ORLANDO, Fla. – At the 61st ASH annual meeting late-breaking abstracts session, researchers from Boston Children’s Hospital reported that three adult patients who had received an autologous transplant of gene-edited hematopoietic stem cells lacking BCL11A produced high levels of functional hemoglobin and had reduced disease symptoms for at least eight months after transplantation.
ORLANDO, Fla. – In an effort to get sickle cell disease (SCD) researchers, drug developers, patients and regulators all on the same page, the American Society of Hematology (ASH) and the FDA have released new recommendations aimed at establishing uniform global standards for clinical trial endpoints to evaluate new therapies.
Less than two weeks after giving the go-ahead to Novartis AG for Adakveo (crizanlizumab) to reduce the frequency of vaso-occlusive crises (VOCs) in adult and pediatric patients ages 16 and older with sickle cell disease (SCD), the FDA cleared – well ahead of its Feb. 26, 2020, PDUFA date – Oxbryta (voxelotor), from Global Blood Therapeutics Inc. (GBT), for SCD in adults and pediatric patients ages 12 and up.
DUBLIN – Crispr Therapeutics AG has delivered what appears, so far at least, to be a safe, functional cure for the first patient enrolled in each of its phase I/II trials of lead CRISPR/Cas9 gene editing therapy CTX-001, in beta-thalassemia and in sickle cell disease.
About two months ahead of the priority review action date, Novartis AG scored FDA clearance for Adakveo (crizanlizumab), previously known as SEG-101, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients ages 16 and older with sickle cell disease (SCD).
