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Despite Its Nascent Technology, MicroRNA Field Adds New Players

While younger than its RNAi counterpart, the microRNA-based drug field continues to attract new companies interested in taking advantage of the small, non-coding RNAs for therapeutic applications.
In the past 12 months alone, at least three pure-play miRNA drug-development companies have made their debut. The newest of these is Miragen Therapeutics, which was founded last summer by University of Texas Southwestern Medical Center researcher Eric Olson, the University of Colorado at Boulder’s Mike Bristow, and former Dharmacon executive Bill Marshall.
As reported by RNAi News last week, the company earlier this month closed an $8 million Series A financing round that will support its efforts to develop drugs against cardiovascular disease and related muscle disorders (see RNAi News, 5/22/2008).
The specific details about Miragen’s pipeline have not yet been publicly disclosed, but Marshall recently told RNAi News that the firm expects to initially go after heart failure, a condition he said “has many different etiologies and potential [points of] intervention” (see RNAi News, 5/15/2008). The firm also plans to define a specific clinical indication over the next six months, he added.
Despite these open questions, one thing is relatively certain: Miragen’s initial drug candidates will be designed for direct delivery to the heart.
“The standard of care for heart failure in a variety of different cardiovascular disorders is catheterization,” Marshall explained. “So we’re going to leverage some semi-invasive, mechanical delivery technologies that allow us to do pseudo-local delivery.”
Another newcomer to the miRNA drugs space is Mirna Therapeutics, which was officially spun out as a subsidiary of Asuragen last month (see RNAi News, 4/3/2008).
When Asuragen was created out of the diagnostics and services components that Applied Biosystems chose not to buy when it acquired Ambion’s research products operations in early 2006 (see RNAi News, 1/5/2006), it was expected to leverage its small RNA expertise to develop both therapeutics and diagnostics based on miRNAs (see RNAi News, 1/12/2006).
Two years later, however, the company began the process of spinning out its miRNA drugs assets into Mirna, which included making a $3 million investment in the new entity (see RNAi News, 1/24/2008).

Although Mirna has made few announcements about its plans, the company’s website indicates that cancer will be its primary interest, with a focus on non-small cell lung cancer, metastatic prostate cancer, and acute myeloid leukemia.

Matt Winkler, Asuragen’s founder, CEO, and CSO, told RNAi News at the time that the move would enable Asuragen to focus on its core diagnostics and services operations.
Although Mirna has made few announcements about its plans, the company’s website indicates that cancer will be its primary interest, with a focus on non-small cell lung cancer, metastatic prostate cancer, and acute myeloid leukemia.
In addition, Mirna has exclusive access to inventions developed by Yale University researcher Frank Slack related to the use of miRNAs, including let-7, to regulate oncogenes (see RNAi News, 1/11/2007). The deal with Yale was originally struck by Asuragen, but passed along to Mirna during the spinout process.
Around the same time that Miragen and Mirna were being established, RNAi heavyweight Alnylam Pharmaceuticals and Isis formed a joint venture called Regulus Therapeutics to discover, develop, and commercialize microRNA-targeting drugs (see RNAi News, 9/13/2007).
Giving Regulus a decided edge in its endeavors is its intellectual property portfolio, which includes IP from both Alnylam and Isis related to therapeutic applications of miRNAs. According to the company, its IP estate includes over 900 patents and patent applications related to chemical modification of oligonucleotides.
In the few months that it has been operational, Regulus has also been the first miRNA therapeutics shop to forge a miRNA-specific alliance with a big pharma: In April, the company reported that it had partnered with GlaxoSmithKline to discover, develop, and market miRNA drugs for inflammatory diseases such as rheumatoid arthritis and inflammatory bowel disease (see RNAi News, 4/17/2008).
Under the arrangement, Regulus was paid $20 million up front, and stands to receive up to $144.5 million in milestones, as well as royalties.
Although interest in miRNA drugs is just starting to heat up, one company — Rosetta Genomics — has been working in the space since 2000 when it was founded to develop miRNA-detection technologies.
In late 2005, RNAi News reported that the Israeli company planned to become a US-based, publicly traded miRNA drug and diagnostics developer (see RNAi News, 11/4/2005).
Thus far, Rosetta’s goals have largely been on track: The company has opened facilities in New Jersey and completed a roughly $26 million public offering (see RNAi News, 3/1/2007 and 3/8/2007). It is also nearing the launch of its first miRNA-based diagnostic (see RNAi News, 5/22/2008) and continues working on miRNA drugs for liver cancer and infectious diseases including HIV and hepatitis C.
Also working in the miRNA drugs field is Danish oligo drugs firm Santaris Pharma, which was created in 2003 through the merger of Cureon and Pantheco. While the company is not strictly a miRNA drug developer, Santaris is exploring the potential of its locked nucleic acid technology to silence the small RNAs and could potentially be the first to test such a drug in humans.
This week, Santaris announced that it had begun phase I testing of its lead miRNA-targeting drug, an investigational HCV therapy called SPC3649, making the company the first to move such a drug into human trials.
Also, late last year, Santaris secured a partnership with GlaxoSmithKline to develop LNA-based antiviral drugs (see RNAi News, 12/20/2007). Although the arrangement is not specifically centered around miRNA therapeutics, the deal does give the British big pharma an option to acquire SPC3649.

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