News

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…

If the U.S. Food and Drug Administration (FDA) approves the experimental sickle cell disease (SCD) gene therapy lovotibeglogene autotemcel (lovo-cel), its developer Bluebird Bio plans to sell the voucher it will receive that can be redeemed to speed up the review of a future application of a different…

Researchers at the University of Tennessee Health Science Center (UTHSC) have received a grant from the U.S. Department of Agriculture to support a specialized education program for improving the care of people with sickle cell disease (SCD) in medically underserved areas of the U.S. Led by principal investigator Sara…

A $12.2-million grant awarded to the New York-based Mount Sinai Health System will enable a nationwide observational study to help better personalize sickle cell disease (SCD) treatments. “Sickle cell traditionally has been a neglected disease, but it benefited from a flurry of innovation over the last decade and…

The number of red blood cells that produce the abnormal hemoglobin S (HbS), which characterizes sickle cell disease (SCD), is reduced by the experimental gene therapy BCH-BB694, as is their tendency to take a sickle-like shape. That’s according to single-cell data from seven patients in an ongoing Phase…