miRNA Chemistry and Delivery
The activity of therapeutic miRNAs can be enhanced by chemical modifications and by an adjuvant that protects the oligonucleotide from degradation in biofluids, prevents rapid clearance by the kidneys and makes sure that the therapeutic miRNA reaches the diseased tissue. These adjuvants, widely referred to as “delivery technologies”, enable a practical route of drug administration. The preferred route of administration for most therapeutic miRNAs is systemic delivery, because it provides the most effective dissemination of the therapeutic. Local administration is an alternative that – depending on disease and location – can be equally effective.
Mirna Therapeutics has an active chemistry program to develop miRNA therapeutics with enhanced pharmacokinetic properties. In addition, Mirna has been evaluating multiple delivery technologies to identify those that endow the therapeutic miRNAs with the ability to target local tumors and tumor metastases. Mirna has exclusive rights for the use of a lipid-based vehicle in the field of therapeutics. This technology shows a favorable safety profile in pre-clinical animal studies and has the potential to be easily translated into clinical applications. With a well developed miRNA chemistry, potent miRNA mimics and effective delivery systems, Mirna is well positioned for bringing miRNA therapeutics into the clinic. The Company anticipates filing its first IND in 2011.