HSCs collected from matched healthy donor
Changing the Paradigm of Cancer Thinking
A word from our scientific founder Dr. Siddhartha Mukherjee:
We seek to make healthy cells invisible to targeted therapies
The traditional tumor target paradigm aims to treat hematological malignancies such as acute myeloid leukemia (AML) by focusing on the specificity and potency of therapies that kill cancer cells expressing a target. However, this paradigm is limited by the expression of tumor targets on healthy cells, resulting in on-target toxicity. On-target toxicity has led to the development failure of many targeted therapies and is often a key mechanism limiting therapeutic use or dose.
Our unique approach aims to help patients fight cancer by unlocking the full potential of targeted therapies with curative intent.
Our proprietary platform changes the traditional tumor target paradigm. Leveraging our expertise in hematopoietic stem cell (HSC) biology and genome engineering, we genetically modify HSCs to remove surface targets expressed by cancer cells and provide these cells as engineered hematopoietic stem cell (eHSC) transplants to patients. We believe that once these cells complete this process and engraft into the patient’s bone marrow, the HSCs and their blood cell progeny will be treatment resistant to targeted therapies and thereby unlock the potential of these therapies to selectively destroy cancerous cells while sparing healthy cells.
Our goal is to replace standard HSC transplants with next-generation, treatment-resistant eHSCs that unlock the potential of highly potent targeted therapies.
We are currently developing our lead eHSC product candidate, VOR33, and our companion therapeutic VCAR33, which together, we believe, have the potential to transform the treatment paradigm for AML and other myeloid malignancies.
The patient is prepared to receive a hematopoietic stem cell transplant and HSCs are collected from a matched, healthy donor.
Genome engineering is used to removed desired surface targets
Patient receives Vor 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 unrecognizable by targeted therapies
Our Proprietary Vor Platform
Leveraging our expertise and recent advances in stem cell biology, genome engineering and targeted therapies, we have built a technology platform to realize our vision of an engineered patient that allows for selective cancer targeting with highly potent companion therapeutics. 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 the following:
Leveraging Stem Cell Biology and Manufacturing Expertise
HSCs are cell types that have unique biology and require specialized genome engineering know-how, as well as specific handling and manipulation processes. We have built considerable expertise manipulating the genes of these cells and have also designed a highly efficient manufacturing process for eHSCs, enabling a rapid vein-to-vein time.
Applying Genome Engineering of Hematopoietic Stem Cells
We have assembled a team with extensive experience in applying genome engineering technologies to HSCs, which display distinct DNA repair mechanisms compared to rapidly dividing cells. Since HSCs are very long-lived, we have developed a series of assays designed to minimize and well-characterize any off-target edits. Furthermore, we have tailored our cell manufacturing process to leverage the most suitable genome engineering technologies for the cells we are creating.
Unlocking the Full 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 cell therapies, whose utility and applicability have previously been limited, in part, by on-target toxicity. We are designing and developing companion therapeutics, including VCAR33, that are specifically optimized for use with our eHSCs in the post-transplant setting.