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DBL%20Hendrix%20small.png College chemistry, 1983

Derek Lowe The 2002 Model

Dbl%20new%20portrait%20B%26W.png After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek email him directly: derekb.lowe@gmail.com Twitter: Dereklowe

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In the Pipeline: Don't miss Derek Lowe's excellent commentary on drug discovery and the pharma industry in general at In the Pipeline

In the Pipeline

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January 13, 2014

Alnylam Makes It (As Does RNAi?)

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Posted by Derek

I've written about Alnylam, one of the flagship RNA interference companies, a few times around here. A couple of years ago, I was wondering if they'd win the race to come up with results that would keep the doors open.

Well, if you haven't been keeping up with the news in this space, they made it. Sanofi has just bought a large stake in the company, on the strength of the recent clinical results with patisiran, an RNAi therapy for the rare disease transthyretin-mediated amyloidosis (ATTR). Alnylam has a lot on their schedule these days, and the Sanofi deal will provide a big boost towards getting clinical data on all these ideas. Congratulations to them, and to RNAi in general, which has had a lengthy (and often overhyped) growth phase, and now might be starting to realize its promise.

Update: more on the story here.

Comments (7) + TrackBacks (0) | Category: Business and Markets | Drug Development


COMMENTS

1. Teddy Z on January 13, 2014 1:45 PM writes...

It's interesting that this was reported today when it was also reported today that Merck punted siRNA to Alnylam (link in my URL). So, Merck lost a billion dollars in value on its 7 years of holding siRNA, but Alnylam is a good bet.

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2. MoBio on January 13, 2014 1:51 PM writes...

Interesting that 'making it' does not mean 'having a drug approved by the FDA/EU'....

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3. Anchor on January 13, 2014 2:06 PM writes...

@2 I agree with you! I mean this siRNA drug makes use of nano particle for its delivery and at the end of the day when things work as planned, you ask at what cost? Rest is assured if the cost is high (as in the case of many cancer drugs that are coming of the pike) then it is not worth it and it spells disaster for the whole area.

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4. Andrew on January 13, 2014 3:43 PM writes...

@2 - making it also doesn't appear to require safety data for more than 2 doses of a chronic product or any clinical efficacy data on any of their programs. And now what, close to $10b market cap when you factor in the new shares to MRK and SNY?

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5. Lu on January 13, 2014 6:24 PM writes...

rare disease transthyretin-mediated amyloidosis (ATTR)

Reminds me of a drunk man looking for lost keys under a streetlight: it's not where he lost them but it's where they are easier to search for.

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6. Anonymous on January 13, 2014 6:29 PM writes...

Alnylam makes it... for the next 5 months.

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7. Gerry Atrickseeker on January 24, 2014 6:17 PM writes...

One of the major problems with siRNA was the inability to effectively deliver these molecules to their intracellular targets. One approach to this has been to load the siRNA into cationic lipid nanoparticles. However, this often resulted in considerable toxicity. This is one of the big reasons that the majors exited the field. Recently Alnylam, as well as several academic labs, have been making ligand-siRNA conjugates that promote siRNA uptake by receptor- mediated endocytosis. An advantage of this approach is that ligand-siRNA conjugates are chemically defined molecules, unlike complex, multi-component nanoparticles. However, the jury is still out on the therapeutic viability of this approach.


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