Conference Day Two

8:00 am
Morning Networking Coffee

8:50 am Chair’s Opening Remarks

  • Uli Stilz VP, Novo Nordisk Bio Innovation Hub

Elevating Strategies to Increase siRNA Biodistribution & Improve Intracellular Performance

9:00 am Efficient On-Target Delivery of Oligonucleotides to the Liver & Beyond: in vitro & in vivo Performance of Sapreme’s Endosomal Escape Enhancers


  • Endosomal escape remains a bottleneck for efficient oligo therapeutics delivery
  • Sapreme has developed a series of endosomal escape enhancing compounds, derived from a natural product evolved to mediate endosomal escape
  • Optimized for conjugation to targeting ligand and payload through industry standard chemistries, the Sapreme compounds lend themselves to facile incorporation into existing compounds and proven manufacturing methods

9:30 am Neural Extracellular Vesicles for Selected & Protected Transport of siRNAs


  • Neural extracellular vesicles (EV) have high levels of tropism for neurons
  • Internally and externally loaded siRNAs in EVs can effectively knock down transcript levels in targeted cells
  • Effect of EV carrying siRNAs in various organ tissues

10:00 am
Morning Networking Break

11:00 am Discovery of a Minor Allele-Specific siRNA for PNPLA3 I148M to Treat NASH


  • Human genome wide association studies confirm the association of the rs738409 single nucleotide polymorphism (SNP) in the gene encoding protein patatin like phospholipase domain containing 3 (PNPLA3) with nonalcoholic fatty liver disease (NAFLD); the presence of the resulting mutant PNPLA3 I148M protein is a driver of nonalcoholic steatohepatitis (NASH)
  • While Pnpla3-deficient mice do not display an adverse phenotype, the safety of knocking down endogenous wild type PNPLA3 in humans remains unknown
  • To expand the scope of a potential targeted NAFLD therapeutic to both homozygous and heterozygous PNPLA3 rs738409 populations, we report the identification of a minor allele-specific siRNA

11:30 am Development of Molecular Vectors for Targeted Delivery of RNAi

  • Michel Khrestchatisky Research Director, CNRS, Director, Institute for Neurophysiopathology, Co-Founder & Scientific Counsel, Vect-Horus


  • Transport via high-capacity endocytic receptors as an efficient process for cell and tissue targeting of RNAi
  • Development of small peptide- and VHH-based vectors that target such receptors for efficient tissue and intracellular RNAi delivery
  • Application to targeted delivery of RNAi in different organs

12:00 pm
Networking Lunch

1:00 pm Round Table Discussion – Effective Cell & Tissue Specificity for siRNA Therapeutics

  • Julia Alterman Assistant Professor, RNA Therapeutics Institute, University of Massachusetts Chan Medical School
  • Ioanna Mylonaki Head of Preclinical R&D, Sixfold Bioscience
  • Vignesh N. Hariharan Post Doctoral Associate, Khvorova Lab RNA Therapeutics Institute
  • Daniel O’Reilly Post Doctoral Associate, Khvorova Lab RNA Therapeutics Institute
  • Qi Tang Post Doctoral Associate, Khvorova Lab RNA Therapeutics Institute


With siRNA therapies combating liver diseases, it is now important to realise the opportunities of such therapeutics applications beyond hepatic delivery. We will discuss new platform technologies focussed on aspects of siRNA therapeutics for target specific delivery beyond the liver

  • What does the future look like for the next generation of siRNA therapeutics beyond the liver?
  • Where do we expect innovations to come from and what challenges are ahead of us?
  • What challenges need to be addressed to overcome the target specificity for complex tissues?

1:45 pm
Afternoon Networking Break

Advancing the Future Outlook & Opportunities for siRNA Therapeutics

2:15 pm Panel Discussion – From Theory to Reality: Targeting Cancer with siRNA Therapeutics


In the past year, siRNA therapeutics have rocketed as a widely promising potential therapy for several diseases. With the major shift of interest in oncology, it is critical to now consider the viability of targeting the tumors for therapeutic intervention & debate the important next steps to ensure siRNA cancer drugs become a reality

  • Addressing the latest improvements and case studies on drug delivery systems for siRNA targeted delivery for targeting tumours with great success
  • How to robustly develop tumour models for complex siRNA delivery intratumorally for rapidly evolving cancers with appropriate
  • How to increase patient tumour responses by sensitizing the immune system with combination therapies

3:00 pm Bi-Specific shRNA that Simultaneously Downregulates mTOR & STAT3

  • Jinwoo Choi Professor & Co-CEO, Kyung Hee University & Curigin


  • Introducing a new approach for RNAi therapy using Bi-specific shRNA
  • Using our bioinformatic platform to determine prominent genes in specific diseases such as mTOR and STAT3 genes in cancer
  • Determine the therapeutic effect of naked Bi-specific shRNA vs using a viral carrier for Bi-specific shRNA

3:30 pm RNAi 2.0: Massively Parallel Functional Platforms De-Risk & Accelerate R&D


  • Quantitative functional screens identify single-digit pico-molar hits and optimize PK properties
  • Fit-for-purpose synthetic biology systems validate leads and teach the rational design prediction engine
  • Extra-hepatic targeting, using prophylactic and/or treatment regimens are becoming a reality

4:00 pm Chair’s Closing Remarks & End of 3rd RNAi – Based Therapeutics Summit 2022

  • Uli Stilz VP, Novo Nordisk Bio Innovation Hub