We’re aiming to treat sickle cell disease and beta thalassemia with an investigational gene-editing approach that is supported by well-understood genetics.
The inherited hemoglobinopathies sickle cell disease (SCD) and β-thalassemia result from mutations in a gene that encodes a key component of hemoglobin, the oxygen carrying molecule in blood. These genetic disorders of hemoglobin (hemoglobinopathies) result in reduced life expectancy and currently require lifetime treatment, such as regular blood transfusions and frequent hospitalizations.
We advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018, and this treatment is now approved in some countries for certain eligible people living with SCD or TDT.
Our approach to treat SCD and β-thalassemia is designed to switch back on expression of a different form of hemoglobin called fetal hemoglobin (HbF), which is naturally present in all people before birth. Increased levels of HbF are intended to substitute for the diseased adult hemoglobin in patients with SCD and β-thalassemia, with the goal of reducing or eliminating symptoms.
¹Sedrak, A., & Kondamudi, N. (n.d.). Sickle cell disease - statpearls - NCBI bookshelf. National Library of Medicine. https://www.ncbi.nlm.nih.gov/books/NBK482384/
²Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010;5:11. doi:10.1186/1750-1172-5-11
3Sayani F, Kwiatkowski JL. Increasing prevalence of thalassemia in America: Implications for primary care. Ann Med. 2015;47(7):592-604. doi:10.3109/07853890.2015.1091942
To learn more about the full range of investigational therapies we are developing, visit our pipeline page.
For patients or family members who want to learn more about the clinical trials, please visit the link below for more information to discuss with a doctor.
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