Nature4 年
One-time gene therapy for sickle cell disease
Aruvant is developing ARU-1801, a one-time, potentially curative gene therapy for sickle cell disease and β-thalassemia. In an ongoing clinical phase 1/2 study, ARU-1801, administered with only ...
pharmaphorum15 天
Commercialization of novel gene-editing technology in beta-thalassemia
Earlier this year, the US Food and Drug Administration (FDA) approved the first potentially curative gene therapy to treat beta-thalassemia. While multiple companies have been working to bring ...
来自MSN11 个月
SFDA approves first gene therapy for sickle cell anemia and thalassemia
RIYADH — The Saudi Food and Drug Authority (SFDA) has approved Casgevy, the first gene therapy for sickle cell anemia and thalassemia diseases in patients 12 years of age and older. It is a cell ...
来自MSN11 个月
FDA approves Vertex’s gene-edited therapy for beta thalassemia
"FDA approves Vertex’s gene-edited therapy for beta thalassemia" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has ...
BBC1 年
Casgevy: UK approves gene-editing drug for sickle cell
In a world first, medical regulators in the UK have approved a gene therapy that aims to cure two blood disorders. The treatment for sickle cell disease and beta thalassemia is the first to be ...
Business Wire17 天
Long-Term Follow-Up Data Continue to Support Beti-Cel as a Potentially Curative Gene ...
About ZYNTEGLO® (betibeglogene autotemcel) or beti-cel ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene therapy approved for the treatment of beta-thalassemia in adult and pediatric ...