RNAi: Yep, Antisense All Over Again

Yesterday, New York Times reporter Andrew Pollack [covered][1] the pharmaceutical industry’s recent rush to the lifeboats of the siRNA/RNAi ship:

When RNA interference first electrified biologists several years ago, pharmaceutical companies rushed to harness what looked like a swift and surefire way to develop new drugs.

Billions of dollars later, however, some of those same companies are now losing their enthusiasm for RNAi, as it is called. And that is raising doubts about how quickly, if at all, the Nobel Prize-winning technique for turning off specific genes will yield the promised bounty of innovative medicines.

In particular, Pollack says companies have been spooked by the apparent side-effects of RNAi:

One obstacle is that the double-stranded RNA snippets, perhaps because they do resemble viruses, can wake up certain immune system sentinels and set off an immune response.

Such responses can be an unwelcome side effect in some cases. In other cases, like in treating cancer or infections, an immune response might be welcomed — but might also obscure whether the gene silencing itself is working.

I recall someone else pointing out exactly that problem a couple of years ago. Since I’m obviously on a roll with predictions here, I’ll throw out another one: the last company standing in the RNAi field – probably Alnylam – will soon be able to buy up all of the relevant intellectual property and talent in RNAi at fire-sale prices. They’ll then restructure into a small, lean operation that will eventually make a modest profit with some niche therapies. In other words, they’ll do what Isis did.

Place your bets.

[1]: http://www.nytimes.com/2011/02/08/science/08rna.html?_r=1&ref=science&pagewanted=all