How can we target emerging oligonucleotide therapies for success?

[Oleksandr Koniev, CC BY-SA 4.0 via Wikimedia Commons]

The potential of oligonucleotide therapeutics is vast, as illustrated by the growing number of drugs coming to market and the increase in noise and interest in this field. Oligonucleotide therapeutics hold promise to silence aberrant expression, modulate gene splicing or activate the expression of previously undruggable targets across a range of diseases. As the field has progressed, increased understanding has allowed researchers to overcome significant challenges in therapeutic function, from half-life control and extension to target specificity. Despite these advances, the primary challenge remains of targeted delivery to a specific cell type or tissue.

Encapsulation to direct conjugation: A new dawn in targeted delivery

Many have focused delivery around viral vectors and lipid nanoparticles (LNP) to encapsulate the oligonucleotide and protect it from deg…

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