In late November, Reuters reported a milestone in medical history: a gene therapy drug could go on sale in Germany next year, after winning the approval of European regulators two years ago. The drug, Glybera, by a Dutch firm called UniQure — currently being scrutinised by Germany’s federal joint committee — would be the first commercial use of gene therapy in the Western world (China has had a gene therapy drug for a specific form of cancer in the market since 2004).
This marks a potential turning point in an area of medicine that has been the subject of highs and lows over more than two decades of clinical trials.
Gene therapy — which can involve a number of things, including replacing a malfunctioning gene or introducing a new gene with the ability to fight a disease — has been in conceptual development for far longer. Its origins could be said to go back as early as the 1920s, well before the discovery of the structure of DNA, when a British scientist, Frederick Griffith, put forward what he described as the “transforming principle”; he successfully converted a non-virulent strain of bacteria into a virulent one, after injecting mice with both.