Our Platform Aspect Ratio 1200 384

Our Platform

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

Leveraging our scientific expertise in the development of Chimeric Antigen Receptor T (CAR-T) cells, recent advances in stem cell biology, and genome engineering, our approach to the treatment of blood cancers 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 the Vor Bio platform are:

Stem Cell Biology Aspect Ratio 600 400

Stem cell biology and
manufacturing expertise

We have built an extensive understanding of the biology of Hematopoietic Stem Cells (HSCs) to enable our eHSCs 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 have invested in internal in-house clinical GMP manufacturing capabilities and facilities that allow us to leverage our expertise and to 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.

Platform Car T Aspect Ratio 600 400

Unlocking the potential of
targeted therapies

We believe our eHSCs are a solution to the lack of tumor-specific targets and enable selective cancer targeting. Our solution allows for treatment with potent agents, such as CAR-T therapies, whose utility and applicability have previously been limited, in part, by on-target toxicity. We are designing and developing targeted therapies that are optimized for use with our eHSCs in the post-HCT setting.

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.

Traditional Paradigm

Traditional Paradigm
for Drug Development

Target cancer antigens to kill cancer cells

The Problem


Few unique cancer antigens, so drugs kill both cancer and healthy cells through on-target toxicity

Vor Unique Approach

Vor Bio Paradigm:
Engineered HSCs (eHSCs)

Remove target expression on healthy cells so that killing is cancer-specific

Related Publications

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

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

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

The Science Behind the Vor Bio platform

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


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