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 ...

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 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 ...

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 ...

CASGEVY is a non-viral ... and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin ...