MicroRNAs are a family of small (19-25 nucleotides) noncoding RNA molecules that regulate gene expression at the post-transcriptional level. MicroRNAs inhibit gene expression by complementary base pairing with sequences mainly located in the 3’ untranslated region (3’ UTR) of the target messenger RNAs, leading to translational repression or messenger RNA degradation. A key recognition element is a perfect match in the seed sequence comprising nucleotides 2-8 at the 5’ end of the microRNA. MicroRNAs are classified as families, defined by the conservation of the seed region, with many family members highly conserved from nematodes to humans, which already suggests the importance of microRNA function during evolution.
Since microRNAs only require partial complementarity for target recognition, any single microRNA is able to subtly regulate multiple messenger RNAs. The combined effect of single microRNAs and their combined target networks results in significant effects on the phenotypic outcome of relevant human medical disorders such as cancer, neurodegenerative diseases, metabolic disorders and cardiovascular diseases. Mirabilis Therapeutics is focused on developing microRNA therapeutics, drugs that target aberrantly expressed microRNAs in disease areas of high unmet medical need. By regulating microRNA function, the use of antisense oligonucleotides as microRNA inhibitors for the treatment of cardiovascular diseases produces therapeutically beneficial results by restoring proper target gene regulation. Since current heart failure pharmacotherapy only has a marginal impact on long-term prognosis of the disease, there is both room and need for the development of innovative bio-therapeutics. Development of microRNA-modulating drugs also requires optimization of their chemical and pharmacological properties. Mirabilis Therapeutics scientists have vast experience in pharmacologically modulating microRNAs using oligonucleotides, pre-clinical and clinical development, formulation and regulatory affairs.