Explore the Agenda
8:00 am Registration & Coffee
9:00 am Workshop A
Understanding Target Biology & Finding Novel Targets for RNAi to Advance Specific Delivery
As RNAi developers push beyond the liver, identifying the right receptor is crucial in achieving efficient and selective extra-hepatic delivery. This workshop will explore emerging approaches for discovering and validating novel receptors across a range of target tissues and disease indications. Attendees will examine why receptor expression alone is often insufficient for successful targeting, considering factors such as receptor internalisation, trafficking pathways and tissue accessibility. The session will also compare receptor-mediated delivery strategies against non-targeted approaches, helping attendees understand how different delivery technologies can improve specificity, potency and clinical translation.
Join this workshop to:
- Dive into the discovery and validation process for new receptors for cell-specific delivery
- Understand why receptor expression alone is insufficient for successful targeting
- Compare receptor-mediated versus non-targeted delivery approaches
12:00 pm Lunch Break & Networking
1:00 pm Workshop B
Modifying Novel Oligonucleotide Chemistry for Improved PK/PD Effects
RNAi therapeutics are constantly expanding into increasingly challenging tissues and chronic disease indications, making optimising oligonucleotide chemistry essential for improving both therapeutic performance and commercial viability. This workshop will explore how novel chemical modifications can enhance stability, circulation time and tissue exposure, enabling more durable gene silencing and less frequent dosing. Attendees will examine emerging approaches designed to overcome key barriers such as RNA degradation, poor intracellular trafficking and limited endosomal escape, while balancing potency, safety and manufacturability. The session will also discuss
how advances in chemistry are helping developers extend the clinical lifespan of RNAi assets, reduce dose burden for patients, unlock applications beyond traditional liver-directed therapies and, with an off-target strategy lens, how these decisions are assessed and defended through the regulatory process.
Join this workshop to:
- Assess chemical modifications that enhance durability and circulation time
- Improve therapeutic performance while reducing dose burden
- Extend the commercial and clinical viability of RNAi assets
- Overcome challenges like endosomal escape or RNA degradation