8:00 am Online Registration & Explore the Digital Platform

8:25 am Chair’s Opening Remarks

Improving Future Gene Therapy Analytical Development Strategies

8:30 am Defining, Developing & Managing a Functionally Relevant Set of Assays for AAV Vectors

Synopsis

Discussing the challenges of AAV gene therapy analytical development

  • With no industry defined assay package, how best to decide which panel of assays are needed for release, stability, and characterization
  • Using platform approaches to achieve faster timelines
  • Exploring sampling plan strategies considering volume required

8:50 am Analytical Comparability Requirements During the Development & Commercialisation of Gene Therapy Products

  • Clare Blue Director of Analytical Development, Biogen

Synopsis

  • What is analytical comparability?
  • Requirement for analytical comparability during and post clinical development
  • Regulatory expectations
  • Defining comparability acceptance criteria
  • Bridging studies and assay performance monitoring to ensure comparability

 

9:10 am Panel Discussion: Addressing the Current Challenges of Analytical Development Between Phases

Synopsis

Hear from gene therapy developers at different stages of the development timeline, from manufacturing to commercialization. Topics to discuss include:

  • Encouraging the use of novel technologies and breaking free from traditional approaches
  • Maximising the use of limited materials
  • Overcoming the standardization issue and lack of reference material
  • Comparability: what improvements can be made?
  • Brexit: What are the consequences for gene therapy developers?

9:40 am Virtual Speed Networking

Synopsis

Specifically designed to connect you with new contacts from the most active companies in the field, the, now virtual, renowned Speed Networking session will be one of the most valuable hours you will spend at Gene Therapy Analytical Development Europe.

10:30 am Short Break: Explore Exhibition Booths & Private Chat Time

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with your sponsors, discover their technologies and set up private conversations with fellow attendees to form connections for future partnerships.

Improving Potency Assay Development to Demonstrate Clinical Relevance

11:00 am Case Study: Developing a Functional Potency Assay to Demonstrate a Link to Effectiveness

  • Elina Hessels Senior Scientist, Lead Assay Development, UniQure

Synopsis

  • Background potency testing
  • Approaches to be used for potency testing
  • Case study on a potency test development for a Phase I/II product

11:20 am Phase-Appropriate Analytical Control Strategies for Recombinant AAV-Based Gene Therapy Products: Case Studies in the Characterization of a Process-Related Impurity and Development of an In-Vitro Potency Assay

  • Eric Yearley Principal Scientist, Analytical Development, BridgeBio

Synopsis

  • Overall analytical control strategy: CQAs assessment and corresponding assays, stability program and benefits of implementing a CRO for phase I analytical development
  • Example of an in-house in vitro potency assay to measure protein expression
  • Current development of an enzymatic in vitro potency assay
  • Development of an assay to measure residual detergent for in process and DS/DP samples

11:40 am Using Infectivity Assays as a Surrogate for Potency Assessment of Viral Vectors

  • Marina Feschenko Director, Analytical Development- Gene Therapy/Biologics, Biogen

Synopsis

• Infectivity, expression, and functional potency are three major biological activities of viral vectors. Do we need all three for each programme?
• When and how can we use infectivity for in vitro potency assessment?
• Implementation of a novel ddPCR-based method for measuring infectivity
• Relative transduction versus infectious titer assays

12:00 pm Live Q&A & Panel Discussion

  • Marina Feschenko Director, Analytical Development- Gene Therapy/Biologics, Biogen
  • Elina Hessels Senior Scientist, Lead Assay Development, UniQure
  • Eric Yearley Principal Scientist, Analytical Development, BridgeBio

12:30 pm Analytical Technologies Interactive Discussion

  • Ian Anderson Senior Manager, Analytical Sciences, Allergan
  • David Dobnik Scientific Associate, Head of Laboratory for Therapeutic Viruses, National Institute of Biology
  • Doyoung Lee Senior Scientist, Lead UK DRI Vector Core, King’s College London

Synopsis

Participate in a focussed roundtable discussion to discuss key analytical technologies in an intimate environment. Each table will be led by an industry leader with experience in using the relevant methodology, so this is your opportunity to have your burning questions answered and benchmark your approach with your colleagues in the field. Roundtables will discuss the use of the following approaches:

  • Digital PCR vs qPCR
  • Analytical Chemistry
  • Next Generation Sequencing
  • Cell-Based Assays

1:00 pm Lunch Break

The Switch to Digital PCR

1:30 pm Digital PCR – State of the Art Vector Genome Titering Or Even More?

