Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania (Penn Medicine) 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, a cardiologist, geneticist and gene editor at Penn, were named to TIME100 Health 2026, TIME’s annual list of the 100 individuals who most influenced global health this year.
The honor recognizes the CHOP–Penn team behind a historic breakthrough that could enable gene editing to treat previously untreatable rare diseases. Last year, a child diagnosed with a rare genetic disorder was successfully treated with a customized CRISPR gene editing therapy. The infant, KJ, was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. After spending the first several months of his life in the hospital, on a very restrictive diet, KJ received the first dose of his bespoke therapy in early 2025.
The treatment was administered safely, and he is now growing well and thriving. The case was detailed in a study published by The New England Journal of Medicine and was presented at the American Society of Gene & Cell Therapy Annual Meeting in New Orleans.
The TIME100 Health list is an annual recognition by TIME of 100 individuals who are significantly shaping the future of global health. Launched as a specialized, annual list in 2024 to highlight innovators, leaders, and advocates across various health sectors, it honors scientists, doctors, policymakers, and activists.
Watch KJ’s full story here.
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Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania (Penn Medicine) 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, a cardiologist, geneticist and gene editor at Penn, were named to TIME100 Health 2026, TIME’s annual list of the 100 individuals who most influenced global health this year.
The honor recognizes the CHOP–Penn team behind a historic breakthrough that could enable gene editing to treat previously untreatable rare diseases. Last year, a child diagnosed with a rare genetic disorder was successfully treated with a customized CRISPR gene editing therapy. The infant, KJ, was born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. After spending the first several months of his life in the hospital, on a very restrictive diet, KJ received the first dose of his bespoke therapy in early 2025.
The treatment was administered safely, and he is now growing well and thriving. The case was detailed in a study published by The New England Journal of Medicine and was presented at the American Society of Gene & Cell Therapy Annual Meeting in New Orleans.
The TIME100 Health list is an annual recognition by TIME of 100 individuals who are significantly shaping the future of global health. Launched as a specialized, annual list in 2024 to highlight innovators, leaders, and advocates across various health sectors, it honors scientists, doctors, policymakers, and activists.
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