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Gene Editing: Off-Target Effects Should Be A Key Safety Focus During Development, US FDA Says

 
• By Sue Sutter

Clinical development programs for human gene therapy products that incorporate genome editing should address the potential risks of off-target editing as well as unintended consequences of on-target effects, agency says in new draft guidance.

Missed target
Therapeutics incorporating gene editing require special attention to off-target effects. • Source: Alamy

The US Food and Drug Administration wants developers of gene therapy products that incorporate human genome editing to pay special attention to safety issues resulting from both on- and off-target effects of editing.

In a new draft guidance, the FDA recommends that clinical development programs for human genome editing (GE) products address the risks associated with the gene therapy product itself, as...

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