Pre-Conference Workshop Day

Tuesday, February 13, 2024

8:00 am Registration

8:29 am

8:30 am Optimizing Gene Therapy Comparability Study Design for Effective Development

  • Scott Burger Principal Consultant, Advanced Cell & Gene Therapy


Ensuring successful gene therapy development and regulatory approval relies on well-designed comparability

studies. This session will delve into the significance of strategic study planning, enabling developers to make wellinformed

decisions at critical junctures.

Attend this session to:

  • Explore the importance of initiating comparability study design at the early stages of gene therapy development to establish robust process controls and ensure product stability
  • Understand how breaking down comparability studies into multiple parts can provide incremental data insights and enhance decision-making
  • Analyze study design adaptations to accommodate complex manufacturing processes involving multiple sites
  • Learn best practices for study design to align with international regulatory guidelines and facilitate successful approval of gene therapy products

10:30 am Morning Break & Refreshments


11:00 am Enhancing Gene Therapy Comparability through Statistical Solutions

  • Wendy Larson Director - Regulatory Affairs Chemistry, Manufacturing & Controls, Astellas
  • Anita Knott Senior Director - Regulatory Affairs Chemistry, Manufacturing & Controls, Astellas


Learn from the experts as they provide insights, practical solutions, and real-world examples to enhance the

comparability of gene therapy products. Learn how statistical considerations can help overcome challenges

in gene therapy comparability studies and ultimately contribute to the success of these groundbreaking


Get involved and delve into:

  • Navigating limited batches and statistical significance in comparability assessments
  • Exploring statistical methodologies for meaningful comparisons with gene therapy data sets
  • Discussing the importance of providing comprehensive narratives alongside statistical values to facilitate regulatory understanding and acceptance
  • Optimizing analytical methods to minimize variability and enhance comparability outcomes
  • Case studies and best practices for addressing statistical and variability challenges effectively

1:00 pm Lunch


2:00 pm Strategic Learnings from Cell Therapy: Applying Study Design and Regulatory Experience to Gene Therapy Comparability

  • Blair McNeill Senior Vice President & Head of CMC, Sumitovant Biopharma


Join us for an interactive workshop where experts from the cell therapy and gene therapy fields will share

their experiences, best practices, and key learnings. Discover how strategic insights from cell therapy can be

harnessed to enhance study design and regulatory success in gene therapy comparability assessments:

Join this workshop session to:

  • Compare and contrast study design approaches between gene therapy and cell therapy
  • Learn from cell therapy regulatory experiences and how they can inform gene therapy comparability studies
  • Understand the similarities and differences in regulatory requirements for both therapies to optimize interactions with regulatory bodies
  • Discuss novel analytical methods and study designs for assessing immune function in gene therapy, drawing inspiration from cell therapy advancements
  • Assess the transferability of cell therapy learnings to gene therapy, considering the complexities and variables involved