Skip to main content
          Skip to FDA Search
          Skip to in this section menu
          Skip to footer links


U.S. flagAn official website of the United States government

					Here’s how you know
          			Dot gov**The .gov means it’s official.**Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you're on a federal government site.
							      SSL**The site is secure.** The **https://** ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. 
			U.S. Food and Drug Administration
				
        Search
            Menu
							
						Search FDA
									
									Submit search 
		Featured
							Contact FDA
	                FDA Guidance Documents
	                Recalls, Market Withdrawals and Safety Alerts
	                Press Announcements
	                Warning Letters
	                Advisory Committees
	                En Español

					Products
            Food
							Drugs
							Medical Devices
							Radiation-Emitting Products
							Vaccines, Blood, and Biologics
							Animal and Veterinary
							Cosmetics
							Tobacco ProductsTopics
            About FDA
							Combination Products
							Regulatory Information
							Safety
							Emergency Preparedness
							International Programs
							News and Events
							Training and Continuing Education
							Inspections and Compliance
							Science and ResearchInformation For
            Consumers
	            Patients
	            Industry
	            Health Professionals
	            Federal, State and Local Officials 
 In this section:

                Workshops, Meetings & Conferences (Biologics)

Workshops, Meetings & Conferences (Biologics)

                        Webinars and Outreach Home
          Vaccines, Blood & Biologics
          News & Events (Biologics)
          Workshops, Meetings & Conferences (Biologics)
          Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products - 12/16/2022Workshops, Meetings & Conferences (Biologics) Workshop
    Event Title
              Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products


                  December 16, 2022

  

    
  Share
    Post
    Linkedin

    Email
    PrintOn This Page
      Meeting Information
                Event MaterialsDate:
          
  


     
           

December 16, 2022   

 
 
        
                                      Time: 
                                      
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.

Background:

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 AnalysesRecordings

Genome Editing Workshop, Recording 1
	Genome Editing Workshop, Recording 2DATE:

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

LOCATION:

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

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.

FOR FURTHER INFORMATION CONTACT:

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).





Event Materials

  Title
            File Type/SizeAssessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products Workshop Tentative Agenda
              pdf               (107.25 KB)Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products Workshop Speaker Bios
              pdf               (435.29 KB)Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products: An FDA Public Workshop
              pdf               (181.33 KB)

                                                                                  Content current as of:
      08/24/2023
         
                   
Regulated Product(s)
		 BiologicsMore Meetings,
      Conferences, and
      Workshops
        
        Workshops, Meetings & Conferences (Biologics)

          
                        Webinars and Outreach
 
 Footer Links
    FDA Archive
          About FDA
          Accessibility
      Visitor Information
          Website Policies / Privacy
          No FEAR Act
          Vulnerability Disclosure Policy
      FOIA
          HHS.gov
          USA.gov
    Contact FDA
      Follow FDA on Facebook
      Follow FDA on X
      Follow FDA on Instagram
      Follow FDA on LinkedIn
      View FDA videos on YouTube
      Subscribe to FDA RSS feeds
    FDA Homepage
    Contact Number
      1-888-INFO-FDA (1-888-463-6332) Back toTop