Genetic Heterogeneity in Genome Editing; Public Workshop

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Genetic Heterogeneity in Genome Editing; Public Workshop

What is the impact of human genetic heterogeneity on human genome editing and best practices for assessing genetic heterogeneity?

When and where

Date and time

Location

Online

About this event

  • 8 hours
  • Mobile eTicket

The goal of this one-day FDA led 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. 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.