Doctors Built a One-of-a-Kind Gene Therapy for a Sick Baby — in Just Six Months
An infant with a rare metabolic disorder received a CRISPR treatment designed specifically for him, start to finish, in record time.
When a baby was born with a dangerous metabolic condition called CPS1 deficiency, doctors did something that would have been science fiction a few years ago: they designed a personalized in-vivo CRISPR therapy aimed at his exact mutation.
From concept to FDA clearance to the treatment reaching the patient took just six months — a turnaround that hints at a future where the rarest diseases get custom-built cures, one child at a time.