Lindsey Shapiro, PhD, science writer —

The global burden of sickle cell disease (SCD) may be greater than previously thought, with mortality rates up to 11 times higher than earlier estimates, a recent analysis reported. Particularly vulnerable are people living in sub-Saharan Africa and children younger than age 5. Failures to properly diagnose sickle cell…

A new gene-editing approach called prime editing was able to selectively correct the genetic deficit underlying sickle cell disease (SCD) in stem cells collected from patients, a recent study reported. “We were able to directly fix the sickle cell mutation in human hematopoietic stem cells (HSCs) … which we…

Despite widespread use of therapies that can influence fertility, few young people with sickle cell disease (SCD) have documented discussions with their doctors about their treatments’ potential effects on reproductive health, a new U.S. study found. While most patients were counseled about the potential to pass SCD on to…

Vertex Pharmaceuticals and CRISPR Therapeutics have completed an application to the U.S. Food and Drug Administration (FDA) seeking  approval of exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The rolling biologics license application, which was initiated late…

Hemex Health has been awarded $3 million to bring Gazelle, its point-of-care, rapid diagnostic testing device for sickle cell disease (SCD) and beta thalassemia, to market in the U.S. The company will use the three-year award from the National Heart, Lung, and Blood Institute (NHLBI) to…

An MRI technique to quantify the amount of fat in the bone marrow of people with sickle cell disease (SCD) may be a promising way to track disease severity, a recent study indicates. Patients with more signs of red blood cell destruction (hemolysis) in standard blood tests had less…

Vertex Pharmaceuticals will use ImmunoGen technology to develop less aggressive conditioning approaches for exagamglogene autotemcel (exa-cel), its experimental gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. Vertex will pay ImmunoGen $15 million up front to use its antibody-drug conjugate (ADC) technology to develop…

The risk of death from end-stage kidney disease (ESKD) has generally declined in the U.S. over the last 20 years — but not as much for people with sickle cell disease (SCD). Patients with the genetic disorder still have a higher risk of mortality than those without SCD,…

Activation of a protein critical for the body’s response to low oxygen leads to increased levels of fetal hemoglobin in adults — a finding that could offer new therapeutic avenues for people with sickle cell disease (SCD) or beta-thalassemia, a study says. In cell cultures of red blood cells…

The prevalence of a genetic disorder affecting red blood cells, called glucose-6-phosphate dehydrogenase (G6PD) deficiency, was equally high among people with sickle cell disease (SCD) and healthy blood donors in the Democratic Republic of Congo (DRC), a study has found. In addition, among SCD patients, the presence of G6PD…