Rebecca Ahrens-Nicklas, MD, PhD
Associate Professor of Pediatrics, is the 2026 recipient of the Luigi Mastroianni, Jr. Clinical Innovator Award. Dr. Rebecca Ahrens-Nicklas is a pioneering physician-scientist whose work is creating new therapeutic possibilities for children with rare and previously untreatable genetic diseases. Her most groundbreaking achievement was the development and delivery of the first personalized gene-editing therapy for an infant with CPS1 deficiency, demonstrating that a treatment tailored to an individual patient’s genetic variant could move from therapeutic design to clinical use within a matter of months. Beyond this landmark case, she has built an innovative program spanning gene therapy, precision gene editing, and inherited metabolic disease, while developing the clinical and translational infrastructure needed to bring these advances to patients. Through this work, Dr. Ahrens-Nicklas is establishing a new model for individualized genomic medicine with the potential to expand life-changing treatment options for patients with ultra-rare diseases. Learn more about Dr. Ahrens-Nicklas