7:00 am Registration and Morning Networking

Synopsis

7:50 am Chairperson’s Opening Remarks

7:59 am
SETTING AND SATISFYING THE ACCEPTANCE CRITERIA FOR DEMONSTRATING COMPARABILITY

8:00 am Case Study: Overcoming Regulatory Challenges in Comparability Design

Synopsis

  • Lessons learned from satisfying regulatory feedback regarding comparability design and execution
  • Best practices when adopting a collaborative approach to meet authority expectations
  • Assessment of the common pitfalls in comparability design that result in re-testing and tips to mitigate risk and set up for success

8:30 am Setting up for Success: How To Prepare Ahead of Time to Meet Regulatory Expectations

  • Meghan Brown Executive Director, Regulatory Affairs, CMC, Sarepta Therapeutics

Synopsis

  • Best practices to working with regulatory authorities when demonstrating comparability
  • Assessment of the regulatory expectations when designing potency assays
  • Creating clarity around the current standards in methods for comparability testing

9:00 am Panel Discussion: Understanding The Nuances in Comparability Package Expectations at a Market Level

  • Janet Glassford Quality Assessor, MHRA
  • Omar Tounekti Manager, Gene Therapies Division in the Biologic and Radiopharmaceutical Drugs Directorate (BRDD), Health Canada
  • Yoko Momonoi Head of Regulatory CMC Cell Therapies, Takeda

Synopsis

  • How are regulatory authorities aligning expectations to prevent market specific disparities in comparability criteria?
  • What are the ‘need to know’ differences in comparability requirements globally that need to be considered?
  • Why are the specifications for product release changing and how are the acceptance criteria for comparability tests deviating? Are there any leniencies provided for variable quality attributes?
  • What is the guidance when it comes to overcoming the bottleneck of material retention?

9:50 am Structured Networking

Synopsis

Join us for face-to-face networking. We will pair you up with fellow attendees so you can get one on one time with the brightest minds working in the gene therapy field in order to establish meaningful business relationships to pursue for the rest of the conference.

10:20 am Morning Coffee Break

REINFORCING COMPARABILITY DESIGN TO MITIGATE RISK IN GENE THERAPY DEVELOPMENT

10:50 am Holistic approach to Comparability

Synopsis

    • What is comparability and what triggers the need for comparability?
    • What is clinical comparability? What is Analytical comparability?
    • Impacts of comparability on your overall program
    • Are there ways to limit comparability risk?
    • How can Resilience help your program?
      • Platform approaches for process and analytical
    • Slowing down to go faster
      • Program or portfolio approach?
      • Are there ways to streamline?

 

11:20 am Virtual Presentation: A-Cell: Best Practices for the Manufacture of Cell Therapies

  • Josephine Lembong Manager, Science & Industry Affairs, Alliance for Regenerative Medicine

Synopsis

  • Establishing Quality Target Product Profile and Critical Quality Attributes early are critical to managing comparability across products and process changes during development
  • Risk and criticality assessment tools are used throughout product development to establish processing operations, design space, and control strategy to ensure the process delivers a quality product in a reproducible process
  • A comparability plan to introduce manufacturing changes should be developed as early as possible in product development, preferably before a phase 1 trial
  • The acceptability of comparability depends on the knowledge of the product, availability of well-defined CQAs that are informative for product safety and efficacy, and timing of when the major change is introduced in the product development cycle

11:50 am Case Study: Assessing the Type & Magnitude of Change in Order to Develop an Appropriate Comparability Test

  • William Werner Sr. Director Analytical Assay development, Astellas Gene Therapies

Synopsis

  • Addressing comparability dependent on the extent of change, the impact on critical quality attributes and the linkage to implications on safety and efficacy

12:20 pm Analytical to Clinical: Ensuring Analytical Method Validation Supports Clinical Determination

Synopsis

  • How to generate value added data that ensures clinical determinations and endpoint assessments are based on the analysis from a robust analytical method validation
  • Define what method validation really entails and the attributes that are critical to understand
  • Review the controls that are fundamental for clinical determination and the components that support validation methods
  • Reflect on how to apply the Quality By Design principles in practice and provide a roadmap to pathway success

12:50 pm Lunch & Networking

2:19 pm
REINFORCING COMPARABILITY ASSESSMENTS WITH STATE OF THE ART ANALYTICAL TOOLS

2:20 pm Quality Attributes and Extended Characterization of Gene Vectors Using Light Scattering

  • John Champagne Senior Applications Scientist and Northeast Regional Manager, Wyatt Technology

Synopsis

  • The new LNP Analysis method enables size-based nucleic acid payload
  • MD-SEC and MD-FFF are essential tools for measuring LNP size, concentration, payload, and product quality
  • MD-SEC and MD-FFF are automated, robust, easy to adopt, minimum hands-on time, less prone to experimental errors

2:50 pm Overcoming the Limitations of Antiquated Technologies

  • Rachael Ahern Associate Director, Quality Control, Tessera Therapeutics

Synopsis

  • How to manage variabilities in data caused from the methods commonly used in drug characterization
  • Evaluate the impact of transitioning to more cost effective and higher throughput technologies on comparability assessments
  • Discuss the best approaches to take for late stage comparability tests when early methodology becomes obsolete or no longer fit for purpose

3:20 pm Afternoon Networking Break

3:50 pm Virtual Presentation: Characterization of Lentiviral Vectors Using Capillary Electrophoresis Platform Technology

Synopsis

  • Overview the key characteristics of lentiviral vectors (LLVs) and their applications in cell and gene therapy
  • Introduce a new workflow for p24 based titer assessment and protein profiling of structural and non-structural proteins
  • Introduce an additional workflow for the size and purity analysis of the lentiviral genome
  • Discuss the use of these workflows to analyze the genome and proteome of lentiviral vector in-process samples

4:20 pm Application of Asymmetric Flow Field-Flow Fractionation coupled to Multi-Angle Light Scattering (A4F-MALS) for the Characterization of AAV Products

Synopsis

  • Development of an A4F-MALS method to evaluate important quality attributes of AAV products, including purity, percent aggregation, capsid particle concentration, and putative empty/full ratio will be discussed
  • Key examples of its application on AAV samples to assess batch-tobatch consistency, effects of accelerated stress, and stoichiometry of AAV-mAb complexes will be presented

4:50 pm Serotype Associated Impurities Under a Production Platform

Synopsis

  • Discovery Viralgen powerful and scalable platform for the production of AAV of diverse serotypes. Numerous batches have been already manufactured in the past years.
  • Understand the key role of data when using a production platform and the value that this data adds to the Viralgen platform process
  • Discuss the comparison of associated impurities (e.g. HCDNA, Full/empty) clustered by serotypes

5:20 pm Keeping up with the Analytics: A Case Study on Viral Titer Method Evolution During The Product Lifecycle & its Impact on Comparability Studies

Synopsis

  • Impacts of changing product development stages and the need for evolving analytics
  • Discussion of CMC, Regulatory and Clinical challenges for viral titration analytical method development during product development lifecycle
  • How to address the requirements of viral titer analytics for different stakeholders during a product lifecycle

5:50 pm Chairperson’s Closing Remarks