8:15 am Online Registration & Virtual Coffee Networking

8:50 am Chair’s Opening Remarks


9:00 am Immunogenicity Risk Assessment for AAV Gene Therapies – the What, the Why, & the How


• Immunogenicity Risk Assessment is a commonly used tool for monoclonal antibodies
• The approach consists of identifying potential risks followed by risk evaluation and identification of a risk mitigation strategy
• The outcome is an appropriate, cost-effective, testing approach to describe the immunogenicity of AAV gene therapies and its impact on safety and efficacy

9:30 am Intrathecal Gene Therapy for Giant Axonal Neuropathy: Immunological Considerations


• A case study exploring the first in human intrathecal delivery of AAV9 gene therapy for CNS targeting
• Formulating an immuno-modulatory strategy to mitigate the innate, anticapsid & anti-transgene immune response after intrathecal delivery
• Exploring how immune modulation strategies can be translated to other indication areas and routes of gene therapy delivery

9:50 am A Review of the Classical Gene Therapy Landscape


  • Analysis of the current trial landscape by vector serotype and targeted indication
  • Consideration of what the future classical gene therapy landscape may look like

10:20 am Live Q&A – Ask the speakers your questions

10:50 am Virtual Speed Networking & Morning Break


Recreating face-to-face networking in the virtual world. We will pair you up with fellow attendees at so you can get face-to-face time with the brightest minds working in the gene therapy field in order establish meaningful business relationships to pursue for the rest of the conference.

11:30 am Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays


• All humans may be exposed to natural Adeno-Associated Virus (AAV) infections resulting in an immune response against the virus prior to treatment
• High prevalence of pre-existing anti-AAV immunity may be expected, leading to limitation for AAV-based gene therapy treatment
• Recommendations for the development and validation of cell-based anti-AAV NAb detection methods will be presented


12:00 pm Immunogenicity Assay Challenges and Strategies for Gene Therapy

  • Yanmei Lu Senior Director Bioanlytical Sciences, Sangamo Therapeutics


• Evaluate most appropriate methods for developing and validating assays used to detect gene therapy immunogenicity
• Developing assays to better understand gene therapy immunogenicity
• What challenges must be overcome to develop high performing assays

12:30 pm Safety Bioanalytics in LHON Clinical Studies: Technical Challenges & Solutions & Outcomes

  • Pierre Burguiere Director - Analytical Development & Nonclinical Research, GenSight Biologics


• LHON disease and Gensight GS010 gene therapy & clinical trials (overall design & safety of bioanalytical assays)
• Two examples of challenges faced regarding bioanalytical protocols being used: i) qPCR specificity for assessment of viral particles systemic biodissemination; ii) Impact of blood sampling and blood cells isolation on physiological state of PBMCs used for cellular immune response measurement
• Technical solutions developed to overcome the encountered challenges
• Brief overview of safety bioanalysis outcomes and conclusions

1:00 pm Practical Guide for Immunogenicity Assays: a CRO perspective


ꔷ Leveraging the CRO-Sponsor relationship

ꔷ What Sponsor needs to provide for a successful Immunogenicity assay development

ꔷ Case study of assay development and validation process: bio

1:20 pm Live Q&A – Ask the speakers your questions

1:50 pm Lunch & Networking

2:50 pm Monitoring the Immune Response to Gene Therapy for Inherited Retinal Disease


• Assessing the risk of immunogenicity for gene therapy in ophthalmic disease and the impact on the bioanalytical strategy
• Considerations and challenges for development and validation of assays for monitoring immune responses to gene therapy
• Rapid advances in bioanalysis for cell and gene therapy: opportunities and challenges from the perspective of a small to medium size enterprise

3:10 pm Navigating the immune responses to CRISPR-associated nuclease Cas9

  • Zuben Sauna Principal Investigator & Reviewer, US FDA


• Unique challenges while assessing the immunogenicity risk to Cas9
• The need of validated reproducible assays that are fit-for-purpose
• The need for well characterized reagents and for controlling contaminants and impurities
• Model systems for evaluating the immunogenicity of Cas9

3:30 pm Live Q&A – Ask the speakers your questions

3:50 pm Chair’s Closing Remarks

4:00 pm End of Day One