TL;DR
Some children are born with a broken gene that slowly takes their sight, starting in early childhood and getting worse for years. On Oct. 21 the FDA cleared a one-time gene therapy for one of those genes to be tested in people for the first time, its maker says. If it works, a single treatment given early could save vision a child would otherwise lose, and it adds one more inherited disease to the short but growing list where the goal is a one-time fix instead of a lifetime of decline. No one has received it yet, and most treatments at this stage never reach patients.
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 6 milestones, starting with "The first person receives Opus Genetics' RDH12 gene therapy by 2027"; our labeled speculation on what it adds up to; and every source.