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Vertex Achieves Gene-Editing Milestone With First Ever CRISPR Therapy Approval

 
• By Andrew McConaghie

Great Britain is first to approve the groundbreaking CRISPR-based therapy for sickle cell and beta thalassemia. Vertex is yet to reveal its price, however, and wants to play down early expectations of its commercial potential.

Vertex building logo

Vertex and CRISPR Therapeutics have achieved a historic milestone with the approval of the first ever CRISPR/Cas9 based treatment, their sickle cell gene therapy and transfusion dependent beta-thalassemia treatment Casgevy.

The conditional approval granted by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) opens up a new era of gene-editing based therapies, with the hope of transforming treatment in hundreds of

Key Takeaways

  • The UK regulator’s approval of Casgevy makes it the world’s first ever CRISPR-based gene therapy

  • US approval is expected in early December, but Vertex has looked to lower expectations about its commercial potential

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