Dr. Rebecca Ahrens-Nicklas

New Capabilities

Leading expansion to umbrella trial for 7 urea cycle disorders

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 that didn't exist when he was diagnosed. The treatment was designed, manufactured, approved, and delivered in 180 days.

Updated Jan 7