Exploring and developing new pathways to transform the treatment of sickle cell disease
We are committed to advancing next-generation investigational therapies for sickle cell disease (SCD) to transform the treatment of this devastating lifelong disease – both through internal R&D and via collaborations with academic centers and industry experts in the field.
Syros Pharmaceuticals: Inducing Fetal Hemoglobin to Treat SCD and Beta Thalassemia
We are collaborating with Syros Pharmaceuticals to discover, develop and commercialize new investigational medicines that induce fetal hemoglobin to treat SCD and beta thalassemia using Syros’ leading gene control platform. Fetal hemoglobin is present in the red blood cells of the developing fetus but is replaced by adult hemoglobin after birth in most people.1 The presence of fetal hemoglobin is known to exert protective effects on the red blood cells of patients with SCD and beta thalassemia and mitigate the clinical manifestation of these diseases. In SCD, Syros’ approach to inducing fetal hemoglobin involves switching on the gamma-globin gene, typically turned off at birth, to make healthy red blood cells. Controlling the expression of a single gene represents one of the promising ways to identify next-generation investigational therapies to treat SCD and beta thalassemia.
Beta thalassemia is a genetic blood disorder caused by mutations in the beta-globin gene, which results in reduced or no production of the protein beta-globin, a key component of hemoglobin.2 Because beta thalassemia impacts hemoglobin and thus the delivery of oxygen to tissues and organs throughout the body, beta thalassemia can lead to severe anemia.2 People with this inherited disease may be dependent on blood transfusions for life.2
- Sankaran, V. et al. Cold Spring Harb Perspect Med. 2013 Jan 1;3(1):a011643.
- Genetics Home Reference. Beta thalassemia. May 2019. National Organization for Rare Disorders. Beta thalassemia. https://rarediseases.org/rare-diseases/thalassemia-major. Accessed February 24, 2020.