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Good times indeed. But they’re long gone now. A perfect storm of a US biotech bear market, concerns about the efficacy and safety of treatments, and rather overhyped expectations have left gene therapy looking less like a silver bullet and more like a slightly leaky water pistol. And a very expensive one at that. The recent exit of Amicus from the gene therapy world is just one example of reality biting in this space.
But the dream is still alive. New delivery methods, allowing better targeting and lower dosing, for example, are being investigated to address some of the big issues. Not to mention a much broader view of the types of diseases that might be addressable through genetic medicines, beyond the rare conditions that have attracted much of the attention. It’s also not fair to say that there’s been no progress in the area, with some recent in vivo gene editing success in amyloidosis.
In our recent digging into the topic, we’ve been speaking to executives at a number of genetic medicine companies, and it’s clear that there is no shortage of belief in the future of the technology. There is, however, significant divergence in approach which is interesting. In fact, even the language starts to vary pretty quickly, from gene therapy to gene editing to perhaps the most ambitious, gene coding. Getting to grips with the differences between these three gets somewhat sticky, but we think it’s useful to try to pin down the definitions.
Those who truly believe in the vision of gene therapy are of the opinion that one day it may become as common as other mainstream therapies. If that is true, there is a long and winding path between us and that brave new world. And the bricks to lay that road need funding by the very investors who have already started to get cold feet on the whole idea.
The next few years need to show some solid progress to secure that investment. If these new approaches succeed, the potential impact on a huge number of diseases (not to mention the coffers of those who invest) will be almost immeasurable. If such successes do not materialise, even the most cutting-edge technology might not be able to revive the sector.
The full report features interviews with Generation Bio, Saliogen, Code Bio, Verve, and Excision Biotherapeutics and you can read it here.