Track Title: Oligonucleotide Discovery, Preclinical and Clinical
Advancing RNA Therapeutics from Bench to Bedside
Explore cutting-edge developments in oligonucleotide therapeutics, from innovative chemistry and design principles to targeted delivery systems and clinical breakthroughs. This track brings together leading researchers and industry experts to showcase the latest advances in antisense oligonucleotides, siRNA, mRNA, circular RNA, and emerging modalities.
About This Track
The oligonucleotide therapeutics field is experiencing unprecedented growth, with RNA-based medicines transforming the treatment landscape for genetic diseases, rare disorders, and complex conditions. This comprehensive track spans the entire development continuum—from discovery chemistry and preclinical optimization to innovative delivery systems and clinical translation.
Cutting-Edge Chemistry
Learn about novel chemical modifications that improve stability, specificity, and efficacy.
Breakthrough Modalities
Explore mRNA vaccines, circular RNA, base editing, and protein upregulation platforms
Precision Delivery
Discover innovative conjugates, nanoparticles, and peptide-based systems for targeted therapy
Clinical Translation
Hear real-world data from ongoing trials in rare diseases, CNS disorders, and genetic conditions
Regulatory Insights
Gain perspectives from the EMA on the development and approval of RNA therapeutics
Scalable Manufacturing
Address cost and production challenges for personalized N=1 therapeutics
Advancing Oligonucleotide Therapeutics: Key Challenges & Solutions
Challenge 1: Chemical Optimization & Stability
Master advanced chemical modifications and design principles that enhance nuclease resistance, target specificity, and therapeutic efficacy across diverse oligonucleotide modalities
Challenge 2: Targeted Delivery
Overcome biodistribution barriers with innovative conjugation strategies, nanoparticle platforms, and organ-specific delivery systems that enable precise tissue targeting beyond the liver
Challenge 3: Emerging Modalities
Explore next-generation platforms including circular RNA, base editing, mRNA therapeutics, and protein upregulation technologies that expand the therapeutic potential of nucleic acid medicines
Challenge 4: Clinical Translation & Scalability
Address manufacturing bottlenecks, regulatory requirements, and cost barriers to accelerate programs from preclinical development through clinical trials and commercial deployment
Track Focus Areas
Oligonucleotide Chemistry & Design
Discover the latest chemical innovations and design strategies that enhance the therapeutic potential of oligonucleotides.
Sessions:
Oligonucleotide Chemistry and Discovery to Clinic
Chemistry of Antisense Oligonucleotides for the Correction of Unproductive Splicing
Design and Chemical Optimization of Activating Oligonucleotides for Therapeutic HbF Induction in Sickle Cell Disease
Applying Small Molecule Drug Design Principles to Improve the Drug-like Properties of siRNA
Elongating siRNA Duration of Action by Enhanced Nuclease Resistance: End Stabilization Revisited
Emerging RNA & Oligonucleotide Modalities
Explore next-generation platforms and novel therapeutic modalities that are expanding the boundaries of RNA medicine.
Sessions:
Emerging RNA and Oligonucleotide Modalities
smartRNA: A First-in-Class Scalable RNA Therapeutics Platform for Protein Upregulation
Next-generation mRNA Vaccines and Therapeutics
EMA Perspectives on mRNA Vaccines and Therapeutics
Precision RNA Splicing through Targeted Pseudo-exon Activation: A Novel Platform Unlocking Broad Therapeutic Potential
Novel Circular RNA Therapeutics for Infectious and Non-infectious Diseases
Targeted Delivery Systems
Learn about innovative delivery technologies that enable tissue-specific and cell-specific targeting of oligonucleotide therapeutics.
Sessions:
Targeted Delivery of Oligonucleotides
Organ Specific Delivery of Oligonucleotides Using Nanofitins
siRNA Conjugate Mediated Delivery to Disease Specific Cell Types Occurs through a Novel Mechanism
Peptide-Targeted Delivery of Oligonucleotide Therapeutics
PEP-NP: Peptide Based Nanoparticle-platform for Targeted Delivery of Functional RNAs
Brain Biodistribution and Neuronal Gene Silencing of Three Intranasally Delivered siRNA Constructs in Rhesus Monkeys
xPhore: A Platform for Delivering Therapeutic Nucleic Acids
Analytical Methods & Quality Control
Discover advanced analytical techniques and quality control strategies for comprehensive characterization of oligonucleotide therapeutics and mRNA products.
Sessions:
Delivery, Analytical and CMC Strategies for Oligonucleotides and mRNA
Neutral Backbones and Dendrons for Enhanced Therapeutic Oligonucleotides Delivery
The Bayer LNP Platform for the Delivery of Therapeutic RNAs
Maintaining Diastereomeric Control in Phosphorothioate siRNAs: Integrated Analytical and Manufacturing Approaches for Reliable Clinical Development
Addressing Challenges in the Development and Quality Control of Individualized mRNA Products
Synthesis and Characterization of Stereopure Chimeric Oligonucleotides
LC-MS Innovation for Impurity Profiling of Oligonucleotide Therapeutics: Ion-Pair Hydrophilic Interaction Chromatography (HILIC) and FOCUS Two-Dimensional LC (2DLC) MS Methods
Preclinical & Clinical Development
Gain insights into the translation of oligonucleotide therapeutics from preclinical studies to clinical trials and regulatory approval.
Sessions:
- AIR-001: A Highly Potent and Precise GalNAc-conjugated RNA Base Editing Treatment for Alpha-1 Antitrypsin Deficiency
- AIC468, A First-in-class Antisense Oligonucleotide Targeting BKV in Kidney Transplant Patients
- Oligonucleotide Preclinical and Clinical Updates
- FiCAT: Precise Gene Writing System for Next-generation CAR-T Cell Therapy Production
- Targeting miRNAs for Disease Modification in Multifactorial CNS Disorders
- Genetically Validated Targets and the Next Wave of RNAi Therapeutics: Progress from the Alnylam Pipeline
- Beyond Silencing: ASOs as Protein Upregulators
- Interim Pharmacokinetic and Pharmacodynamic Results of the Phase 1/2a AVANCE1 Trial on Tricyclo-DNA-Induced DMD Exon 51 Skipping in Duchenne Muscular Dystrophy
- Overcoming the Cost Bottleneck in N=1 Therapeutics: A Highly Affordable, GMP-Compliant CMC Platform for Rapid Clinical Deployment
Who Will Benefit from This Track?
- Medicinal chemists
- RNA biologists
- Drug delivery scientists
- Preclinical and translational researchers
- Clinical development professionals
- Regulatory affairs specialists
- Pharmaceutical and biotech executives
- Academic researchers in nucleic acid therapeutics
- Investors and business development professionals