Programme : Presentations by Session
Short modified oligonucleotides such as ASOs are successfully used as gene-targeted drugs, developments that were made possible by chemistry advancements to improve in vivo stability and specificity, while reducing toxicity. There is currently a strong focus on improving productive uptake in physiologically relevant tissue, where the chemical matter is at interplay with the packaging of the cargo in novel formulations.
The available toolbox of chemical probes and drugs has recently expanded to include a plethora of approaches for achieving targeted protein degradation. The fundamental science driving this exciting new field is the generation of molecules capable of modulating or engineering new protein-protein interactions, which subsequently exploit the ubiquitin-proteasomal system or other protein degradation pathways. In this session we will explore technologies that are critical for in-depth studies of these interactions.
The future of life science research sits with our ability to fully embrace big data, which is increasingly used for better understanding treatment responses but also for better characterising cellular model systems. Here we will explore the future of compound-induced fingerprinting, where details on the model system become an integral part of this interpretation.
The concept of using chemistry to interrogate biology is fundamental to the chemical biology community. We are now going beyond this goal, with efforts to create new biology, for example by the introduction of non-naturally occurring building blocks. In this session we will explore this line of thinking and potential future applications.
The starting session will focus on the biological profiling of treatment response to oligonucleotides and the underlying characterisation of cellular model systems to achieve this in a rational manner. This includes the application of different techniques for understanding molecular mechanisms behind mRNA modulation and changes to their levels using e.g. RNA sequencing approaches to understand safety aspects. We will also explore novel developments in spatially resolved biology to understand the sequence of events from endosomal uptake, release and delivery to site of action.