Thursday, August 28
For the most up-to-date agenda, please download the full event guide
7:00 am Morning Check-In: Served with Coffee & Light Breakfast
7:55 am Chair’s Opening Remarks
DREAMS TO REALITY: ADVANCING ASO & SIRNA INNOVATION THROUGH CLINICAL DEVELOPMENT & BEYOND
8:00 am Reduced ALN-APP for Alzheimer’s Disease & Cerebral Amyloid Angiopathy
Synopsis
- Look at work upstream to reduce amyloid production
- Demonstrate target engagement with the first siRNA to be used in the brain
- Highlight preclinical findings from AD models and CAA, highlighting reduced cognitive decline in mice and reduced bleeding
- Delve into clinical data highlighting target engagement and plans for CAA
8:30 am A Landscape Review of Oligonucleotide Therapy Development for Neurological Disorders
Synopsis
- The drug landscape, displaying critical data-driven therapeutic and clinical trial highlights, as well as current trends
- A look at the developer landscape, allowing for understanding of the commercial market involving these drugs’ development
- The outlook for the future of oligonucleotide therapies for the CNS from a preclinical and clinical perspective
9:00 am Panel Discussion: Turning Setbacks into Success: Lessons Learned from Clinical Trial Failures to Strengthen CNS Oligonucleotide Development
Synopsis
- Explore common causes of clinical trial failures in CNS oligonucleotide programs, from delivery barriers to patient heterogeneity
- Share strategies for improving target validation, biomarker selection, and preclinical models to better predict clinical outcomes
- Discuss best practices for early safety assessment, managing off-target effects, and optimizing dosing strategies
- Examine how aligning trial design and development plans with regulatory expectations can de-risk clinical progression
- Highlight real-world examples of how insights from failed programs have shaped more resilient second-generation approaches
10:00 am Speed Networking
Synopsis
Connect face-to-face with C-suite executives, discovery biologists, oligonucleotide chemists, DMPK scientists, pharmacologists, CMC experts and platform tech specialists, working across diverse pipelines and indications, from pharma, biotech and academia, united in a common goal of unleashing the disease modifying potential of oligonucleotides in the CNS
10:30 am Morning Break & Refreshments
REFINING TfR1 & ANTIBODY OLIGONUCLEOTIDE CONJUGATES (AOCs) FOR MORE POTENT, SAFER & DURABLE BRAIN DELIVERY
11:00 am Optimizing Both Conjugate & Oligonucleotide Design for Greater Target Cell Affinity & Blood-Brain Barrier Permeability of siRNA Payload
Synopsis
- Focus on ligand optimization over high throughput screenings to improve the balance of affinity for target cell with permeability across the blood-brain barrier
- Modify PK/PD profiles to reduce peripheral tissue distribution by improving receptor specificity to the blood-brain barrier
- Explore alternate regions beyond the FAB region for Transferrin Receptor mediated transcytosis across the blood-brain barrier
- Discuss the impact of the blood-brain barrier on distribution to different brain regions as compared to intrathecal injection as illustrated through ISH imaging analysis
11:30 am Innovative Approaches for Systemic Delivery of Metabolically Stable siRNA to the Brain
Synopsis
- Discuss approaches for enhancing metabolic stability and targeted delivery of siRNA
- Examine conjugation strategies for generating stable antibody-oligonucleotide conjugates
- Explore preclinical and clinical data demonstrating success of oligonucleotides for CNS indications
- Share our recent data on systemic delivery of siRNA-conjugate to the brain
12:00 pm Lunch & Networking Break
1:00 pm TfR1-Mediated Delivery of Oligonucleotides to Address Neuromuscular Diseases: Translating Research into Clinical Functional Improvement
Synopsis
- The FORCE™ platform leverages Transferrin Receptor 1 (TfR1) to enable delivery of therapeutic oligonucleotides to muscle and the CNS
- Discuss preclinical data in models of DM1 and DMD to demonstrate that FORCE™ delivers broad functional improvement in muscle and CNS phenotypes
- The FORCE™ platform achieved POC in the ACHIEVE and DELIVER trials in DM1 and DMD, demonstrating translation of preclinical research into clinical functional improvement
DISCOVERY BIOLOGY, OMICS & FORWARD THINKING: TOOLKIT TO REFINE NEXT GENERATION OLIGONUCLEOTIDE STRATEGY IN THE CNS
1:30 pm Session Reserved for Luxna Biotech
2:00 pm Roundtable Discussion: Future Applications of Oligonucleotides in Neuroscience: What’s the Next Target Disease Area?
Synopsis
- Outline disease areas with recent progress and innovation, and opportunities for cross-learnings
- Compare multimodal oligonucleotides approaches (ASOs, siRNAs, and more), diverse conjugates mapped out against opportunities in different disease areas
- Assess the potential of oligonucleotides in neuropsychiatric and neurodevelopmental disorders. Can lessons from neurodegeneration be applied?
2:30 pm Scientific Poster Session
Synopsis
Revel in the truly unmatched innovation in the oligonucleotide R&D stratosphere, showcasing preclinical and clinical findings for multimodal approaches, across neurodegeneration, neuromuscular, epilepsies, neurogenetic indications and more. If you’re interested in showcasing your own research, approach or technology, get in touch at info@hansonwade.com
3:00 pm Afternoon Break & Refreshments
REFINING OLIGONUCLEOTIDE CHEMISTRY & FORMULATION FOR IMPROVED STABILITY, HALF LIFE, DURABILITY, BINDING AFFINITY & SCALABILITY
3:30 pm Biomimetic Chemistry of siRNA Delivery to CNS
Synopsis
- Synthetic small interfering RNAs (siRNAs) are potent inhibitors of gene expression. These molecules are perfect examples of biomimetic chemistry as synthetic siRNAs act through the natural RNA interference (RNAi) pathway
- To deliver therapeutic siRNAs for CNS applications, we have developed approaches that include extensive chemical modification of the siRNAs and lipidic nucleic acids (siRNA-lipid conjugates)
- This presentation will cover the chemical biology of RNA therapeutics including the chemical modifications employed in each RNA strand and novel motifs to ensure uptake into cells, recognition of these modifications and motifs by the slicer enzyme Argonaute-2 and its recognition for potency improvement, silencing efficiency, on-target specificity, safety, and metabolic stability due to nucleolytic degradation
4:00 pm Targeting Complement C3 via Intrathecal siRNA: A Novel Approach to Neuroinflammation in Neurodegeneration
Synopsis
- Early discovery and translational journey of Apellis’ siRNA program targeting complement C3 for neurodegenerative diseases
- Insights from siRNA screening and optimization to enable potent, durable knockdown of C3 in non-human primates (NHPs), setting the stage for clinical translation
- Exploring the potential of intrathecal complement inhibition as a first-line therapy and its synergistic use in combination with other disease modifying therapies