The assay demonstrated impressive results in distinguishing between the two conditions, as it identified 95% of the samples accurately. Similar to other miRNA markers, the miRNAs utilized in this test can be detected in formalin-fixed paraffin embedded (FFPE) tissues, enabling fast and wide-scale retrospect analysis as well as the opportunity to be combined with standard pathological procedures. Earlier this year, Asuragen established its Mirna subsidiary, a company focused on miRNA therapeutics. This step is similar to what Isis and Alnylam did with Regulus, as Asuragen provided initial capital infusion and transferred the intellectual property to Mirna. The long term goal is entering into drug development deals with larger pharmaceutical companies.

Last week, Danish biotech company Santaris (another GSK partner) announced the launch of the first ever clinical trial for the evaluation of a miRNA-targeted drug. The drug is evaluated as a potential treatment for Hepatitis C Virus (HCV), as its target ( miRNA-122) has been shown to affect virus replication in liver cells. The trial is already in progress and will include a maximum of 48 healthy volunteers for the evaluation of the drug’s safety and other factors. So far, the company reports, results are encouraging and phase II trials in actual HCV patients are planned.

Another newcomer to the miRNA arena is Colorado based Miragen, which has recently completed $8 million equity placement from several VC firms. Miragen will initially focus on identifying targets related to cardiovascular diseases, primarily heart failure.

miRNAs are making their way from the periphery to the heart of the pharmaceutical industry in two routes: They are being evaluated as therapeutic targets as well as diagnostic targets. In the long run, miRNA-targeted drugs should have greater financial importance, but in the foreseeable future, diagnostic products represent the genuine opportunity to generate meaningful recurring revenues. Candidates from the GSK-Regulus deal, for instance, are expected to enter the clinic in three years time, and from that point, it should take on average 10 years to get a product to the market. Needless to say, the process of developing any drug is very expensive and characterized by countless delays and an extremely high likelihood of failure. Developing miRNA-targeted drugs should be twice as challenging due to the lack of experience and the nascence of the field.

As is the case with other disruptive technologies, there are still plenty of unresolved issues with respect to the wide-scale commercialization of miRNA-targeted drugs. One of these issues is the safety profile of such drugs, as every miRNA gene has multiple target genes and is involved in tens or hundreds of biological pathways. Another issue has to do with validating the right miRNA to be targeted, as not every miRNA can serve as a drug target. In addition, there are more general hurdles, which are not specifically related to for miRNA, such as getting the drug to the right organs or tissues and choosing the right technology for modulating miRNA expression. These hurdles will certainly make the road towards miRNA therapeutics a very bumpy one.