CHOP-Penn Gene Therapy Collaboration

New Capabilities

As of May 2026: Preparing umbrella trial for 7 urea cycle disorders in 2026

In August 2024, KJ Muldoon was born with a death sentence encoded in his DNA—a single broken letter among three billion that left his body unable to process protein. Six months later, he walked out of Children's Hospital of Philadelphia, cured by a gene-editing therapy designed, manufactured, approved, and delivered in 180 days.

Updated May 19