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 ...

Its efficacy is proven, but selling the treatment has been an uphill battle. Casgevy is essentially a vial of a patient's stem cells that have been edited outside their body to permanently produce ...

“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 ...