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To Attend

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

Speaker

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.

Learning Objectives

1. Describe the principles of gene therapy and the mechanism of AAV-mediated gene delivery, including AAV vector structure, transduction pathway, and the regulatory landscape of approved AAV gene therapy products.
2. Identify the major immunological barriers to AAV gene therapy, including pre-existing antibodies, innate and adaptive immune responses to capsid and transgene, and their impact on patient eligibility and treatment outcomes.
3. Explain research approaches to address AAV immunogenicity, including computational capsid engineering, sex-based immunological studies, post-translational modification effects, and advanced analytical methods for improved product characterization and safety assessment.

Continuing Medical Education (CME) Credit

As CME credit is being offered through the Johns Hopkins University School of Medicine, those wishing to obtain CME credit must create a user profile on the Hopkins Cloud CME platform. If you have never had a CME certificate/credits awarded and need a user profile created, please log onto https://hopkinscme.cloud-cme.com and click the “Sign Up Now” button. DO NOT create a new account if you have already received credit for Johns Hopkins CME activities at any time. Click on Forgot Password if you already have an email on file with the JHU OCME office. Instructions on how to claim the CME credit will be provided during the Grand Rounds presentation, but participants must already have a Hopkins Cloud CME user profile before the presentation. 

Accreditation Statement

The Johns Hopkins University School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Credit Designation Statement

The Johns Hopkins University School of Medicine designates this live activity for a maximum of 1 AMA PRA Category 1 Credit. Physicians should claim only the credit commensurate with the extend of their participation in the activity. This regularly scheduled series has been approved for a 52-week cycle beginning October 23, 2025.

Other Credits

Reciprocity exists with the following boards that accept AMA PRA Category 1 Credits™ from organizations accredited by the ACCME:

• American Nurses Credentialing Center (ANCC)
• American Academy of Nurse Practitioners National Certification Program (AANP)
• National Commission on Certification of Physicians Assistants (NCCPA)
• Psychologists: JHUSOM is authorized by the Maryland State Board of Examiners of Psychologists as a provider of continuing education. The JHUSOM maintains responsibility for this program.
• Social Workers: The Maryland Board of Social Work Examiners certifies that this program meets the criteria for 1 credit hour of Category 1 of continuing education for social workers licensed in Maryland. The JHUSOM is an approved sponsor of the Maryland Board of Social Work Examiners for continuing education credits for licensed social workers in Maryland.

These professionals should submit their transcript to their respective boards to convert their credits to CEUs.

Policy on Presenter and Provider Disclosure

It is the policy of the Johns Hopkins University School of Medicine that the presenter globally disclose conflicts of interest and any discussion of unlabeled/unapproved uses of drugs or devices during their presentation(s). The Johns Hopkins University School of Medicine OCME has established policies in place to identify and mitigate relevant conflicts of interest prior to this educational activity. Detailed disclosure will be made prior to presentation of the education.