Issue 9/2012

  • Is gene therapy finally coming into its own?

    In this issue:


    Is gene therapy finally coming into its own?

    After a balancing act that went down to the wire with regulators – and left its predecessor company bankrupt – uniQure biotherapies now looks set to become the first pharma company to gain approval for a gene therapy in Europe. the concept of ‘curing’ rare inherited diseases by altering a patient’s genome in key cells has been around for decades. But disastrous, highly-publicised failures to harness the technology in the past frightened off developers and patients alike. Now that looks set to change.

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