8:15 am Online Registration & Virtual Coffee Networking

8:50 am Chair’s Opening Remarks


9:00 am Understanding & Characterization of Immune Responses to AAV Gene Therapy & Preclinical & Clinical Risk Mitigation Strategies

  • Tony Arulanandam Vice President & Head of Gene Therapy Nonclinical Research, PTC Therapeutics


• Understanding innate and adaptive immune responses to AAV gene therapy
• Characterization of the immune response and strategies to monitor immune responses to AAV gene therapy
• Preclinical and clinical risk mitigation strategies of AAV gene therapy

9:30 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

10:00 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

10:30 am Live Q&A – Ask the speakers your questions

11:00 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.


12:00 pm 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

12:30 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

1:00 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

1:30 pm Live Q&A – Ask the speakers your questions

  • Boris Gorovits Senior Director, Pfizer
  • Pierre Burguiere Director - Analytical Development & Nonclinical Research, GenSight Biologics
  • Yanmei Lu Senior Director Bioanlytical Sciences, Sangamo Therapeutics

2:00 pm Lunch & Networking

2:30 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

3:00 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

4:00 pm Chair’s Closing Remarks

4:10 pm Virtual Networking & Happy Hour


The time of day we’ve all been waiting for! Grab a beverage of your choice from your home bar and use this final networking opportunity to follow up on connections made earlier in the day, ask that burning question to a speaker whose talk got you thinking and forge relations that could lead to your next collaboration.

5:30 pm End of Day One