Explore the Agenda

7:30 am Check In, Coffee & Light Breakfast

8:25 am Chair’s Opening Remarks

Advancing CNS Oligonucleotide Delivery Through Viral Tropism, Deep Brain Penetration & Cell Specific Selectivity

8:30 am Targeted MAPT Silencing via Brain Shuttle–Enabled siRNA Delivery in Alzheimer’s Disease

Vice President, Research, Ossianix
  • Targeted MAPT Silencing via Brain Shuttle–Enabled siRNA Delivery in Alzheimer’s Disease
  • Optimising CNS delivery of therapeutic payloads using TXP1, a novel second‑generation, brain‑selective shuttle engineered for enhanced transcytosis across the BBB
  • Identifying a potent MAPT‑targeting oligonucleotide for tau silencing and optimising its site‑specific conjugation to TXP1 to enable efficient CNS delivery and robust knockdown
  • Demonstrated in vivo efficacy, with potent MAPT knockdown observed in a PoC study in transgenic mice following intravenous administration of TXP1‑MAPT

9:00 am Optimising AAV Delivery of Gene Supplement & Silencing Payloads to the Brain for Neurodegenerative Disorders

Chief Scientific Officer, AviadoBio
  • Adeno-associated viral vectors offer the potential of a one-time treatment targeting mutant genes or disease-critical pathways
  • Delivery of any genetic medicines to the brain must address multiple challenges. This presentation will address issues of target choice, cell-specific expression, entry across the blood/CSF-brain barriers and dose-related toxicity for AAV capsids

NEW DATA

9:30 am Roundtable Discussion: Into the Thick of It: Deep Brain Penetration & Cell-Specific Precision

  • Evaluate strategies to achieve robust oligonucleotide penetration into deep brain regions while maintaining controlled CNS distribution
  • Examine approaches to enhance neuron-specific uptake over glial populations, improving cell-type precision and therapeutic impact
  • Assess the limitations of current delivery systems, including transferrin receptor-mediated approaches, and discuss emerging shuttle technologies for deeper and more targeted brain access
  • Leverage imaging and PK/PD modelling to quantify regional and cell-specific exposure, enabling early differentiation between delivery efficiency and target biology
  • Address safety and translational considerations, balancing deep CNS exposure, cell selectivity, and risks of neurotoxicity and peripheral off-target effects

10:30 am Morning Break & Refreshments

Formulation Strategies to Maximise CNS Oligonucleotide Delivery & Cellular Target Engagement

11:00 am Engineering Targeted CNS Shuttles to Overcome Endosomal Escape Barriers in Oligonucleotide Therapeutics

Chief Development Officer, Sapreme Technologies
  • Exploring targeted strategies for precision delivery, focusing on how route of administration (local/systemic), intracellular trafficking, and endosomal sequestration constrain the translation of oligonucleotide exposure into functional potency
  • Preclinical in vivo proof-of-concept data establish a mechanistic framework linking oligonucleotide exposure, endosomal escape, and productive target engagement, independent of target organ or tissue 
  • Positioning delivery and endosomal escape as central bottlenecks, we discuss emerging mechanistic insights and translational considerations relevant to CNS-directed applications, emphasizing targeted approaches that improve on-target intracellular availability

NEW DATA

11:30 am Precision RNA Splicing Through Targeted Pseudo-Exon Activation: A Novel Platform Unlocking Broad Therapeutic Potential

Chief Development Officer, Inverna Therapeutics
  • A novel RNA splicing modulation platform that selectively activates pseudo-exons to precisely control gene and protein expression at the source of disease
  • Targeted oligonucleotides function as an innovative molecular switch, displacing repressive RNA-binding proteins to enable pseudo-exon inclusion during splicing, deliberately altering mRNA and downstream protein production
  • Powered by a proprietary map spanning over 90% of spliced protein-coding genes with pseudo-exons, the platform unlocks a vast therapeutic targeting space with broad applicability across many conditions

11:45 am Beyond the Sequence: Advancing siRNA Leads for CNS Delivery

Director, RNA Therapeutics & Translational Science, Aerska
  • Aerska is developing siRNAs for CNS indications
  • Aerska’s lead program is APP Program for the treatment of Alzheimer’s Disease
  • Aerska will be presenting their approach in siRNA lead development to identify a potent sequence and the delivery strategy to reach CNS

12:00 pm Lunch

Engineering Clinical Confidence In CNS Oligonucleotides: Delivery, Safety & Translation

1:00 pm From Preclinical Evidence to Early Clinical Studies: Advancing VO659 for Polyglutamine disorders

CSO, Vico Therapeutics
  • Development of VO659: outlining the path from candidate selection through IND-enabling activities and entry into Phase 1/2a studies
  • Broader implications of the VO659 program for advancing next-generation oligo therapies targeting CNS disorders
  • Beyond VO659: innovating ASO chemistry to improve CNS uptake

1:30 pm Roundtable Discussion: Device-Based CNS Delivery Strategies for Oligonucleotides

  • Evaluate whether implantable and intrathecal delivery offer clear advantages over systemic approaches for CNS targeting
  • Identify when device-based delivery outperforms systemic or ligand-mediated strategies
  • Debate whether implantation limits use to rare diseases and how innovation could broaden applicability
  • Examine key factors impacting distribution and reliability, including reservoir stability, catheter design, CSF flow, and dosing parameters
  • Assess trade-offs between device-enabled delivery of simpler oligos and more complex chemical or conjugation strategies

2:30 pm Afternoon Break & Refreshments

Accelerating CNS Oligonucleotide Therapeutics Through Novel Target Discovery & Genetic Validation

3:00 pm Discovering & Validating Novel CNS Targets for Oligonucleotide Therapeutics

Chief Scientific Officer, Harness Therapeutics
  • Strategies to identifying emerging genetic targets beyond the crowded IP space and evaluating their therapeutic potential with different oligonucleotide modalities
  • Overcoming preclinical translation challenges by leveraging humanized and disease-specific models to confirm target relevance, address species-specific phenotypes, and predict clinical efficacy
  • Integrating bioinformatics and AI-driven approaches to prioritise causal targets, enhance mechanistic validation, and accelerate the selection of disease-relevant candidates 

NEW DATA

3:30 pm Panel Discussion: Cracking the CNS’s Code: Finding the GalNAc of the Brain

Novel Technologies & Therapeutics Lead, Lundbeck
Vice President, Research, Ossianix
  • Explore the search for CNS-targeted oligonucleotide ligands and shuttle strategies that could match GalNAc’s efficiency in the liver
  • Evaluate emerging chemistry, conjugation, and receptor-mediated approaches, including methods to validate and optimize chemical modifications for CNS delivery
  • Discuss translational opportunities and challenges in creating next-generation CNS delivery platforms that enable differentiated therapeutic strategies

NEW DATA

4:00 pm Chair’s Closing Remarks

4:05 pm End of Conference