8:00 am Check In, Coffee & Light Breakfast
8:55 am Chair’s Opening Remarks
From Raw Materials to Final Products: Advanced Methods for Stereochemical Comparability & Diastereomer Analysis in Oligo Drugs
9:00 am Methods for Establishment of Stereochemical Comparability in Phosphorothioated Oligos: Analytical Approaches & Development Strategies
Synopsis
• Designing ASO sequences with different stereochemical content
• Evaluation of three analytical methods CD, NMR, and NP1/LCMS for assessing
stereochemistry
• Developing a robust comparability criteria and strategy based on analytical outputs to
assess and control stereochemistry across batches
9:30 am Development of an AEX Method for the Analysis of the Diastereomers in Oligonucleotide
Synopsis
• Case study exploring how a series of oligonucleotide 4 and 5 base shortmers were
evaluated for their diastereomer profile using a Strong Anion Exchange method
• Exploring how the development of the method evaluated a variety of parameters
including temperature, pH, MP buffer type and concentration
10:00 am Analytical Control Strategy for a Stereodefined Oligonucleotide Platform: An Integrated Approach from Raw Materials to Drug Substance
Synopsis
• Delve into Wave’s pioneered, stereodefined oligonucleotide chemistry across several
clinically viable modalities
• The comprehensive control strategy for stereoisomers that encompasses analytics of
raw materials, processes and finished drug substance
• Discussing the range of methodologies supporting the overall stereochemistry control
strategy
10:30 am Morning Break & Networking
Maximizing Efficiency Through Deep Characterization of Oligo Drug Product to Align With Regulatory Expectations
11:30 am Oligonucleotide Drug Product Characterization, Optimization & Development
Synopsis
• Proactive approaches to establish effective control strategies in early-stage programs.
• Considerations regarding drug product formulations
• Regulatory considerations for drug product manufacturing for successful submission
and market approval
12:00 pm Characterization of Oligonucleotides from Building Blocks to Higher-Order Structures
Synopsis
• Separation and Characterization of Product-Related Oligo Impurities Using Various
Analytical Technologies
• Proposed Solution for Overcoming the Challenges in Impurity Separation and
Characterization for Analytical Control
• Proposed Structure of an Unknown Impurity by High-Resolution Mass Spectrometry
(HRMS/MS) – a case study and characterization of High-Order Structures (HOS)- a
case study
12:30 pm Lunch Break
Turbocharging Oligo Development: Leveraging Advanced Modelling & Collaborative Innovation to Streamline Processes & Drive Efficiency
1:30 pm Panel Discussion: Lowering the Barrier to Entry for Oligonucleotide Drug Development
Synopsis
• How can companies in the oligo space collaborate to ease entry for small biotechs
• Exploring attempts to lower costs of analytical development and manufacturing and their subsequent impacts • Balancing innovation of analytical methods and technology versus cost- saving practises
2:30 pm Rapid, Automated Residence Time Distribution (RTD) Modeling for SolidPhase Oligonucleotide Synthesis (SPOS) using Process Data
Synopsis
• Using the column inlet and outlet conductivity signals during coupling can provide
information about flow through the column during each cycle without the need for
interrupting the synthesis
• RTD modeling and fitting can be automated and performed in seconds, with potential for
real-time implementation
• This approach involves some drawbacks which limit applicability somewhat
• Syntheses can be compared to assess the interdependencies of process equipment/
materials/conditions, flow character, and analytical results
3:00 pm Afternoon Break
Streamlining Drug Substance & Drug Product Comparability: Ensuring CQA Consistency & Managing Process Changes in Oligo Development
3:30 pm Ensuring Critical Quality Attributes (CQA) Consistency in Oligo Drug Development: Managing Analytical Considerations Through Drug Comparability
Synopsis
• Regulatory agencies expectations for comparability assessments to manage changes
in the manufacturing process and ensuring CQAs are maintained through the product
development process
• Analytical comparability of drug substances, methods that not only compare CQAs in
the release specifications but also conduct additional in-depth characterization and
impurity profiling
• The importance of comparability assessments that include representative batches
before and after process modifications, along with toxicology batches
4:00 pm Panel Discussion: Producing a Comprehensive Drug Comparability Framework
Synopsis
• Creating a consistent impurity profile across manufacturing and vendors through a robust comparability assessment
• Taking a proactive approach to comparability submission packages- thinking beyond just your manufacturing steps
• Developing strategies for effectively managing and documenting any changes in compliance with agency guidelines,
while proactively engaging with regulatory bodies to ensure the drug’s continued effectiveness and adherence to quality
standards