Day One

Exploring how priorities have shifted across the ecosystem, from proving modality feasibility to solving delivery, scalability and commercialization challenges, and what this means for the next generation of RNA therapeutics.

• Gain insight into the evolving innovation landscape, including how changing investment priorities, strategic collaborations and platform consolidation are influencing the future direction of oligonucleotide therapeutics and partnership opportunities
• Delving into the scientific, manufacturing and strategic considerations that determine whether innovative delivery technologies successfully translate from platform concepts into approved medicines

• Gain first-hand insights into the scientific and translational advances that are redefining blood-brain barrier delivery and creating new opportunities for oligonucleotide therapeutics beyond the liver
• Explore where meaningful progress has been made in extrahepatic and CNS delivery over the past two years, distinguishing genuine breakthroughs from the challenges that continue to limit clinical translation
• Set the strategic context for the summit by understanding the opportunities, bottlenecks and innovation priorities that will shape the future of oligonucleotide delivery

As this community unites, this session will provide valuable networking time with your peers,

enabling you to forge new and lasting connections.

• Analyzing engineering principles underpinning successful extrahepatic oligonucleotide delivery and how different approaches overcome tissue-specific barriers
• Drawing practical lessons from recent publications to identify common themes and emerging best practices
• Compare the strengths and limitations of conjugated and non-conjugated approaches to identify which strategy is best suited to different disease indications and development goals

• Combining a SNP-specific siRNA with an inflammatory target to tackle both the underlying genetic driver and disease progression in fibrodysplasia ossificans progressiva
• Developing multimeric oligonucleotide architectures to simultaneously address multiple disease-driving pathways
• Demonstrating therapeutic potential through both preventative and therapeutic models, including local and systemic delivery

• Repurposing transferrin receptor 1 (TfR1)-binding aptamers as targeting ligands for systemic siRNA delivery
• Achieves selective and durable siRNA activity in skeletal muscle following a single systemic dose
• Establishes aptamer–siRNA conjugates as a promising platform for next-generation targeted oligonucleotide therapeutics

An interactive panel discussion bringing together experts developing next-generation delivery technologies to debate the future of transferrin receptor targeting, emerging alternatives and the scientific challenges that remain in achieving effective delivery

• Compare the strengths and limitations of IgG, Fab, VHH and bispecific transferrin receptor targeting strategies to understand where each approach is demonstrating the greatest translational potential
• Debate whether continued optimisation of transferrin receptor biology is enough or if the field must identify new receptors and targeting mechanisms to unlock the next generation of oligonucleotide-based therapeutics
• Put your questions directly to the panel and gain practical insights into the opportunities, remaining barriers and future direction of transferrin receptor delivery

• Explore how target-agnostic ligand discovery platforms, including mRNA display and DNA-encoded libraries, are uncovering previously unexplored receptor targets for selective oligonucleotide delivery
• Understand how AI and machine learning approaches applied to real screening datasets are being used to optimise ligand properties, improve binding characteristics and enhance delivery performance
• Gain practical insights into how emerging ligand discovery technologies can be integrated into your own oligonucleotide development programmes to expand targeting opportunities beyond current approaches

• Showcase the application of AI-designed miniproteins for siRNA delivery to muscle, including evidence of functional target knockdown
• Examine the key challenges in translating receptor binding and cellular uptake into effective intracellular delivery and pharmacology
• Consider how targeted miniprotein delivery could expand the reach of oligonucleotide therapeutics to tissues and cell types that remain difficult to access

This is your opportunity to contribute to the conversation and share your cutting-edge research with this community while discovering exciting work carried out by your peers. To submit a poster, please contact info@hansonwade.com.

The next generation of oligonucleotide therapeutics will require more than incremental improvements to existing delivery approaches. This interactive innovation lab will bring together researchers to explore emerging receptor targets, linker design strategies and the critical chemistry challenges that influence delivery performance, stability and therapeutic potential

• Bring your own receptor targeting or linker chemistry challenges to the discussion and gain practical insights from peers working through similar delivery bottlenecks
• Explore how linker design impacts conjugate stability, tissue distribution, cellular uptake and intracellular release
• Discuss emerging opportunities in novel receptor targeting and how underexplored delivery mechanisms could expand the reach of oligonucleotide therapeutics
• Leave with new perspectives, potential solutions and valuable connections to advance your own delivery programs