Researchers will focus on the development of personalized gene‑editing therapies for four groups of rare, liver-related genetic disorders

PHILADELPHIA, July 9, 2026 /PRNewswire/ — Children’s Hospital of Philadelphia (CHOP) announced today that it has been awarded a five-year up to $38.9 million award from the Advanced Research Projects Agency for Health (ARPA‑H) THRIVE program to support the advancement of personalized gene therapy. The award will fund work led by Rebecca Ahrens-Nicklas, MD, PhD, Director of CHOP’s Gene Therapy for Inherited Metabolic Disorders Frontier Program, and Lindsey A. George, MD, CHOP’s Director of Clinical In Vivo Gene Therapy and an attending physician in the Division of Hematology, together with colleagues and partner institutions, including Kiran Musunuru, MD, PhD, MPH, ML, MRA, Co-Director of the Orphan Disease Center (ODC), a partnership between CHOP and Penn Medicine. THRIVE is led by ARPA-H Program Manager Daria Fedyukina, Ph.D.

The ARPA-H award, which builds on the historic treatment of baby KJ Muldoon, whose first ever personalized gene-editing therapy was led by Ahrens-Nicklas and Musunuru, will focus on the development of individualized gene-editing therapies for four groups of rare, liver‑related genetic disorders. These include urea cycle disorders (dangerous newborn ammonia buildup), organic acidemias (toxic metabolites and metabolic crises in infants), severe blood clotting (e.g. protein C deficiency), and bleeding (e.g. hemophilia A) disorders. The CHOP‑led team proposed a carefully designed program to turn a real-time, personalized gene‑editing approach into a scalable platform to treat infants and children with rare, life‑threatening liver‑related genetic diseases, while managing safety, regulatory, manufacturing, and access challenges.

“Current care for these patients typically requires lifelong special diets, regular infusions, or liver transplants, which carry big risks and delays. Tragically, many infants die or have major morbidity before a treatment is possible,” said Ahrens-Nicklas. “Formal clinical trials are needed to evaluate if gene editing therapies are safe and if they work.”

The five-year plan is organized into three core components. CHOP will build and refine its base editing and prime editing lipid nanoparticle systems to create reliable tools for precise gene correction. CHOP will carry out clinical trials, pursue regulatory approvals, engage payers, and expand treatment access by implementing therapies at community sites and remote hubs.

“We are excited to pursue this work and to contribute to the broader goal of advancing the development of transformative individualized gene editing therapies,” said George.

About Children’s Hospital of Philadelphia:

A non-profit, charitable organization, Children’s Hospital of Philadelphia was founded in 1855 as the nation’s first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania, New Jersey, and Delaware. CHOP also operates the Middleman Family Pavilion and its dedicated pediatric emergency department in King of Prussia, the Behavioral Health and Crisis Center (including a 24/7 Crisis Response Center) and the Center for Advanced Behavioral Healthcare, a mental health outpatient facility. Its unique family-centered care and public service.

Contact: Ashley Moore
Children’s Hospital of Philadelphia
(267) 426-6067
moorea1@chop.edu

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SOURCE Children’s Hospital of Philadelphia