Roundtable Breakout Discussions

These interactive discussion groups are open to all attendees, speakers, sponsors, & exhibitors. Participants choose a specific breakout discussion group to join. Each group has a moderator to ensure focused discussions around key issues within the topic. This format allows participants to meet potential collaborators, share examples from their work, vet ideas with peers, and be part of a group problem-solving endeavor. The discussions provide an informal exchange of ideas and are not meant to be a corporate or specific product discussion.


Immunogenicity Assessment and Clinical Relevance

TABLE: Assessing Vaccine Efforts for COVID-19
Moderator: Theresa Goletz, PhD, President, Theresa Goletz Consulting

  • Clinical candidates: Current outlook for safety and efficacy
  • Regulatory interactions and guidance during a public health emergency
  • Innovation's role in the response to COVID-19 and vaccine development efforts

TABLE: Value of Bioanalytical PMRs – Learnings from Late Stage Clinical Studies
Moderator: Soumi Gupta, PhD, Director, Translational Sciences, BioMarin Pharmaceutical Inc.

  • What types of bioanalytical PMRs have you received?
  • What is the incidence of ADA and clinical relevance of ADA of your biologic?
  • Have the redeveloped bioanalytical ADA assays improved the clinical interpretation, risk assessment of clinical decision making?
  • How are you incorporating ADA testing in the post-marketing testing?
  • Have you been released from PMRs? What was the process?

TABLE: Pre-Clinical Immunogenicity Risk Assessment
 Moderator: Andrea Ferrante, MD, Principal Research Scientist, Eli Lilly & Co.

  • Holistic approach to immunogenicity risk assessment
  • Rapid evolution and improvement of in-silico tools
  • Optimize use of in vitro assays
  • Relevance of animal models
  • Tailor the assessment strategy to the specificity of the compound

TABLE: Assessing the Clinical Relevance of ADA
Moderator: Eric Wakshull, PhD, CEO, Eric Wakshull Consulting

TABLE: AAV Gene Therapies and Redosing
Moderator: Barry Byrne, PhD, Director and Associate Chair, Child Health Research Institute, University of Florida


Immunogenicity Prediction and Control

TABLE: Overcoming Technical Issues with Assays to Assess Innate Immune Response Modulating Impurities
Moderator: Daniela Verthelyi, MD, PhD, Chief, Laboratory of Immunology, CDER, FDA

  • What are the best testing platforms?
  • Overcoming formulation issues
  • Assessing assay design

TABLE: Immunogenicity Prediction in The Real World: Feedback from the Users
Moderator: Bernard Maillere, PhD, Research Director, Immunology, CEA
Immunogenicity teams are invited to share their own experiences

  • Is there a global or case-by-case strategy to assess the risk of immunogenicity?
  • How is the choice of prediction technologies guided?
  • What are the warning signs to discontinue the development?
  • Are all studies internalized or outsourced?
  • Is there any feedback on the prediction strategy of using internal vs. external clinical data?

TABLE: Mechanisms of Immunogenicity of Gene Therapy Products
Moderator: Ronit Mazor, PhD, Principal Investigator, CBER, FDA

  • Capsid VS. transgene immunogenicity
  • Clinical mitigation strategies 
  • Vector tropism or targeting to specific organs and its impact on immune response
  • CpG content of transgene and vector

TABLE: Different Strategies to Predict Immunogenicity
Moderator: Sivan Cohen, PhD, Scientist, Genentech

  • Development and optimization of T cell assay
  • Can we rely on in silico algorithms to predict immunogenicity?
  • What are the best controls to use in ex vivo T cell assays to predict immunogenicity?
  • How sensitive are the immunogenicity prediction assays?

TABLE: Viability of Immune Tolerance Strategies for the Treatment of Human Disease
Moderator: Stephen Miller, PhD, Professor of Microbiology-Immunology, Feinberg School of Medicine, Northwestern University

  • What are the appropriate disease indications for testing tolerance therapy in initial clinical trials?
  • What factors are important in developing the ideal approach for tolerance induction
  • What immune tolerance induction approaches are currently in development for disease prevention and therapy?
  • What are the similarities and differences in the mechanism(s) of action by the different tolerance approaches?

TABLE: New Molecular Insights for Antibody and Protein Drug Immunogenicity
Moderator: Brandon DeKosky, PhD, Assistant Professor, The University of Kansas 

  • Unique immunogenicity features of antibodies compared to other classes of protein drugs
  • Elucidating the mechanisms of epitope spreading
  • Immunogenicity of injected proteins vs. gene therapies
  • Optimal monitoring and assay development for immunogenicity analysis


TABLE: Potency Assays
Moderator: Catherine Liloia, Associate Director, Cell Lab, PPD, Inc.