In China gene-editing trial, 2 children improved, 1 died and 1 withdrew

AI Summary

A gene-editing drug was given to four Chinese boys with a rare inherited disease: two showed improved ability to walk, one had no gait improvement and withdrew, and one died 81 hours after treatment following a severe systemic reaction. The small trial presents sharply mixed outcomes and a serious safety concern.

Four seriously ill Chinese boys who would almost certainly have died in their 20s from a rare inherited disease have received the same highly anticipated gene-editing drug. More than a year later, a set of cruel and contradictory data emerged. Two boys gained the strength to walk farther. One boy showed no improvement in gait and subsequently withdrew from the trial. And the fourth boy’s heart stopped beating 81 hours after receiving the medication intravenously, owing to a severe systemic...