Baby KJ

Overview

Baby KJ is an infant cured of a fatal metabolic disorder by a personalized CRISPR therapy made by Danaher companies. Baby KJ was born with a fatal inborn error of metabolism and received a personalized CRISPR therapy manufactured by Aldevron, IDT, and Cytiva in roughly six months, about three times faster than standard timelines. After treatment KJ is reported to be at home and developing normally, an outcome Blair stressed was an exceptional N-of-1 that required regulators and partners to make exceptions and is not yet scalable.