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, Ethics and Governance,” focuses several times on the predictable appeal...