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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the potential risks of off-target editing as well as unintended consequences of on-target effects, agency says in new draft guidance.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In a new draft guidance, the FDA recommends that clinical development programs for human genome editing (GE) products address the risks associated with the gene therapy product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 well as the additional risks associated with the GE

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