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…
News
A new gene-editing approach called prime editing was able to selectively correct the genetic deficit underlying sickle cell disease (SCD) in stem cells collected from patients, a recent study reported. “We were able to directly fix the sickle cell mutation in human hematopoietic stem cells (HSCs) … which we…
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…
Children with sickle cell disease (SCD) might be at risk for severe obstructive sleep apnea (OSA) even if they’re a normal weight, according to a study. This contrasts with obesity being a widely known risk factor of OSA in the general population. OSA occurs when the natural relaxation of…
Despite widespread use of therapies that can influence fertility, few young people with sickle cell disease (SCD) have documented discussions with their doctors about their treatments’ potential effects on reproductive health, a new U.S. study found. While most patients were counseled about the potential to pass SCD on to…
Kier “Junior” Spates is hosting a new podcast to empower and educate people living with sickle cell disease. Called “Living Your Life,” the show can be found on YouTube or on the podcast website. The first episode, which premieres April 18, features comedian Nathaniel Stroman, better known as…
A Sickle Cell Carrier Status Awareness program has been launched to help identify people who carry genetic variants associated with sickle cell disease (SCD). Established through a collaboration between the Sickle Cell Foundation of Georgia (SCFG), the Morehouse School of Medicine, and the human genetic testing company 23andMe,…
Bluebird Bio has provided the U.S. Food and Drug Administration (FDA) with information regarding the commercial manufacturing of its experimental gene therapy for sickle cell disease (SCD) in preparation for requesting its approval. The company had expected to submit a biologics license application seeking the approval of…
Vertex Pharmaceuticals and CRISPR Therapeutics have completed an application to the U.S. Food and Drug Administration (FDA) seeking approval of exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The rolling biologics license application, which was initiated late…
Health Canada has approved an extended-release formulation of Ferriprox (deferiprone) to treat iron overload in people with sickle cell disease (SCD) and other blood disorders. The key benefit of the new formulation, according to the therapy’s maker, Chiesi Global Rare Diseases, is that it reduces the medication’s dosing…
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