Novel Oligonucleotide Therapeutics and Their Delivery

Dear Colleagues,

New oligonucleotide therapeutics are becoming increasingly important. Recent approval of parenteral nanoparticle oligonucleotide formulations such as the Pfizer and Moderna COVID RNA vaccines, siRNA therapies such as, givosiran, patisiran, and aptamer therapies like pegaptanib highlight the importance and the increasing success of oligonucleotide delivery. While the promise of antisense oligonucleotide therapies have been anticipated since the late 90s, new oligonucleotide therapies are being produced, including those that induce exon skipping, novel aptamers, and the potential for delivery of gRNA for CRISPR gene editing. All of these promising oligonucleotide technologies converge on an important requirement: effective drug delivery methods that can get the oligonucleotide drugs into cells and target the drug to the right tissues. Nanoparticle delivery technology has advanced to meet some of these challenges and newer technologies are being developed that have extended half-lives, and target nanoparticles to particular tissues using “smart” materials, and chemical conjugation. This Special Issue covers the latest information on novel oligonucleotide therapeutics and their delivery. We are seeking original contributions and review articles in these areas.

Dr. Ted Lakowski
Dr. Alejandro Martínez-Åguila
Guest Editors

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