B-cells, B-lymphocytes and plasma cells
Many different cell types are important when dealing with genetic medicines. We are focusing on B-cells, as they have unique properties that make them specially suited for the development of new genetic therapies.
B-cells have the following unique properties:
- B-cells can be easily isolated from PBMCs that are abundant when collecting blood samples from individuals
- B-cells are well suited for the production and secretion of proteins in the human body
- In the human body B-cells are long-lived and they can migrate to many different tissues and locations
We have developed an efficient ex vivo genome editing procedure that can be used in combination with our RITDM™ gene editing technology. B-cells can be developed to make proteins that can become the basis for new genetic medicines.
B-cells are some of the bodies’ most efficient secretors of proteins. This can be useful in situations where a genetic disorder is causing the absence of specific proteins (for example factor VIII or IX in the case of hemophilia or the enzymes IUDA or IDS in the case of lysosomal storage disorders). In such cases the gene coding for the missing protein could be brought to expression in B-cells taken from the individual involved and the altered cells can be given back intravenously.
Another area of potential is to genetically modify the specificity of the antibodies that the B-cells secrete. B-cells are the bodies producer of antibodies that normally protect against many diseases, infections and they even can play role in cancer surveillance as inherent prevention against tumor growth. We are exploring the use of our RITDM™ gene editing technology to modify the binding specificity of antibodies. This could be useful in many situations where there is knowledge on the binding specificity in preventing and curing specific diseases.