News

Bluebird Bio has provided the U.S. Food and Drug Administration (FDA) with information regarding the commercial manufacturing of its experimental gene therapy for sickle cell disease (SCD) in preparation for requesting its approval. The company had expected to submit a biologics license application seeking the approval of…

Vertex Pharmaceuticals and CRISPR Therapeutics have completed an application to the U.S. Food and Drug Administration (FDA) seeking  approval of exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The rolling biologics license application, which was initiated late…

Health Canada has approved an extended-release formulation of Ferriprox (deferiprone) to treat iron overload in people with sickle cell disease (SCD) and other blood disorders. The key benefit of the new formulation, according to the therapy’s maker, Chiesi Global Rare Diseases, is that it reduces the medication’s dosing…

Salveo Therapeutics and the newsmagazine African Sickle Cell News and World Report — known as Sickle Cell News — are collaborating to promote a new free application (app) that seeks to empower sickle cell disease (SCD) patients to take more control of their health and wellbeing. The overarching aim…

Hemex Health has been awarded $3 million to bring Gazelle, its point-of-care, rapid diagnostic testing device for sickle cell disease (SCD) and beta thalassemia, to market in the U.S. The company will use the three-year award from the National Heart, Lung, and Blood Institute (NHLBI) to…

Higher levels of hemoglobin — the protein in red blood cells responsible for oxygen transport — were associated with a lower risk of end-organ damage with sickle cell disease (SCD), a retrospective study has found. The risk was higher for patients with hemoglobin levels below 7 g/dL and increased…

The Sickle Cell Disease Association of America (SCDAA) is collaborating with several pharmaceutical companies in a new initiative aimed at increasing awareness of the blood disorder and promoting clinical trial participation. Along with the SCDAA, the Sickle Cell Disease Collaboration of Advocates for Research, Education, and Science — officially…

An MRI technique to quantify the amount of fat in the bone marrow of people with sickle cell disease (SCD) may be a promising way to track disease severity, a recent study indicates. Patients with more signs of red blood cell destruction (hemolysis) in standard blood tests had less…

Suburban Propane Partners recently teamed up with the American Red Cross, its longtime national collaborator, to host a blood drive in Boston to boost sickle cell disease (SCD) awareness and encourage blood donations. The event was attended by Willie Gross, Boston’s former, and first, African American police commissioner. Some…

AddMedica and Abacus Medicine Pharma Services have partnered to market and distribute the sickle cell disease (SCD) therapy Siklos (hydroxyurea) to patients ages 2 and older across Benelux — the economic region of Belgium, the Netherlands, and Luxembourg. In entering into the partnership, the companies agreed that Siklos…