Children’s Hospital of Philadelphia Receives $38.9 Million ARPA-H Award for Personalized Gene Editing Research

Children’s Hospital of Philadelphia has been awarded a five-year up to $38.9 million grant from the ARPA-H THRIVE program according to a press release. The funding supports research into personalized gene editing therapies targeting four categories of rare liver-related genetic disorders.

The program is led by Dr. Rebecca Ahrens-Nicklas and Dr. Lindsey A. George, along with collaborators including Dr. Kiran Musunuru from the Orphan Disease Center, a partnership between CHOP and Penn Medicine. The research expands on previous work involving the first personalized gene editing treatment for an infant patient.

The effort will focus on developing therapies for urea cycle disorders, organic acidemias, blood clotting issues such as protein C deficiency, and bleeding disorders such as hemophilia A. The team aims to create a scalable platform for personalized treatments while addressing safety, regulatory, and manufacturing challenges.

CHOP’s plan includes three core goals: advancing base and prime editing through lipid nanoparticle systems, conducting clinical trials and pursuing regulatory approvals, and expanding access by deploying therapies to community and remote clinical sites.