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Choice of mRNA
Build to last
A single mRNA molecule, as the short-living copy of a single gene, can produce up to a few thousand therapeutic protein molecules on-site, making it very powerful and yet precise. The predictable behaviour and tunability allows for truly personalized medicine. And the relatively short lifetime of mRNA makes it much safer and more controllable than DNA, although this also poses a challenge.
Therefore, our mRNAs are modified in unique ways to optimize their stability and lifetime, and thereby maximize the therapeutic potential of each correctly delivered mRNA molecule.
We focus on target such as cytokines, transcription factors, bioactive peptides. These mostly have shorter mRNAs with associated benefits including possibilities for co-formulation, chemical production, and easier delivery.
Lipid nanoparticles (LNPs) are increasingly gaining attention for the delivery of mRNA. They are composed of various lipids that build the basic particle structure, encapsulate the negatively charged mRNA via complexation, assist membrane fusion and aid endosomal escape.
At Mercurna, we are working with a proprietary LNP platform to optimize LNP formulation(s) with high efficiency mRNA release into the cytosol of desired cells. Consequently, we can tune expression of the therapeutic protein(s) of choice.
A precision medicine
Targeted delivery of mRNA offers enormous benefits for reaching the desired cell types and tissues, as unspecific uptake into undesired cells and organs (mainly liver and spleen) is difficult to overcome with systemic administration of mRNA-lipid nanoparticle formulations.
Our lead formulation consists of a unique targeting peptide that is derived from an endogenous protein present in our blood. This binds with high affinity to a specific (for IP-reasons undisclosed) receptor present in the glomerulus of the kidney.
By composing the outside of our lipid nanoparticles with this peptide, we can target drug delivery to the kidney, rather than any other organ, and as a consequence avoid many of the side-effects common in the current treatments.