Lindsey Shapiro, PhD,  science writer—

Certain blood biomarkers of fat dysregulation, inflammation, and red blood cell destruction, known as hemolysis, may be used to predict the short-term risk of acute chest syndrome (ACS) in people with sickle cell disease (SCD). That’s according to a new study from France, in which scientists identified these…

Riociguat, a medication used to treat forms of pulmonary hypertension, was found to safely lower blood pressure in people with sickle cell disease (SCD) who had risk factors for poor outcomes, according to findings from an early clinical trial. “Our results are encouraging, and open the door to…

Authorities in the U.K. have preliminarily recommended that Casgevy (exagamglogene autotemcel) not be reimbursed for eligible patients with sickle cell disease (SCD) under its national public health program based on uncertainties about the gene therapy’s cost-effectiveness. The drafted decision from the National Institute for Health and Care…

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…

On Dec. 8, the U.S. Food and Drug Administration (FDA) approved two cell-based gene therapies for sickle cell disease (SCD), a groundbreaking decision that now offers patients more than one potentially curative option that targets the underlying cause of the disease. Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel) — formerly…

A European Medicines Agency committee has issued a positive opinion on the gene-editing therapy Casgevy (exagamglogene autotemcel) in treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The Committee for Medicinal Products for Human Use (CHMP) recommendation will be considered by the European Commission in making a final…

New regulatory decisions in the U.S. and the U.K. have made Casgevy (exagamglogene autotemcel) the first gene-editing therapy to be approved for adults and adolescents with sickle cell disease (SCD) who experience painful vaso-occlusive crises (VOCs). With these recent approvals, the therapy from Vertex Pharmaceuticals and…

Note: This story was updated Dec. 12, 2023, to clarify that Lyfgenia treats vaso-occlusive events, including painful vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) has approved the gene-editing therapy exagamglogene autotemcel (exa-cel) — now known as Casgevy — for treating sickle cell disease (SCD). The therapy…

Note: This story was updated Dec. 12, 2023, to clarify that Lyfgenia treats vaso-occlusive events (VOEs), including painful vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) has approved lovotibeglogene autotemcel, a gene therapy from Bluebird Bio known as lovo-cel, for treating sickle cell disease (SCD) patients ages…

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…