July 9, 2026 —
The U.S. government has awarded $43 million to Philadelphia-based researchers through a new federal effort to advance scalable gene therapy and gene editing approaches for rare genetic diseases.
The funding comes through THRIVE, a new $160 million program from the Advanced Research Projects Agency for Health, or ARPA-H, designed to support precision genetic medicines that can be adapted across multiple rare diseases. The program aims to enable platform approaches capable of testing multiple treatments for multiple diseases within a single clinical trial framework.
Children’s Hospital of Philadelphia received $39 million to develop a scalable platform for treating rare genetic liver-related diseases in infants and children. The CHOP team includes Rebecca Ahrens-Nicklas, who worked with Kiran Musunuru of Penn Medicine on the first personalized gene-editing treatment for an infant known as Baby KJ. That therapy was designed to correct the genetic mutation driving the child’s rare metabolic disease and was associated with improved liver function.
The new ARPA-H funding will expand that work beyond a single patient or single diagnosis. The CHOP-led program will focus initially on liver-related genetic diseases, including urea cycle disorders and rare coagulation disorders. The goal is to develop a platform in which the same core therapeutic approach can be adapted to target different disease-causing genetic variants.
The five-year plan includes preclinical and clinical studies to evaluate the safety and efficacy of individualized genetic medicines. The team also plans to work on regulatory pathways, payer engagement, and implementation models that could allow therapies to be delivered at community sites and remote hubs, expanding access beyond major academic centers.
The CHOP team also includes Lindsey George, a gene therapy researcher focused on bleeding disorders. Together with Ahrens-Nicklas and Musunuru, the group aims to build a more generalizable framework for pediatric rare disease gene editing.
Separately, ARPA-H awarded $4 million to GEMMABio, a University of Pennsylvania spinout founded by gene therapy pioneer Jim Wilson, to support AI-guided design of gene therapies. In partnership with ProFluent, GEMMABio will use artificial intelligence to design base editors that could be rapidly adapted for different patients and diseases.
GEMMABio’s initial focus is on rare liver diseases with significant unmet need, including Maple Syrup Urine Disease and homozygous familial hypercholesterolemia, or HoFH. The award will support preclinical studies of the company’s technology.
The Philadelphia awards highlight a broader shift in rare disease genetic medicine: moving from one-off bespoke therapies toward reusable platforms that can be scaled, standardized, and potentially made more affordable. With most rare diseases still lacking approved treatments, ARPA-H’s THRIVE initiative aims to address one of the central challenges in the field—how to bring precision genetic medicines to very small patient populations in a practical and sustainable way.
