Through transcriptional activation, RNAa therapeutics promise a revolution in our ability to modulate previously undruggable targets.
Through transcriptional activation, RNAa therapeutics promise a revolution in our ability to modulate previously undruggable targets.
RNA ACTIVATION
HITTING THE ON SWITCH
We are pioneering a new class of medicines called small activating RNA (RNAa) therapeutics. RNAa therapeutics share many features with other RNA therapies but work through an entirely distinct mechanism called RNA activation.
THE FULL POTENTIAL OF BIOLOGY
By working at the gene level, RNAa medicines are able to restore a cell’s own biology, such as powerful transcription factors, currently “undruggable” by conventional medicines.
NEW WAYS TO TREAT DISEASE
RNAa medicines enable entirely new approaches to treating disease. Our first RNAa medicine has been evaluated in over 130 patients and has achieved clinical Proof of Concept.
RNAa TECHNOLOGY & PLATFORM
Through transcriptional activation, RNAa therapeutics promise a revolution in our ability to modulate previously undruggable targets.
ENDOGENOUS ACTIVATION MECHANISM
RNAa therapeutics recruit endogenous transcriptional complexes to a target gene, leading to increased expression of naturally processed mRNA and upregulation of the target protein.
BIOINFORMATICS DISCOVERY ENGINE
MiNA has developed a powerful bioinformatics platform to design RNAa therapeutics for targeted gene activation. RNAa sequences recognize elements upstream of the mRNA transcription initiation site and downstream of the polyA site. Furthermore, the platform does not rely on specific knowledge of noncoding RNA complexity at the target gene.
TARGET FLEXIBILITY
RNAa therapeutics can upregulate intracellular or secreted proteins for therapeutic benefit and have been shown to increase protein levels for both naturally expressed and epigenetically silenced targets.
MECHANISM OF ACTION
1.
RNAa is loaded onto Argonaute proteins (Ago) in the cytosol and the active complex translocates into the nucleus.
2.
Inside the nucleus the Ago complex associates with either promoter elements of the target gene or natural antisense transcripts which serve as a docking site.
3.
By recruiting endogenous transcription complexes, including RNA Polymerase II (Pol-II), the Ago complex activates the gene’s transcription of messenger RNA (mRNA).
4.
The new mRNA is naturally processed into protein. MiNA has demonstrated the therapeutic potential of up-regulating several proteins in a diverse range of pre-clinical models.
RNAa THERAPEUTICS
RNAa therapeutics are small oligonucleotide drugs, similar in chemical structure to RNAi. MiNA has pioneered the pharmaceutical development of RNAa medicines including the use of medicinal chemistry and drug delivery technologies. The culmination of MiNA’s experience has resulted in accelerated pharmaceutical development cycles from target selection to IND comparable with mature oligonucleotide modalities.
INTELLECTUAL PROPERTY
MiNA has a dominant IP position in RNAa therapeutics and is building a comprehensive patent portfolio spanning compositions of matter and methods of use covering a large number of RNAa compounds. Our portfolio is built around inventions from our founder Pål Sætrom that have been assigned to MiNA by the Norwegian University of Science and Technology.
In addition, we have exclusively in-licensed fundamental patents from UT Southwestern Medical Center that broadly cover RNA activation therapeutics including issued European patent no. 2641970, “Modulation of gene expression by oligomers targeted to chromosomal DNA”.
MiNA collaborates with leaders in drug delivery and has exclusively in-licensed SMARTICLES® liposomal delivery technology for use in clinical candidate MTL-CEBPA.
