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 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 ...

The new gene therapies for sickle cell disease—including the gene-editing treatment Casgevy, based on research at Boston Children's Hospital—have been game-changing for the patients who have ...

The company has just one product on the market, Casgevy, which treats transfusion-dependent beta-thalassemia and sickle cell disease, two rare blood disorders. Casgevy has been on the market for a ...

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 ...

And in December, it shelved a sickle cell treatment similar to Casgevy, the treatment approved in late 2023 from Vertex and CRISPR Therapeutics. Casgevy’s slow rollout — it appears to have ...

CRISPR Therapeutics stock slipped in November following its third-quarter report. Casgevy, a once-and-done treatment for sickle cell disease and beta-thalassemia, earned FDA approval last December ...

Though the stock soared at the onset of 2024, thanks to the FDA approval of its one-shot gene therapy Casgevy for two blood disorders, much of these gains have pared mainly due to the waning ...