Synopsis

  • Digital PCR: The Basic Principles
  • Reducing variation: why is digital PCR considered better than qPCR?
  • Potential drawbacks of this innovative technique
  • Strategies of designing the transgene target sequences and the individual influence on the obtained results

1:50 pm Exploring Droplet Precision for Analytical Development & AAV Quantification

  • Dianna Maar Product Manager, Digital Biology Group, Bio-Rad Laboratories
  • Koen De Gelas Regional Sales Specialist ddPCR (BeNeLux & UK), Bio-Rad Laboratories

Synopsis

  • New optimized ddPCR workflow and ITR assay for AAV
  • Showcasing quality and quantity data duplexing ITR with GOI
  • Introducing the newest ddPCR instrument QX ONE

2:10 pm Applications of ddPCR for the Characterisation of AAV Vectors

Synopsis

• Process manufacturing and Quality Control of AAV vectors
• VG titers- qPCR vs ddPCR
• Evaluation of genome integrity with 2D ddPCR

2:30 pm Live Q&A & Panel Discussion

3:00 pm Afternoon Refreshments

Assessing Analytical Methods for Gene Therapy Characterisation

3:30 pm Session Reserved for Beckman Coulter

3:50 pm Assessing Analytical Tools for the Characterisation of Empty, Partial & Full AAV Particles

  • Ian Anderson Senior Manager, Analytical Sciences, Allergan

Synopsis

  • Analytical Ultracentrifugation (AUC): What value does AUC provide if not suitable for GMP environment?
  • Discussing the limitations such as low sensitivity and requiring a lot of material
  • Determining the high-resolution value and benefit of using HPLC

4:10 pm Quantify Viral Vector Attributes with Light Scattering

Synopsis

  • Viral vectors, such as adeno-associated virus (AAV) and lentivirus (LV), are widely used in gene and cell therapies.
  • In order to ensure the safety and efficacy of these emerging therapeutics, it is critical to fully characterize the viral vectors throughout the development process, as well as quantify their critical quality attributes (CQAs) across manufacturing and quality control.
  • In this webinar, we discuss how dynamic light scattering (DLS) and multi-angle light scattering combined with size exclusion chromatography (SEC-MALS) or field-flow fractionation (FFF-MALS) characterize AAV, LV, and other gene delivery vectors.
  • Biophysical properties and attributes covered by these techniques include size, concentration, aggregation, stability and capsid content.

4:30 pm Mass Spectrophotometry Based Analytical Methods for AAV Protein Characterisation & Residual Protein Determination

  • Uditha deAlwis Executive Director Analytical Sciences (AD/QC), Sarepta Therapeutics

Synopsis

  • AAV protein identity and post translational modification determination in a denaturant free system
  • A method that utilizes the natural denaturation temperature of AAV to achieve capsid protein recovery and high-resolution mass analysis
  • Host cell and media component quantification at low ng/mL levels. The presence of host cell protein debris and the determination of these components has been conducted by ELISA methods
  • A novel method of identifying all proteins that may be contaminating an AAV preparation and then quantifying the contaminates will be discussed

4:50 pm Live Q&A & Panel Discussion

  • Uditha deAlwis Executive Director Analytical Sciences (AD/QC), Sarepta Therapeutics
  • Ian Anderson Senior Manager, Analytical Sciences, Allergan

5:20 pm Explore the Platform & Set Up Private Calls

Synopsis

Grab a drink and visit the exhibitions available, set up private conversations with your fellow online attendees and network from the comfort of your own home.

6:00 pm End of Day One