Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA of Children’s Hospital of Philadelphia (CHOP) were named to the 2026 TIME100 list of the world’s most influential people, CHOP announced on Apr. 16. The recognition highlights their role in developing a personalized CRISPR-based gene editing therapy that marked a significant milestone in treating rare diseases.
The inclusion on the TIME100 list brings attention to advances in gene editing therapies for conditions previously considered untreatable. In February, Ahrens-Nicklas and Musunuru’s team marked one year since an infant known as KJ became the first person worldwide to receive this type of personalized treatment for severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. According to CHOP, KJ is now meeting developmental milestones and is thriving.
Ahrens-Nicklas directs CHOP’s Gene Therapy for Inherited Metabolic Disorders Program while Musunuru serves as Co-Director of the Orphan Disease Center—a partnership between CHOP and Penn Medicine. Both are working with regulatory agencies such as the Food and Drug Administration and other experts in the field to advance what they describe as a “plausible mechanism” framework. This approach could allow variant-specific editors to be treated as a single platform technology and potentially speed up approvals for therapies targeting rare pediatric diseases.
The researchers have also been named to the 2026 TIME100 Health list. They will be honored alongside other awardees at a gala event scheduled for Thursday, April 23 in New York City that gathers prominent figures from various sectors including science and medicine.
This recognition underscores ongoing efforts by CHOP’s Gene Therapy program not only to innovate but also broaden access so more patients with rare genetic disorders may benefit from emerging treatments.
