A novel CRISPR/Cas9 Based Mutation Correction Method for Hemoglobinopathies

Unmet Medical Need:  Sickle cell disease is the most common hemoglobinopathy caused by many mutations in HBB (hemoglobin subunit beta). The only curative option is bone marrow transplant from a matching donor, which has its own complications, such as high morbidity caused by graft-versus-host disease and a high risk of infertility.

Envisioned Healthcare Product:  An improved CRISPR/Cas9 gene editing approach that corrects the HBB mutations in stem cells of patients with SCD and will increase the efficiency of the therapy and reduce the off-target effects.

Stage of Development: Proof of Concept 

Collaborative Research Team: Suleyman Ucuncuoglu, PhD and Wendy Chung, MD, PhD

Funding Cycle: 2019-2020