LANDMARK PATIENT-SPECIFIC GENE EDITING STUDY WINS TOP PRIZE AT ANNUAL CLINICAL RESEARCH AWARDS

Annual competition of major clinical research breakthroughs illustrates the return on the nation's investment in the health and welfare of its citizens.

WASHINGTON, D.C. , DC, UNITED STATES, May 18, 2026 /EINPresswire.com/ -- 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.

“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.

In addition to the award, named after CR Forum’s Founding Vice Chair Dr. Herbert Pardes, Dr. Rebecca Ahrens-Nicklas and her team also received a $7,500 cash prize.

Two additional studies received Distinguished Clinical Research Achievement Awards and $5,000 each:
• "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.
• "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.

The 2026 Top Ten Clinical Research Achievement Awards showcase clinical and translational research advances that benefit the health and welfare of all Americans, and reflect the influential work being done by investigators at over 60 research institutions and partner institutions around the world.

Andrea Van Hook
Clinical Research Forum
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Patient In-Vivo Gene Editing to Treat a Rare Disease

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