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

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 ...

Last month, Vertex and CRISPR Therapeutics announced that the FDA expanded the Casgevy label to treat transfusion-dependent beta thalassemia (“TDT”) in patients aged 12 years and older.

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 ...

CRISPR Therapeutics and Vertex Pharmaceuticals are also conducting separate late-stage studies to expand Casgevy’s label in pediatric patients with SCD and TDT.

Vertex Pharmaceuticals said the U.S. Food and Drug Administration has approved its Casgevy drug for the treatment of transfusion-dependent beta thalassemia in patients 12 years and older.

S hares of CRISPR Therapeutics CRSP have lost more than 30% in the past year. Though the stock soared at the onset of 2024, thanks to the FDA approval of its one-shot gene therapy Casgevy for two ...