Marisa Wexler, MS,  senior science writer—

FTX-6058, an investigational treatment for sickle cell disease (SCD) and beta-thalassemia being developed by Fulcrum Therapeutics, showed efficacy at raising the levels of fetal hemoglobin in cellular and animal models of the diseases, according to new preclinical data announced by the company. Fulcrum also stated…

Medications that activate the protein soluble guanylyl cyclase (sGC) may have promise in the treatment of sickle cell disease (SCD), a new study in cells and animal models indicates. The study, “Beneficial Effects of Soluble Guanylyl Cyclase Stimulation and Activation in Sickle Cell Disease Are…

Using a newly developed assay, researchers identified seven U.S. Food and Drug Administration (FDA)-approved medications that may be effective, with minimal safety issues, in treating sickle cell disease (SCD). Their findings were reported in the Journal of Clinical Medicine, in the study, “Large-Scale Drug Screen Identifies…

The SECURE-SCD registry, which is collecting de-identified data on individuals with sickle cell disease (SCD) who develop COVID-19, will be expanding to include information on children and adults in Africa. The expansion was funded by a $100,000 grant from the Doris Duke Charitable Foundation (DDCF) awarded to…

A European Medicines Agency (EMA) committee has adopted a positive opinion on Novartis‘s Adakveo (crizanlizumab) for the prevention of vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended the conditional approval of…

Blood stem cell transplant from unrelated donors is a feasible strategy in people with sickle cell disease (SCD), with better outcomes when blood markers are matched between donor and recipient, a study suggests. The study, “The role of HLA matching in unrelated donor hematopoietic stem cell…

FT-4202 — an investigational treatment for sickle cell disease (SCD) — has a favorable safety profile and the expected biological effects, providing preliminary support of efficacy, according to data from an ongoing Phase 1 clinical trial. Forma Therapeutics, which is developing FT-4202, plans to start a global Phase…

Global Blood Therapeutics (GBT) plans to submit a marketing authorization application to the European Medicines Agency (EMA) asking that Oxbryta (voxelotor) be approved for treating hemolytic anemia in people with sickle cell disease (SCD) who are 12 or older. The submission is anticipated by…

Global Blood Therapeutics (GBT) will ask the U.S. Food and Drug Administration (FDA) to allow children with sickle cell disease (SCD), ages 4 to 11, to also be treated with Oxbryta (voxelotor). The company announced plans to file an application requesting extended approval after a Type B meeting with FDA…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Forma Therapeutics‘ FT-4202 for the treatment of sickle cell disease (SCD) This designation is given to investigational therapies intended to treat rare conditions (those that affect fewer than 200,000 people in the U.S.). It provides companies…