Story perspectives

First Patient Walks After Groundbreaking CRISPR Therapy

12/19/2025

Summary

Story summary

KJ Muldoon, treated with a personalized CRISPR therapy at Children's Hospital of Philadelphia, has taken his first steps.
KJ became the first patient to receive this CPS1-deficiency therapy developed by Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunuru.
After ten months in the hospital, he was discharged in June and celebrated his first birthday at home.
KJ's case is prompting discussions on new models for approving personalized therapies for rare diseases.

1 of 1

Story summary

KJ Muldoon, treated with a personalized CRISPR therapy at Children's Hospital of Philadelphia, has taken his first steps.
KJ became the first patient to receive this CPS1-deficiency therapy developed by Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunuru.
After ten months in the hospital, he was discharged in June and celebrated his first birthday at home.
KJ's case is prompting discussions on new models for approving personalized therapies for rare diseases.