Margarida Azevedo, MSc,  —

Novo Nordisk has obtained worldwide rights to EpiDestiny’s sickle cell disease treatment EPI01. The disease-modifying therapy, consisting of decitabine and tetrahydrouridine, is aimed at  helping patients increase fetal hemoglobin levels to replace the hemoglobin that’s defective in the disease. The idea is to prevent red blood cells from becoming sickle-shaped,…

The European Medicines Agency recently granted orphan drug status to Sancilio Pharmaceuticals’ Altemia for the treatment of sickle cell disease (SCD) in pediatric patients, the company announced. Altemia is a specific formulation of lipids designed to replenish the elements needed for healthy red blood cells that are damaged…

The European Medicines Agency (EMA) has signaled that it is ready to begin reviewing Emmaus Life Sciences’ Xyndari as a possible treatment for sickle cell disease (SCD). The signal was the agency’s validation of the company’s marketing authorization application (MAA) for Xyndari. Xyndari increases the amount of free glutamine in the…

Gamida Cell recently presented updated data from its ongoing Phase 1/2 clinical trial on an experimental alternative to bone marrow transplants in patients with sickle cell disease (SCD). Elbit Imaging, of which Gamida is an indirect shareholder, announced the information at the 2018 BMT Tandem Meetings in…

Emmaus Life Sciences has reached an agreement with the Centers for Medicare and Medicaid Services that will allow coverage of Endari (L-glutamine oral powder) to reduce acute complications of sickle cell disease (SCD) in adults and children 5 years and older. “This treatment is considered very important by many…

England’s National Health Service (NHS) has selected 138 entrepreneurs to design and deliver new technological solutions for healthcare challenges – one of which was appointed to tackle conditions like sickle cell disease (SCD) using a voice-recognition mobile app. The support is part of the NHS Clinical Entrepreneurs…

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…

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…

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…

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…