Scientific achievement, access questions define the world’s first gene editing approval

Less than 12 years after the first publications indicating that CRISPR-Cas9 can precisely edit the human genome, the technology has gained its first drug approval. With the green light for Vertex to begin marketing Casgevy to treat sickle cell disease and transfusion-dependent thalassemia, the U.K. is securing its position as a cell and gene therapy leader. 

It’s a transformational scientific and drug development breakthrough that opens the door to more therapies involving the curative modality. However, the next challenge is bringing the new drug to eligible patients in a timely manner. ...