News

A Phase 3 clinical trial evaluating whether adding red blood cell exchange to standard-of-care improves outcomes for adults with high-risk sickle cell disease (SCD) is still recruiting at sites across the U.S., as well as in France and Brazil. The study, called Sickle Cell Disease and CardiovAscular Risk Red…

Health regulators in the Kingdom of Bahrain have approved the gene-editing therapy Casgevy (exagamglogene autotemcel) to treat people with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This decision makes Bahrain, a small island nation in the Persian Gulf, the second country to approve Casgevy. The therapy was…

Skin grafts can ease pain associated with leg ulcers and improve wound healing in people with sickle cell disease (SCD), according to a study in France. The procedure consists of the surgical collection of a piece of healthy skin on the body that is then transplanted to the injured…

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…

The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has conditionally approved the gene-editing therapy exa-cel (exagamglogene autotemcel), under the brand name Casgevy, for people with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The authorization covers adults and children, ages 12 and older, with SCD and experiencing…

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…

The Phase 1/2 BEACON trial, which is testing the gene-editing cell therapy BEAM-101 in people with sickle cell disease (SCD), is expected to dose its first patient before the year’s end. According to Beam Therapeutics, which is developing the therapy for fetal hemoglobin production, the trial is…

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…

Being hospitalized due to a vaso-occlusive crisis (VOC) is associated with a significant reduction in quality of life for children with sickle cell disease (SCD), and has a special impact on their physical functioning, a study finds. The negative effects were particularly higher in the first months after…

An advisory committee to the U.S. Food and Drug Administration (FDA) has completed its meeting on exa-cel — formally exagamglogene autotemcel — ahead of the agency’s approval decision on the gene editing therapy, expected next month. The meeting was scheduled as part of the FDA’s review of exa-cel, which is…