Steve Bryson, PhD,  science writer—

A naturally-occurring protein called hemopexin, also an investigational therapy for acute vaso-occlusive crises (VOCs) in sickle cell disease (SCD), safely reduced the signs of such crises in cell and animal models, a study has demonstrated. These findings support an ongoing Phase 1 trial (NCT04285827), sponsored by the…

The United Arab Emirates (UAE) Ministry of Health has approved Endari (L-glutamine) for sickle cell disease (SCD), according to Emmaus Life Sciences, the therapy’s developer. During the five-month review process, which was originally expected to take up to one year, the oral therapy was available by early…

Dosing will soon start in a Phase 1/2 trial of GPH101, an experimental gene-editing therapy designed to correct the genetic mutation that causes sickle cell disease (SCD). The first participant had been enrolled in the study late last year, but due to the recent surge of the SARS-CoV-2…

Red blood cell biomarkers may predict sickle cell disease (SCD) severity and the risk of vaso-occlusive crisis, according to a study that evaluated the FA-WB-VCAM blood test. “Our latest research indicates that one of our clinically available red blood cell health tests called Flow Adhesion of Whole Blood to…

Oxbryta (voxelotor), a daily oral therapy for hemolytic anemia associated with sickle cell disease (SCD), is now available to eligible patients in the U.K. under an early access program. It received a positive scientific opinion by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) under the Early…

Fewer adults and children with sickle cell disease (SCD) have sought medical care for disease-related events during the COVID-19 pandemic, resulting in lower hospitalization rates, but longer hospital stays, according to data collected from a specialty center in Southern California that cares for a large number of SCD patients.

Researchers at the University of York in the U.K. have been awarded a $3.1 million (£2.3 million) grant to accelerate research supporting stem cell gene therapy to treat sickle cell disease (SCD). The grant was awarded by the nonprofit Bill & Melinda Gates Foundation to track genetic changes in…

Higher doses of FTX-6058, an investigational oral treatment for sickle cell disease (SCD), effectively raised hemoglobin messenger RNA (mRNA) levels above those found at lower doses in healthy volunteers participating in a Phase 1 trial, new data suggests. Increases in hemoglobin mRNA — the molecule cells use as…

IMR-687 (tovinontrine), an experimental oral therapy for sickle cell disease (SCD), reduced the rate of painful vaso-occlusive crisis (VOC), lowered hospitalization due to VOCs, and extended the time to a first VOC in SCD patients, according to new data from an open-label extension of a Phase 2 clinical trial.

Enrollment is underway for a Phase 1/2 trial evaluating the safety and efficacy of EDIT-301, an experimental gene editing cell therapy for people with sickle cell disease (SCD). The trial, called RUBY (NCT04853576), had been cleared for launch by the U.S. Food and Drug Administration earlier this…