News

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…

Because many individuals of African ancestry don’t know they have the sickle cell trait or what it means to be a carrier, 23andMe and the nonprofit organization Sickle Cell 101 are collaborating to expand an awareness program. With the collaboration, up to 1,000 U.S. adults of African…

Treatment with the investigational gene-editing therapy OTQ923 led to the sustained production of fetal hemoglobin and prevented vaso-occlusive crises (VOCs) in three adults with severe sickle cell disease (SCD), according to data from a Phase 1 study. The treatment involved collecting red blood cell precursors, called hematopoietic stem…

Preventive red blood cell transfusions may protect against complications associated with pregnancy in women with sickle cell disease (SCD), a 13-year study has found. Findings indicate that the rates of premature births, as well as SCD complications, such as vaso-occlusive crises (VOCs) and acute chest syndrome (ACS), are…

Point-of-care tests can be used to accurately diagnose sickle cell disease (SCD) in resource-limited settings such as sub-Saharan Africa, according to a recent study. “Based on our experience and this study, we support the use of these [point-of-care] tests as standalone diagnostic tests for the early diagnosis of SCD…

HPC International has released new editions of Hope & Destiny, a book series that aims to help people with sickle cell disease (SCD) navigate life with the disorder. “We’re excited to get the new Hope & Destiny editions into the hands of the patients who need them, furthering our goal to…

Corticosteroids taken during pregnancy are a risk factor for more severe vaso-occlusive crises (VOCs) in women with sickle cell disease (SCD), according to a study in France. Particularly, this risk was higher in women with a more severe SCD type and evidence of more severe disease. Newborns exposed…

Advocates, patients, caregivers, doctors and others impacted by sickle cell disease (SCD) are gathering in September to bring attention to the struggles and needs of those living with the condition, and the importance of research and developing treatments. National Sickle Cell Awareness Month was established 40 years ago…

To help bring new therapies more quickly and cost effectively to those with sickle cell disease (SCD) in sub-Saharan countries — where patients bear a significantly high SCD burden — the U.S.-based biotechnology company Functional Fluidics is planning to expand its operations into Africa. The expansion is expected…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on FTX-6058, an investigational oral treatment for sickle cell disease (SCD) that’s being developed by Fulcrum Therapeutics. With the hold lifted, Fulcrum is planning to resume enrolling patients in a Phase 1b clinical trial…