8am - 5.45pm EDT

8:50 am Chair’s Opening Remarks

BETTER UNDERSTANDING THE IMMUNE RESPONSE TO GENE THERAPY PRODUCTS

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

Synopsis

  • 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

Synopsis

  • 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

Synopsis

• 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

Synopsis

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

Synopsis

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

HARNESSING THE POWER OF IMMUNOASSAYS TO BETTER MEASURE IMMUNE RESPONSE

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

  • Travis Harrison Vice President Bioassay Solutions, Precision for Medicine

Synopsis

• 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

Synopsis

  • 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

Synopsis

  • 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

Synopsis

  • 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?

Synopsis

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

5:45 pm DAY END