Saturday, June 14, 2008

RNAi Therapeutics: RNA Research Blossoms in Economically Challenging Times

As the war in Iraq continues to cannibalize investments into the US health-care system and particularly funding for basic research, and spiraling commodity prices and the bursting of the credit bubble conspire to create risk-averse capital markets in which small biotech companies are struggling to fund R&D, RNA research is blossoming while RNAi Therapeutics appears to be one of the very few areas in biotech financing where money is still readily available.

This view is not only supported by the exploding number of RNA-related research publications, not to a small degree triggered by interest in RNAi and microRNAs, but also anecdotally by the exponentially increasing numbers of official registrants of the Bay Area RNA Club meetings. An impromptu meeting for RNA researchers to gather and schmooze over RNA science every half a year or so, it drew more than 250 RNA enthusiasts this past week to the UCSF Mission Bay Campus across the street from the expanding Merck subsidiary Sirna Therapeutics in San Francisco, also the place that had just hosted the Qiagen HT RNAi user meeting the week before. At the same time, the corporate RNAi world was assembled for the Beyond Genome conference at the lofty Fairmont Hotel.

Of course, the big meeting taking place concurrently in San Francisco was the enormous ADA conference. Although RNAi was not really represented there, this should change in the coming years as metabolic disease together with other liver disease and cancer is emerging as one of the hot areas for first-generation systemic RNAi Therapeutics. RNAi Therapeutics appears to be the logical answer as the genome revolution is yielding serious therapeutic targets for metabolic disease and cancer on an almost daily basis. Recent papers studying non-alcoholic steatohepatitis with AAV RNAi (a very potent technology for persistently knocking down genes in the liver, at least in mice) or the surprising identification of transcription factor XBP1 in regulating lipogenesis in the liver are just two examples. The latter is also particularly interesting as targeting XBP1 may require tissue-specific delivery, and the availability of RNAi delivery technologies such as SNALP RNAi which can carry up to 95% of the injected dose to the liver.

Given the potential for innovation, rational drug development, and more efficient and shortened development time-lines, it is therefore not surprising that one of the very few areas in Big Pharma R&D not affected by big cutbacks is RNAi Therapeutics. Think Merck, GSK, Pfizer, Abbott, Takeda, Novartis… a list of those Big Pharma companies not participating in RNAi Therapeutics research would probably be more informative at this point.

I even wouldn’t be surprised to see Genentech, based on their increased presence at RNAi conferences, make themselves less reliant on protein-based therapeutics and throw their support behind RNAi Therapeutics relatively soon, probably before 2010. Genentech, the pioneer and poster child of personalized cancer therapeutics, would be the logical, unnamed Big Pharma that Tekmira mentioned has been evaluating their SNALP delivery technology. I should apologize for mentioning SNALP RNAi so often, but by following the Alnylam-Tekmira, I believe one can gain invaluable insights into current RNAi Therapeutics trends.

I am confident that the intense efforts in RNA research in academia and industry will provide a fertile soil on which RNAi Therapeutics will continue to thrive, and may not only survive the very real economic downturn, but given the pressures experienced by Big Pharma and the healthcare system even benefit from it. Merck, Roche, Takeda and others are not the result of clever business development removed from science, but a consequence of such efforts.

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By Dirk Haussecker. All rights reserved.

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