Conference Agenda

8:50 am Chair’s Opening Remarks


9:00 am Clinical Enrolment Assay to Detect Pre-existing Neutralizing Antibodies to AAV6 With Demonstrated Transgene Expression in Gene Therapy Trials

  • Liching cao Director - Bioanalytical Operations, Sangamo Therapeutics


  • Clinical enrollment cut off determination for pre-screening patients supporting AAV6 gene therapy trials
  • Does assay format matter? NAb or TAb approach for clinical trial eligibility criteria
  • Explore AAV6 seroprevalence evaluations

9:30 am Employing AAV immunogenicity predictive models for risk assessment of gene therapy products


  • Learn about the use of novel assays and platforms that can be implemented during the developability of gene therapy products
  • Develop and implement novel predictive immune assays for product characterization and risk assessment of critical quality attributes (CQA) testing
  • Understand the mechanism related to AAV-mediated immune responses which can provide potential solutions to modulate vector immunogenicity

10:00 am Morning Refreshments


Pre-clinical Evaluation of Immunogenicity and Transduction

11:30 am Reduction of peripheral transduction of AAV9 delivered to the CNS

  • Roberto Calcedo Vice President - Preclinical & Immunology, Affinia Therapeutics


  • IVIG pre-treatment block peripheral organ transduction of AAV9 delivered to the CNS
  • IVIG pre-treatment reduces humoral response to AAV9 vector
  • AAV9 transduces DRGs (Dorsal Root Ganglion) directly from the CNS compartment

12:00 pm A QSP approach for Modeling and Species-Translation of Biodistribution, Transduction and Efficacy of AAV-based Gene Therapies

  • Jatin Narula Principal Scientist Biomedicine Design, Pfizer


  • Data-driven identification of Dose, Serotype, Construct, Target tissue and Species dependencies of AAV treatment response
  • Role of modeling in preclinical evaluation of AAV gene therapies – optimizing biodistribution study designs
  • Use QSP modeling frameworks for species translation and minimum effective dose determination


Immunogenicity Responses to Alternate Routes of Administration

11:30 am The Effect of Administration Route on Immune Response & How Viable Alternative Routes of Administration May Be to Circumvent Immunogenicity


  • Assessing the impact of pre-existing immunity on effective intramoral AAV administration

12:00 pm Modulating AAV-Mediated Immune Response Depending on Route of Administration & Underlying Disease


  • Consideration for prophylactic immunomodulation/ immunosuppression depending on AAV-based gene therapy route of administration
  • Finding a subtle balance between safe immunomodulation and sufficient suppression of AAV-driven immune response
  • Additional safety considerations as they relate to immunosuppression when treating frail patient populations

1:00 pm Lunch & Networking


Exploring Immune Profiling Methodologies to Better Assess Immunogenicity

2:30 pm Circumventing Immunogenicity in Gene Therapy With Svec, a Novel SV40 Based Viral Vector Platform


  • The crucial role of immunity and tolerance in gene therapy
  • Features and development of the SVec platform in Haemophilia
  • Opening new frontiers in gene therapy: Potential to restore tolerance in autoimmunity

3:00 pm In Vitro Immunogenicity Assessment of AAV Gene Therapy Vectors


  • Selecting less inflammatory AAV vector candidates
  • Identifying immunogenic epitopes in AAV capsids
  • Setting up the tools for immunosafety assessment


Immunogenicity Monitoring in a Clinical Setting

2:30 pm Clinical Immunogenicity Assessment Strategies for Cell & Gene Therapy

  • Priya Chockalingam Vice President & Head of Clinical Bioanalytics & Translational Sciences, Beam Therapeutics


  • Discussing LNP based gene therapies
  • Examining Allogenic CART therapies in the context of clinical assessment strategies and cross learning opportunities
  • Clonality in allogenic CART therapies

3:00 pm Applying an Immunogenicity Risk-Based Approach to Gene Therapy Products


  • Current regulatory expectations
  • Immune responses to gene therapies
  • Evaluation and mitigation strategies

3:30 pm Afternoon Refreshments & Networking


4:00 pm Understanding & Engineering Immune Responses for Better Gene Therapies


  • Review of current state of immune suppression and immune responses to gene therapy, particularly adaptive immune responses
  • Novel immune modulatory strategies
  • Development of engineered T regulatory cells for mitigating immune responses to gene therapy

4:30 pm Roundtable Discussion: If Re-Dosing Is a Reality, Then What Must The Field Be Prepared to Face?


The immune response to systemic use of gene therapy is challenge that looms over the field, particularly as questions regarding durability of gene therapy products means
that re-administration seems to look like an increasingly likely reality for many diseases targeted by this drug.

Re-dosing or re-administration is limited by a unique set of challenges itself. The biggest of which being the formation of an anti-AAV and anti-transgene immune response. Consequently, in cases where re-dosing is desirable or unavoidable, immunogenicity stands to be the biggest challenge that the field must work to overcome.

As such this roundtable discussion will set out to address:

  • Durability of gene therapy products and its implications for re-dosing
  • Immune modulation and immunosuppression strategies as an approach to dampen and mitigate immune response
  • If re-dosing is a reality, then what must we be prepared to face?
  • What effect does repeat exposure have on the immune response?
  • The challenges of patient safety in re-dosing
  • What can we expect the need and feasibility of re-dosing to be in the next 10 years?

5:30 pm Chair’s Closing Remarks

5:40 pm End of Conference