Explore the Agenda
BREAKFAST BRIEFING
8:00 am 101-Oligos for CNS
This session is designed to equip experienced drug developers and multidisciplinary teams with the technical knowledge required to enter the CNS oligonucleotide space. Whether you have recently transitioned from peptides or small molecules, are exploring oligonucleotides for CNS targets, or want your team to have a shared understanding, this session provides a practical foundation. Attendees will gain a clear understanding of oligonucleotide chemistry, pharmacokinetics, cellular uptake, and CNS-specific challenges. The session will cover key considerations in target selection, delivery strategies, translational modelling, and biomarker integration, providing a practical framework to evaluate and advance CNS oligonucleotide programmes.
WORKSHOP A
9:30 am The Oligo Conjugate Code: Engineering, Chemistry, Cargo & Clinical Translation of Oligos for CNS
This workshop will focus on the practical design and optimisation of oligonucleotide conjugates, exploring what works, what doesn’t, and the innovative strategies currently being applied in the field. Examine multiple targeting modalities including carbohydrates, lipids, peptides, and receptor antibodies, and learn how the choice of linker chemistry influences tissue-specific delivery, efficacy, and safety. The session will highlight real-world lessons from trial-and-error approaches, literature benchmarks, and emerging AI/ML tools, equipping attendees to design conjugates that are clinically feasible and optimised for CNS targeting.
Highlights Include:
- Which targeting modalities are prioritised for CNS, and why?
- How conjugation chemistry affects oligonucleotide stability, efficacy, and delivery; and how this is being validated
- What mechanistic lessons can be learned from failed versus successful conjugates
- How linker-cargo design can maximise therapeutic index and minimise off-target effects
- Where AI/ML can guide conjugate selection versus where empirical validation is required
- How to balance chemical feasibility, manufacturability, and safety for clinical-grade conjugates
12:30 pm Lunch
WORKSHOP B
1:30 pm Exploring Second Generation Blood Brain Barrier Shuttles
Participants will examine innovative strategies to improve CNS penetration beyond transferrin receptor approaches, optimise molecular functionalisation for active transport, and leverage predictive models and organoid technologies to accelerate development while minimising systemic exposure and toxicity. The session will highlight practical insights for designing shuttles that balance deep brain delivery, regional specificity, and safety as the field prepares for clinical translation.
Highlights Include:
- Explore second-generation BBB shuttles to improve CNS penetration and regional specificity
- Design oligo conjugates for active transport without compromising activity
- Maximise brain delivery while minimising systemic exposure and off-target effects
- Use organoids, lab-on-chip, and in silico models to accelerate development
- Efforts to apply AI and machine learning to streamline shuttle design and reduce experimental trial-and-error
- Combine chemistry, dosing, and delivery strategies to achieve safe and effective CNS therapeutics