The UK made history by regulating the authorization of a CRISPR-based gene-editing therapy, becoming the first country in the world to do so. The government recently announced that a new treatment for sickle-cell disease and transfusion-dependent β-thalassemia was authorized by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients aged 12 and over. This new treatment, Casgevy, is the first medicine ever to be permitted to use the CRISPR gene-editing tool, a technology that edits genes by precisely cutting DNA and then harnessing natural DNA repair processes to modify the gene in the desired manner.
Sickle cell disease and β-thalassemia are genetic conditions caused by errors in the genes for haemoglobin, impacting individuals with African, Caribbean, or Mediterranean, south Asian, southeast Asian, and Middle Eastern ethnic backgrounds. The new treatment, Casgevy, is designed to edit the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin. This process involves removing stem cells from bone marrow, editing them in a laboratory, and then infusing them back into the patient, with the potential for life-long results.
Casgevy was granted an Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the MHRA, which is an initiative to accelerate the timeline for new drugs to get marketing authorization in the UK. The regulation is significant as it sets the stage for continued breakthroughs in gene-editing technologies in other countries. However, concerns have been raised that the MHRA could shift from being a watchdog to an “enabler” of the pharmaceutical industry due to its pledge to automatically approve medicines.
Vertex Pharmaceuticals and CRISPR Therapeutics have lauded the authorization of Casgevy, which is also under review by the European Medicines Agency, the Saudi Food and Drug Authority, and the U.S. Food and Drug Administration (FDA). However, there is a potential for the MHRA’s shift in regulatory standards to compromise its role as a watchdog for public safety. Consultant pathologist Dr. Clare Craig has argued that there is no public outcry for faster drug approvals and that the priority should be maintaining a rigorous, safe approval process, given the potential risks and ethical implications of gene-editing technology.