News

The U.S. Food and Drug Administration (FDA) approved Endari (L-glutamine) last summer, but now the medicine is available by prescription to most patients in the U.S. who have sickle cell disease. Endari, an oral L-glutamine therapy, was developed by Emmaus Life Sciences to reduce acute complications of…

Voxelotor, a potential treatment for sickle cell anemia (SCA), has been given breakthrough therapy designation (BTD), Global Blood Therapeutics (GBT) recently announced. Voxelotor (previously called GBT440) is a potential once-daily oral medicine for SCA patients. It was designed to increase hemoglobin’s binding of oxygen, which keeps red blood…

Emmaus Life Sciences has developed a sickle cell disease therapy that the United States has approved but regulators in much of the world are still reviewing. Until other countries approve L-glutamine, those living there will be unable to obtain the treatment for the blood disease, which is painful, causes organ damage…

Bluebird bio announced encouraging interim results from the ongoing Phase 1 clinical trial testing its investigational gene therapy LentiGlobin for patients with severe sickle cell disease (SCD). The results were presented at the 59th Annual Meeting of the American Society of Hematology (ASH), held Dec. 9-12 in Atlanta,…

Crizanlizumab (SEG101), manufactured by Switzerland’s Novartis, delays the time to first sickle cell pain crisis (SCPC) in adults with sickle cell disease, according to results from a subgroup analysis of the Phase II SUSTAIN study (NCT01895361). Researchers presented the abstract, “Crizanlizumab 5.0 Mg/Kg Increased the Time to First on-Treatment…

The sickle disease of 83 percent of adolescents improved after 16 weeks of taking Global Blood Therapeutics‘ voxelotor, preliminary results of a Phase 2a clinical trial indicate. Another important finding was that voxelotor prevented irregularly shaped oxygen-carrying hemoglobin cells from clumping in more than half of those treated. The clumping prevents…

Prolong Pharmaceuticals has completed enrolling patients in an ongoing Phase 2 clinical trial of  Sanguinate, a treatment for the painful blocked blood vessel episodes that occur in sickle cell disease. The cause of the episodes, known as vaso-occlusive crises, is abnormally shaped red blood cells  bunching up and blocking…

Researchers have found that a new dosing regimen of the drug hydroxyurea results in levels of fetal hemoglobin above 20 percent in sickle cell anemia patients, and reduces hospitalizations by at least two-fold. Hydroxyurea has been shown to be of clinical benefit to children with sickle cell anemia (SCA) and is…

Vertex Pharmaceuticals and CRISPR Therapeutics recently announced a joint collaboration to co-develop and co-commercialize CTX001 as a gene-editing treatment of diseases of hemoglobin, including β-thalassemia and sickle cell disease (SCD). The announcement follows the presentation of preclinical data for CTX001 at the American Society of Hematology…

Intellia Therapeutics has used gene editing to replace an oxygen-carrying component in blood that is defective in sickle cell disease. The work involved editing the BCL11A gene in a mouse model of the disease to produce an effective version of the oxygen-carrying component, hemoglobin. Intellia discussed the project at the…