Margarida Maia, PhD,  science writer—

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…

A clinical project designed to test whether masitinib, AB Science’s experimental oral therapy for sickle cell disease (SCD), can resolve complications associated with the inherited disorder has been awarded €9.2 million (about $10 million) in public funding in France. The funding comes as part of a larger investment of €160…

Children and young adults with kidney failure due to sickle cell disease (SCD) wait nearly two times longer for a kidney transplant and have higher mortality than those whose kidney failure is not caused by SCD, a study found. “Children in the U.S. with SCD now have higher survival…

More than 1 in 3 children and adolescents with sickle cell disease (SCD) develop retinopathy, or damage to the blood vessels in the retina, the light-sensitive layer of tissue at the back of the eye, according to a new U.S. study. In addition, nearly one-tenth of eyes with retinopathy…

For nearly half of people with sickle cell disease who took part in the Phase 2 part of a clinical trial called RISE UP, treatment with the oral medication mitapivat brought about a significant increase in levels of hemoglobin — the oxygen-carrying protein in red blood cells — new…

The U.S. Food and Drug Administration (FDA) is reviewing an application that seeks the approval of exagamglogene autotemcel, a CRISPR-based gene-editing therapy known as exa-cel, to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The rolling biologics license application was completed by Vertex Pharmaceuticals and CRISPR…

A protein called NLRP12 appears to act as a switch that turns on inflammation and tissue damage when it senses free heme, which is released from broken red blood cells during an infection and in other diseases like sickle cell disease (SCD). This protein could therefore be a potential…

People in Ghana with sickle cell disease (SCD) use prevention, monitoring, diagnosis, and treatment to self-manage their condition, but how this is done differs between adults and children, a study finds. People with SCD spoke more about doing things to stay healthy and treat problems that come up, rather…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EDIT-301, an experimental cell-based gene-editing therapy given as a one-time infusion for the treatment of sickle cell disease (SCD). The designation is given to spur the development of therapies for diseases affecting fewer than 200,000 people…

Bluebird Bio is seeking approval from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate lovotibeglogene autotemcel — lovo-cel for short — for adults and children, ages 12 and older, with sickle cell disease (SCD). The company has filed a biologics license application (BLA) to…