Our Platform Aspect Ratio 1200 384

Our Platform

We have built a proprietary technology platform

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 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-HSCT 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 platform and expertise.

Traditional Paradigm

Traditional Paradigm
for Drug Development

Target cancer antigens to kill cancer cells

The Problem

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

In Depth Assessment of Off-target Editing by CRISPR/Cas9 in VOR33, an Engineered Hematopoietic Stem Cell Transplant for the Treatment of Acute Myeloid Leukemia

The Science Behind the Vor Bio platform

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

Pipeline

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