News

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…

The U.S. Food and Drug Administration (FDA) has cleared for use Trinity Biotech’s Premier Resolution System, an automated analyzer that can quickly and accurately detect and quantify hemoglobin variants. Hemoglobin is the protein that red blood cells use to carry oxygen through the bloodstream. Several hemoglobin variants identified…

A new, interactive application touted as the first of its kind can help people with sickle cell disease (SCD) learn about and communicate with their care team about their pain attacks. Called Pinpoint, the app is the product of a partnership between healthcare purchased services provider HPC…

Editas Medicine has expanded its partnership with Azzur Cleanrooms on Demand (COD) for the manufacturing of EDIT-301, its experimental cell-based gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. If approved, the scaling up of EDIT-301 needs to follow current good manufacturing practice (cGMP) standards — set…

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…

The European Commission has decided to revoke its conditional approval of Adakveo (crizanlizumab) for sickle cell disease (SCD) — meaning its developer, Novartis, will have to remove the antibody-based therapy from the market in all 27 countries that are part of the European Union. The decision came after…

A gene editing technology called adenosine base editing (ABE), wherein a single DNA building block is changed to increase fetal hemoglobin, may be more useful for treating sickle cell disease (SCD) than other gene editing techniques, a study suggests. “We showed that not all genetic approaches are equal. Base…