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
Funding Cycle: 2019-2020