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  4. Workshop on Integration Site Analysis During Long Term Follow-Up for Gene Therapies with Integrating Viral Vectors - 11/14/2024
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Workshop | Virtual

Event Title
Workshop on Integration Site Analysis During Long Term Follow-Up for Gene Therapies with Integrating Viral Vectors
November 14, 2024

Date:
November 14, 2024
Time:
9:30 AM - 5:00 PM ET
Organized By:
Organizer

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting a virtual scientific public workshop on the clinical use of integration site analysis (ISA) during long term follow-up following administration of gene therapies with integrating viral vectors. In this workshop, FDA will convene a panel of external experts to discuss the risk of insertional mutagenesis and best practices for ISA method design, data analysis, and clinical interpretation. The workshop participants will include research scientists with experience in vector design and ISA, as well as clinician-scientists with expertise in clinical interpretation of ISA data.

Meeting Logistics and Registration:

Focus for this Workshop

As of 2024, twelve approved and hundreds of investigational cell-based gene therapies use integrating viral vectors. While stable integration may have considerable therapeutic benefits, integration into genomic DNA can alter host gene expression and may contribute to secondary malignancies via insertional mutagenesis. Additionally, the choice of vector (e.g., lentivirus versus gammaretrovirus) and cell substrate (e.g., T cells versus CD34+ cells) may also contribute to this risk.

ISA is the established approach to identify vector insertion sites. However, ISA method design and data interpretation are highly complex and product-specific. Moreover, ISA methods may be costly and challenging to perform. There is a critical need to understand the best practices for product design and ISA method design, execution, and interpretation to facilitate efficient evaluation of complex gene therapy products and protect the public from potential serious adverse events.

This workshop will include discussions on the following topics:

  • Effects of Cell Substrate and Vector Design on the Risk of Vector Insertion-Related Adverse Events
  • ISA Methodology, Assay Design, and Analytical Capabilities
  • ISA Analysis, Quality Control, and Data Reporting
  • Clinical Follow-Up for Integrating Viral Vectors
  • Using ISA to Inform Clinical Decisions

For current guidance on the role of ISA in clinical follow-up, please refer to section V.F “Special Considerations Regarding Integrating Vectors” of the FDA Guidance for Industry “Long Term Follow-Up After Administration of Human Gene Therapy Products” dated January 2020 (https://www.fda.gov/media/113768/download).

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