8am - 5.45pm EDT

8:50 am Chair’s Opening Remarks


9:00 am Evaluation Of Antibody-Based Immunity Against Viral Gene Therapy Modalities

  • Boris Gorovitz Vice President, In vitro Pharmacology, Biomarker Discovery & Bioanalysis at Development Sciences, Sana Biotechnology


  • High prevalence of pre-existing anti-gene therapy has been reported and is typically evaluated prior to treatment. Induction of post-dose antibody response is expected
  • Appropriate assessment of potential subjects often includes evaluation for the neutralizing of the total antibody anti-gene therapy
  • Recommendations for NAb and TAb methods, including suggestions for key assay parameters will be discussed

9:30 am Role Of Innate Immunity In Cd8+ T Cell Responses In AAV Gene Transfer

  • Roland Herzog Riley Children’s Foundation Professor of Immunology, Indiana University


  • PAMPs: TLR9 sensing of the viral genome
  • Role of dendritic cell subsets in CD8+ T cell activation
  • DAMPs: TLR9-independent responses

10:00 am Cleanrooms on Demand – A novel approach to accelerate cGMP manufacturing for Gene Therapies


• Current industry challenges in terms of gene therapy manufacturing capacity and capability
• Existing options for early phase clinical manufacturing and the challenges (Build vs Buy)
• Azzur’s Cleanrooms on Demand hybrid model designed to accelerate gene therapy products

10:30 am Have The Speakers Answer Your Questions

  • Boris Gorovitz Vice President, In vitro Pharmacology, Biomarker Discovery & Bioanalysis at Development Sciences, Sana Biotechnology
  • Roland Herzog Riley Children’s Foundation Professor of Immunology, Indiana University
  • Ravi Samavedam President & COO, Azzur Group

11:00 am Speed Networking & Morning Break


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.

12:00 pm PANEL: Better Understanding Mechanisms of Immune Response in Different Areas of the Body & its Implications for Delivering Gene Therapy


This panel will explore the latest understandings of the immune response and challenges associated with systemic delivery


12:30 pm Development of a Neutralizing Antibody Assay as a Diagnostic

  • Travis Harrison Vice President Bioassay Solutions, Precision for Medicine


• Assays that measure neutralizing antibodies (NAb) have become an essential part of the development process for many gene therapeutics
• When NAb assays are used to determine eligibility for treatment, there are heightened regulatory concerns
• Development strategy for use of a NAb assay as a diagnostic assay for a pivotal clinical trial and subsequent use as a companion diagnostic

1:00 pm Regulatory considerations for immune responses to gene therapies

  • Andrew Byrnes Chief, Gene Transfer and Immunogenicity Branch, US FDA


  • Codevelopment of anti-vector antibody assays
  • Potential impact of product quality on immunogenicity

1:30 pm Lunch & Networking

2:30 pm Performances of Biosafety Analytical Assays Commonly Used in Gene Therapies Development


  • Introduction of biosafety analytical assays commonly used in gene therapy development such as monitoring of cellular and humoral immune responses of biodissemination
  • Presentation of some intrinsic limitations and their impact on the biological relevance of these analytical protocols to monitor patients biosafety in clinical trials using gene therapy (AAV)
  • Discussion of the potential need for the gene therapy industry to develop novel biosafety analytical assays in close collaboration with regulatory authorities

3:00 pm Strategy to Monitor an Anti-AAV Cell Based Transduction Inhibition Assay Supporting Clinical Trials

  • Liching Cao Director, Bioanalytical Operations, Sangamo Therapeutics


  • Challenges in assay maintenance and monitoring
  • Sharing Case studies to illustrate how to monitor clinical enrollment assay and to bridge reagents without the need to adjust enrollment cutoff

3:30 pm Afternoon Break & Poster Session

4:00 pm Cas9 Immunogenicity: The Need for Fit-For-Purpose Non-Clinical Assays Prior to Introducing CRISPR Genome Editing into Clinics

4:30 pm Have The Speakers Answer Your Questions

5:00 pm PANEL: Do We Have Good Enough Bioanalytical Tools?


Exploring current bioanalytical tools available for measuring immune response and assess whether current methods are up to scratch

5:45 pm DAY END