Prof Bola Owolabi, of NHS England, called it a "monumental step forward", and said the one-off treatment Casgevy, also known as Exa-cel, "holds a very real prospect of a cure". A confidential ...

The FDA has lost no time in approving Vertex Pharma and CRISPR Therapeutics pioneering gene-editing therapy Casgevy for its second use, approving the drug for transfusion-dependent beta ...

The deal is notable because the U.K.’s cost-effectiveness watchdog initially raised concerns about the durability of the treatment, called Casgevy, in a preliminary ruling last year, saying it ...

Vertex and CRISPR Therapeutics have won the backing of NICE for their gene-editing therapy Casgevy as a treatment for the blood disorder beta thalassaemia, a few months after turning it down for ...

The newest gene therapy treatment, Casgevy, treats sick cell disease and transfusion-dependent beta thalassemia. Its addition means that CHLA provides the most cell and gene therapy treatments for ...

“Building on the success of CASGEVY’s launch, we continue to broaden our portfolio across oncology, autoimmune and cardiometabolic indications. 2025 promises to be a milestone-rich ...