A historic first in biotech
This week, the industry experienced a watershed moment when the U.K.’s MHRA granted the first regulatory authorization of a CRISPR-based gene-editing therapy in the world.
Exa-cel (now branded Casgevy), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, was greenlit for the treatment of sickle cell disease and transfusion-dependent beta thalassemia. (The ‘conditional marketing authorization’ tag means that the MHRA will monitor the safety of the treatment through real-word data and post-authorization safety studies conducted by the drugmakers.)
In what the Alliance for Regenerative Medicine called a "seminal moment in the history of biotech" the landmark decision could transform the treatment of sickle-cell disease and β-thalassaemia.
Both inherited blood disorders are caused by errors in the genes for hemoglobin. According to the MHRA, Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning hemoglobin. This is accomplished by taking stem cells from the patient’s bone marrow, editing them using CRISPR and then infusing them back into the patient.
It’s a one-time treatment and the results have the potential to be life-long — although the approval was based on the two trials’ primary efficacy interim analysis, which was 12-months post treatment.
Casgevy uses the gene-editing tool which earned its two developers, Emmanuelle Charpentier and Jennifer A. Doudna, the Nobel Prize in Chemistry 2020. According to the Royal Swedish Academy of Sciences, the CRISPR/Cas9 genetic scissors have had a “revolutionary impact on the life sciences" and "may make the dream of curing inherited diseases come true.”
The FDA is slated to deliver its decisions of exa-cel in SCD on December 8 and TDT on March 30, but the UK nod is a good sign that the FDA will give the drug the thumbs up.
The UK approval is also incredible news for those suffering from SCD and TDT and may pave the way for further applications of CRISPR therapies in the pursuit of cures for genetic diseases — all of which is news worth celebrating. —Karen Langhauser