TUESDAY, OCTOBER 4 | 2:55 PM
TABLE 1: 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 2: 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
TABLE 3: Immunogenicity of CAR T Therapies
Alexander Kozhich, PhD, Director, Bristol Myers Squibb
- Immunogenicity risk assessment
- What is relevant - humoral or/and cellular immunogenicity
- Bioanalytical strategy and methods
TABLE 4: 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
THURSDAY, OCTOBER 6 | 9:30 AM
TABLE 1: 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 2: 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?
CANCELLED: TABLE 3: The Complex Landscape of the MHC-II Ligandome
Laura Santambrogio, PhD, Professor, Associate Director, Precision Immunology, Weill Cornell Medicine
- The multiplicity of translational and post-translational mechanisms affecting the MHC-II restricted immunopeptidome
- How to quantitatively assess the MHC-II ligandome and why it's important to know epitope copy numbers
- MHC-II presented tolerogenic and immunogenic peptides
TABLE 4: 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 5: 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?