Lindsey Shapiro, PhD, science writer —

A scientist whose discoveries paved the way for the development of an investigational gene therapy for sickle cell disease (SCD) and other blood disorders has been awarded the third Elaine Redding Brinster Prize in Science or Medicine. Stuart Orkin, MD, received the award — given annually to a researcher…

Researchers have developed a new way of delivering gene-editing therapies for people with blood disorders such as sickle cell disease (SCD), which could improve treatment safety and lower costs if eventually adopted in the clinic. Instead of a standard method that requires a round of chemotherapy before externally-edited cells…

Researchers at Rice University in Texas have been awarded $2.6 million to further investigate questions surrounding the safety and efficacy of gene editing as a potentially curative treatment for sickle cell disease (SCD). The four-year grant from the National Institutes of Health (NIH) was awarded to Gang Bao, PhD,…

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…