Lindsey Shapiro, PhD, science writer —

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…

A research team is conducting two projects to learn more about neurological problems, including cognitive impairment and stroke, in people with sickle cell disease (SCD). Led by Hyacinth I. Hyacinth, PhD, at the University of Cincinnati, the team will focus on how various anti-inflammatory medications might act to prevent…

Interfering with two newly identified proteins — NFIA and NFIX — involved both directly and indirectly in silencing fetal hemoglobin in red blood cells, could be a new therapeutic approach for sickle cell disease (SCD), according to researchers. Their hypothesis is that inhibiting the production or activity of these…

ARU-1801, Aruvant Sciences’ experimental gene therapy for sickle cell disease (SCD), led to the production of fetal hemoglobin in red blood cells and significantly lowered the number of vaso-occlusive crises (VOCs) in a group of five people with severe SCD, according to data from the ongoing Phase…