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RNAi - Next Phase or Next Fad?

I have long told our investors to avoid RNA interference stocks because the science is unproven. If you want to speculate, you can do so on more proven science.

I have also written that investing based on the hope of a major partnership or buyout is a sucker's game. Well, these two rules backfired on me this week and bite me on the butt. Alnylam (ALNY) announced a ginormous (just added to the official Webster'; Dictionary, much to my kids' pleasure and my discomfort) drug deal with Roche.

I don't care! You need the metaphorical leather butt to do what I do -- nevertheless, in the interest of public service I have put together a list of names you can investigate on your own and get more hits on this blog as investors frantically search for RNA stocks.

• Alnylam (ALNY)
• AP Pharma (APPA)
• Cytrx (CYTR)
• Genta (GNTA)
• Isis Pharmaceuticals (ISIS)
• Invitrogen (UVGN)
• Nastech (NSTK)
• Sangamo Biosciences (SGMO)
• Sentek (SNKTY)

This is your research job and many of these companies seem to have done as much work on their press releases as they have on RNAi.

What is RNAi?

In the past decade or so, RNA -- a cousin of DNA (look it up on Wikipedia, I can't do everything for you here) - has been shown to have a much more prominent role in cellular activity as a regulator of behavior. Modify RNA behavior and you can modify a disease.

A couple of products are wending (not winding -- look that one up too) their way through the labs and development process. The closest one, for age related macular degeneration, is from what used to be called Sirna, now part of Merck, the first RNAi deal to bite me you know where.

Nothing else appears close. If I sound cranky, I am - my butt is beginning to ache.

Comments (2)

Adam:

You can also add to the list AstraZeneca since they just recently purchased Silence Therapeutics for $402M. Not quite half as big as the Merck-Sirna deal. I am coming to you from Boulder, CO..."RNA-town" for lack of a better moniker. RNAi, siRNA and miRNA has started and will continue to be validated by big pharma!

Jay Schneiders:

You should be aware that SGMO is not founded on RNAi technology. SGMO's ZFP technology works at the DNA level and consequently far more precise (than RNA interference) in its ability to regulate gene expression. SGMO can up-regulate, down-regulate, correct abnormal genes, and knock-out undesired genes. RNAi can only down-regulate.

Furthermore, SGMO dominates the IP in its space (DNA platform) whereas RNAi technology is fragmented. That's why SGMO has been more "under the radar". Its recently announced partnership with Sigma may start to raise the profile.

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