Wednesday, August 27
For the most up-to-date agenda, please download the full event guide
BIOLOGY TRACK
CHEMISTRY TRACK
Designed for CMOs, research VPs, neurobiologists, molecular biologists, drug discovery scientists, translational experts, preclinical development, RNA biologists, nonclinical safety and target discovery validation experts
Tailor-made for CSOs, chemistry VPs, medicinal and structural chemists, DMPK & PKPD scientists, oligonucleotide/RNA chemistry leads, drug formulation experts, nucleic acid and modality experts
8:30 am Morning Check-In: Served with Coffee & Light Breakfast
9:00 am Workshop A: Streamlining Preclinical Assessment from Dish, to Rat, to NHP for Improved Oligonucleotide Translation in the CNS
Synopsis
As oligonucleotide therapeutics advance in neurodegeneration and ophthalmology, optimizing in vitro and in vivo assays is critical to improving delivery, minimizing off-target effects, and ensuring translatability into clinical success. This workshop will explore best practices in assay development for RNA-based therapeutics, from high-throughput screening (HTS) strategies to NHP validation.
Join this session to:
- Refine in vitro assays to better demonstrate deep brain penetration to target cells in the human system
- Design relevant cellular models, use transcriptomics for off-target analysis, and ensure preclinical assays translate into clinical outcomes
- Compare what has worked for in vitro vs what will be used for in vivo
- Strategies for assessing unintended gene knockdowns, systemic distribution challenges, and immune response screening for off-target and toxicity profiling
- Lessons from recent oligonucleotide failures due to cytokine-driven inflammation and novel chemistries to mitigate immune activation
9:00 am Workshop B: Optimizing the Oligonucleotide Structure: Unravelling the Role of Linkage & Sugar Modifications in CNS Therapeutics
Synopsis
Given the pivotal role of linkages and sugars in the development of oligonucleotide developments for CNS, this workshop will explore the latest advancements, challenges and strategies to enhance the safety and efficacy of CNS-targeted siRNAs, ASOs and RNA aptamers.
Join this session to:
- Alter accessibility of 3’ and 5’ ends to create transient shapes improving delivery, specificity and potency of ASOs
- Adjust prime positioning in sugar’s phosphate back bone modifications, to protect from endonuclease degradation
- Understand the optimal applications for siRNA and ASO technology
- Perfect polarity and charge for optimized binding and bloodbrain barrier crossing
- Explore emerging trends in linkage and sugar ring modifications and future directions
- Evaluate the impact of sugar ring modifications on hybridization kinetics and binding affinity, for the oligonucleotide as crucial modifiers of target engagement in CNS tissue
12:00 pm Lunch Break & Networking
1:00 pm Workshop C: Harnessing ASO & siRNA Innovation for Cell Specific Delivery & Distribution
Synopsis
Oligonucleotide therapeutics offer a powerful approach to modulating gene expression in the CNS, but precise cell-type targeting and broad distribution, remain key challenges. This workshop will explore how different oligonucleotide modalities, siRNA, ASOs, and aptamers, can be optimized for CNS penetration, cell-specific uptake, and long-lasting efficacy.
Join this session to:
- Understand how oligonucleotide stability and degradation impact long-term CNS exposure
- Investigate how ligand and conjugate modifications influence selective uptake in neurons, astrocytes, and microglia
- Assess how IT injection strategies can be refined to ensure broader and deeper brain distribution
- Evaluate current limitations in predicting oligonucleotide biodistribution and improving in vivo screening strategies
1:00 pm Workshop D: Refining the Next Antibody- Oligonucleotide Conjugates (AOC) & Delivery Chemistries
Synopsis
Antibody-oligonucleotide conjugates (AOCs) represent a cutting-edge approach to improving targeted RNA therapeutics, offering enhanced precision in delivery, while overcoming key barriers such as biodistribution and cellular uptake. This workshop will focus on the latest advancements in AOC design, with a particular emphasis on brain shuttle strategies and delivery chemistry to optimize therapeutic outcomes for neurological diseases.
Join this session to:
- Leverage Transferrin Receptor mediated transport to enhance CNS penetration
- Ensure consistent Drug-Antibody Ratio (DAR) strategies to improve therapeutic efficacy
- Optimize scalable conjugation chemistry, following advances in linker chemistry, conjugate stability, and manufacturing scalability
- Translate insights from antibody-drug conjugates (ADCs) and other targeted delivery platforms to enhance AOC development
- Match disease areas with the most effective targeting strategies to ensure efficient drug uptake by diseased cells
- Enhance payload delivery and endosomal escape strategies to improve intracellular trafficking and prevent degradation before reaching the intended RNA target