7:50 am Chair’s Opening Remarks

CMC CHALLENGES IN ANALYTICAL DEVELOPMENT

8:00 am Advances in Analytics to Streamline the Production of Viruses for Gene Therapy and Vaccination

Synopsis

  • Overview of analytical methods for characterisation of vaccines and gene therapy vectors’ critical quality attributes
  • Highlight of the main challenges in analytics to assist bioprocess development (UPS and DSP)
  •  Perspectives on integration of critical analytical tools in vaccine and gene therapy frameworks

8:30 am Detection of Replication Competent Viruses in Viral Vector Products

Synopsis

Production of viral vectors carries with it the risk of the generation of replication
competent viruses
• The European Pharmacopeia and the US FDA has issued guidance concerning the
testing of these vectors for replication competent viruses
• Robust detection assays are necessary in order to screen these products to ensure
safety
• We have established platform assays for all 4 virus families and expanding them to
new strains and serotypes

9:00 am Joint Q&A Session

9:30 am Morning Refreshments & Speed Networking

BIOASSAYS & MOLECULAR BIOLOGY

NAVIGATING THE CHALLENGES OF LIMITED MATERIALS

10:30 am Key Points in Developing qPCR and dPCR Based Methods for AAV Vector Quantification

Synopsis

  • Reference material characterisation
  •  Multiplexing in qPCR and Dpcr
  • A common step: sample preparation

11:00 am New Analytical and Biophysical Tools to Minimise Sample Consumption for Release and Characterisation Assays of AAV-Based Products

Synopsis

  •  AAV products comprise a protein capsid and a nucleic acid packaged inside.
  • The smaller manufacturing scales of AAV-based products make it particularly challenging to develop methods with very limited sample availability.
  •  We evaluate new technologies which require minimal sample amounts to inform critical quality attributes and we demonstrate how they are orthogonal to the traditional methods.

11:30 am Key CMC Challenges and Development Approaches for AAV Gene Therapy: Pre-Clinical to IND

Synopsis

  • The key CMC deliverables amid the pre-clinical to clinical phase of AAV gene therapy product development
  • Analytical challenges will be emphasised, including production yield vs. volume requirements, batch comparability, and stability

12:00 pm Joint Q&A Session

ANALYTICAL CHEMISTRY

DEFINING ANALYTICAL APPROACHES TO EXTENDED CHARACTERISATION

10:30 am PANEL DISCUSSION: Challenges of Extended Characterisation with Limited Samples

Synopsis

  •  Analytical approaches to extended characterisation
  • Applications of extended characterisation
  • Which Assays to use with limited sample availability
  • How Many are needed to provide sufficient data on key quality parameters for an acceptable production lot

11:10 am Extended Characterisation of Lentiviral Vectors: Closing the Gap with AAV

Synopsis

  • Extended characterisation is required to demonstrate product understanding of impurities and impact to efficacy.
  • Lentiviral vectors (LVV) combine the complexity of AAV and extracellular vesicles, and AAV techniques are not always directly applicable to LVV.
  • Case studies will be presented describing current methods and future potential for LVV characterisation assays.

11:40 am Mass photometry – a new method for analysis of biomolecules and AAV’s

Synopsis

  • Introduce mass photometry as a technology
  • Share specific examples including for the determination of empty / full ratios in the analysis of AAV’s

11:50 am Joint Q&A Session

12:30 pm Lunch & Networking

CONTROL STRATEGIES & METHOD LIFE CYCLE MANAGEMENT

1:30 pm Future In-Vivo GT Control Strategy: Need for Adequate (Pre-) cQAs of AAV Based GT Products

Synopsis

  • Current and future GT control strategy
  • Assessing adequate CQA’s

2:00 pm A QC Perspective: Method Life Cycle Management and Considerations for Gene Therapy Programs

2:30 pm Speaker TBC

3:00 pm Joint Q&A Session

CHARACTERIZATION OF AGGREGATES &
IMPURITIES

1:30 pm AAV Vectors: Product Related Impurities Characterisation

Synopsis

  •  Identifying product related impurities for AAV vectors
  • Analytical approaches to characterization of impurities

2:00 pm Control of Critical Analytical Reagents

Synopsis

Which analytical reagents should be considered critical analytical reagents?
This talk will discuss which and how they should be evaluated and managed
Dossier expectations at approval, and post approval changes

2:30 pm LC-MS Characterization to Support AAV Gene Therapy Development

  • Xiaoying Jin Head of GMU Characterization, Global CMC Development, Sanofi

Synopsis

• Capsid protein heterogeneity may impact transgene
expression
• LC-MS method improvement
• Structure-function studies

3:00 pm Joint Q&A Session

3:30 pm Virtual Networking & Scientific Poster Session

CRITICAL QUALITY ATTRIBUTES IDENTIFICATION, ASSESSMENT & CONTROL STRATEGIES

4:00 pm Analytical Tools to help the production of your Gene Therapy products

  • Dr Stephen Lock Senior Marketing and Market Development Manager, Biopharma, EMEAI, SCIEX

Synopsis

  • Understand how new analytical tools are helping to optimize your cell culture media
  • Learn how to characterize viral proteins in your gene therapy products to get a better understanding of their potency
  • Discover how to profile empty vs full viral particles using less than 5 µL of sample in under an hour

4:30 pm A Top-Down Approach to Gene Therapy Product Characterisation: Addressing Critical Quality Attributes from Intact Capsids to Amino Acid Modifications

  • Ian Anderson Associate Director Analytical Sciences, Allergan

Synopsis

  • Biophysical techniques for empty full and aggregation analysis
  • Intact mass and peptide map analysis for VP subunit characterisation
  • Multiple Attribute Monitoring: moving LC-MS from characterisation to QC release testing

5:00 pm Joint Q&A Session

  • Ian Anderson Associate Director Analytical Sciences, Allergan
  • Dr Stephen Lock Senior Marketing and Market Development Manager, Biopharma, EMEAI, SCIEX

5:20 pm Chairs’ Closing Remarks