Join Us for a Live ‘Tweetchat’ on the Future of RNAi, With Guest John Maraganore of Alnylam

9/28/11Follow @xconomy

Usually when I want to chat with someone, I still pick up that 19th century contraption called the telephone or sometimes meet in person. Each of those communications methods has its merits, but you can bring a lot more voices to a conversation through a real-time chat like the ones happening now on Twitter.

So now I’m getting ready to moderate what should be an informative and fun live chat on Twitter about a hot current topic in biotech—the future of RNA interference technology. I’m happy to say that one of the industry’s leaders, Alnylam Pharmaceuticals CEO John Maraganore, has agreed to take the plunge online and field questions from me, and anybody else who wants to join.

Here are the basics. This free and open chat will start at 1 pm Eastern/10 am Pacific on Tuesday October 4th, and will go for about 30 minutes. For those of you familiar with Twitter, I’ll be sending off short questions from my personal account—@ldtimmerman and from @xconomy. Maraganore, alas, doesn’t yet have his own personal account. But he will be on hand to answer questions in real-time from his company’s account—@alnylam, with some help from Alnylam’s communications director, Cynthia Clayton @claytoncomm. We will keep track of the running dialogue of questions and answers in a single column under the hash tag #RNAichat.

For those readers out there who haven’t yet signed up for Twitter accounts, we’re going to set up a livestream on Xconomy where you can watch the back-and-forth. And, if you’re not on Twitter but would like to pass a question to Maraganore, I’m giving you an out. You can send me the question at ltimmerman@xconomy.com and I’ll put it out there for Maraganore to answer on Twitter. But I will want to identify you and your company, since that’s the way it would appear if you were asking the question yourself.

I haven’t yet figured out exactly what I want to ask yet, but I do know there has been plenty to talk about lately in RNAi. For those of you less familiar, RNA interference has electrified scientists for several years, and its co-discoverers won the Nobel Prize in 2006. The excitement comes from the technology’s potential to hit biological targets of disease that are currently inaccessible by conventional small-molecules or larger biotech drugs. The pharmaceutical industry wrote some very big checks to the emerging biotech companies in the space for a while, although in recent years companies have run into tough technical challenges with delivering these new RNAi molecules where they need to go in cells. Merck and Roche are a couple of the high-profile pharma companies that have made significant cuts in the past year. Just this past week, Worcester, MA-based RXi Pharmaceuticals (NASDAQ: RXII) split into two companies as investors have lost interest, or patience, in its strategy to come up with useful RNAi drugs.

Maraganore has heard the chorus of skeptics for some time now, yet his company continues to forge ahead in what he has proclaimed will be the “RNA decade.” This week, Alnylam (NASDAQ: ALNY) advanced into clinical trials with a novel RNAi drug for lowering cholesterol in patients with severely high cholesterol scores. It is the company’s fourth experimental drug program to advance in clinical trials. Want to ask him why? Or why he’s kept the faith when others have walked away? That should be pretty easy to do in 140 characters or less. See you online at 1 pm Eastern/ 10 am Pacific on Oct. 4.

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  • R. Jones

    You’ve said it before Luke, what Biotech is missing is guts and a willingness to stir the pot.

    I think of RNAi research as a “Beer and Pizza Diet”. A fat man goes to his doctor for advice on losing weight. The doctor tells him to eat pizza and drink beer for every meal. One month later the fat man returns to the doctor and he’s gained weight.

    “What kind of beer did you drink?”

    Light beer? Stout? IPA? What about the pizza? Thin crust? Chicago style?

    The combinations can go on forever. With RNAi the “beer” is RNA. Looped? Nucleotide analogs? The “pizza” is delivery. Lipids? Peptide libraries? Every RNAi company has different options.

    At Alnylam they use conjugates, antibodies, liposomes, and peptides, “to name a few” in their delivery. They have siRNAs including lengths from 15 to 49 nucleotides, so-called “overhang” or “blunt end” features, longer forms or so-called “dicer substrates” as well as many additional siRNA design and functional features; and all siRNAs with or without chemical modifications used to introduce “drug-like” features.

    It makes ones head spin the think of how many ideas have been tried by Alynlam and others. Along the way Big Pharma has given up. Big Pharma was the fat man who just got fatter.

    Stir the pot and make a list of how many RNAis’ have been proposed in the literature and company websites. How many delivery systems are there? Why should we believe Alynlam? It’s time to admit that something is rotten in Denmark.

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