Marta Figueiredo, PhD, science writer —

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.

A Phase 1 trial evaluating the safety and pharmacological properties of TAK-755 — Takeda’s experimental therapy for painful vaso-occlusive crises (VOCs) — in adults with sickle cell disease (SCD) is actively recruiting patients for its intermediate dose group. SCD patients, ages 18–65 years, who had no VOCs in the…

GBT601 and inclacumab, experimental therapies from Global Blood Therapeutics (GBT) for sickle cell disease (SCD) and associated vaso-occlusive crises (VOCs), were generally safe and showed signs of biological activity in healthy volunteers, according to data from Phase 1 clinical trials. These findings will be presented by GBT through two…

Long-term treatment with Oxbryta (voxelotor) safely and effectively reduced anemia and red blood cell destruction (hemolysis), while keeping a low rate of painful vaso-occlusive crises (VOCs), in adolescents and adults with sickle cell disease (SCD). That’s according to nearly three years of data from the Phase 3 HOPE…

Regulators in Brazil have approved Ferriprox (deferiprone), Chiesi Global Rare Diseases’ iron-binding oral treatment, for blood transfusion-induced iron overload in people with sickle cell disease (SCD) or other anemias. Ferriprox’s use was also approved in combination with other iron-binding, or iron-chelating, treatments for people with thalassemia major, a severe…

The Sickle Cell Disease Foundation (SCDF) will use a $1 million grant to educate sickle cell disease (SCD) patients living in the western U.S. about the COVID-19 vaccine and assist them in accessing vaccination programs. The goal is to increase the number of SCD patients in western U.S.

Children with sickle cell disease (SCD) as young as age 4 could be prescribed Oxbryta (voxelotor) if the U.S. Food and Drug Administration approves a supplemental new drug application (sNDA) submitted by the therapy’s developers. Global Blood Therapeutics (GBT) is asking the FDA to expand the use of…

The University of California, San Francisco (UCSF) has been awarded $8.39 million to fund a Phase 1/2 clinical trial of CRISPR_SCD001, the first non-viral and CRISPR-Cas9-based gene editing therapy for sickle cell disease (SCD). Researchers will use the gene-editing technology to attempt to correct a genetic mutation in the blood…

A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.

Global Blood Therapeutics (GBT) is currently focused on expanding the use of Oxbryta (voxelotor) to children, ages 4–11, with sickle cell disease (SCD). But research into its use with younger children, as well as studies into other potential SCD therapies, are also underway. “Sickle cell disease patients of all ages worldwide…