Experimental Treatments for Sickle Cell Disease


ACZ885 (canakinumab) acts as an anti-inflammatory. It is an antibody that binds to and inhibits the action of a protein called interleukin-1 beta (IL-1β), which normally works to trigger inflammation. Through this mechanism, ACZ885 is intended to reduce inflammation in sickle cell disease and potentially reduce the pain experienced by patients. A Phase 2 trial has recently been completed.

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ARU-1801 is an experimental gene therapy being developed to treat sickle cell disease and a blood disorder known as beta-thalassemia. ARU-1801 is designed to increase the number of functional red blood cells by inserting a modified version of the gene coding for gamma-globin, a subunit of fetal hemoglobin directly into the patients’ own hematopoietic stem cells. ARU-1801 is under investigation in a Phase 1/2 trial.

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CTX001 is being developed to treat inherited disorders of hemoglobin such as sickle cell disease and beta-thalassemia. CTX001 uses gene-editing technology to make a genetic change to increase the production of fetal hemoglobin in patients’ red blood cells. A Phase 1/2 trial found that it safely and effectively increased the levels of fetal hemoglobin and prevented vaso-occlusive crises in three people with severe sickle cell disease.

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FT-4202 is a potential disease-modifying therapy for sickle cell disease. It is a small molecule designed to bind to pyruvate kinase-R in red blood cells to increase its activity. As a result, 2,3 DPG levels decrease. This raises hemoglobin’s affinity for oxygen in sickled red blood cells, helping them to deliver more oxygen to tissues and organs. Positive results were recently reported in a Phase 1 trial for the investigational therapy.

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Memantine has shown potential in patients and in preclinical studies to treat sickle cell disease. In sickle cell patients, the N-methyl-D-aspartate receptors on red blood cells are overactive, leading to too much calcium intake. Memantine inhibits the receptor, preventing calcium ions from entering the cell. It has been tested in a Phase 2 trial, with positive results.

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Riociguat is an investigational oral medication that stimulates an enzyme called guanylate cyclase, an enzyme that produces cyclic guanosine monophosphate (cGMP) upon activation by nitric oxide, which is an important regulator of vascular function in the lungs and other parts of the body. cGMP functions as a smooth muscle relaxant that causes the blood vessels to relax and widen. It is currently being tested in a Phase 2 trial for sickle cell disease.

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Ticagrelor is an approved blood thinner, or compound that prevents blood platelet clumping. It also is a potential treatment for sickle cell disease. Researchers are investigating whether ticagrelor could be an effective way to reduce blood vessel blockages in patients with the disease. A Phase 3 trial in pediatric patients was terminated upon the recommendation of an independent data monitoring committee.

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