Day Two
8:00 am Morning Coffee & Check-in
8:55 am Chair’s Opening Remarks
9:00 am Panel Discussion: Overcoming Design Challenges in Next-Generation Oligonucleotides to Maximize Selectivity, Potency & Clinical Impact
Synopsis
- What design strategies are most effective in mitigating off-target effects?
- How do teams prioritize stability vs. delivery vs. potency?
- What are the emerging trends in oligonucleotide chemistry for next-generation therapeutics?
Emerging Modalities, Innovative Technologies Enabling Diverse Therapeutic Strategies for Enhanced Disease Targeting
10:00 am Aptamers: Precision Intracellular Modulators Offering Targeted Protein Control Beyond Gene Silencing
Synopsis
- Overview of aptamer structure and target binding capabilities inside cells
- Challenges in chemical stability and strategies to improve molecular retention in vivo
- Potential for success, sharing success stories in the therapeutic space, rather than just the diagnostic space
10:30 am Morning Break & Networking
11:30 am Cancer Immunotherapy Using RNA Aptamers for Cell-Selective Delivery or Intracellular Target Inhibition
Synopsis
- Expanding therapeutic horizons of aptamers beyond conventional targets on cell surface
- Leveraging innovative oligonucleotide chemistries to enable novel cancer treatment strategies
- Advancing conjugation technologies to enhance delivery and address complex myeloid cell biology
12:00 pm Maximizing Therapeutic Succes: Choosing the Right Oligonucleotide, Delivery Vehicle & Administration Route
Synopsis
- Shuttle peptide platform: Efficient endosomal escape driving the development of PMO-based therapeutics
- Development of a non-surgical therapeutic for basal cell carcinoma administered by intralesional injection
- Efficient delivery to airway epithelial cells, advancing inhaled PMO therapies for muco-obstructive lung diseases
12:30 pm Lunch & Networking
Innovating RNA Editing & Strengthening CMC Foundations to Enable the Next Wave of Oligonucleotide Therapies
1:30 pm From Chemical Design Optimization to Clinical Application: Advancing ADAR RNA Editing in Liver and CNS
Synopsis
- ADAR-mediated RNA editing as a versatile therapeutic approach to precisely correct disease-causing RNA mutations, modulate protein expression levels, or alter protein function to prevent or treat disease
- Optimization of editing oligonucleotides (EONs) through chemical modifications to enhance editing efficiency, specificity, and cellular uptake
- Translation of optimized EON strategies into therapeutically relevant applications addressing high unmet medical needs in the liver and CNS.
2:00 pm Navigating CMC Complexity: Overcoming Manufacturing & Cost Barriers to Advance Next-Generation Oligonucleotide Therapeutics
Synopsis
- Why multi-component designs present unique manufacturing and regulatory hurdles to understand the CMC burden of novel oligos
- New approaches to reduce cost and complexity: Including, enzymatic ligation, and emerging scalable manufacturing technologies
- Building global manufacturing readiness: Evaluating current capacity and capabilities to meet growing demand for large-scale, GMP-compliant oligonucleotide production