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Some Cold Water

There's been growing excitement about gene therapies after a lull in the field, but MIT's Technology Review reports that many treatments are years away from patient use.

It sifted through to find that there are only six late-stage gene therapy trials currently open or recruiting patients and a further five late-stage trials that have been completed. The rest, it says, are in phase I trials. In addition, Tech Review notes that none of those completed trials have posted their results to the site yet, and the US Food and Drug Administration has not approved any gene therapy, through regulators in Europe and China have.

Weill Cornell Medical College's Ronald Crystal tells Tech Review that these low numbers of late-stage trials is likely in part because academic researchers have conducted a lot of gene therapy research and they often don't have the resources to conduct later trials. At the same time, Spark Therapeutics' Katherine High notes that the technology is complex.

Still, Tech Review says gene therapies for monogenic disorders appear to be having success and that Spark's treatment for a condition that causes blindness is expected to be the first on approved for use in the US.