Biotech Values

[urche]

pithy post on RNAi

I admit my bias (as a clinician, not a hard scientist)which is not optimistic about RNA interference strategies becoming commercially successful in my investing lifetime.

In that light, I share a pithy post from one of the sagest posters on the Motley Fool Biotech message board. Note, in particular, the last paragraph/footnote.

I'm going to go out on a limb here and predict that, 10 years from today, there will not be a single approved drug based on RNAi technology*.

As Dan correctly points out, the problem is cell penetration, or more correctly, delivery, and it is exactly the same problem that has stymied antisense technology for all these years (really decades now). How many people remember that one of the most successful biotech companies, GILD, started life as an antisense company, but gave up in disgust? It's always been clear that antisense works, if only you could get enough of the damn stuff to the right place at the right time.

While it appears that RNAi is more potent than antisense, it is pretty much equally undeliverable. I wouldn't place a lot of faith in promised solutions to this problem. The antisense field has been making those same promises for years, and all we have to show for it is a single marketed drug that sell ~$100K/year, and probably doesn't even work by an antisense-mediated mechanism anyway.

RNAi is great in the lab (at least in tissue culture--it sucks in animals). Unfortunately, there's no money in that.

Fushi

*Excluding drugs that target diseases of the eye. Drugs injected into the eye tend to stay there, solving the delivery problem--which is why the number one discovery program at most RNAi companies targets macular degeneration. If there is an approved drug in 10 years, and it isn't for macular degeneration, it will be for a liver disease (hepatitis?), which is where all the RNAi injected into the blood stream ends up.