8:20 am Chair’s Opening Remarks


8:30 am An Introduction to AAV Immunogenicity Measurements & Mitigation Strategies


  • Reviewing factors that can impact the immunogenicity of AAV vectors
  • Do we have the appropriate assays for measuring and predicting the immunogenicity of candidate vectors
  • Outlining new immune mitigation strategies

9:00 am Panel Discussion: Aligning Immunomodulation Regimens with Overall Development Goals


  • Outlining early your desired downstream immune response to better inform your regimen selection
  • Key considerations in AAV vector types for this selection process
  • Linking patient population characteristics to the regimen

9:30 am Speaking Position Reserved for Precision for Medicine

10:00 am Morning Refreshments & Speed Networking


This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the gene therapy field and establish meaningful business relationships to pursue for the rest of the conference


Novel Platforms for Risk Prediction & AAV Generation

11:00 am An Adaptive in Silico Immunogenicity Prediction Platform

  • Konstantin Piatkov Senior Scientist, Immunogenicity, Drug Metabolism & Pharmacokinetics Group Lead, Takeda


  • De-immunization effort for the mitigation of immunogenicity risk using in silico analysis
  • Emulating immune response in patients using computational tools
  • Integrative approach to improve the predictability of immunogenicity assessment

11:30 am Machine Learning to Identify the Next Generation of AAV

  • Adrian Veres Co-Founder & Chief Scientific Officer, Dyno Therapeutics


  • What key benefits does machine learning hold over traditional AAV capsid engineering methods?
  • Multi-property optimization to both increase transduction efficacy and reduce the immune response
  • Comparing model evaluation results between machine-guided sequences and traditional methods.
  • Approaches to funnelling through capsid libraries to identify top-performing candidates


Innovative Immunogenicity Studies in Clinical Trials

11:00 am Clinical Trial Design Considerations to Overcome Immune Responses to the Transgene

  • Benjamin Greenberg Vice Chair, Translational Research; Director, Transverse Myelitis, NMO & CONQUER Program, University of Texas Southwestern Medical Center


  • Clinical trial design, prevention, and monitoring of the anti-transgene immune response.
  • Overcoming the key assay development challenges where no positive control exists
  • Exploring readouts from a Phase I Batten disease clinical trial

11:30 am Exploring the Immune Response to AAV Gene Therapies in Immune Privileged Organs


  • Definition of an immune privileged organ
  • Evading immune response in the eye (retina) and inner ear (cochlea)
  • Opportunities for clinical applications

12:00 pm Lunch Break & Networking


Levelling up the Translatability of Preclinical Models

1:00 pm Developing Novel Preclinical Models to Accurately Recapitulate Human Physiology

  • Graça Almeida-Porada Professor - Regenerative Medicine, Wake Forest Institute for Regenerative Medicine


  • An overview of the use of organoids, MPS, and the cell types and processes used in their development
  • Unique considerations for enabling their use in AAV gene therapy preclinical models
  • Performance analysis of organ-on-a-chip platforms in modelling the innate immune response

1:30 pm Evaluating the Impact of Genetic Diversity and Immunogenicity on the Efficacy of AAV Gene Therapy


  • Outlining key reasons for the lack of predictive murine models
  • Using genetically diverse collaborative cross (CC) mice to elucidate drug toxicity and host-pathogen factors that lead to infection persistence or clearance
  • Administering an AAV8 vector to 16 strains of CC mice and evaluating the effects of genetic diversity and adaptive immune responses on efficacy


Overcoming Pre-Existing Immunity

1:00 pm IgG Cleaving Enzymes as a Potential Solution to the NAb Challenge

  • Lena Winstedt Gene Therapy & Global Franchise Lead, Hansa Biopharma


  • An overview of Imlifidase: it’s MOA and translatability for use in conjunction with gene therapies
  • Analyzing data from its use in in-human gene therapy trials
  • Considerations for treatment timing and dosing post-adminstration

1:30 pm A Guide to Combination Therapies: Are IgG Cleaving Enzymes Alone Enough for AAV Gene Therapy Transfer?


  • Introduction to methods to reduce humoral immune response to AAVs
  • Limitations in the use of IdeS in a clinical setting
  • Overcoming IdeS limitations through the use of combination therapies

2:00 pm Afternoon Refreshments & Networking


2:30 pm Utilizing a Risk-Driven Bioanalytical Strategy Prior to Beginning Assay Development

  • Vibha Jawa Executive Director, Bristol Myers Squibb

3:00 pm Fit-For-Purpose Support of Pre-existing Antibody Assessment From Use in Preclinical Development to Use as Companion Diagnostic


  • Purpose of bioanalytical and diagnostic assay
  • How to set an appropriate cut-off for patient selection?
  • Characterization and validation of methods
  • Assay Journey: from preclinical use to use as companion diagnostic

3:30 pm Hope for the Best but Prepare for the Worst: Considerations for Gene Therapy Treatment in Populations with Pre-existing Anti-AAV Antibodies


  • Anti AAV antibody assays or CDx; Context of use: pre-existing versus treatment induced anti-AAV antibodies
  • Benefit risk assessment based on medical need and route of administration
  • Benefit risk assessment based on pre-existing antibodies
  • Patient exclusion or plan to overcome pre-existing immunity

4:00 pm Panel Discussion: The Value & Challenge in Industry Standards for Pre-Screening Assay

  • Vibha Jawa Executive Director, Bristol Myers Squibb
  • Joshua Friedman Vice President, Head of Translational Science & Companion Diagnostics, Spark Therapeutics


  • Standardizing anti-AAV antibody pre-screening: what level of antibody presence should be deemed as too high?
  • Exploring the need for industry-standard antibody measurements to accelerate progress in the field
  • Key challenges and potential solutions for establish reference standards

4:30 pm Chair’s Closing Remarks & End of Day 1