The world’s first patient to successfully receive a CRISPR gene-editing treatment was discharged from Children’s Hospital of Philadelphia on June 2, ABC News reported.
KJ Muldoon, a 10-month-old who was born with severe carbamoyl phosphate synthetase 1 deficiency, received the first infusion of the bespoke therapy in February. CHOP and Philadelphia-based Penn Medicine collaborated on his treatment, and he responded well to follow-up doses in March and April, his clinicians said.
The rare metabolic disease disrupts the body’s urea cycle, which is the breakdown and removal of nitrogen, and usually results in death within the first few months of life.
He quickly reached a weight appropriate for his developmental stage and “graduated” from the hospital in a blue cap and gown, ABC News reported.
“While KJ will need to be monitored carefully for the rest of his life, our initial findings are quite promising,” Rebecca Ahrens-Nicklas, MD, PhD, director of CHOP’s gene therapy for inherited metabolic disorders frontier program, said in a May statement.
