In China gene-editing trial, 2 children improved, 1 died and 1 withdrew
Four seriously ill Chinese boys who would almost certainly have died in their twenties from a rare inherited disease 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…
In a gene-editing trial for a rare inherited disease, four Chinese boys received the same highly anticipated drug. After a year, the results showed mixed outcomes. Two boys gained the ability to walk farther, while one boy showed no improvement and withdrew from the trial. Regrettably, the fourth boy's heart stopped 81 hours after receiving the medication due to a severe immune reaction and respiratory failure.
Cell published the clinical trial results, detailing the promising yet perilous nature of experimental treatments. The therapy, developed by Shanghai-based HuidaGene Therapeutics, aimed to restore dystrophin expression in patients with Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease. With Orphan Drug Designation from the FDA, the gene-editing therapy, HG302, had shown promising results in preclinical mouse studies.
However, the trial on the four boys aged four to eight revealed varying degrees of success and significant risks.
Written by urgent.news from Reuters Business via SCMP's reporting — not their text. Machine-written — may contain errors; check the original before relying on it.
