8:00 am Check-In & Coffee

8:55 am Chair’s Opening Remarks

  • Elben Guimaraes Senior Manager - Manufacturing - Gene Therapy, Ultragenyx


9:00 am Navigating the Transition: From CMO Manufacturing to In-House Manufacturing in Cell and Gene Therapy Production

  • Elben Guimaraes Senior Manager - Manufacturing - Gene Therapy, Ultragenyx


  • Discussing the benefits of having additional in-house manufacturing capabilities alongside with CMO for cell and gene therapy production
  • Highlighting the need for flexibility and control in the manufacturing process for cell and gene therapies
  • Emphasizing the unique challenges and opportunities of constructing a largescale cell and gene therapy facility

9:30 am Navigating Variability and Scalability Challenges in AAV production

  • Rafal Garus Associate Director MSAT BT Analytical Methods, Europe, Bayer


  • Emphasizing the need for improved analytical methods and variability measurement to ensure product consistency
  • Addressing the challenge of maintaining product consistency when operating at various production scales
  • Discussing potential strategies and approaches to address the challenges related to product quality and scalability

10:00 am Method Validation in Gene Therapy: Ensuring Consistency Across Sites

  • Shibu Poulose Senior Scientist - QCAT Gene Therapy, CSL Behring


  • Exploring the intricacies of transferring methods from originating sites to CMOs
  • Learning about the key elements and best practices required to ensure that test results consistently meet established standards, regardless of the testing site
  • Discovering strategies and techniques for successfully addressing the critical role of method comparability in gene therapy development and regulatory compliance

10:30 am Morning Break and Speed Networking


Our speed networking is the ideal opportunity to get face-to-face time with the brightest minds working in gene therapy comparability and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against industry leaders and establish meaningful business relationships to pursue for the conference and beyond.

11:30 am Navigating the Difficulties of Initial Forecasting and Scaling Predictions


  • Navigating how to complete initial forecasting and scale predictions for approval
  • Addressing the feasibility of manufacturing phase 3 pivotal material during early stages
  • Strategies to adjust and address comparability issues with the FDA

12:00 pm Panel Discussion: Navigating the Complexities of Gene Therapy Manufacturing: Ensuring Comparability Across Scales and Locations


  • Addressing variations in manufacturing process to ensure consistent product quality and discussing strategies for managing and minimizing process variability
  • Exploring the critical role of raw materials in gene therapy manufacturing
  • Strategizing how to safeguard product quality throughout the supply chain

12:30 pm Lunch & Networking


1:30 pm Round Table Discussions: Analyzing the Latest Update from the FDA Review on the Comparability Guidelines

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


Join us for an enlightening session as we delve into the recent update from the FDA on comparability guidelines and its potential implications for the evolving landscape of gene therapy development. In this session, we will explore diverse viewpoints from experts in the field, shedding light on the key facets of the FDA’s latest guidelines and how they may impact the gene therapy industry.

Explore diverse viewpoints on the FDA’s latest comparability guidelines and their potential impact on gene therapy development

Investigate the practicality and effectiveness of the FDA’s recent guidelines in streamlining the comparability assessment process

Navigate the relationship between the guidelines and the risk assessment strategies employed by industry stakeholders

Strategize how the FDA’s perspectives on comparability will evolve over time

2:15 pm Leveraging ELISA and PCR for Potency Assay Development in AAV-Related Gene Therapy to Aid Comparability


  • Exploring challenges in characterizing and analyzing AAV-related gene therapy products
  • Designing ELISA-based assays for quantifying AAV vector titers and assessing their functional activity
  • Strategies for designing PCR-based assays to validate gene expression and therapeutic payload delivery

2:45 pm Afternoon Break

3:30 pm Fireside Chat: Navigating Process Changes and the Assurance of Comprehensive Testing to Minimize Challenges in Gene Therapy Comparability

  • Sreeti Choudhury Associate Vice President, Analytical Sciences and Quality Control in Cell and Gene Therapy, Castle Creek Biosciences, Inc.
  • Samarendra Mohanty President and Chief Scientific Officer, Nanoscope Therapeutics Inc


  • The potential impact of process changes on product quality and consistency
  • Identification of challenges that arise when implementing process changes
  • Developing and executing comparability protocols to systematically evaluate the impact of process changes
  • Strategies for assessing and mitigating risks associated with process changes


4:00 pm Roundtable Discussion: Gene Therapy Comparability Regulatory Engagement Strategies and Navigating International Regulatory Variances for Global Comparability


  • Delve into the regulatory tools available for managing comparability in the dynamic world of gene therapy, including the strategic roles of meetings, comparability protocols, and staggered filing approaches amidst rapid development and changes
  • Examine the implications of the evolving FDA comparability guidance for gene therapy products, emphasizing key aspects amid ongoing public comment periods
  • Analyze the differences in comparability requirements across international regulatory agencies, gaining a nuanced understanding of the global landscape
  • Learn effective strategies to ensure that gene therapy products meet diverse standards for comparability on an international scale
  • Gain insights into building robust communication channels with various regulatory bodies, fostering collaboration and ensuring successful approvals worldwide

4:45 pm Chair’s Closing Remarks

  • Elben Guimaraes Senior Manager - Manufacturing - Gene Therapy, Ultragenyx