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Gene therapy nears approval in Europe
"Now, for the first time, a treatment exists for these patients that not only reduces this risk of getting severely sick, but also has a multi-year beneficial effect after just a single injection."
- UniQure chief executive officer Jorn Aldag
Europe is on the cusp of approving a gene therapy for the first time, in what would be a landmark moment for the field.
Gene therapies alter a patient's DNA to treat inherited diseases passed from parent to child.
The European Medicines Agency has recommended a therapy for a rare genetic disease which leaves people unable to properly digest fats.
The European Commission will now make the final decision.
The idea of gene therapy is simple: if there is a problem with part of a patient's genetic code then replace that part of the code.