Our Science
Transforming Treatment Through Targeted mRNA Therapeutics
Our approach
Unlocking the Power of mRNA for Inflammatory Disorders
Delivering medicines to the correct cells is one of the foremost challenges in modern therapeutics. At Mercurna, we have developed a proprietary platform that enables targeted mRNA delivery, opening new treatment possibilities for diseases where conventional therapies fall short.
Our strategy integrates three key innovations:
Efficient delivery systems: Optimized for stability, efficient encapsulation of therapeutic mRNA, and reduced off-targeting
Cell-Specific targeting ligands: Innovative molecules that guide delivery vehicles to the cells driving inflammation and disease progression.
Therapeutic mRNA payloads: Designed to modulate immune responses or restore protein function in immune-mediated disorders.
By combining these elements, our platform overcomes systemic delivery barriers, minimizes off-target effects, and fully harnesses the transformative potential of mRNA medicines for patients with immune-mediated diseases.
Platform Technology
mRNA therapeutics
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A single mRNA molecule, as the short-living copy of a single gene, can generate thousands of therapeutic protein molecules at the site of action – combining both power and precision.
Our focus is on targets such as cytokines, transcription factors, and bioactive peptides. These typicaly have shorter mRNA sequences, which offer distinct advantages: compatibility with co-formulation, streamlined chemical synthesis, and enhanced delivery efficiency.
Delivery
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Efficient delivery systems are essential to unlock the full potential of mRNA therapeutics. Lipid nanoparticles (LNPs) have become the gold standard for delivery. LNPs shield mRNA from enzymatic degradation, facilitate cellular uptake via endocytosis, and promote endosomal escape to ensure translation in the cytoplasm. Yet, systemic administration often leads to non-specific uptake by organs like the liver and spleen. At Mercurna, we developed a formulation with reduced aspecific uptake and make use of a modular architecture that allows integration of targeting ligands.
Targeting
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Targeted delivery is critical for directing therapies to the intended cell types and tissues. Precise delivery is critical to ensure therapies reach the intended cells and tissues, increasing therapeutic efficiency and reducing side effects.
Our lead formulation features a proprietary kidney-targeting that binds with high affinity to a receptor located in the glomerulus. By decorating our LNPs with this peptide, we can enable delivery to kidney cells, thereby minimizing off-target effects commonly associated with current treatments.
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Relevant publications
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Martinez-Balsalobre et al (2023) Beyond current treatment of Fanconi Anemia: What do advances in cell and gene-based approaches offer?
Blood Rev. https://doi.org/10.1016/j.blre.2023.101094
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Bondue et al (2023) Evaluation of the efficacy of cystinosin supplementation through CTNS mRNA delivery in experimental models for cystinosis.
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Van Asbeck et al (2021) Protein expression correlates linearly with mRNA dose over up to five orders of magnitude in vitro and vivo.
Biomedicines. https://doi.org/10.3390/biomedicines9050511
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Van Asbeck et al. (2020) Kidney-targeted therapies: A quantitative perspective. J. Control