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

Its first treatment, Casgevy, earned approval from the Food and Drug Administration (FDA) to treat sickle cell disease in December 2023. Last January, the FDA approved Casgevy again to treat ...

Casgevy works by enabling people to produce ... In the new work, Orkin and his colleagues explored the structure and chemistry of the BCL11A protein, looking for ways to hit it.

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 ...

The disease alters the structure of hemoglobin ... but none are as promising as Lyfgenia and Casgevy. These two novel therapies can decrease or potentially eliminate pain crises in patients.

Vertex offers up to $70,000 in fertility services assistance for patients undergoing Casgevy treatment. HHS claims Vertex’s program incentivizes patients to choose its gene therapy over competitors.