platform

We have built a proprietary platform for the treatment of Acute Myeloid Leukemia (AML) and other blood cancers.

We have built a technology platform to realize our vision that allows for selective cancer targeting with highly potent targeted therapies by leveraging our expertise and recent advances in stem cell biology and genome engineering.

Our approach is in stark contrast to conventional approaches that have focused solely on developing the therapeutic and have faced clinical limitations due to toxicities. The key components of our proprietary platform are stem cell biology and manufacturing expertise, genome engineering to HSCs, and unlocking the potential of targeted therapies.

Stem Cell Biology Aspect Ratio 600 400

Leveraging stem cell biology and manufacturing expertise

We have built an extensive understanding of the biology of HSCs to enable our shielded transplants to retain their cellular viability and functionality during manipulation. In addition, we have built process development expertise centered around HSCs, enabling us to process these cells quickly, precisely, reproducibly, and efficiently for patients. We are continuing to develop our in-house clinical GMP manufacturing capabilities and facilities to further allow us to leverage our expertise and maintain strategic control over the manufacturing process.

Platform Genome Engineering Aspect Ratio 600 400

Applying genome
engineering to HSCs

Recent developments in genome engineering allow permanent changes to DNA in cells and all their progeny. We have assembled a team with extensive experience in applying genome engineering technologies to HSCs, which display distinct DNA repair mechanisms compared to many other cell types. We possess expertise in a variety of genome engineering technologies including CRISPR-Cas9, CRISPR analog enzymes and base editing, and we are capable of multiplex editing using a variety of techniques, all with the objective of creating various shielded transplants.

Platform Car T Aspect Ratio 600 400

Unlocking the potential of
targeted therapies

We believe our shielded transplants are a potential solution to the lack of tumor-specific targets and can enable selective cancer targeting. We are directly developing CAR-T therapies, which we believe is the most potent form of targeted therapies available. Our CAR-T product candidates are uniquely generated from prior healthy transplant donors where these cells are healthy, more stemlike compared to other cell sources, and exactly matched to the patient since the patient’s blood and immune system was previously reconstituted by the transplant donor. Our objective is to create highly potent CAR-T therapies that are designed to strongly proliferate and persist in patients, in order to drive prolonged relapse-free survival or induce cures in patients.

Our goal is to replace the patient’s HSCs with next-generation, treatment-resistant eHSCs that unlock the potential of highly potent targeted therapies by leveraging our new treatment platform and CAR-T cells, stem cell biology, and genome engineering expertise.

Current Challenge with Standard of Care Transplants for AML

Stem Cells from Healthy Donor

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

Re-emerging Cancer Cells

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

Vor Bio's Shielded Stem Cells

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.

Cells targeted with ADC and CAR-T therapy and shielded cells

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.

Related Publications

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Poster Presentation

ESGCT – 2022 – Poster Presentation P150

October 11, 2022

Efficient knockout of both CD33 and CLL-1 by multiplex genome editing of human hematopoietic stem cells enhances the potential of next- generation transplants for acute myeloid leukemia (AML) treatment

Poster Presentation

ISMB – 2022 – Poster Presentation 859

July 10, 2022

TransACT enhances detection and characterization of translocation events from high-throughput sequencing data at base-pair resolution for gene editing products

Poster Presentation

EHA – 2022 – Poster Presentation 1429

June 9, 2022

Multiplex Deletion of Myeloid Antigens CD33 and CLL-1 by CRISPR/Cas9 in Human Hematopoietic Stem Cells Highlights the Potential of Next-Generation Transplantation for AML Treatment

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The Science Behind the Vor Bio platform

We are applying proven technologies in new ways. Learn more about the science behind our approach.

Learn About The Science
Pipeline

We are working on programs to bring our approach and the possibility of a cure to patients with high-risk blood cancers.

See Our Pipeline