The UK’s Medicines and Healthcare Regulatory Agency (MHRA) approved Casgevy for sickle cell disease in 2023, followed by ...

The gene-editing treatment will be available to certain patients with sickle cell disease in England as Vertex commits to collecting additional data.

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 ...

The launch of Casgevy represents a pivotal moment for CRISPR Therapeutics. While initial uptake has been described as slow due to the lengthy treatment process, analysts note that the increase in ...

Advances in CRISPR technologies promise to accelerate the burgeoning pipeline of gene-editing therapies and broaden access to these disease-altering therapies.

However, Casgevy is an ex vivo treatment, meaning patients’ cells are edited outside the body in the treatment process. Download the free BBJ app for important news alerts on your phone.

The gene therapy Casgevy, which will now be offered to some patients in England, could help people live disease-free, experts ...