The Advanced Research Projects Agency for Health (ARPA-H) has announced a new round of funding through its THRIVE program, providing up to $160 million over five years to organizations developing personalized genetic medicines. THRIVE, which stands for Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines, is designed to support the development of new platforms for individualized therapies, particularly for rare pediatric diseases.
Under the program, recipients are required to begin first-in-human clinical trials by the third year of their projects. At least one of those trials must use an umbrella clinical trial model that includes multiple products tailored to different patients.
Explaining the need for the initiative, THRIVE program manager Daria Fedyukina, Ph.D., said, “Our current system is built to develop one drug for one disease tested in clinical trials designed specifically for that drug and that disease.” She added that patients with rare genetic diseases, especially children, cannot wait for this traditional approach. Fedyukina also said the umbrella trial model will involve patients and their families in helping shape clinical trials and is intended to help treatments reach those who need them rapidly and affordably.
Among the largest awards is funding of up to $38.9 million for the Children’s Hospital of Philadelphia (CHOP). The hospital was part of the team that last year captured the world’s attention by treating baby KJ Muldoon with a custom-built gene therapy created to correct the genetic mutation responsible for his rare metabolic disease.
According to CHOP, its THRIVE-supported effort will focus on building platform infrastructure, conducting clinical trials, and seeking regulatory approval for personalized gene therapies targeting four rare genetic liver diseases. The project will be led by Rebecca Ahrens-Nicklas, M.D., Ph.D., and Lindsey George, M.D., CHOP’s director of clinical in vivo gene therapy. Kiran Musunuru, M.D., Ph.D., who co-led the development of KJ Muldoon’s treatment with Ahrens-Nicklas, will also contribute to the work.
The Innovative Genomics Institute at the University of California, Berkeley, another participant in KJ Muldoon’s treatment, will also receive ARPA-H funding. The institute will use the support to advance therapies for genetic immune diseases.
Gemma Biotherapeutics is another recipient. The company was launched in 2024 by gene therapy pioneer James Wilson, M.D., Ph.D., following his departure from the University of Pennsylvania. Gemma will work with Profluent Bio to apply machine learning to the design of new base-editing medicines for rare liver diseases. Wilson said he believes the collaboration could lead to a new base-editing approach that may be applied across diverse mutations and genetic diseases, while potentially reducing costs and increasing access globally.
Several other research institutions were selected for awards. St. Jude Children’s Research Hospital will work on bone marrow failure disorders, while Massachusetts General Hospital will develop treatments for rare blood vessel diseases.
The Broad Institute received an award of up to $34.5 million to lead a team developing custom treatments for rare pediatric epilepsy. Partners in the effort include The Jackson Laboratory and CHOP.
Stanford University is the final recipient named by ARPA-H. Its project will focus on epidermolysis bullosa, a group of rare genetic skin diseases that cause fragile skin, frequent blistering, and other complications.
ARPA-H Director Alicia Jackson, Ph.D., said the program addresses one of medicine’s most difficult challenges and could change the trajectory of genetic disease, expand access to advanced treatments, and reinforce U.S. leadership in the future of medicine.
ARPA-H has announced funding of up to $160 million to accelerate the development of personalized genetic medicine technologies. The initiative aims to support groundbreaking research that enables tailored treatments based on an individual’s genetic profile. Through this investment, ARPA-H seeks to foster innovation that can improve outcomes for patients with rare diseases, inherited disorders, and other conditions where precision medicine offers significant potential.
H2: ARPA-H Launches Major Genetic Medicine Initiative
The new ARPA-H funding program is designed to encourage researchers, biotechnology companies, and academic institutions to develop next-generation genetic therapies. By providing substantial financial support, ARPA-H hopes to accelerate scientific discoveries and shorten the timeline from laboratory research to clinical application.
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