Opportunities and Challenges of Immunogenicity Assays for AAV Gene Therapies

Recorded: November 2022

This webinar will discuss immunogenicity assays as an integral part of large molecule bioanalysis. Presentations will focus on the practical aspects of developing and validating tests to assess both humoral and cellular immunogenicity. Based on our experience and case studies we will discuss both the advantages and challenges faced by bioanalytical labs while working on such studies.

Agenda

• Challenges with immunogenicity assessments as it pertains to AAV Gene Therapies
  • Regulatory guidance and industry practices related to these products
  • Steps of the development, from early preclinical assessment to the qualified clinical assays
  • Pre-existing immunity and impact on the assay development
  • Identification of appropriate controls for the clinically meaningful assay
• Humoral Immunogenicity testing
  • Multitiered approach to immunogenicity ADA testing
  • Assay Validation parameters
  • Developing a right cutpoint as a critical step of ADA assays
    
• Role of cell-based assays in evaluating immune response: ELISpot and Nab Assays
  • Advantage and challenges of ELISpot technology
  • Case study: Validation of Nab assay for CGT
  • Statistical analysis of the data

Moderated by: 

Benjamin Bronfin, Sr. Director of Operations at Pharmaron

Speakers:

Key Opinion Leader: Zoya Gluzman-Poltorak PhD, VP Therapeutic Development, ASC Therapeutics

Dr. Gluzman-Poltorak has over 20 years of executive-level leadership experience in early development biotechnology companies, advancing several gene, cell and protein-based products from idea through pre-clinical into clinical development in multiple therapeutic areas.  In 2017 Dr. Gluzman-Poltorak pioneered ASC Therapeutics Division to lead the cutting-edge AAV-based genome editing, gene and cell therapy products. Under her leadership ASCTx completed IND-enabling pre-clinical and analytical program for ASC618, AAV based Gene Therapy Hemophilia A program, in less than 1 year.

Dr. Gluzman-Poltorak received her PhD in Cell Biology and Genetic Engineering and MBA from the Technion, Haifa, Israel.

Pharmaron Speakers: 

Kate Patterson, Associate Director BioAnalytical

Kate is the Associate Director of Bioanalytical group at the Boston site of Pharmaron. Ms. Patterson holds a B.Sc. in Animal Science from UMASS, Amherst and has been working at Pharmaron for 13+ years. She has extensive experience in developing and validating e immunoassays like ELISA, Western Blot, ADA, Enzyme activity tests, Luminex etc and served as a PI on multiple GLP, GCP, and GMP projects.

Babette Haven, Associate Director BioAssay

Babette is an experienced scientist and a leader of Bioassay group. She is responsible for different types of cell-based assays including Flow Cytometry, ELISpot, Nab, compound screening etc. Ms. Haven was previously employed at Santa Cruz Biotechnology and received her B.A. in Chemistry from the University of California, Santa Barbara.