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Webcast | Virtual

Event Title
FDA Grand Rounds – Adeno-associated Virus-mediated Gene Therapy: Advances, Immune Challenges, and Research Innovations
February 19, 2026

Date:
February 19, 2026
Time:
12:00 p.m. - 1:00 p.m. ET

Webcast Recording

For technical assistance please contact: Rakesh.Raghuwanshi@fda.hhs.gov

Speaker

Ronit Mazor, PhD

Ronit Mazor, PhD
Principal Investigator & CMC Reviewer
Gene Transfer and Immunogenicity Branch
Division of Gene Therapy 2
Office of Gene Therapy
Office of Therapeutic Products
Center for Biologics Evaluation & Research
U.S. Food and Drug Administration

About the Speaker

Dr. Ronit Mazor is a principal investigator and lab head in the FDA’s Gene Transfer Immunogenicity Branch in the Center for Biologics Evaluation and Research, where she leads a multi-disciplinary research team focused on evaluating and mitigating immunogenicity of gene therapy products. She serves as a primary reviewer of chemistry, manufacturing and controls submissions for gene therapy products and BLA chair for first-in-class gene therapy products. Dr. Mazor's scientific contributions include high-impact publications, patents, and several FDA awards including the Center Director Awards. Her research encompasses T cell immunology, algorithm-based HLA binding predictions, investigation of sex-based immunological differences in gene therapy outcomes, and development of tolerogenic approaches to enhance therapeutic efficacy. Prior to joining FDA, she conducted research at the National Cancer Institute developing immune tolerance strategies for cancer immunotoxins and served as a research lead at AstraZeneca focusing on immunogenicity prediction for therapeutic proteins. Dr. Mazor holds a Ph.D. in Molecular Immunology from Tel Aviv University through the NIH Graduate Partnership Program and completed her postdoctoral training at the National Cancer Institute.

About the Presentation

This lecture will introduce gene therapy technology and examine the immunological challenges facing AAV gene therapy, including pre-existing immunity, innate and adaptive immune responses, and transgene immunogenicity. It will highlight CBER's regulatory science research addressing these barriers through computational capsid engineering, sex-based immunological studies and additional factors affecting product characterization and safety assessment.

 

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