UK approves CRISPR gene editing therapy in global first

The UK has become the first country to approve a therapy based on CRISPR gene editing, with the regulator authorizing a treatment for sickle cell disease and beta thalassemia.

The Medicines and Healthcare products Regulatory Agency has approved the therapy, called Casgevy, which was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The drug could be used to replace bone marrow transplants.

The UK regulator has promised to focus on speeding the most innovative treatments to market after being given permission from next year to cut its workload by following other countries’ recommendations on approvals of other drugs. It had been struggling to keep up because of a lack of resources after the UK left the EU, where it had been a key part of the bloc’s regulatory agency.

CRISPR is a flexible and efficient gene editing tool based on the bacterial immune system, which has sped from the initial discovery to an approved drug in just 11 years. Scientists Jennifer Doudna and Emmanuelle Charpentier showed that CRISPR could be used to disrupt, delete, or correct genetic errors in 2012, leading to a Nobel Prize in 2020.

Read the original article in The Financial Times.