WITHIN DAYS after KJ was born in Philadelphia in August 2024 it was clear that one thing was incorrect. He was not consuming and slept an excessive amount of. Blood checks revealed sky-high ranges of ammonia, a poisonous substance the physique often expels. Genome sequencing confirmed that he had a uncommon genetic illness referred to as carbamoyl-phosphate synthetase 1 (CPS1) deficiency, which regularly kills in infancy, and for which no good neonatal remedy exists. Then one in all his docs steered one thing radical: a gene-editing drug designed particularly for him.
