What are miRNAs and what makes them interesting for Mirna Theraputics?

Recently, we sat down with TIDES speaker Dr. David Brown, the Director of Discovery at Mirna Therapeutics.  He discussed at length Mi-RNAs and pre-clinical development programming.  Today's featured question is:

What are miRNAs and what makes them interesting for your therapy?

David responded: MicroRNAs are small RNAs, they function as guide sequences within eukaryotes. They are typically about 20-22 nucleotides in length. In the human genome – there are about 1500 micro-encoding genes that have been identified so far. They play very important roles in early development and tissue differentiation. It has been noted that there are specific micro RNAs that function to facilitate cardiovascular development and neurodegenetive development, immunological development, metabolism, inflammation, aging and so on and so forth. 

It has also been noted that micro RNAs are often differentially expressed in disease states. So, there are specific diseases that correspond to microRNAs being altered. It turns out that this alteration tends to play a role in the realm of the disease. So, for instance, there are individual micro RNAs that function as tumor suppressors and their down regulation leads to the development of tumors in cancer patients. There are other microRNAs that function as oncogenes. Their up regulation, of course, corresponds with the development of tumors in patients. 

Probably most importantly to us, where studies have been done using either microRNA mimics for microRNAs that are down-regulated in disease or micro RNA inhibitors for microRNAs that are up-regulated in disease, it’s been noted in animal models of these diseases that these microRNA inhibitors or microRNA mimics can actually cause a therapeutic response. That is to say, if you have microRNAs that are associated with cardiovascular development, for instance, you can inhibit that microRNA and have a therapeutic response in an animal model of cardiovascular disease. 

In essence, the way we view micro RNAs is that they were really the world’s first oligonucleotide therapeutics. We were just fortunate enough to be here at a time when microRNAs were discovered. Really, all that we are doing is trying to identify those microRNAs that have a therapeutic role in humans. Now we are developing mimics or inhibitors for those microRNAs and using those as treatments.

Listen to David's full podcast here.
Read the full transcript here.

You can find out more from David at the TIDES Summit, taking place May 12-15, 2013 in Boston, MA. He will be presenting Efficacy, PK and Tox Data Supporting the Move to IND for Mirna Therapeutics on Wednesday, May 15. For more information on this session and the rest of the program, download the agenda. If you'd like to join David in Boston, as a reader of this blog when you register to join us and mention the code TIDES13JP, you'll save 20% off the standard rate! Stay tuned for more excerpts from David's podcast.

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