Oct 1, 2007: RNA interference—an innovation that quickly moved from discovery to a technology that enables drug discovery, drug development and therapeutic synthesis has big pharma in attention. A report by market analyst Frost & Sullivan indicates that the worldwide RNA interference market is estimated to be $328 million by 2010.
Abbreviated as RNAi, the applications of this technology are witnessing a steep rise with the rapid uptake among researchers in biotechnology and pharmaceutical companies. Between 2003 and 2005 RNAi segment witnessed active collaborations, licensing and buyouts, attracting VC
interest.
Today, more than 20 companies are already involved in RNAi-based drug discovery programs, among which about 10 are exclusively working on RNAi.
World’s biggest pharmaceutical firms like Merck, GlaxoSmithKline, Pfizer, Abbott, AstraZeneca, Roche, Novartis and Bristol-Myers Squibb have put their monies on RNAi and believe that this technology will bring new products to their pipeline.
According to Frost & Sullivan the three main applications of RNAi are in drug target validation, basic research and therapeutics. Among these, target validation will be the most common use for RNAi in the foreseeable future. RNAi also facilitates the study of functional genomics in mammalian cells, a key to developing personalized medicines.
What makes the technology so hot?
RNAi has the potential to address significant medical needs, many of which cannot effectively be addressed with small molecules or antibodies, the current major classes of drugs. RNAi is a natural process of gene silencing. It has the ability to down-regulate protein levels in a gene specific manner.
The main advantage of this technology is that it does not affect cells that are not targeted and it allows researchers to study the molecular effects of modulating expression at the level of individual genes. It is also said to be a cost-effective method for rapid identification of gene function. RNAi is rapidly becoming the preferred method for knocking out the expression of only targeted genes. It is useful for assigning gene function and in signalling pathway analysis.
Therapeutics based on RNAi technology target the cause of diseases by potently silencing specific messenger RNAs (mRNAs), thereby preventing disease-causing proteins from being made. Hence, by harnessing RNAi, a major new class of therapeutics and diagnostics can be created.
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What is RNAi?
Discovered in 1998 by Craig Mello and Andrew Fire, RNA interference is heralded as a major scientific breakthrough in the past decade, which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNA interference (RNAi) allows scientists to silence the expression or effect of a gene being studied. It is a mechanism for RNA-guided regulation of gene expression in which double-stranded RNA inhibits the expression of genes with complementary nucleotide sequences. Conserved in most eukaryotic organisms, the RNAi pathway is thought to have evolved as a form of innate immunity against viruses and also plays a major role in regulating development and genome maintenance.
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RNAi powers Alnylam
Backed by the inventors of RNAi technology, Alnylam Pharmaceuticals is spearheading the business of RNAi-based therapeutics. The company has to its credit the maximum number of collaborations and has received maximum funding in the RNAi segment.
Alnylam is leveraging its capabilities to build a broad pipeline of RNAi therapeutics. Its most advanced program is in phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. In addition, the company is developing RNAi therapeutics for the treatment of influenza, hypercholesterolemia, and liver cancers, among other diseases. By December 2007, Alnylam would announce the outcome of its phase II clinical trial on their lead RNAi product for treating the infant respiratory disease caused by Respiratory Syncytial Virus (RSV infections). Researchers believe that the potential of RNAi would be testified with the outcome of this company’s phase II trials.
Pharma biggies like Novartis, Biogen Idec and GSK are all collaborating with this forerunner. Even medical device major Medtronic has struck a deal with Alnylam. The company also signed a deal valued at over $1 billion with Roche. This is said to be the largest drug discovery alliance in biotech history and broadest collaboration on RNAi therapeutics to date.
Alnylam was also awarded a $38.6 million contract over 33 months from the United States Defense Threat Reduction Agency (DTRA) to develop a broad spectrum RNAi anti-viral therapeutic for the treatment of viral hemorrhagic fever. Viral hemorrhagic fevers are considered by federal agencies to be high priority agents that pose a risk to national security because they can be easily disseminated from person to person, result in high mortality rates, and require special action for public health preparedness.
The most recent collaboration took place in September 2007 when Alnylam and Isis Pharmaceuticals announced the formation of Regulus Therapeutics Inc., a biotechnology start-up that will focus on commercializing drugs made with microRNA technology. The collaborators felt that this joint venture made sense given the two companies’ expertise in drug development and in RNAi. With all these mergers and alliances Alnylam is leading the business of RNAi.
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Reagent suppliers move in
Not only is the mainstream business doing well but also the associate companies supplying reagents are bullish on RNAi. Reagent and analytical and screening instrument manufacturers have sensed a growing demand from RNAi researchers and scientists. Companies such as Invitrogen, Sigma-Aldrich, Dharmacom, Qiagen, Ambion have all set up divisions that work specifically towards the preparation of reagents associated with RNAi technology.
The RNAi reagent supply market also witnessed a wave of consolidation. In 2005, Fisher Scientific acquired Dharmacon for $80 million and Sigma-Aldrich acquired Proligo. In 2006, Ambion sold its research products division to Applied Biosystems for $273 million. In the same year Abbott signed a collaboration with Dharmacon to develop new therapeutics based on RNAi technology. Such is the power of RNAi.
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Multi-billion dollar market on the horizon
Scientists and industrialists opine that even if a fraction of RNAi’s promise is realized, the technology could lead to billions of dollars in new drugs. Optimism on the technology is rising, as exciting results from late stage clinical trials are announced. It is perceived that in future RNAi therapy could probably be the most successful for diseases caused by a single gene or a small number of genes for use as a complementary therapy in treatment of complex multigenic disorders. In fact the importance of RNAi has spurred academicians and event organizers to hold an exclusive event on RNAi as a theme. RNAi Europe, which was held in Barcelona, Spain in Spetember 2007, is a good example.
Analysts also perceive that if drug delivery–a major hurdle for RNA-based therapeutics–is solved, the technology could throw open a multi-billion market. The company that succeeds to launch the first RNAi-based drug will definitely be a run-away hit.
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