Day One

• Discussing safety profiles, stability and translational outcomes across different oligo subtypes including ASO’s, siRNA, saRNA, aptamers and more
• Comparative safety and toxicity considerations across subtypes including off target effects, immunogenicity and long-term durability
• Industry perspective on room for innovation and strategies to stand out in a crowded therapeutic landscape
• What differentiates next-generation modalities and how biopharma can differentiate themselves in an increasingly competitive space

This informal session provides the perfect opportunity to connect with industry pioneers and key opinion leaders. Establish meaningful connections to build upon at the rest of the conference and gain exclusive first-hand insights into the latest research and developments driving progress in the space. 

• Development and validation of PBPK models for predicting in vivo CNS exposure, distribution, and cellular engagement
• Characterizing cell-type–specific targeting in the CNS through consideration of biological barriers, cellular diversity, and molecular design
• Bridging preclinical insights to clinical application and identifying gaps  

NEW DATA

• Interpreting CNS exposure and tissue distribution across species
• Translating preclinical PK into human dose and dosing frequency
• Balancing effective target engagement with safety margins 

• Discover mechanistic drivers of neurotoxicity by moving beyond simple correlations with sequence motifs and using advanced assays such as calcium oscillation and multielectrode array (MEA) systems to understand potential longterm CNS safety risks
• Evaluating these predictive neurotoxic models and advances necessary before the first human trials
• Explore safer oligonucleotide design strategies by identifying potentially neurotoxic sequences early, optimising backbone and end modifications, and prioritising chemistries before in vivo testing 

• Application of RNA-based editing technologies to achieve targeted modulation within the CNS, including brain distribution and editing characteristics distinct from knockdown approaches
• Translational preclinical evidence from disease models and non-human primate studies supporting progression toward clinical development
• Case study of a Rett syndrome program, highlighting the data package and strategic considerations that led to candidate nomination and IND-enabling plans

• Discover strategies to optimise oligonucleotide-peptide or oligonucleotide antibody conjugates for reproducible, scalable GMP production
• Balancing complex conjugate chemistry with manufacturing priorities: learnings from the successes and the failures
• Evaluate sustainable and cost-effective manufacturing approaches, including enzymatic synthesis and green chemistry alternatives, to support long-term clinical development

Want to share your work but not ready for the big stage just yet? The Scientific Poster Session is your prime time to share your work with peers from various backgrounds, all focused on oligonucleotides for CNS. Gain feedback on how you can accelerate the progression of your drug pipelines and build connections for potential collaborations to take your therapeutic from discovery to market.

• Exploring PF/PD profiling of Contera’s lead ASO in non-human primates, including both juvenile and adult cohorts, with longitudinal measurements over 28 weeks to characterise tissue distribution, target engagement, and systemic exposure
• Validating target engagement and compound selectivity using non-invasive proton spectroscopy biomarkers
• Discover how extended preclinical evaluation in non-human primates informs translational development for establishing benchmarks for dosing, pharmacodynamic responses, and optimisation strategies for clinical readiness 

NEW DATA

• Design dosing strategies around CNS distribution and target engagement, using high initial doses for deep regions and maintenance dosing to sustain effect
• Select administration routes based on risk-benefit, balancing invasiveness, BBB penetration, and systemic toxicity
• Integrate PK/PD modelling, molecular design, and delivery devices to maximise efficacy while maintaining safety for chronic CNS therapy