Conference Day Two

Day Two

Thursday June 17, 2021

Registration Opens & Chair’s Opening Remarks

Discussing Oligonucleotides: What can We Learn from Selective Delivery?

9:30 am Efficient, Targeted Cytoplasmic Delivery of Oligonucleotides via Sapreme’s Endosomal Escape Enhancers

  • Guy Hermans Chief Executive Officer, Sapreme Technologies

Synopsis

  • Outlining Sapreme’s SPT001 compound enhances endosomal escape of targeted bioconjugates
  • Demonstrating data on how adding SPT001 to antibody-oligo conjugates or GalNAc-oligo conjugates enhances oligo potency by up to 1000x, without impacting the specificity of tissue delivery

9:55 am Targeting DNA Damage Response Genes with Oligonucleotides for Therapeutic Modulation of Somatic Instability in the Brain

Synopsis

  • Discussing how genes in the DNA damage response (“DDR”) pathway are potent genetic modifiers of Repeat Expansion Disorders (REDs), such as Huntington disease (HD), myotonic dystrophy, spinocerebellar ataxias, and operate upstream of disease genes
  • Outlining how Triplet targets this fundamental unifying pathway with the goal to slow somatic repeat expansion and potentially delay or prevent onset and progression in multiple REDs
  • Exploring the administration of DDR-targeting ASOs via multiple routes to non-human primates was well tolerated and drove significant and sustained knockdown in disease-relevant brain regions, including the striatum

10:20 am Treating Diseases with Antibody Oligonucleotide Conjugates: Combining the Selectivity of Antibodies with the Specificity of Oligonucleotide Drugs

  • Arthur Levin Chief Scientific Officer, Avidity Biosciences

Synopsis

  • Antibody oligonucleotide conjugates utilize the selectivity of antibody delivery and the specificity of oligonucleotide therapeutics to treat diseases
  • AOC1001 is designed to treat myotonic dystrophy type 1
  • Other AOCs are in development to address muscle disease and other disorders

10:45 am Live Panel Q&A – Ask the Speakers your Burning Questions!

11:00 am Morning Break & Speed Networking

Exploring Novel Technologies & Approaches for Targeted & Effective Delivery

12:00 pm LUNAR™: A Novel, Potent and Safe Platform for the Delivery mRNA 

  • Padmanabh Chivukula Chief Scientific Officer & Chief Operating Officer, Arcturus Therapeutics

Synopsis

  • LUNAR nanoparticles can encapsulate a wide range of RNA molecules (300–15,000 kDa)
  • A self-transcribing and replicating RNA (STARR™) based vaccine (LUNAR®-COV19) has been developed to prevent SARS-CoV-2 infection.
  • A single dose led to robust antibody responses, with high neutralizing antibody titers

12:25 pm DegradaBALL® Platform DDS Technology for siRNA Therapeutics

  • Dal-Hee Min Chief Technology Officer & Co-Founder, Lemonex Inc

Synopsis

  • DegradaBALL® is a modular DDS platform based on biocompatible porous nanoparticle
  • DegradaBALL® allows highly effective and long-lasting RNAi with lower dose and fewer injections
  • DegradaBALL® is compatible with various administration routes and maintains its structure and function for a long time, even after storage at room temperature

12:50 pm Novel Approaches for Targeted & Lung Delivery of RNAi

Synopsis

  • Outlining novel mechanisms of active targeting for effective RNAi therapies
  • Discussing apatamers as ligands for targeted delivery of RNAi
  • Exploring novel delivery techniques for the lung delivery of RNAi

1:15 pm Live Panel Q&A – Ask the Speakers your Burning Questions!

  • Sean Bedingfield Research Engineer, New Therapeutic Modalities, Cambridge, Eli Lilly and Company
  • Dal-Hee Min Chief Technology Officer & Co-Founder, Lemonex Inc
  • Elias Fattal Professor,, University of Paris-Saclay
  • Padmanabh Chivukula Chief Scientific Officer & Chief Operating Officer, Arcturus Therapeutics

1:30 pm Networking Lunch

2:30 pm Designing Conjugated siRNAs for Extrahepatic Delivery

  • Annabelle Biscans Associate Principal Scientist, Oligonucleotide Chemistry Team, AstraZeneca

Synopsis

• Exploring lipid conjugation for improving siRNA extrahepatic delivery
• Discussing the impact of lipid structure, siRNA structure and linker
chemistry on siRNA distribution and efficacy
• Outlining lipid conjugates for muscle siRNA delivery

2:55 pm Outlining Amgen’s Program Updates Harnessing RNAi for Therapeutic Application

Synopsis

  • Discussing recent successes and learnings from RNAi projects
  • Outline challenges of successful delivery of RNAi
  • What is on the horizon for Amgen?

3:20 pm Live Panel Q&A – Ask the Speakers your Burning Questions!

  • Julie Lade Senior Scientist, Amgen
  • Annabelle Biscans Associate Principal Scientist, Oligonucleotide Chemistry Team, AstraZeneca
  • Marc Abrams Vice President, Discovery Research, Dicerna Pharmaceutical

3:30 pm Chair’s Closing Remarks

3:45 pm End of 2nd RNAi Based Therapeutics Summit