Day Two

• Explore the chemistry behind circularised antisense oligonucleotides and understand how these designs differ from conventional linear gapmer ASOs
• Evaluate whether circularisation can meaningfully improve stability, pharmacokinetics and therapeutic durability compared with existing antisense approaches
• Gain deeper technical insight into an emerging chemistry platform that has rapidly gained industry attention but remains relatively unexplored from a development perspective

• Introduce RNA 2’-OH modification as a versatile post-synthetic conjugation strategy for oligonucleotides that can go beyond what is possible with standard solid-phase oligonucleotide synthesis
• Describe the chemical classes that can drive these reactions, their advantages/disadvantages, and how they may be manufactured
• Applications of this technology to oligonucleotide stability enhancement, delivery, and structure analysis, and where future work may lead to effective drug development

• Explore the rationale and approach for targeting non-coding RNAs to upregulate gene expression for diverse therapeutic applications
• Discuss how the platform enables discovery of antisense oligonucleotides that can increase transcription across a range of therapeutic indications and target tissues.
• Provide case studies on the translation and development of candidate ASOs from preclinical research to the clinic

• Introducing a novel and highly potent ASO modification and the manufacturing considerations associated with its development
• Discussing how process parameters can be optimised to maintain product quality while enabling safe and robust manufacturing
• Exploring how teams can identify and address unexpected chemistry and process bottlenecks during development

Small activating RNA remains one of the most debated areas within RNA therapeutics, with supporters highlighting its potential for durable gene activation whilst critics question its maturity, scalability and clinical translation. This live hot seat will challenge assumptions around saRNA development, bringing opposing perspectives together to examine where science stands today and what needs to happen for broader adoption

• Watch a controversial and evolving technology be critically examined through a live cross-examination rather than a traditional presentation format
• Explore the key scientific and development questions around saRNA, including delivery, durability, specificity and clinical readiness
• Gain a balanced perspective on whether saRNA represents a future therapeutic opportunity or remains an area requiring further validation before significant investment

• Understand why certain tissues can accumulate high levels of siRNA and ASOs while showing limited or no measurable gene silencing activity
• Explore how to distinguish productive versus unproductive intracellular trafficking pathways and identify the factors that determine functional delivery
• Examine emerging approaches to rescue oligonucleotides trapped in non-productive pathways, including dual-receptor strategies and next-generation delivery innovations

• Poly-dxC (exNA-dC) self-assembles into iMotif structures (ex-iM) specifically under the acidic pH condition
• Poly-dxC-modified siRNA self-assembles within the cell in vitro, enhancing the kinetics of siRNA activity without inducing toxic endosomal membrane rupture
• Poly-dxC-modified siRNA improves gene-silencing kinetics in vivo, with substantially enhanced early-time-point silencing in mouse kidney

• Gain insights from a leading researcher in endosomal escape on why overcoming this barrier remains a central challenge for the next generation of oligonucleotide therapeutics
• Understand the mechanistic gap between cellular uptake and functional cytosolic delivery, including why many internalised oligonucleotides fail to reach their intended site of action
• Evaluate emerging endosomal escape strategies and distinguish approaches showing genuine promise from those that remain early-stage or speculative

Understanding what drives successful partnerships is critical for emerging oligonucleotide companies looking to secure investment, collaboration or acquisition opportunities. This candid panel will bring together pharma business development, external innovation and investment leaders to discuss what truly differentiates programs that attract strategic interest, from scientific validation and platform maturity to team capabilities and commercial potential.

• Hear directly from pharma BD and external innovation leaders on the scientific, technical and strategic signals that make an oligonucleotide programme stand out as a partnership opportunity
• Gain insight into how companies should position emerging platforms, technologies and assets to align with evolving pharma priorities and investment strategies
• Ask your questions directly and understand what decision-makers are evaluating when considering partnerships, collaborations and future opportunities