When Baby KJ was introduced to the world last year as the first recipient of a personalized gene-editing treatment, the logical next question was: How can we get to more Baby KJs?
In November, six months after KJ’s debut, top Food and Drug Administration officials Marty Makary and Vinay Prasad published a paper in the New England Journal of Medicine laying out a “Plausible Mechanism Pathway,” for approval of personalized gene editing treatments.
KJ, an infant born with a life-threatening liver disease in Philadelphia, received his therapy only because of a herculean six-month effort, involving dozens of scientists across North America and untold dollars from government and industry. The new pathway was designed to enable such medicines to be scaled, by allowing them to be formally approved and paid for.
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