Science Digest: The Future of Science is Here

And we're already on it.

At Karchem Consulting, we're always on the pulse of innovation. Team KC’s Laboratory Informatics Consultants Haley Gallagher and Helen Flynn cover the latest on RNA Therapeutics.

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Here's what Team KC consultant's Haley Gallagher and Helen Flynn had to say about the latest challenges in RNA Therapeutics:

RNA therapeutics have immense potential, and have already reshaped vaccine development. However, they are still in their early stages and come with challenges that need to be addressed for widespread success in transforming treatment from cancer to genetic disorders. Let’s explore three key hurdles: stability, targeting, and modulation.

1. Stability: Ensuring RNA Stays Functional

RNA molecules are naturally unstable, which makes them difficult to utilize in therapeutic applications. More traditional modalities such as lipid nanoparticles (LNPs) have been widely used in mRNA vaccines, their use for broader RNA therapies is still an area of active research, with ongoing efforts to improve their efficiency and stability. A recent review on LNPs for RNA delivery outlines both the progress and formulation challenges that remain in improving in vivo performance.

Alternatively, emerging RNA modifications, such as pseudouridine can reduce immune recognition and enhance translational efficiency. Showing promise in increasing the stability of RNA, particularly transfer RNA (tRNA) by regulating splicing and stabilizing folded RNA structures.

2. Targeting: Precision is Key

RNA-based therapies require precise targeting to avoid off-target effects and increase therapeutic efficacy. Dipeptide-antisense oligonucleotide (ASO) conjugates are emerging as a promising strategy to enhance specificity by delivering RNA therapeutics directly to cytoplasmic targets. Another exciting area of research is self-amplifying RNA (saRNA) technology, which can replicate within the body to provide a sustained immune response. Furthermore, extended nucleic acid backbones are being engineered for small interfering RNA (siRNA) therapies to protect RNA from degradation.

3. Flexibility: RNA Modulation

To reach their full potential, RNA therapies must effectively modulate gene expression. ADAR (Adenosine Deaminase Acting on RNA) is an enzyme that modifies RNA by converting specific adenosine (A) bases into inosine (I), which cells interpret as guanosine (G). This technology, combined with a target guide RNA sequence, allows for the correction of disease-causing mutations in the genome by targeting highly specific editing sites within an mRNA transcript. Other technologies, such as epigenetic editors utilizing CHARM (Coupled Histone tail for Autoinhibition Release of Methyltransferase) can modulate gene expression via methylation or demethylation transiently causing phenotypic changes without permanently altering DNA. 

These emerging RNA technologies hold immense promise, but they also introduce new complexities in laboratory data management, analysis, and integration. From tracking nuanced modifications like pseudouridylation to managing complex delivery constructs, researchers face more pressure to ensure data is analysis-ready.

As the industry continues to evolve, the need for robust informatics solutions has never been greater. Whether you are exploring the potential of pseudouridylation, circRNA, or other RNA-based innovations, ensuring the integrity and scalability of your data is key to unlocking new breakthroughs.

Our expertise in implementing software solutions like ELNs, LIMS, and SDMS can help your team navigate the complex data management needs of these advanced RNA research fields. By digitizing lab data, we empower your organization to unlock discoveries and accelerate progress. Let's work together to bring your cutting-edge research to life—contact us today