8:00 am Chair’s Opening Remarks

Improving Future Gene Therapy Analytical Development Strategies

8:15 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

8:45 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

 

9:15 am Session Reserved for Bio-Rad

9:45 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?

10:15 am Speed Networking

Synopsis

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

11:00 am Morning Refreshments

Improving Potency Assay Development to Demonstrate Clinical Relevance

11:30 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

12:00 pm AAV Titration ELISAs: a Robust & Reliable Tool for AAV Quantification

12:30 pm 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

1:00 pm Lunch & Networking

Exploring the Use of Next Generation Sequencing for Different Applications

2:00 pm Accurate & Sensitive Identification of Nucleic Acid Impurities in AAV Vector Samples by High-Throughput Sequencing

  • David Dobnik Scientific Associate, Head of Laboratory for Therapeutic Viruses, National Institute of Biology

Synopsis

  • Using HTS to identify all nucleic acid impurities present in AAV vectors
  • Highlighting differences between short and long read platforms
  • Case study on AAV vectors, focusing on encapsidated impurities
  • Discussing the limitations of this method and its regulatory implications

2:30 pm Analytical Technologies Roundtable Discussion

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

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:

3:15 pm Afternoon Refreshments & Networking

Assessing Analytical Methods for Gene Therapy Characterisation

3:45 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

4:15 pm Session Reserved for BIA Separations

4:45 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

5:15 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

5:45 pm Chair’s Closing Remarks

6:00 pm Scientific Poster Session

Synopsis

The scientific poster session is an ideal opportunity to communicate your new results and expertise to a niche audience of industry experts, get feedback from peers and colleagues in the industry and learn how others in the field have been tackling similar challenges.