Accelerating the clinical translation of hematopoietic stem cell gene editing to correct hemoglobin disorders

Hemoglobinopathies such as sickle cell disease (SCD) affects millions of people around the world and is caused by a mutation in the globin gene. There is currently no cure available for patients with SCD. One strategy that can be used to cure SCD is by correcting the mutation in the globin gene in blood stem cells. This strategy has shown promise in early human clinical trial data. However, a major limitation of this strategy is that the process is currently inefficient and blood stem cells are naturally resilient to gene editing correction. We at Canadian Blood Services have developed a patented technology that allows us to expand blood stem cells and simultaneously make them more amenable to editing. In this application, we propose to isolate blood stem cells from patients with SCD and expand them using this novel technology. We will then edit the SCD mutation within the expanded blood stem cells and demonstrate that the edited cells behave like normal stem cells that can produce healthy red cells. The findings from this project will enable us to enhance gene-editing therapies currently tested in clinical trials for SCD and many other blood-borne disorders. Importantly, this will break the long-term reliance of many patients for blood transfusion products, thereby improving the efficient and appropriate utilization of blood cell products, while maintaining the safety of the blood system.

MAGANTI, Harinad

Canadian Blood Services

Graduate Fellowship Program

Ontario

$70,000