Modifications and Delivery

Delivery of nucleic acids is one of the most important success factors and largest hurdles for the continued advancement of miRNA therapeutics. At miRagen, we are making significant progress developing several approaches for the delivery of miRNA therapeutics, both internally and through collaborations with leading technology companies and academic institutions. In preclinical models, we have demonstrated that systemic administration is capable of delivering miRNA modulators into target cells at clinically relevant doses.  We are confident that delivery solutions for miRNAs to the heart are now accessible.

Chemical modifications are an essential area of focus. Potency, safety and duration of our miRNA inhibitors and mimics are key factors in determining the type of modifications used and their position in the miRNA modulator. We are developing novel modifications and modification patterns as well as in-licensing modifications for further study.