Wednesday, April 20, 2011

Sifting Through the Noise in RNAi Therapeutics Delivery

Hardly a day goes by without news of delivery deals, or universities issuing a press release that their scientists have just published on a revolutionary and ‘novel’ delivery technology that, by the way, is available for licensing. To be fair, public relations is a necessary evil of drug development and even academia these days, and don’t expect any company to blink and admit to their weaknesses.

This, however, poses a real problem for RNAi Therapeutics as the noise causes investments to be diverted away from the most deserving technologies, technologies that are more than just wishful theoretical thinking and with reasonable paths towards the clinic. There is, of course, also a place for the more audacious technologies, but this is what universities are for.

When reading about a new delivery breakthrough or deal announcements from sources you have hardly heard of before, it pays to browse through the peer-reviewed and patent literature to get a first impression of whether there is anything of substance to start with. Rest assured, a new delivery technology, especially if ground-breaking, won’t successfully go from scratch into the clinic within the next 10 years.

I don’t want to name any deal or company in particular, but I would also caution you not to put too much faith into the delivery claims of those biotech companies that are run by management and Directors that constantly chase the latest and hottest in science in order to generate constant buzz around their companies so that more shares and options can be issued and dumped on retail investors. Be wary of those companies that get regularly promoted in cheap analyst reports listed on Yahoo! and Google Finance pages, companies that select academic collaborators not based on how they can contribute technically, but based on politics and fame, and where companies are happy to have rumors spread on message boards.

When asked by a newspaper reporter what, in my mind, were the key publications in RNAi Therapeutics delivery over the last 3 years, I answered that it’s not been one or two, but the series of papers describing the transitioning and refinement of SNALP delivery technology from the first non-human primate proof-of-concept data reported in 2006 in Nature into the clinic today. Needless to say, despite the criticality of these de-risking events, also as it relates to the overall investment climate in RNAi Therapeutics for the coming years, this wasn’t picked up as being quotable.

There is no easy solution to this capital allocation problem. All one can hope for is that some of the more deserving technologies can build a network of supporters that, among the boom and bust cycles of the industry, will see them through to at least early clinical studies where they will have to prove their mettle.


Gene Genie said...

The technology that works is shrna , this can be triggerable or reversable so si rna is a dead end except for a few applications , Benetic and the CSIRO invented RNAI and chose shrna , it has taken the market 8 years and 6 billion dollars to get to the same spot , shrna has no delivery option limitations in fact the converse is true. Now Benitec is fully funded for the next 3 years it should accelerate in its leading position regarding rnai , dont forget the HIV data , the first breakthrough rnai clincal program. Targets now include viral , inflammatory, cancer and soon genetic with Duchennes this will prove the universal application of the Benitec and CSIRO patent estate.

Anonymous said...

Avoiding names when tekmira competitors are involved is in a perfect concord with previous reports about TKMR/ALNY relation.

Anonymous said...


Calando was the first to provide evidence of effective therapeutic delivery of RNAI in humans, yet it has been unable to find a partner to help fund the clinical development of this promising delivery system. Are you aware of any serious interest in this technology, and what is your assessment of the value of this system?

Dirk Haussecker said...

Without serious in-house research, it will be difficult for Calando to find a partner for its delivery system. I would agree, however, that Calando's was the first direct demonstration of RNAi activity in Man.

Benitec: Yes, quite encouraging turn for the company. The strong resource-driven Aussie economy has probably helped a bit in encouraging taking risks with innovation. I wish them well.

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