Day One - Wednesday August 28, 2024

8:00 am Check-In & Welcome

9:00 am Chair’s Opening Remarks

Charting the Uncharted: Regulatory Guidance for a New Era of Oligonucleotide Development

9:10 am Panel Discussion: Outlining Current Industry Trends in Oligonucleotide Development

Synopsis

  • Discussing the movement away from the development of oligonucleotides for rare diseases
  • Capitalizing on opportunities and changes we anticipate in the future and newer complex oligos coming into pipelines
  • Outlining challenges of scale, impurity characterization and qualification

10:00 am The Use of Risk Assessment to Evaluate the Quality Impact of Raw & Starting Materials Used in mRNA Manufacturing

Synopsis

  • Understanding the quality requirements and analytical testing panels for raw and starting material used in mRNA manufacturing
  • Assessment product quality and impact of raw material related risks and develop risk mitigation strategies to support mRNA manufacturing  

10:30 am Speed Networking & Coffee Break

Synopsis

Dive into this speed networking session to meet 100+ fellow colleagues and break the ice. This is your opportunity to meet like-minded fellow colleagues, share your challenges and spark collaborations. 

Debating Analytical Methods & Validity for Alternate Oligonucleotide Types

11:30 am Advancing Oligo-Conjugates Through Analytical Development

  • Mona Bill Senior Scientist, Analytical Chemistry, Silence Therapeutics

Synopsis

  • Reimagining analytical development methods based on structure of the conjugated moiety
  • Exploring functional assays to measure activity of the conjugate to ensure complete regulatory compliance and efficacy
  • Validating methods to quantify the conjugate and potential degradation products with HPLC or LC-MS

12:00 pm Ensuring Sequence Specificity as ASO Production Increases

  • Anna Dudkina Head of Analytical Development, Tessera Therapeutics

Synopsis

  • Assessing and maintaining sequence specificity to ensure ASO activity and gene-silencing
  • Rethinking formulation and characterization methods

12:30 pm Lunch & Networking

Upstream Innovation: Analytical Development of Oligonucleotides to Enhance Processes

1:30 pm Strategies for Enhancing Oligonucleotide Drug Formulation With Patient Adherence in Mind

Synopsis

  • Conducting stability assessments to address potential degradation products and de-risk larger scale production
  • Optimizing process and excipient selection for potential novel formulas to ensure safety
  • Navigating formulations, such as choice of salt and pH, to increase administration options and improve patient adherence

2:00 pm Application of NMR for PMO Characterization & Process Control Strategy

  • Bao Cai Executive Director, Process Development, Sarepta Therapeutics

Synopsis

Characterizing phosphorodiamidate morpholino oligomer chemistries to improve product consistency and efficacy

2:30 pm Session Reserved for Wuxi Tides

3:00 pm Afternoon Break & Scientific Poster Session

Synopsis

This is an informal session to help you connect with your peers in a relaxed atmosphere and forge new and beneficial relationships. With an audience of oligonucleotide enthusiasts eager to hear the latest cutting-edge advancements, you will have the opportunity to display a poster presenting your own work. Additionally, you will have the chance to review others’ posters displaying novel approaches to impurity assessments, analytical tools, and manufacturing for IND submission and post-regulatory approval into market.

Delving Into Downstream AD & CMC Toolboxes

4:00 pm Development of AEX & HILIC as Orthogonal Separation Methods to IPRP for the Characterization of Purity & Impurities in Oligonucleotide Products to Ensure Quality

Synopsis

  • Exploring novel chromatography protocols to improve separation and purification in CMC
  • Finding the right methods to separate nucleotides with minimal damage that maintains yield integrity
  • Establishing quantity and quality of oligonucleotides within a drug product for continuous quality control and process monitoring

5:00 pm Development & Application of 2D HPLC for PMO Analysis & Impurity Profiling

  • Tao Wei Associate Director - Ribonucleic Acid Process Development, Sarepta Therapeutics

Synopsis

  • Introducing advanced analytical techniques for comprehensive analysis and separation of complex mixtures in PMO formulations, aiding in the identification and quantification of impurities with high resolution and sensitivity

5:30 pm Chair’s Closing Remarks & End of Day 1