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Trending Topics at Oligonucleotide Therapeutics Society (OTS) 2025
This year’s annual meeting in Budapest showcased recent advancements in the field of oligonucleotide therapeutics.
Oligonucleotide Therapeutics Society (OTS) brings together leading researchers and industry experts across various therapeutic areas, including oligonucleotide therapies such as antisense oligonucleotides (ASOs), siRNA therapies, aptamer technologies, mRNA therapies, and vaccines, as well as RNA/DNA editing. Research presentations, insightful discussions, and networking opportunities at OTS enable knowledge sharing and collaboration across the RNA therapeutic community.
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Here are a couple of trends that stood out for our RNA experts at the OTS Annual Meeting 2025.
The Route to Patients is Established
Over the past few years, several oligonucleotide therapeutics have received marketing approval, establishing this as an important drug class bring benefit to patients. Data presented at OTS 2025 reinforced this clinical success:
- Phase 1 data for a clinical study (ANQUR) testing QRL-201 (QurAlis) demonstrated a half-life in CSF of approximately 100 days in patients. QRL-201 is an investigational ASO that targets STMN2 mis-splicing to treat amyotrophic lateral sclerosis (ALS). The trial is currently ongoing in Canada, the United Kingdom, Ireland, Belgium, the Netherlands, and Germany.
- Zorevunersen (Stoke Therapeutics), an investigational ASO targeting SCN1A gene haploinsufficiency, also demonstrates disease-modifying effects and is well tolerated in patients with Dravet syndrome. The clinical data presented demonstrated a reduction in seizure activity and improvements in cognition, behavior, and quality of life, showing great promise for the development of disease-modifying drugs targeting neurological disorders.
- Outside of neuroscience, a self-replicating RNA vaccine developed for rabies by Replicate Bioscience was shown for the first time to have an equivalent or superior durability of immune responses to traditional vaccines.
- ALN-6400 (Alnylam Pharmaceuticals), an investigational small interfering RNA (siRNA) that reduces liver-derived plasminogen, was shown to have favorable results in Phase 1 clinical studies. This novel therapy could represent a universal treatment for various bleeding disorders.
Solving the Challenge of Precise Delivery Requires Diverse Solutions
The delivery of RNA therapeutics to their target cells and the intracellular uptake of these therapies have long been challenges for drug development companies. It’s common to conjugate ASOs and siRNA therapies to improve bioavailability and target specific cells and tissues. At OTS, several companies, including Ionis Pharmaceutics, Souffle Therapeutics, and Pepgen, presented novel solutions in the form of new conjugation ligands, including lipids, peptides, and antibodies. These will enable ASOs and siRNA as incredibly versatile molecules to better engage with their therapeutic targets, ultimately resulting in improved medicines for patients.
Novel Mechanisms of Action Expand the Versatility of Oligonucleotides
Beyond mRNA knockdown, RNA splice-switching or exon skipping, and DNA editing, multiple presentations highlighted novel molecular mechanisms being explored by several organizations in both preclinical and clinical research. This includes RNA editing (Wave Life Sciences and AIRNA Bio), aptamers (Robomic), and divalent siRNAs (UMass Chan Medical School). The expanded toolkit certainly opens the door for more therapeutic strategies utilizing oligonucleotides to benefit patients across a range of rare and more common disorders.