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Wednesday, September 04, 2013

RAiN BioScience and The Disappearing Bees

RNAi and all RNA drug projects are doomed to fail. RNA and gene expression are too complex for mere mortals to regulate. We must leave that up to our DNA and the wisdom within. That doesn't mean that human beings won't succeed at selling each other drug products that tout RNA as the API. It just means that something other than scientific evidence will be needed to convince the FDA that you have a treatment for some disease. Cargo Cult Science will be needed. Once approved, RNAi proponents will have been vindicated. In the end, RNA will enter the body and quickly be reduced to an assortment of nucleic acids.

The questions I want to bring up today is 1) what happens to the PhD and laboratory staff who work on dead end Cargo Cult projects like RNAi and 2) what can we learn about their disappearance?

Most RNAi companies called it quits in the past five years. There is one company that has survived is called Serepta Therapeutics. From Wikipedia:

Incorporated in 1980, the company maintains some laboratory capability in Bothell, Washington and Corvallis, Oregon. As of 2008, the company has 170 issued medical patents, and over 150 patents pending.[1] The company changed its name from AVI BioPharma and stock symbol from AVII in July 2012 to Sarepta Therapeutics and SRPT respectively.
I wonder how many of those patents are gene patents? 

 The company is a great example of a Cargo Cult Airport. They have rearranged their airport many times in many ways. The company began with the name AntiVirals Inc. in 1980. Later they changed the name to AVI BioPharma and then later to Serepta Therapeutics. They have changed locations from around Oregon, then a big shift to Seattle Washington (to attract the big brains) and then to Cambridge MA to attract "the right people". The Seattle move was a bust. They have burned through over $320M since 1980 and they have yet to bring a drug to the market.

What happened to the PhDs who were not invited to Cambridge? They are now known as RNAi experts, not as research professionals. Therefore they need to do something with RNAi.

Enter RAIN BioScience, a new Seattle biotech started by two discarded Serepta PhDs. Imagine the interview process at Serepta when the employees were arguing to keep their jobs. Serepta management took their patents and technology to the east coast. Those people who established the legitimacy of the science, however, were brought in to question. The two guys who started RAiN, somehow, did not make the cut.

The company, RAiN, is still a mystery. Yesterday on Biospace it was announced that Seattle had this new biotech in town.  RAiN BioScience is a Delaware corporation that will make therapeutics for unmet medical needs using antisense technology. Antisense drugs cannot enter cells well so RAiN BioScience will develop technology to help the RNA reach their intracellular sites of action. The project will be validated with an anti-cancer target.

So that is where two of the RNAi PhD castaways washed up. They are not alone however. Lets look at a few of their predecessors.

PhaseRx, an RNAi Delivery Technology company, founded in 2007 by a few local Seattle suspects. Two PhDs came from Marina Biotech, an RNAi company with "world class expertise" in delivery. From their website:

PhaseRx was conceived on the basis of using novel polymer technology to deliver siRNA. The Company's proprietary technology is a new type of synthetic, multi-functional polymer exclusively licensed from the University of Washington. The polymers come from the laboratories of Patrick Stayton and Allan Hoffman at the University of Washington, which have world-class expertise in fine-tuning the polymers to meet intracellular and in vivo delivery needs.

They too aim to treat cancer. They are several years ahead of RAiN but the two project in their pipeline are still in the early preclinical research phase. The latest news release was in January of 2011 and they have no jobs listed. Financing beyond their initial $19M has not been reported.

Agave Pharma Inc. was founded in 2009 by a former PhD from Marina Biotech. They have 4 employees and they specialize in novel nucleic acid delivery technology for RNAi therapeutics. Very little information can be found on their progress. 

Halo Bio RNAi Therapeutics founded in 2011 by Todd Hauser offers an RNA drug that consists of three separate double-stranded RNA regions that form a star. Indeed their website is pretty slick, even offering an Ap for your I-pad. When you click on "delivery" however, nothing comes up. The website also seems to exclude common details such as management, directors, location... It is not clear if they are still in business. 
Part II  

There is documentary on the disappearance of honey bees worldwide. It's called Vanishing of the Bees. In this film the first warnings of the vanishing of the bees came from bee keepers. Men with their boots on the ground who open up their cages to find that their bees are gone. I submit that the rest of the world is not going to hear about the Vanishing of Biotehnology from the likes of Xconomy or the greatly reduced staff at the local newspaper. It won't come from the workforce who take their lumps and come back for more. They won't rock the boat. It will come from me, the Cargo Cult Scientist! Or someone else who gives a crap. There is a story to be told out there about the comings and goings of the biotechnology, the PhDs and their science. The science is questionable. The way in which we deal with it is far more telling. 100 years from now RNAi will mostly be forgotten because it is Cargo Cult. We'll learn more about RNA and it's role in gene expression regulation, but this little history of jumping the gun to make our fortunes from RNAi will fade away. Useless poetry. The machinations of a dying science and its practitioners however, will serve as a warning. 

The lesson I have learned here in Seattle is that RNAi delivery became the scapegoat for the jumping of the gun. It is a big issue among the PhDs closest to the laboratory. It cost them their jobs. We have no way of knowing what would have happened had they gotten their RNA to the target cells, but we know that the new pseudo companies intend to address this issue. We also know that the PhDs are starting to pile up at this very juncture, delivery. I haven't even mentioned the consultants here in town. 

Why haven't they all gotten together to figure this thing out? Why so many silos? Where is the RNAi book club of Seattle? Do they all intend to work alone on promise of RNAi delivery and ignore the hype?

I know they are dying. Like the bees, they just aren't there anymore. My LinkedIn account shows me what my newspaper and what Xconomy does not see. They are disappearing. They pop their heads up in companies like RAiN but you don't hear much about them beyond the initial hype. My old co-workers aren't updating their accounts with exciting new jobs. The little sparks like RAiN BioScience would be fun to watch grow, but if PhaseRx, Agave, Halo and all of the other unreported efforts are any example, RAiN will simply fade. 

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