The challenge of delivering RNA-interference therapeutics to their target cells

From Nature, 16 October 2019:

The commercial story of RNA interference (RNAi) harbours more plot twists and unexpected demises than television fantasy drama Game of Thrones.

As in all good dramas, there was someone — or something — that, just as things were looking promising, showed up to foil everyone’s plans, in this case hindering efforts to safely get the RNAi into the right cells to go about its work.

Christopher Anzalone, chief executive of Arrowhead Pharmaceuticals, a company focusing on RNAi therapies, based in Pasadena, California, said: “The world saw this as a new panacea and the world goes crazy, the market goes crazy.”

Then, about ten years ago, it dawned on people that getting the therapeutic strands of RNA into the right cells, so they could do their job of interfering with the target gene, was a greater challenge than first thought. Read more.

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