In a monumental stride toward combating genetic diseases, Vertex Pharmaceuticals has achieved a groundbreaking feat with the UK’s approval of Casgevy (exagamglogene autotemcel), the world’s first clustered regularly interspaced short palindromic repeats (CRISPR)-based drug for sickle cell anaemia (SCA) and β-thalassaemia.

Casgevy allows for precise modifications to be made in the DNA sequence, offering the potential to correct genetic mutations responsible for these inherited blood disorders. The UK’s approval of Casgevy opens new possibilities in the global landscape of genetic medicine that leverages CRISPR technologies.

According to GlobalData, the global cell and gene therapy market is expected to reach $80bn by 2029, with a high compound annual growth rate of 51.6% between 2023 and 2029. With CRISPR-based treatments officially penetrating the therapeutic space, Vertex Pharmaceuticals will likely gain a dominant position in the global cell and gene therapy market.

Currently, the company is seeking FDA approval of Casgevy for SCA. While Casgevy has been disrupting the genetic medicine space, the limiting factor for its widespread usage is its high cost. Estimates suggest that the treatment could cost approximately $2m per patient.

As Vertex Pharmaceuticals spearheads the CRISPR therapeutic space with the approval of Casgevy, continuous research, ethical oversight, and collaborative efforts between scientists, policymakers, and healthcare providers are required to further guarantee the safety and efficacy of this drug.