The US regulatory agency (FDA, according to its acronym in English) approved this Friday the first drug based on CRISPR/Cas9, the tool that allows carrying out ‘genetic editing’. The drug has received the green light to correct the alteration in the DNA that causes sickle cell disease, a hematological disorder.
This is the second regulatory decision on this compound, after the recent approval by the British regulatory agency (MHRA), which occurred more than two weeks ago and which also contemplated its use in another blood disease, transfusion-dependent beta-thalassemia. The medication in question is Casgevy (exa-cel), de Vertex Pharmaceuticals.
The British agency was ahead of the US agency and the European EMA – whose advisory committee for Medicinal Products for Human Use will also rule in a few weeks on this advanced therapy – when it came to endorsing this pioneering genetic treatment.
Es “a historic approval”in the words of the geneticist Kay Daviesfrom the University of Oxford, as it “opens the door to new applications of CRISPR therapies in the future for the possible cure of many genetic diseases.”
gene therapy exagamglogene autotemcel (exa-cel) uses the CRISPR/Cas9 gene editing tool, the technology that won the 2020 Nobel Prize in Chemistry, which the scientists received Jennifer Doudna y Emmanuelle Charpentier which was developed thanks to the first discoveries made in bacteria by the Spanish Francis Mojica. Not even they imagined that the cut-paste genetically inspired by a defensive system of bacteria would reach the clinic so quickly. This is what Doudna recognized in Nature: “It was clear that having the ability to edit genomes was a powerful tool, but I don’t think any of us could have imagined how quickly this field would advance.”