TL;DR
People born with primary hyperoxaluria type 1, a rare inherited liver defect, make too much of the compound that forms kidney stones, and for many the result is a lifetime of painful stones and kidneys that can fail. A gene editor given once, by infusion, lowered that compound in the urine by up to 61.8% a year later in 11 patients aged 2 to 59, its maker YolTech reports; in plain terms, the best responders had roughly 6 in 10 fewer units of it a year after one dose, and their kidney function held steady. If that holds in larger trials, one treatment could stand in for a lifetime of managing the disease. It is also one more sign that single-dose gene edits made in the liver are spreading from one rare disease to the next, with the first FDA decision on such a treatment due March 10, 2027.
Below the line, for paid subscribers: The full story: where it stands, how we got here and what's dated next; our Odds board on 5 milestones, starting with "A one-time gene-editing infusion for primary hyperoxaluria type 1 is approved for patients by 2031"; our labeled speculation on what it adds up to; and every source.