Pre-Conference Workshop Day
Pre-Conference Day
Tuesday 3rd December 2024
7.00 Check In, Coffee & Light Breakfast
8.00 Workshop A
Exploring Opportunities for Systemic Antisense Delivery with Receptor-Mediated BBB Shuttles
Synopsis
Due to their large size and virus-like nature eliciting immune defences, oligonucleotide therapeutics have been restricted in their ability to be delivered systemically and cross the blood-brain barrier, but what if they could be conjugated to brain-penetrating proteins and shuttled across as cargo?
Push the boundaries of ASO delivery beyond intrathecal injection and uncover new opportunities for the application of shuttle-mediated systemic delivery in this forward-thinking workshop covering:
- Emerging examples of existing shuttle platforms being applied to oligonucleotides to achieve efficacious delivery across the blood-brain barrier
- Considerations for the potential of peptide-based oligonucleotide conjugates leveraging blood-brain barrier receptors to internalize the oligonucleotides
- Next steps for successful translation of these animal and NHP studies
9.30 Morning Break & Refreshments
10.00 Workshop B
Unlocking the Potential of CED Infusion: A Gateway to Neurological Breakthroughs
Synopsis
Embark on a journey through the innovative landscape of convection-enhanced delivery (CED), where cutting-edge techniques meet clinical challenges head-on. This workshop offers a deep dive into the latest advancements in CED infusion and their transformative potential for neuro-oncology and neurodegenerative conditions.
Key Takeaways:
- Elevate Clinical Outcomes: Learn how strategic preclinical studies can serve as a cornerstone for achieving higher success rates in clinical trials
- Tailored Treatment Strategies: Discuss the integration of indication-specific CED infusion methods with the current standard of care, particularly in neuro-oncology
- Optimized Patient Selection: Contribute to the conversation on establishing robust criteria for selecting patients for chronic, intermittent infusion therapies in oncology
Hosted By:
12.00 Lunch & Network Break
1.00 Workshop C
Reviewing Recent Progress in Focused Ultrasound Technology & Broadening its Clinical Applications
Synopsis
Focused ultrasound is being increasingly explored to enhance therapeutic uptake, by temporarily increasing blood-brain barrier permeability, to treat a range of CNS disorders, beyond glioblastoma.
Explore possibilities for further applications of focused ultrasound-mediated drug delivery to the CNS and contemplate the evidence for:
- Safe and efficacious demonstrations of microbubble-based blood-brain barrier opening, with increased uptake of therapeutic material into the CNS during ultrasound pulses
- Restoration of blood-brain barrier flow following temporary opening
- Maintaining selectivity for the therapeutic while avoiding peripheral exposure through careful timing of blood-brain barrier opening
2.30 Afternoon Break & Refreshments
3.00 Workshop D
Unleashing the Potential of Non-Viral Delivery Vectors & Debating Their Optimal Route to the Brain
Synopsis
As lipid nanoparticles and liposomes attract growing interest as innovative vehicles for enhanced therapeutic delivery to the brain, their pharmacokinetic profiles and distribution within the CNS remain relatively unknown. Additionally, addressing their tolerability shortcomings will be crucial for optimizing these modalities for clinical application.
Furthermore, with systemic, local injection and nose-to-brain being explored as potential RoAs, which will prove optimal for eventual clinical success?
Discuss all this and more in this collaborative session covering:
- Optimizing nanoparticle design for specific blood-brain barrier penetration
- Ensuring effective brain tissue transfection of CSF-administered LNPs
- Exploring opportunities for nasal administration of nanoparticle delivery vectors