Marta Figueiredo, PhD, science writer —

Multiple daily doses of GBT601, Global Blood Therapeutics (GBT)’s experimental oral therapy for sickle cell disease (SCD), were generally well tolerated and showed promising pharmacological and efficacy signals in six patients in a Phase 1 trial. These results support further evaluation of GBT601 as the potential successor to GBT’s…

Exposure to systemic steroids — frequently used to treat asthma or ease inflammation — is significantly associated with a higher risk of painful vaso-occlusive crises (VOCs) leading to hospitalization in children and adults with sickle cell disease (SCD), a French study shows. “Individuals living with SCD often suffer…

While most adolescent boys and young men with sickle cell disease (SCD) report wanting biological children in the future, few are aware of the impact their disease and its treatments can have on their fertility, a small, pilot U.S. study suggests. Similar knowledge gaps were found for these patients’…

A team of researchers has created the most comprehensive atlas, or roadmap, to date of each step of human blood stem cell development — helping them identify the previously unknown molecular signature of blood stem cells and ending a longstanding debate on these cells’ specific cellular origin. The data, open…

Imara is discontinuing the clinical development of tovinontrine (IMR-687), its experimental oral therapy for sickle cell disease (SCD) and beta-thalassemia, based on interim data from two Phase 2b clinical trials. While the therapy was generally safe and well tolerated in adults with both inherited blood disorders, it resulted in…

A team of international researchers has received a $3-million grant from the National Institutes of Health (NIH) to sequence the genome — the whole genetic code — of children with sickle cell disease (SCD) in Ghana. The award was granted to the project’s principal investigator, Solomon Fiifi Ofori-Acquah, PhD,…

Researchers have ruled out a phenomenon called clonal hematopoiesis as the potential cause of the increased risk of blood cancer in people with sickle cell disease (SCD) by analyzing the genetic data of more than 3,000 SCD patients and 71,000 unaffected people. In clonal hematopoiesis, people acquire mutations that cause…

Oxbryta (voxelotor) has become the first approved therapy in the European Union to target the underlying cause of sickle cell disease (SCD) for patients 12 and older. The medication, given as a once-daily oral tablet, suppresses the sickling and destruction of red blood cells that underlies the disease. Eligible…

Blood vessel narrowing due to external or internal stimuli, and the resulting reduced blood flow, may be the main drivers of the painful vaso-occlusive crises experienced by people with sickle cell disease (SCD). Inducing stimuli for such blood vessel narrowing include mental stress, pain, and extreme temperatures, either low…

The U.S. Food and Drug Administration (FDA) has agreed to expand the use of Oxbryta (voxelotor) — the first approved therapy targeting the underlying cause of sickle cell disease (SCD) — to children as young as 4. The therapy previously was approved for SCD patients ages 12 and older.