8:50 am Chair’s Opening Remarks

8:55 am
OVERCOMING MANUFACTURING HURDLES TO REVOLUTIONIZE GENE THERAPY COMPARABILITY

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

Synopsis

  • 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 Manufacturing, Science & Technologies BT Analytical Methods, Bayer

Synopsis

  • 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

Synopsis

  • 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

Synopsis

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

Synopsis

  • 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

Synopsis

  • 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

1:00 pm
NAVIGATING REGULATORY CHALLENGES IN GENE THERAPY COMPARABILITY

1:30 pm Roundtable Discussion: Navigating Regulatory Considerations and Challenges in Pre and Post-Approval Gene Therapy Comparability

Synopsis

  • Explore strategies for engaging regulatory agencies in the context of gene therapy comparability, focusing on when and how to initiate discussions for pre- and post-approval changes
  • Discuss the implications of the evolving FDA comparability guidance for gene therapy products, highlighting key aspects even in the face of ongoing public comment periods
  • Delve into the regulatory tools and mechanisms available for managing comparability in the pre- and post-approval world of gene therapy, including the role of meetings, comparability protocols, and staggered filing approaches amidst rapid development and changes

2:00 pm Mastermind: Exploring the Key Differences Between International Regulatory Agencies to Understand Comparability on a Global Scale

  • Melody Dai Senior Director, Regulatory Affairs Gene Therapy, Adverum Biotechnologies

Synopsis

  • Examine the differences in comparability requirements across international agencies
  • Learn effective strategies to ensure your gene therapy products meet diverse global standards for comparability
  • Gain insights into building effective communication channels with various regulatory bodies to facilitate successful approvals

2:30 pm
LEVERAGING CMC EXPERIENCES TO BETTER INFORM COMPARABILITY

2:45 pm Navigating Challenges in CMC Package Submissions

Synopsis

  • Addressing the challenge of limited batch data, including the proposal to manufacture additional batches for compatibility testing
  • Emphasizing the need for forward-thinking strategies in transitioning from experimental processes to commercially viable ones
  • Exploring the regulatory landscape and how regulatory agencies, such as the FDA, respond to innovative approaches
  • Highlighting the importance of risk assessment in justifying the analytical approach and how this can influence regulatory decision-making

3:15 pm Afternoon Break and Poster Session

Synopsis

This is your chance to join the conversation to share your groundbreaking research to leaders from biotech and pharma.

4:00 pm
INNOVATIONS IN GENE THERAPY PROCESS DEVELOPMENT TO AID COMPARABILITY/center>

4:15 pm Leveraging ELISA and PCR for Potency Assay Development in AAV-Related Gene Therapy to Aid Comparability

Synopsis

  • 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

4:45 pm Panel Discussion: Navigating Process Changes and the Assurance of Comprehensive Testing to Minimize Challenges in Gene Therapy Comparability

Synopsis

  • 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

5:15 pm Chair’s Closing Remarks