Through transcriptional activation, saRNA therapeutics promise a revolution in our ability to modulate previously undruggable targets.

Through transcriptional activation, saRNA 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 saRNAs (small activating RNAs). saRNAs 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, saRNAs 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

saRNA medicines enable entirely new approaches to treating severe diseases. Our first saRNA medicine is currently in clinical testing in patients with advanced liver cancer.

OUR saRNA TECHNOLOGY & PLATFORM

Through transcriptional activation, saRNA therapeutics promise a revolution in our ability to modulate previously undruggable targets.

ENDOGENOUS ACTIVATION MECHANISM

saRNAs 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 saRNA for targeted gene activation. saRNA 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

saRNAs 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.

saRNA 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.

saRNA THERAPEUTICS

saRNAs are small oligonucleotide drugs, similar in chemical structure to siRNAs. MiNA has pioneered the pharmaceutical development of saRNA 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 saRNA and is building a comprehensive patent portfolio spanning compositions of matter and methods of use covering a large number of saRNA 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 and UC San Francisco 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.

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