US FDA's Authority Over CRISPR Is Adequate, NASEM Finds, But Off-Label Challenge May Emerge

National Academies’ report on the fast-advancing genome-editing technologies points out that ways to improve musculature in dystrophy patients will almost certainly raise issues about treatment use in narrow population versus enhancement use in broader population. In most cases, however, specificity of genome-edited products will help limit use of the new therapies to targeted populations.

A recently released report by a committee of the National Academies of Science Engineering Medicine (NASEM) on the advance of genome-editing technologies (accelerated by CRISPR-Cas9) finds that muscle generation under investigation in the muscular dystrophy area is likely to present an off-label use challenge to FDA from the new therapies when and if they reach commercial use.

Addressing the potential use for genome-edited products for enhancement instead of treatment, the report, “Human Genome Editing: Science,...

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