News

Masitinib clinical program in SCD awarded €9.2M in France

A clinical project designed to test whether masitinib, AB Science’s experimental oral therapy for sickle cell disease (SCD), can resolve complications associated with the inherited disorder has been awarded €9.2 million (about $10 million) in public funding in France. The funding comes as part of a larger investment of €160…

1st gene-editing therapy for SCD, called Casgevy, approved in UK

The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has conditionally approved the gene-editing therapy exa-cel (exagamglogene autotemcel), under the brand name Casgevy, for people with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The authorization covers adults and children, ages 12 and older, with SCD and experiencing…

Mount Sinai gets $12M NIH grant for study to personalize treatments

A $12.2-million grant awarded to the New York-based Mount Sinai Health System will enable a nationwide observational study to help better personalize sickle cell disease (SCD) treatments. “Sickle cell traditionally has been a neglected disease, but it benefited from a flurry of innovation over the last decade and…