Vertex Resolves To Reverse English Funding Rejection For CRISPR Sickle Cell Gene Therapy

The health technology assessment institute, NICE, is not yet ready to recommend Casgevy for sickle cell disease and says it wants more data. Meanwhile, an access agreement relating to the treatment’s use for transfusion-dependent β-thalassemia is making progress in England, as are reimbursement talks for SCD in other European countries.

3d graphic of sickled blood cell
Casgevy is a one-off gene therapy for sickle cell disease • Source: Shutterstock

Vertex is hoping to reverse NICE’s interim decision to recommend against funding its CRISPR gene editing therapy, Casgevy, for severe sickle cell disease (SCD) in England because of uncertainties over the clinical and economic evidence submitted.

The company, which developed the one-off treatment with CRISPR Therapeutics, said it was disappointed by the health technology assessment institute’s...

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