Vertex Pharmaceuticals (Nasdaq: VRTX) announced today a reimbursement agreement with NHS England for eligible sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY® ...

January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...

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 ...

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 ...

It is thought that 1,700 people could be eligible after the National Institute for Health and Care Excellence (Nice) approved Casgevy for certain patients with the genetic condition. NHS officials ...

It is estimated that 1,700 people could be eligible after the National Institute for Health and Care Excellence (Nice) approved Casgevy for certain patients with the genetic condition. NHS officials ...

Only some patients with the disease will be eligible to receive Casgevy. The treatment is designed to work by editing the faulty gene in a patient’s bone marrow stem cells. The cells are taken out of ...

Casgevy, also known as exa-cel, was the first treatment to be licensed using gene-editing tool Crispr (Alamy/PA) A £1.65 million treatment has been approved for use for some NHS patients ...