Britain's health regulator said on Wednesday it had revoked a conditional marketing authorisation for Novartis' sickle cell disease drug, Adakveo.
Patients with severe sickle cell disease could soon be freed from vaso-occlusive crises after receiving an infusion of exa-cel, the first gene-editing therapy to use CRISPR technology.
Bluebird bio said on Thursday a large insurer had agreed to cover its newly approved sickle cell disease gene therapy, easing some investor worries about resistance from.
The average age of death for Black US patients with sickle cell disease rose from 39 years in 1999-2009 to 43 years in 2010-2020, reflecting improvements in treatment.