Margarida Maia, PhD, science writer —

The U.S. Food and Drug Administration (FDA) is reviewing an application that seeks the approval of exagamglogene autotemcel, a CRISPR-based gene-editing therapy known as exa-cel, to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The rolling biologics license application was completed by Vertex Pharmaceuticals and CRISPR…

A protein called NLRP12 appears to act as a switch that turns on inflammation and tissue damage when it senses free heme, which is released from broken red blood cells during an infection and in other diseases like sickle cell disease (SCD). This protein could therefore be a potential…

People in Ghana with sickle cell disease (SCD) use prevention, monitoring, diagnosis, and treatment to self-manage their condition, but how this is done differs between adults and children, a study finds. People with SCD spoke more about doing things to stay healthy and treat problems that come up, rather…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to EDIT-301, an experimental cell-based gene-editing therapy given as a one-time infusion for the treatment of sickle cell disease (SCD). The designation is given to spur the development of therapies for diseases affecting fewer than 200,000 people…

Bluebird Bio is seeking approval from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate lovotibeglogene autotemcel — lovo-cel for short — for adults and children, ages 12 and older, with sickle cell disease (SCD). The company has filed a biologics license application (BLA) to…

AddMedica and Abacus Medicine Pharma Services have partnered to market and distribute the sickle cell disease (SCD) therapy Siklos (hydroxyurea) to patients ages 2 and older across Benelux — the economic region of Belgium, the Netherlands, and Luxembourg. In entering into the partnership, the companies agreed that Siklos…

People with sickle cell disease (SCD) may see their physical, mental, and social health get better after a stem cell transplant, and this may help them pursue their personal life goals, a small study found. Still, challenges remained as they “confronted a new and unfamiliar reality,” the researchers…

Bluebird bio will resume its study of lovotibeglogene autotemcel (lovo-cel) in children and adolescents with sickle cell disease (SCD) following the U.S. Food and Drug Administration’s (FDA’s) lifting of its yearlong partial clinical hold for patients under age 18. The temporary holdup stemmed from an investigation into…

A first patient has been enrolled in BEACON, a Phase 1/2 clinical trial into the safety and efficacy of BEAM-101, Beam Therapeutics’ transplant-based gene-editing cell therapy for sickle cell disease (SCD). Recruitment of adults with severe SCD, ages 18–35, continues at Boston Children’s Hospital in Massachusetts and…

Vertex Pharmaceuticals and CRISPR Therapeutics are planning a November launch for a biologics license application (BLA) for their gene-editing therapy exagamglogene autotemcel — known as exa-cel — seeking its approval for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The U.S. Food and Drug Administration (FDA)…