WASHINGTON - The Food and Drug Administration Wednesday granted supplemental approval for a breakthrough CRISPR gene therapy ...

The fresh FDA approval allows the use of the cell therapy in patients as young as two years old with sickle cell disease (SCD ...

Vertex Pharmaceuticals VRTX announced that the FDA has approved its one-shot gene therapy, Casgevy (exagamglogene autotemcel) ...

Children as young as two with either sickle cell disease or thalassemia are now eligible for the gene therapy.

For the first time, children in the US as young as two with the blood disorders sickle cell disease and beta thalassaemia are ...

Vertex Pharmaceuticals VRTX and partner CRISPR Therapeutics CRSP announced that the European Commission has granted conditional marketing approval to their one-shot gene therapy Casgevy for treating ...

Follow this author to personalize your feed and get instant alerts. WHY FOLLOW? Custom Feed: See the stories that matter most to you. Smart Alerts: Get notified about major news as it happens. Update ...

The Food and Drug Administration (FDA) approved Casgevy for the treatment of sickle cell disease (SCD) last December. In January the agency followed up with approval to treat transfusion-dependent ...

The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending a CRISPR gene editing-based treatment for severe sickle cell disease (SCD). The regulator said ...

The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for ...