FDA has issued a new gene editing guidance that gives additional information on expectations for use of next-generation sequencing. The most interesting point for me, and I think for companies developing these products, was a repeated reference to testing for off-targets in samples where the editing rate is \”comparable to the rates proposed in the final drug product\” (presumably meaning the intended editing rate in target tissues when the product is administered). This is in contrast to what many companies are doing, which is testing for off-targets in samples in which editing has been saturated or at some multiple above that.
It will be interesting to see how/if this changes after the guidance comment period, but for now, it is helpful to understand current FDA thinking on how far we need to push the system to understand off-target effects.
FDA Gene Editing Guidance
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