RNA Therapeutics Are Here to Stay


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demonstrated positive preclinical data in hepatitis C and Alport syndrome, an orphan kidney disease driven by genetic mutations with no approved therapy. Regulus plans to establish human proof of concept with RG-101, a compound targeting microRNA-122 for the treatment of hepatitis C, in a Phase 1 study by the end of 2014. If favorable, these results will go a long way toward validating Regulus’ microRNA therapeutic platform and approach to treating disease. Additionally, Regulus is also exploring the utility of identifying microRNAs as biomarkers of human disease.

• Other types of up and coming RNA therapeutics include RNA aptamers, messenger RNA technology, and RNA modulation. RNA aptamers bind with high specificity to a target molecule, and can rival the function and efficacy of therapeutic antibodies. Novartis recently demonstrated its confidence in these next generation ligands by gaining rights to Opthotech’s (NASDAQ: OPHT) aptamer-based treatment for age-related macular degeneration (AMD). RNA-modulating techniques target pre-mRNA processing (alternative splicing) and have significant implications in rare diseases. Both Sarepta Therapeutics (NASDAQ: SRPT) and Prosensa (NASDAQ: RNA) are exploring preclinical and early clinical exon-skipping strategies to treat Duchenne Muscular Dystrophy. Lastly, messenger RNA therapeutics use synthetic mRNAs and have the unique ability to produce intracellular proteins in vivo. Moderna Therapeutics is heading up this research effort, and in January of this year, entered into an agreement with Alexion Pharmaceuticals (NASDAQ: ALXN), collaborating exclusively to develop messenger RNA therapies.

Despite the recent explosion of RNA-based research and positive scientific results, some in the media and investment communities continue to focus on perceived concerns regarding drug delivery and the limited capability of tracking down reliable targets. The recent decisions of Novartis and Merck around their respective RNAi programs are in no way a barometer for the state of the entire platform, but rather reflect internal program priorities. In truth, the science is in a very exciting stage as researchers are quickly getting a better handle on the molecular mechanisms behind RNA technology, allowing companies to identify where RNA-based therapies can work and in which disease areas. New delivery technologies are continuously being developed and mastered with validation being seen in numerous preclinical and clinical studies. For example, in 2013 alone, there were over 7,000 peer review publications on microRNA therapeutic targets, and because the biological knowledge is increasing exponentially, it is projected that there will be approximately 9,000 peer review publications on microRNA therapeutic targets in 2014.

Overall, pharma remains interested and invested, and investors continue to embrace RNA therapies, even through the “expected” fluctuations in the market. Now is an exciting time for the RNA therapeutics space, as biotechs are poised to push treatments through the clinic and to the patient. Antisense, RNAi, microRNA, aptamers, messenger RNA, and RNA-modulation technologies offer a unique opportunity to target a range of human diseases. I believe that we are now experiencing the right combination of technology, intellectual property, leadership, and investment that will further advance RNA therapeutics into powerful medicines to help patients in need.

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Kleanthis G. Xanthopoulos, Ph.D. is the former president and CEO of Regulus Therapeutics, a company developing drugs based on microRNA technology. Follow @

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