News

The European Commission has granted conditional approval to the gene-editing therapy Casgevy (exagamglogene autotemcel) for patients ages 12 and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), two blood disorders. Eligible patients include those with SCD who experience recurrent vaso-occlusive crises (VOCs) and…

Heavier menstrual bleeding and pain during menstrual periods appear to be prevalent among adolescents and young women with sickle cell disease (SCD), affecting their quality of life, but few of these patients use hormonal therapies to help regulate their cycles, a single-site U.S. survey study reported. Knowledge of what…

Treatment with hydroxyurea reduced the incidence of severe or invasive infections by 60% among children in Uganda with sickle cell anemia, the most common and usually the most severe form of sickle cell disease, a recent study reports. “Our investigation provides powerful…

Starting in 2025, the U.S. Department of Health and Human Services (HHS) will introduce a pricing model that aims to make new cell and gene therapies more accessible for people with sickle cell disease who are on Medicaid, the health program for lower income residents jointly funded by the…

England’s National Health Service (NHS) is introducing a new genetic test that seeks to make it safer for people with sickle cell disease and other blood disorders to receive transfusions. “Thousands of people living with sickle cell disease and thalassaemia will be eligible for this new world-first blood…

Comer Children’s Hospital at the University of Chicago will be one of the first centers in the U.S. to offer new gene therapies for sickle cell disease that were approved last year by the U.S. Food and Drug Administration (FDA). “I’m excited on behalf…

A biomedical engineer at Kennesaw State University has been given a $426,000 grant to build a low-cost device helping to predict the risk of a stroke in children with sickle cell disease by using light to monitor blood flow to the brain. Paul Lee, PhD, an assistant professor at…

Ferriprox (deferiprone) showed comparable efficacy to Desferal (deferoxamine mesylate) in reducing blood transfusion-induced iron overload in children and adolescents with sickle cell disease (SCD) or other anemias, a recent study reported. Ferriprox is an oral treatment, while Desferal is administered via a subcutaneous (under-the-skin) infusion. As such, Ferriprox is…

The first patient has been dosed in BEACON, a Phase 1/2 clinical trial of a transplant-based gene-editing stem cell therapy called BEAM-101 that Beam Therapeutics is developing for sickle cell disease (SCD). After the transplant, stem cells began to “engraft,” meaning they started to grow and give rise…

Saudi Arabia’s regulatory authorities have approved Casgevy (exagamglogene autotemcel), a one-time gene-editing therapy, to treat sickle cell disease (SCD) in adults and adolescents, ages 12 and older. The approval by the Saudi Food and Drug Authority (SFDA), the country’s equivalent to the U.S. Food and Drug Administration, also…