Modifying Novel Oligonucleotide Chemistry for Improved PK/PD Effects
1:01 pm - Wednesday 27th January 2027
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
