Day One

• Examining why extrahepatic delivery is the field’s defining challenge
• Focusing investment and research on the right bottlenecks
• Defining the milestones that will signal the arrival of truly routine extrahepatic RNAi delivery

• Repurposing transferrin receptor 1 (TfR1)-binding aptamers as targeting ligands for systemic siRNA delivery
• Demonstrating selective and durable siRNA activity in skeletal muscle following a single systemic dose
• Establishing aptamer–siRNA conjugates as a promising platform for next-generation targeted oligonucleotide therapeutics

New Data

• Defining cardiovascular risk among a population with Atherosclerotic Cardiovascular Disease
• Using bispecific strategies to target appropriate cardiovascular receptors and markers
• Developing new RNAi medicines to reduce global cardiovascular risk

• Understanding disease-modifying mechanisms of action
• Recognizing where target identification meets safety profiles
• GenKardia’s therapeutic position on treating heart failure

• Identifying and developing miRNA-based active ingredients for topical delivery
• Surmounting the obstacles presented by the antagonistic relationship between RNA molecules and human skin
• Designing a delivery vehicle for miRNA-based actives that both protects and effectively delivers the RNA payload to viable cells

• Harnessing the potential of miRNA modulation as a disease-modifying therapeutic strategy with broad biological impact.
• Developing delivery methods inspired by natural mechanisms to improve the targeted distribution of oligonucleotides to metabolically active extrahepatic tissues, including the CNS, muscle, and heart.
• Addressing translational and safety challenges to build confidence in clinical development programs based on miRNA modulation.

Connect with peers in a relaxed atmosphere and continue to forge new and existing relationships while exploring the latest in RNAi-based therapies and research advancements.

To submit a poster, please contact info@hansonwade.com

• Addressing whether investment/funding enthusiasm surrounding cell-type specific delivery is running ahead of the biology and where it is justified
• Considering whether to keep optimizing transferrin receptor shuttles which are heavily clinically validated or make the riskier bet on novel ligands to tune delivery
• Locating where the biggest opportunity for the delivery community to make progress in cell-type specific delivery is (RMT, nanoparticles, RNAi chemistry), and the gaps that need to be filled to get there faster

• Examine emerging metabolic pathways beyond GLP-1 approaches
• Explore advances in adipocyte-specific delivery technologies
• Enable transformative therapies for obesity and metabolic disease

• IFIH1/MDA5 is a genetically and mechanistically validated driver of Type 1 Diabetes, linking innate antiviral sensing to aberrant interferon signaling and autoimmune disease.
• Enabling cell-selective delivery and potent silencing of IFIH1/MDA5 using CD71- targeted Centyrin–siRNA conjugates, providing a strategy to modulate diseaserelevant innate immune signaling
• Demonstrating pharmacodynamic and disease-modifying activity in pre-clinical studies, supporting continued development of targeted RNAi as a potential therapeutic approach for Type 1 Diabetes

New Data