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.

TUESDAY, OCTOBER 4 | 2:55 PM

Immunogenicity Assessment and Clinical Relevance

TABLE: AAV-Based Gene Therapy Modalities
Boris Gorovits, PhD, Vice President, in vitro Pharmacology, Biomarker Discovery and Bioanalysis, Sana Biotechnology

  • What is the current industry experience related to methodologies to determine anti-GTx immunity?
  • Value and main challenges related to harmonization of anti-GTx assays
  • What are the recommendations generated during industry-wide conversations?

TABLE: Strategies to Improve Drug Tolerance and Manage Interference
Arkadeep Sinha, PhD, Principal Scientist, EMD Serono

  • New techniques for improving drug tolerance/target interference
  • Effect of drug tolerance mitigation strategies on cut point factors through reduced variability of naïve sample responses
  • Target selection in the face of glycosylation

TABLE: Clinical Relevance of ADA
Tatyana Yun, PhD, Senior Scientist, Merck

  • Cost of developing assays and new techniques
  • Collecting good data
  • Detecting ADA
  • Feedback from the FDA

THURSDAY, OCTOBER 6 | 9:30 AM

Immunogenicity Prediction and Control

TABLE: Risk Assessment and Mitigation Strategies for Immunogenicity of Life Saving Therapeutic Proteins and Novel Treatment Modalities: Deimmunization and Immune Tolerance Strategies
Amy Rosenberg, MD, Senior Director of Immunology and Protein Therapeutics, Epivax

  • Strategies to prevent or mitigate immunogenicity of therapeutic proteins and application of these strategies to prevent/mitigate immunogenicity of novel modalities: Cell and gene therapies
    • For what clinical scenarios is each strategy (deimmunization, immune suppression, therapeutic drug monitoring, immune tolerance induction) appropriate
    • Retreatment of patients with AAV vectored therapeutics is blocked by humoral immunity to capsid
    • Preexisting immunity to key elements of gene therapies (AAV, Cas9 proteins) may preclude patients from entry into clinical trials
  • Transplantation of gene corrected Autologous Hematopoietic Stem Cells mediated by lentiviral vectors in patients with “inborn errors of metabolism”- strategies for implementation
    • Can lentivirus integration be targeted to avoid integration into oncogenes leading to tumorogenicity? To optimize expression/production of the therapeutic
    • What level and duration of immune suppression will be required post-transplant to prevent rejection of gene corrected Autologous HSC?

TABLE: Immunogenicity Prediction - Translation into the Clinic
Klaudia Kuranda, PhD, Head of Immunology, Spark Therapeutics

  • Are animal models of gene transfer able to predict immune response in humans? While immune-related toxicities seem to be rare in the clinic, is there a possibility that animal models are predictive, but the number of animals used for the preclinical studies is too low?
  • The immunogenicity assay toolbox improves every day. Do we have right assays today to detect anti-AAV or anti-transgene responses in animals? Can in vitro assays be used instead and which one?
  • How can the preclinical data generated be used in the clinic?  What is the true value of the pre-clinical assays and how can this be proved?

Optimizing Bioassays for Biologics

TABLE: Defining a “Phase Appropriate” Bioassay
Nancy Sajjadi, Independent Quality Consultant, Sajjadi Consulting

  • What does it mean to adopt a lifecycle approach to bioassay development and validation? 
  • What matters most to regulators at Phase I, II and III when it comes to measuring potency?
  • How to choose an Interim reference standard(s) and transition to a Primary reference standard and a Working standard?

TABLE: Collaboration with your Statistician
Perceval Sondag, Senior Director of Data Science, Head of Scientific Analytics, Novo Nordisk

  • When should bioassay scientists require help from a statistician?
  • What can bioassay scientists learn to do themselves?
  • When should bioassay scientists perform Design of Experiments?
  • Should bioassay scientists use Statistical Process Control for their assays?


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