Children’s Hospital of Philadelphia (CHOP) announced today that Rebecca Ahrens-Nicklas, MD, PhD, a pediatric geneticist and Director of CHOP’s Gene Therapy for Inherited Metabolic Disorders Program, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, Co-Director of the Orphan Disease Center (ODC), a partnership between CHOP and Penn Medicine, were named to the 2026 TIME100 list of the 100 most influential people in the world. The list recognizes the impact, innovation and achievement of the world’s most influential individuals.
The honor, which recognizes the CHOP–Penn team behind a historic breakthrough at CHOP, could pave the way for gene editing to treat previously untreatable rare diseases. In February, the researchers marked one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the world’s first person to receive a personalized CRISPR-based gene editing therapy. KJ is now meeting developmental milestones and is thriving.
CHOP continues to evolve its Gene Therapy for Inherited Metabolic Disorders program with the goal of broadening access. Ahrens-Nicklas and Musunuru are also working with the FDA and others in the field to advance a “plausible mechanism” framework that could treat variant‑specific editors as a single platform, speeding approvals for rare pediatric diseases.
The researchers, who were also named to the 2026 TIME100 Health list, will be honored alongside other awardees at a gala on Thursday, April 23, 2026, in New York City. The event brings together the world’s leading figures in politics, technology, science, philanthropy, media, business and entertainment.
Watch KJ’s full story here.
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Children’s Hospital of Philadelphia (CHOP) announced today that Rebecca Ahrens-Nicklas, MD, PhD, a pediatric geneticist and Director of CHOP’s Gene Therapy for Inherited Metabolic Disorders Program, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, Co-Director of the Orphan Disease Center (ODC), a partnership between CHOP and Penn Medicine, were named to the 2026 TIME100 list of the 100 most influential people in the world. The list recognizes the impact, innovation and achievement of the world’s most influential individuals.
The honor, which recognizes the CHOP–Penn team behind a historic breakthrough at CHOP, could pave the way for gene editing to treat previously untreatable rare diseases. In February, the researchers marked one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the world’s first person to receive a personalized CRISPR-based gene editing therapy. KJ is now meeting developmental milestones and is thriving.
CHOP continues to evolve its Gene Therapy for Inherited Metabolic Disorders program with the goal of broadening access. Ahrens-Nicklas and Musunuru are also working with the FDA and others in the field to advance a “plausible mechanism” framework that could treat variant‑specific editors as a single platform, speeding approvals for rare pediatric diseases.
The researchers, who were also named to the 2026 TIME100 Health list, will be honored alongside other awardees at a gala on Thursday, April 23, 2026, in New York City. The event brings together the world’s leading figures in politics, technology, science, philanthropy, media, business and entertainment.
Watch KJ’s full story here.
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KJ’s Story
KJ received a first-of-its-kind personalized gene editing therapy at CHOP to treat his urea cycle disorder.
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Ashley Moore
Gene Therapy for Inherited Metabolic Disorders Program