PRE-CONFERENCE DAY - Tuesday July 16, 2024

STREAM A – POTENCY TECHNIQUES & ANALYSIS

9:00am – 12:00pm | WORKSHOP A – POTENCY TECHNIQUES & ANALYSIS

Parallel Line Analysis – How Can we Use it to Establish Relative Potency?

  • Sangeet Lal Senior Manager - Cell-Based Potency Assays Development & Quality Control, 4D Molecular Therapeutics
  • Susan Rutberg Director - Bioassays & Molecular Biology, Sanofi

Synopsis

This interactive workshop delves into the critical aspect of determining relative potency in gene therapy potency assays. Relative potency assessment involves comparing the potency of a sample against a reference standard, crucial for ensuring the efficacy and safety of gene therapy products. The session aims to evaluate the different methods of analyzing dose-response curves and establishing parallelisms to develop a comprehensive understanding of relative potency determination.

Attend to Discuss:

  • Introduction to relative potency: understanding the significance of relative potency in gene therapy and its role in ensuring product efficacy and safety
  • Discussing the relevance of the four parameters in dose-response curves
  • Unpicking the requirements for demonstrating parallelism between the dose-response of your reference standard and test sample
  • Demonstrating the utilization of software for four-parameter fits and exploring different statistical methodologies to establish comparability between sample and reference standards
  • Brainstorming how to generate reproducible dose-response curves to supply a sufficient linear range
  • Case studies and practical applications: analyzing sample dose-response curves and discussing example scenarios

1:00pm – 4:00pm | WORKSHOP C – STRATEGIC POTENCY ASSAY DESIGN

To What Extent Can Emerging Cell-Based Assay Techniques Facilitate Potency Assessment?

  • Hwan June Kang Senior Scientist - Biologics Research & Gene Therapy Analytical Development, Neurocrine Biosciences

Synopsis

Emerging cell-based assay techniques are playing a critical role in advancing potency assessment. This session will delve into the potential and practicalities of integrating cutting-edge methodologies to redefine the landscape of potency assessment.

Attend to Discuss:

  • Outlines of examples of innovative cell-based assay platforms including alphaLISA and highthroughput techniques
  • Discussing the benefits and potentials of these technologies in improving assay specificity, sensitivity, and reproducibility to determine relevance
  • Dissecting case studies and success stories demonstrating the integration of emerging cell-based assay techniques in potency assessment
  • Understanding the practical considerations for implementing and validating new assays
  • Highlighting and potential deficits of new technologies compared to traditional techniques to understand limitations

STREAM B – STRATEGIC POTENCY ASSAY DESIGN

9:00am – 12:00pm | WORKSHOP B – POTENCY TECHNIQUES & ANALYSIS

Are All My Assays Necessary? Rethinking the Conventional Potency Matrix Approach

  • Adnan Arnaout Sr. Principal Scientist, Analytical Development, Prevail Therapeutics

Synopsis

The potency matrix is the conventional approach to measuring product efficacy from the DNA through to the protein level. This workshop aims to critically analyze and redefine the conventional potency matrix approach in gene therapy and explore strategies for streamlining potency assays while meeting regulatory expectations.

Attend to Discuss:

  • Brainstorming the necessity of performing all assays in the conventional potency matrix
  • Evaluating the utility of the TCID50 to determine whether it is too variable for effective potency assessment
  • Discussing regulatory perspectives on the inclusion of mRNA expression assays in the case of existing MoA potency assay platforms
  • Balance the quantity of assays performed with the quality of regulatory submissions

1:00pm – 4:00pm | WORKSHOP D – STRATEGIC POTENCY ASSAY DESIGN

Unpicking the Details of the New FDA Guidance: “Potency Assurance for Cellular & Gene Therapy Products” – Changes, Learnings & Implications

  • Natalie Fekete Manager, Science & Industry Affairs, Alliance for Regenerative Medicine

Synopsis

This workshop aims to dissect the recently released FDA guidance on potency assurance for cellular and gene therapy products, analyze the alterations, extract key learnings, and discuss the broader implications for assay development in the field of gene therapy.

Attend to Discuss:

  • Outlining the major changes made to the new FDA guidance to understand the reasoning behind the updates
  • Making comparisons with the previous guidance and identifying significant shifts or emphasis changes
  • Discussing how the new guidelines should be interpreted and implemented to guide novel assay design approaches
  • Introducing case-study examples of the new guidance in practice
  • Discussing potential hurdles and opportunities in implementing revised guidelines
  • Outlining how the new guidance may influence your potency assay strategy going forward
DAY ONE
DAY TWO