New FDA recommendations support next-generation sequencing-based safety evaluation of off-target editing for personalised gene therapies.

The U.S. Food and Drug Administration has released a new draft guidance, “Safety Assessment of Genome Editing in Human Gene Therapy Products Using Next-Generation Sequencing,” providing a clearer, more structured framework for evaluating the safety of genome editing therapies. At the center of this guidance is a strong and explicit recommendation: next-generation sequencing (NGS) should be the foundation for assessing genomic risk.

Why NGS is now essential for genome editing programs

Genome editing technologies such as CRISPR, TALENs,…