Mirrx Technology

Mirrx Therapeutics holds fundamental IP to so-called Blockmirs that are capable of blocking single microRNA:mRNA interactions, i.e. capable of preventing a microRNA from regulating one particular mRNA, while still allowing the microRNA to regulate all its other mRNA targets.

Blockmirs are steric antisense blockers that bind to specific microRNA binding sites in target RNAs to thereby prevent microRNA binding to the same site. Blockmirs do not recruit any cellular enzymes which mediate degration of target mRNAs.

Thus, if a Blockmir bind to a non-intended RNA, it will only cause an effect if it prevents binding of a microRNA or another cellular factor. This is highly unlikely, wherefore off target effects will hardly be an issue. This is contrary to microRNA inhibitors such as antimirs and antagomirs because of the promiscuity of microRNAs and also contrary to mRNA inhibitors such as RNase H recruiting antisense oligonucleotides and siRNAs that will mediate degradation if they bind to a non-intended target.

Hence, Blockmirs enable modulation of microRNA based gene regulation with exquisite specificity. Importantly, Blockmirs are typically agonists of their target mRNA, i.e. they increase the synthesis of the protein encoded by the target mRNA.

From the number of human genes estimated to be subject to microRNA based regulation, approximately every third human gene may be targeted by a Blockmir.

In addition to the apparent applications as therapeutics, Blockmirs will prove highly important research tools, pivotal to the understanding of microRNA based regulatory pathways and the contributions of single microRNA:mRNA interactions. In due course, science will use Blockmirs to study single microRNA:mRNA interactions, inadvertently leading to the discovery of Blockmirs with therapeutic potential.

Messenger RNA and proteins

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Antisense targeting of mRNA

Designing an artificial antisense oligonucleotide that binds with high affinity and specificity to an mRNA is relatively easy. Therefore, it was recognized at an early stage that antisense oligonucleotides which bind to and affect mRNA activity might…Read More

MicroRNA

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Antisense targeting of microRNA

Therapeutic approaches targeting microRNA pathways have so far focused on inhibiting the activity of a particular microRNA by using antisense oligonucleotide directed at the microRNA (e.g. antimirs and antagomirs) or during microRNA replacement therapy…Read More