U.S. flag An official website of the United States government
  1. Home
  2. Vaccines, Blood & Biologics
  3. News & Events (Biologics)
  4. Workshops, Meetings & Conferences (Biologics)
  5. Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products - 12/16/2022
  1. Workshops, Meetings & Conferences (Biologics)

Workshop | Virtual

Event Title
Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products
December 16, 2022

December 16, 2022
9:00 AM - 4:00 PM ET

The Food and Drug Administration (FDA) Center for Biologics Evaluation and Research is announcing a public workshop entitled “Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products”.

Purpose of the workshop:

The purpose of the public workshop is to discuss the impact of individual genetic heterogeneity on genome editing, and best practices for validating off-target editing events in the context of human genetic heterogeneity. The public workshop will bring together academia, industry, and other stakeholders involved in research, development, and regulation of Genome Edited gene therapy products.


Genome editing products hold promise for the treatment of many diseases with unmet medical need. However, the technologies used to assess the safety of genome editing products, with respect to off-target editing, are continuously evolving. Specifically, it is currently unclear how genetic heterogeneity should be factored in when performing validation of off-target editing events. This is problematic given the possible effect genetic heterogeneity may have on the presence of possible off-target editing sites.

The objective of this workshop is to gather scientific information on current technologies regarding off-target editing analyses for genome edited gene therapy products and how off-target editing events should be validated considering human genetic heterogeneity. The use of comprehensive methods or approaches for determining editing at off-target sites is key for ensuring that patients consistently receive safe and effective products during every stage of clinical development.

Topics for Discussion at the Public Workshop

  • Definition and identification of genetic heterogeneity in genome editing
  • Overview of Approaches/Methods for Analyzing Genetic Heterogeneity
  • Strengths and weaknesses of genome editing off-target methods
  • Validation of Off-target Events
  • Impact of Genetic Heterogeneity (GH) on Off-target Editing Analyses



December 16, 2022
9:00 AM – 4:00 PM EST


This is a virtual webcast only event. The link for viewing/participation will be provided within your Eventbrite confirmation upon registration.


To register for the public workshop, please visit the following website: https://www.eventbrite.com/e/genetic-heterogeneity-in-genome-editing-public-workshop-tickets-429332825037. Registration is free and based on space availability. If you need special accommodations due to a disability, please contact Loni Warren Henderson no later than December 2, 2022 (subject line: Genome Editing Workshop) at the contact information provided below.


Loni Warren Henderson, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 1118, Silver Spring, MD 20993, 240-402-8010, or 240-402-8180, or CBERPublicEvents@fda.hhs.gov (subject line: Genome Editing Workshop).

Back to Top