8:15 am Online Registration & Virtual Coffee Networking

8:50 am Chair’s Opening Remarks

DEVELOPING IMMUNOGENICITY RISK ASSESSMENT AND MODULATION STRATEGIES

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

Synopsis

• 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

Synopsis

• 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

Synopsis

• 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

Synopsis

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.

HARNESSING THE POWER OF ASSAYS TO BETTER MEASURE IMMUNE RESPONSE

12:00 pm Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays

Synopsis

• 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

Synopsis

• 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

Synopsis

• 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

Synopsis

• 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

Synopsis

• 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

Synopsis

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