9:00 am Registration & Morning Coffee

9:25 am Chair’s Opening Remarks

Unveiling Pre-Clinical Data in Rodent Models & Novel Strategies for Bioanalysis to Advance into the Clinic

9:30 am Extrahepatic Delivery With OligoPhore & SemaPhore, Peptide-based Nanoparticles for siRNA & mRNA Therapeutics


  • Noncovalent polyplexes with any RNA that are highly stable in circulation and RNAase resistant
  • Extrahepatic delivery and efficacy for siRNA and mRNA polyplexes have been demonstrated in myriad disease models in vivo, including cancer, atherosclerosis, arthritis, metabolic syndrome, and bowel disease
  • Safety profiles after serial dosing in rodent models to date have been devoid of tissue and organ toxicity, complement activation, and immunological responses to polyplex components

10:00 am LC-MS-based Bioanalysis Strategies to Support Oligonucleotide Therapeutics Discovery


  • Overview of oligonucleotide therapeutics and bioanalysis platforms
  • Quantitative bioanalysis of oligo therapeutics by LC-MS/MS
  • Metabolite profiling of oligo therapeutics by LC-HRMS
  • Summary and future perspectives

10:30 am Morning Break & Scientific Poster Session

Mid Morning Chair: June Park, CEO, siRNAgen Therapeutics

Evaluating Pre-Clinical Disease Models with Improved Selectivity & Translatability for Accelerated Development

11:30 am RNAi-based Modulation of Autoimmunity in the Skin

  • Qi Tang Postdoctoral Associate, University of Massachusetts Medical School


  • Improving drug selectivity through RNAi approaches
  • Preclinical evaluation of therapeutic siRNAs in a skin disease model
  • Human skin explant as a valuable tool for the development of dermatological therapeutics

12:00 pm Using Whole Human Livers to Develop RNA Therapies, for Better Translation


  • Ochre Bio was founded around a simple concept – that novel RNA therapies applied to chronic liver disease require better use of human data
  • With more human data in discovery, more human data in validation, and better clinical trials
  • Discussing Ochre’s human-only liver pipeline, from large-scale sequencing of human livers in Asia, Europe, and the US through to testing lead assets in perfused human livers as the ‘preclinical model’

12:30 pm Round Table: Tackling Toxicology Studies in Animal Models


  • How to show knockdown in rodent species?
  • What are the best models for determining safety and efficacy?
  • Optimizing the dosing strategy to reduce toxicity while achieving a therapeutic effect
  • How can adding a targeting ligand benefit improve efficacy?
  • What chemical modifications can be made to reduce toxicity?
  • How to tackle IND-enabling studies in preclinical pharmacology and toxicology models
  • What tools are available to successfully assess PKPD?
  • What PKPD properties are optimal for the desired effect?

1:15 pm Networking Lunch

Developing Robust Processes to Enable Large Scale Manufacturing to Scale Up for the Clinic

2:15 pm Round Table Discussion: Streamlining Process Development for Accelerated R&D


  • How to ensure the process is nailed down to achieve desired yield and purity levels?
  • What purity levels are optimal to advance through the clinic?
  • Is it a requirement to characterize impurities before moving to phase 2?

3:00 pm Strategies for Rapid Scale-up of Diverse asiRNA Duplexes

  • Debasis Patra Vice President, Senior Director & Head of Chemistry, Manufacturing & Controls, OliX US


  • Our asiRNA portfolio is designed to treat ocular, dermatologic, alopecia and multiple liver diseases; diverse conjugation strategy poses unique chemistry-related challenges
  • Rapid product delivery is often required to meet tight timelines for first-in-human clinical studies while developing robust production processes. Significant issues were encountered during the synthesis of lipid and GalNAc-conjugated strands
  • Phase-appropriate strategies were implemented to address the challenges associated with each program

3:30 pm Lessons Learned in Developing & Scaling Novel Platforms

  • Richard Welch Vice President - Chemistry, Manufacturing & Controls, Sirnaomics


  • A high-level overview of the early phase clinical development of one of our core platforms, including not only the challenges to the early phase and transfer from concept to clinic but indications about the challenges to commercialization as well
  • As part of this review will explore how the use of platform technologies can streamline development and provide savings in time and resources; they must be approached with a full understanding of their limitations as well as their benefits. This applies not only to development but to production planning, scale-up, method development, and phase-appropriate validation
  • Outlining how recent changes in the global supply chain and global regulatory and legal frameworks continue to offer challenges in translating concepts to clinical material. Examples of challenges and possible solutions include alternate suppliers to backfill supply chain shortfalls, developing multiple lines of regulatory options to deal with the constantly changing global environment

4:00 pm Chair’s Closing Remarks

4:15 pm End of 4th Annual RNAi-Based Therapeutics Summit