7:50 am Chair’s Opening Remarks

EXPLORING ADVANCED ANALYTICS IN THE PURSUIT FOR QUANTITATIVE ANALYTICAL RESULTS WITH LOW VARIABILITY

8:00 am Exploring the Evolution of Analytical Techniques for Gene Therapy Drug Development

Synopsis

  • Welcome to the digital age!?
    • Switching from PCR to ddPCR
    • Changing from gel to capillary
  • Old but still Gold!? – Analytical Ultracentrifugation

8:30 am Speaking Position Reserved for BIA Separations

9:00 am Importance of a Comprehensive Analytical Development Strategy for AAV Gene Therapy

Synopsis

  • Avoiding common pitfalls by having robust approach to method development.
  • Identification and continual evaluation of CQAs.
  •  Impact of limited material availability

9:30 am Speaking Position Reserved for Lonza

10:00 am Joint Q&A Session

10:20 am Morning Refreshments & Speed Networking

Synopsis

This session is the ideal opportunity to get face-to-face time with many of the
brightest minds working to advance gene therapies. Benchmark against the
industry leaders and establish meaningful business relationships to pursue for the
rest of the conference and beyond.

BIOASSAYS & MOLECULAR BIOLOGY

OPTIMISING ASSAY DEVELOPMENT,
QUALIFICATION & VALIDATION

11:20 am Immunoassays for AAV Capsid Titre Quantification

Synopsis

  • Comparing ELISA with alternative immunoassay methods
  • Building analytical target profile for capsid titre quantification
  • Testing in-process samples and ensuring serotype-specificity

11:50 am PANEL: Assay Validation for Critical Assays

Synopsis

  • What is required to validate a critical assay?
  • What parameters should be assessed for a critical assay to be validated?
  • When should the process of validation begin?
  • Discussing harmonisation between Europe and the US and different regulatory groups, particularly on validation of assays
  • Regulatory expectations for phase-appropriate validation
  • Standardisation of assay qualification and validation

12:20 pm Critical Aspects of Gene Therapy Method Validation

Synopsis

  • Developing a well-controlled method that works at every site
  • Developing the reagents that are needed for success
  • Deciding when to validate and then when to revalidate
  • Managing transfers, validations and life cycle at remote sites

12:50 pm Joint Q&A Session

ANALYTICAL CHEMISTRY

DETECTION & SEPARATION OF PARTIALLY FILLED CAPSIDS

11:20 am What is Hiding in Partially Filled AAV Particles?

  • David Dobnik Senior Research Associate, National Institute of Biology

Synopsis

  • Different fractions of AAV vectors after ultracentrifugation were characterised
  •  Particle number, vector genomes and presence of impurities was evaluated and compared between fractions
  • Long-read sequencing approach can provide additional information on particle content

11:50 am Speaking Position Reserved for Sciex

Synopsis

12:20 pm Seeking Higher Throughput & Smaller Scale Analytical Techniques

Synopsis

  •  What role can platform assays play in aiming to adhere to shorter timelines?
  •  Introducing automation and greater productivity for more CMC compliant methods
  • Changing mindsets from the classical mythology to new advancements to the analytics field
  •  Regulatory agency guidelines on the use of new technologies

12:50 pm Joint Q&A Session

1:10 pm Lunch & Networking

OVERCOMING POTENCY ASSAY CHALLENGES

2:10 pm Potency Assays: Few Lessons Learned

Synopsis

  • Unexpected Potency Trends
  • System Suitability Criteria, how not to design them
  • Key reagents management

2:40 pm Potency Assays Implementation

  • Connie Tsai Senior Scientist Analytical Development, Novartis Gene Therapies

Synopsis

  • Challenges of potency assay validation
  • Analyzing potency assays for different products

3:10 pm Joint Q&A Session

  • Nasser Sadr Associate Director, UniQure
  • Connie Tsai Senior Scientist Analytical Development, Novartis Gene Therapies

THE AGE-OLD CHALLENGE: EMPTY-FULL CHARACTERISATION

2:10 pm Assessing Best Practice Analytical Tools for Characterisation of Empty/Full Capsids

Synopsis

  • Methods for Empty/Full AAV capsid characterisation
  • Pros and Cons of each method
  • Orthogonal approaches

2:40 pm Speaking Position Reserved for for Charles River

Synopsis

3:10 pm Joint Q&A Session

3:30 pm Afternoon Refreshments & Networking

BEST PRACTICES FOR DEMONSTRATING COMPARABILITY

4:30 pm Comparability Assessment At Various Stages Of Product Development

  • Jaap Twisk Senior Director Analytical Development , UniQure

5:00 pm CMC and Analytical Development To Meet Regulatory Expectations On Gene Therapy Comparability Studies

Synopsis

  • Navigating the regulatory pathway on gene therapy comparability
  • Challenges of comparability studies on gene therapy products
  • Evaluation and analysis of Critical Quality Attributes for Gene Therapy products

5:30 pm PANEL: What Can We Learn From The Rapid Evolution Of Viral Vaccine Development Over The Past Year To Better Inform Gene Therapy Analytical Development

  • Roland Pach Project Lead Analytical Technical, Roche
  • Sofia Carvalho Senior Research Associate, IBET
  • Stephen Kaminsky Professor of Research in Genetic Medicine Associate Director, Weill Cornell Medical College

6:00 pm Joint Q&A Session

6:20 pm Chairs’ Closing Remarks