Gene Editing Therapy Casgevy Now Funded In 8 Countries But French Access Withdrawn

The world’s first CRISPR gene editing therapy, Casgevy, has been made available to patients with sickle cell disease in England, adding to access arrangements in the US, Austria, Bahrain, Germany, Luxembourg, Italy and Saudi Arabia.

Casgevy is the first approved product to use CRISPR/Cas9 technology, which was identified as a gene editing tool in 2012. (Shutterstock)
Key Takeaways
  • Vertex’s CRISPR/Cas9 gene editing therapy Casgevy has been made available in England for patients with sickle cell disease, adding to access agreements in the US, Austria, Bahrain, Germany, Luxembourg, Italy and Saudi Arabia.
  • In France, Vertex previously had an early access arrangement for Casgevy in two indications, but it has since withdrawn from these arrangements.
  • The product is the world’s first CRISPR/Cas9 gene editing therapy to gain regulatory approval, and Vertex says it has seen strong interest in the therapy from eligible patients.

England’s health technology assessment (HTA) agency, NICE, has formally agreed to reimburse Vertex/CRISPR Therapeutics AG’s CRISPR/Cas9-based gene editing therapy Casgevy (exagamglogene autotemcel), also known as exa-cel, to treat...

On 31 January, NICE announced that Casgevy had been approved for use on the National Health Service in England for patients with severe SCD aged 12 years and over who...

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