WEDNESDAY, OCTOBER 16 | 2:20 PM
TABLE 1: Immunogenicity of Peptides
Faye Vazvaei, Executive Director, Merck
- FDA's current thinking ADA strategy on peptides.
- Experience on alignment with the FDA new draft guidance: Clinical Pharmacology Considerations for Peptide Drug Product.
TABLE 2: Recommendations for Harmonization of Neutralizing ADA Testing and Reporting
Michele Gunsior, PhD, Senior Director, Astria Therapeutics
- Harmonization of Reporting: Discuss the necessity for standardized reporting in bioanalytical immunogenicity studies to enhance communication of findings and streamline reviews by industry and health authorities.
- Cross-Industry Collaboration: Review the formation and contributions of a cross-industry group tasked with closing key reporting gaps, including their development of harmonized recommendations and a submission template for agency filings.
- Bioanalytical Report Elements: Examine the essential elements of the bioanalytical report (BAR) for anti-drug antibody (ADA) assays, such as method, reagents, equipment, samples, results, and data analysis, and consider the provided template for structuring the ADA BAR.
TABLE 3: When One Size Does Not Fit All: Reevaluating Immunogenicity Risk Assessments and The 3-Tiered Testing Paradigm
Lauren F. Stevenson, PhD, CSO & Head, Translational Sciences, Immunologix Labs
- Revising our thinking on risk assessments—incidence without impact and the myth of the medium risk molecule.
- Lessons learned from over 20 years of data generation—Perspectives on benefits and challenges with the current testing paradigm.
- Mining all the data from the screening tier—How S/N analysis can help fill in the blanks and turn confusion into clarity.
- Immunogenicity is a biomarker—Understanding context of use ensures we don't try to put square pegs in round holes and fail to measure what matters.
- Rethinking the Nab testing tier—If Nab is important to understand, why wait? Differentiating what may be scientifically interesting vs what affects patient safety and efficacy.
FRIDAY, OCTOBER 18 | 9:30 AM
TABLE 1: HESI/AAPS: Towards Harmonization of in vitro T Cell Assays to Predict Immunogenicity of Biologics
Laurent P. Malherbe, PhD, Executive Director, Eli Lilly and Company
- Reference biologics for in vitro T cell assays
- Factors impacting in vitro T cell assays results
- Interpretation of the in vitro T cell assays results to assess the immunogenicity risk of biologics
TABLE 2: End-to-End Immunogenicity: Risk Assessment and Mitigation
Timothy Hickling, PhD, Investigative and Immunosafety Chapter, Roche
- How to build a risk assessment from the start of a new project?
- What does it take to mitigate product risk during molecule design?
- How realistic is clinical mitigation of immunogenicity?
TABLE 3: Immunogenicity of Next-Generation Modalities, Including Antibody-Drug Conjugates
Alexander Kozhich, PhD, Director, Bristol Myers Squibb Co.
- Immunogenicity risk assessment of novel modalities
- Immunogenicity de-risking next-generation modalities
- Bioanalytical strategy and methods
TABLE 4: Potency Assays for Personalized Therapeutics Like CAR T Cells
Dawn Maier, PhD, Cell and Gene Therapy CMC, AD Preclinical Technical Advisor, DGMAIER Consulting
- What are the types of biological assays available?
- What are the challenges associated with those assays?
- Why is potency for personalized therapy more complex than traditional biologics?
- What are potential surrogate assays?
TABLE 5: The Impact of Glycosylation on the Functional Activity of Therapeutic Proteins
Ravish B. Patel, PhD, General Manager, Technical Operations, Kodo Life Sciences
- Fundamental Understanding:
- What are the primary types of glycosylation (N-linked and O-linked) observed in therapeutic proteins?
- How do different glycosylation patterns affect the pharmacokinetics and pharmacodynamics of therapeutic proteins?
- Clinical Implications:
- In what ways can glycosylation impact the immunogenicity of therapeutic proteins?
- How does glycosylation influence the efficacy and safety profiles of biotherapeutics in clinical settings?
- Manufacturing Considerations:
- What challenges do variations in glycosylation present during the manufacturing process of therapeutic proteins?
- How can bioprocessing techniques be optimized to ensure consistent glycosylation patterns?
TABLE 6: Strategies and Challenges in Developing Functional Potency Assays for Biologics and Gene Therapies
Arkadi Manukyan, PhD, Senior Scientist, Bioassay Development, Sanofi
- How can we ensure that the functional potency assay accurately reflects the therapeutics's mechanism of action while maintaining clinical relevance?
- What are the trade-offs between assay complexity and its ability to predict in vivo efficacy?
- What are the primary challenges in selecting and maintaining an appropriate cell-based model for potency assays?
- How do you address variability in cell lines over time and between different laboratories?
- How do you balance assay sensitivity and specificity, particularly when measuring complex biological responses in gene therapies?
- What strategies can be employed to reduce noise and enhance the signal-to-noise ratio?
- What are the most critical steps to ensure regulatory compliance in validating functional potency assays for novel gene therapies and biologics?
- How do evolving regulatory expectations shape assay development, and what best practices can be adopted to meet these standards?
- How can we best address the inherent biological variability of biologics and gene therapies to develop robust and reproducible potency assays?
- What types of controls and standards are most effective in minimizing assay variability and ensuring consistent results across different production batches?