Oligonucleotide Discovery, Preclinical & Clinical: From Design to Translational Success
As oligonucleotide modalities expand beyond traditional indications, discovery and translational strategies are becoming the key differentiators in clinical success. This track delves into cutting‑edge design, AI‑enhanced discovery, preclinical optimization, and clinical validation for next‑generation oligonucleotide therapeutics. Is your discovery pipeline prepared for tomorrow’s challenges?
What are the key topics for this track?
Precision Oligonucleotide Design & Conditional Activation
Next-generation oligonucleotide therapies are moving beyond simple knockdown toward cell-specific, conditionally activated, and highly selective mechanisms of action. New molecular designs are enabling therapies to remain inert until they reach the intended cell type, dramatically improving safety and expanding the range of druggable targets.
Why this matters now:
As oligonucleotides move into more complex indications, precision at the cellular level is becoming essential to avoid off-target effects and unlock previously intractable biology.
Key Session to Attend:
Conditionally Activated siRNA (CASi): Precision RNA Therapeutics through Cell-specific Activation - Discover how sensor-driven siRNA designs enable cell-specific activation and safer RNAi therapies.
RNA Editing & Transcriptome Rewriting Platforms
RNA editing is emerging as a powerful alternative to permanent gene editing, enabling reversible, programmable, and highly specific modulation of RNA and protein expression. Platforms that recruit endogenous ADAR enzymes are now entering the clinic, opening new therapeutic avenues beyond mutation correction.
Why this matters now:
Clinical validation of RNA editing platforms is accelerating, with liver, CNS, and rare disease programs demonstrating that transcriptome engineering is becoming a viable therapeutic modality.
Key Session to Attend:
Advancing ADAR RNA Editing Technology for Broad Applicability in Precision Medicines - Learn how editing oligonucleotides is being translated into clinical programs with expanding therapeutic scope.
Targeted Delivery & Tissue-Specific Oligonucleotide Therapeutics
Delivery remains one of the most critical barriers to oligonucleotide success. New conjugation strategies, receptor-mediated delivery platforms, and tissue-specific targeting approaches are enabling precise biodistribution to CNS, muscle, lung, liver, and tumors.
Why this matters now:
As oligonucleotide pipelines expand beyond liver-centric delivery, targeting new tissues is essential for unlocking broader clinical impact and treating previously inaccessible diseases.
Key Session to Attend:
FORCE Platform to Enable Delivery of Oligonucleotides for Neuromuscular Diseases - Explore how modular delivery platforms are enabling muscle and CNS targeting with diverse oligonucleotide payloads.
Translational, Clinical & Safety Insights for Oligonucleotide Therapies
As more oligonucleotides enter the clinic, clinical pharmacology, safety profiling, and dosing strategy are becoming key differentiators for success. Understanding tissue half-life, neurotoxicity mechanisms, and early clinical signals is critical to de-risk development and accelerate approval.
Why this matters now:
With multiple oligonucleotide modalities advancing into late-stage trials, translational data is now guiding smarter trial design, dosing strategies, and regulatory engagement.
Key Session to Attend:
Utilization of Oligonucleotides’ Half-Life to Optimize Dosing Regimen - Gain insights into how tissue persistence influences efficacy, safety, and clinical study design.
Who's Speaking on the Oligonucleotide Discovery Track?
FAQs
What does the oligonucleotide discovery and preclinical stage involve?
The discovery and preclinical stage focuses on oligonucleotide design, target validation, delivery strategy, and early safety assessment before entering clinical trials.
This includes chemistry optimisation, in vitro and in vivo testing, tissue targeting, and translational modelling to ensure candidate readiness for human studies.
Why is targeted delivery such a major focus for oligonucleotide therapies?
Targeted delivery determines which tissues receive the therapeutic payload and directly impacts efficacy and safety.
New delivery platforms are expanding oligonucleotide therapies beyond the liver to CNS, muscle, lung, and tumors, unlocking new indications and patient populations.
How is RNA editing different from gene editing?
RNA editing modifies RNA transcripts without altering DNA, making it reversible and potentially safer than permanent gene editing approaches.
ADAR-based editing platforms allow precise single-base changes, enabling disease correction, modulation, or protein reprogramming at the RNA level.
What are the main safety challenges for oligonucleotides in the clinic?
Key challenges include off-target effects, tissue accumulation, neurotoxicity, and long tissue half-life that complicates dosing strategies.
Understanding cellular responses and pharmacokinetics early is critical for safe clinical translation.
Why are CNS and neuromuscular diseases a major focus for oligonucleotide development?
Oligonucleotides can precisely target RNA in tissues where small molecules and biologics often fail.
Advances in delivery, conjugation, and intrathecal administration have made CNS and neuromuscular diseases among the most promising areas for oligonucleotide innovation.
What trends will define oligonucleotide discovery over the next five years?
Key trends in oligonucleotide discovery, preclinical and clinical development include precision activation, RNA editing, advanced delivery technologies, and stronger translational links between preclinical and clinical outcomes.
This track specifically highlights:
Precision and conditionally activated oligonucleotide designs that enable cell-specific activity and improved safety profiles.
RNA editing platforms are entering late-stage and clinical development, moving beyond mutation correction toward broader therapeutic modulation.
Tissue-specific delivery beyond the liver, including CNS, muscle, lung, heart, and oncology applications.
Improved translational models that better predict clinical efficacy, safety, and dosing from preclinical data.
The expansion into oncology, CNS, and rare genetic diseases, which demonstrates the growing versatility of oligonucleotide therapeutics.
Attendees will gain practical insight into how leading biotech and pharmaceutical teams are translating cutting-edge oligonucleotide platforms into real clinical candidates, and what this means for the next generation of RNA medicines.