Pre-Conference Discussion Day

Monday, June 13 2022

Seminar: A-Z Oligonucleotide Bootcamp: Upgrade Your Oligonucleotide Design & Development to Unleash the Full Potential of Oligonucleotide Therapeutics Within the CNS

9:00 am
Breakfast & Registration

Manipulating Oligonucleotide Chemistry to Maximize Therapeutic Potency & Precision

10:00 am Deep Diving into Modifying Chemistry of siRNAs for Optimized Extra- Hepatic Delivery

  • Mano Manoharan Senior Vice President, Innovation Chemistry , Alnylam


  • Revealing key oligonucleotide modifications you can make to optimize RNAi activity
  • Illustrating how chemical modifications can mitigate off-target effects
  • Exploring tips and tricks to employ chemical modifications to upgrade your delivery

10:30 am Investigating Data Driven Approaches to Oligonucleotide Molecule Design


  • Learning how to utilize AI to inform molecule design
  • Characterizing off-target effects
  • Exploring how to incorporate knowledge graphs to support ASO design

11:00 am Discovering Basic Chemical Modifications Required for Deeper Penetration & Better Distribution for Different Oligonucleotide Modalities

  • Raj Pandey Vice President & Head of Chemistry, Cargene Therapeutics


  • Exploring the current landscape of modified monomers and ligands target CNS and different organs
  • Explore the barriers that influence oligonucleotide access to tissue sites,
  • To understand the deeper penetration and biodistribution of ASO and siRNAs

11:30 am
Morning Break & Networking

12:00 pm Revealing Structurally Constrained Internucleotide (E)-Vinyl Phosphonate Backbone Impacts siRNA Potency & Allele Specificity

  • Ken Yamada Assistant Professor, University of Massachusetts Medical School


  • Adding structure-constraining of sugars, such as 2′-fluoro and LNA, has been shown to enhance oligonucleotide stability and affinity to target mRNA
  • Exploring the synthesis and impact of a structurally-constrained inter-nucleotide (E)-vinylphosphonate backbone (iE-VP) where we show iE-VP can modulate metabolic stability, enzyme interactions and siRNA activity
  • Demonstrating how iE-VP induces significant mismatch-discriminating properties without compromising on-target silencing
  • Explaining how our study covers a broad range of interests and paves the path towards fully allele-specific siRNAs for CNS disorders such as Huntington’s disease

Scaling Up & Scaling Out: Discovering How to Maximize Efficiency of Oligonucleotide Manufacturing

12:30 pm Investigating New Horizons for Oligonucleotide Drug Substance Manufacturing for CNS Products

  • Firoz Antia Senior Director & Head of Oligonucleotide Process Development, Biogen


  • Revealing our strategy for drug substance manufacturing for CNS products
  • Exploring emerging technologies we’re developing for drug manufacture and understanding how these can be applied to the CNS
  • Deep diving into convergent synthesis

1:00 pm Lunch Break & Networking

2:00 pm Scrutinizing & Analytical Challenges of Oligonucleotide Delivery Systems & their Translation to the Clinic


  • Understand key points in formulation design and definition of critical quality attributes
  • Analytical methods associated with characterization, stability, and assessment of reproducibility and scalability
  • Evaluation and selection of appropriate delivery systems

2:30 pm Learning How to Work With CMOs & CROs for Effective Oligonucleotide Manufacturing in Line With Regulatory Expectations


  • Executing efficient GMP oligo manufacturing at your CMO
  • Learning how to manage impurities in your oligo drug substance
  • CMC for oligos 101: Incorporating information from regulators to optimize oligo development

3:00 pm Design, Screening & Optimization of Oligonucleotide Therapeutics with Optimal Profiles Across Large-Scale Data Sets of Individual Neurons for Targeting CNS Diseases

  • Luis Williams Executive Director, Head of Biology & ASO Epilepsy Therapeutic Lead, Q-State


  • Exploring antisense oligonucleotide (ASO) design, screening and optimization
  • for CNS applications, with a focus on assessment of ASOs at the cellular level in diverse neuronal types
  • Outlining the development of deep-learning techniques with large scale data sets to characterize desired phenotype rescue and undesired toxicities associated with candidate ASO therapeutics
  • Revealing findings in the context of CNS disorders such as genetic epilepsies and chronic pain conditions

3:30 pm
End of A-Z Seminar