PATIENT-SPECIFIC GENE EDITING STUDY WINS TOP PRIZE AT ANNUAL CLINICAL RESEARCH AWARDS
Other top awardees include a study on the effects of loss of drug coverage on Medicaid beneficiaries and a unique, low-cost intervention that reduces infant mortality from malaria by two-thirds
From right to left: Dr. Harry Selker, Dr. Rebecca Ahrens-Nicklas, and Dr. E. Albert Reece, chair of the Top 10 Awards
Washington, D.C. – May 15, 2026
The Clinical Research (CR) Forum, a non-profit membership association of top clinical research experts and thought leaders from the nation’s leading academic health centers, awarded its most prestigious honor, The Herbert Pardes, MD Clinical Research Excellence Award, to Dr. Rebecca Ahrens-Nicklas and her team at the Children’s Hospital in Philadelphia, for a landmark study demonstrating the successful use of customized, in‑body CRISPR gene editing to treat a newborn with a life‑threatening metabolic disorder. See video.
“This breakthrough provides compelling proof that patient‑specific gene editing can move from concept to reality — opening a transformative new pathway for treating rare genetic diseases that were once thought untreatable,” said Dr. Harry Selker, Chair of the Clinical Research Forum. “We are also deeply inspired by the extraordinary courage and trust shown by the child’s parents, whose willingness to pursue an experimental clinical trial made this historic advance possible.”
The study, Treatment of an Infant with Severe Neonatal-Onset Carbamoyl Phosphate Synthetase 1 Deficiency with a Personalized Gene-Editing Therapy, was recognized during CR Forum’s annual Top 10 Clinical Research Achievement Awards on May 11th in Washington, D.C. The Top 10 Awards highlight outstanding research advances that involve both innovation and impact on human diseases. A complete list of the 2026 Top 10 Award Winners is available on the website at https://www.clinicalresearchforum.org/page/TopTen.
The Herbert Pardes Clinical Research Excellence Award, named in honor of CR Forum Board Vice Chair Dr. Herbert Pardes for his profound impact on clinical research and academic medicine, is awarded to the research study that best shows a high degree of innovation and creativity, advances science, and has an impact upon human disease. Dr. Pardes passed away in 2024 after a distinguished career as Vice Chairman of the Board of Trustees at New York-Presbyterian Hospital and as the long-time Dean of the Faculty of Medicine at the College of Physicians & Surgeons of Columbia University. The award comes with a $7,500 prize.
Two additional studies received Distinguished Clinical Research Achievement Awards for their creativity, innovation, and novel approach that demonstrates immediate impact on the health and well-being of patients:
- Loss of Subsidized Drug Coverage and Mortality among Medicare Beneficiaries, nominated by the University of Pennsylvania, examined what happens when low-income Medicare beneficiaries lose subsidized prescription drug coverage following disenrollment from Medicaid. Dr. Eric Roberts and his team were able to demonstrate a measurable and statistically significant increase in mortality, particularly among individuals with chronic conditions requiring consistent medication use. The findings provide compelling evidence that continuous access to affordable medications saves lives and that coverage policy decisions have direct, real-world health consequences. See video
- Permethrin-Treated Baby Wraps for the Prevention of Malaria, nominated by the University of North Carolina, Chapel Hill, focused on one of the leading causes of infant mortality worldwide – malaria. In this global study, Dr. Ross Boyce and his team showed that treating infant swaddling cloths with permethrin, a mosquito repellent, dramatically reduced malaria infections in babies. The intervention is simple, low‑cost, and easily scalable, and resulted in a two‑thirds reduction in malaria cases among infants. See video
Since 1996, the Clinical Research Forum has enabled the sharing of best practices in clinical research, informed meaningful policy dialogues and increasingly played a national advocacy role in support of clinical research.
CR Forum congratulates all the 2026 Top Ten Clinical Research Achievement Award recipients and finalists for groundbreaking, clinical and translational research advances that benefit the health and welfare of all Americans, and reflect the influential work being done by investigators at 60 research institutions and hospitals across the United States, as well as partner institutions around the world.
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About the Top Ten Clinical Research Achievement Awards
Recognizing the need to celebrate our nation's clinical research accomplishments that involve both innovation and impact on human disease, the Clinical Research Forum conducts an annual competition to determine the ten outstanding research accomplishments in the United States. These major research advances represent a portion of the annual return on the nation's investment in the health and future welfare of its citizens.
About the Clinical Research Forum
The mission of the Clinical Research Forum is to catalyze and sustain an environment in which clinical and translational research and researchers will thrive and advance human health and well-being for all. For more information, visit www.clinicalresearchforum.org.