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 a funding initiative of up to $160 million to accelerate the development of personalized genetic medicine. The investment is designed to support groundbreaking research, encourage collaboration among scientists and healthcare organizations, and advance innovative treatments tailored to each patient’s genetic profile. Through this initiative, ARPA-H aims to strengthen the future of precision medicine and improve outcomes for individuals with complex diseases.
Major Investment in Precision Healthcare
The new funding from ARPA-H represents one of the agency’s significant investments in personalized medicine. By supporting cutting-edge genetic research, the program seeks to overcome scientific barriers and speed the development of therapies that are more effective and precisely targeted.
This initiative reflects the growing importance of genomics in modern healthcare.
ARPA-H Commits Up to $160 Million for Genetic Medicine
The decision by ARPA-H to provide up to $160 million for personalized genetic medicine development marks an important step toward the future of precision healthcare. By investing in innovative research, cross-sector collaboration, and advanced genomic technologies, ARPA-H is helping accelerate the development of treatments that are tailored to individual patients and capable of improving health outcomes on a global scale.
Advancing the Future of Genomic Research
Beyond immediate clinical applications, ARPA-H is helping build the scientific infrastructure needed for future breakthroughs in genomic medicine. Funding may support new technologies, data-sharing platforms, and advanced research tools that enable faster discoveries across the life sciences sector.
These investments strengthen the foundation for continued innovation in precision healthcare.
Looking Ahead
The announcement demonstrates ARPA-H’s commitment to transforming healthcare through high-impact scientific research. As funded projects move forward, researchers will focus on developing scalable genetic medicine solutions that can benefit patients worldwide while advancing the broader field of personalized healthcare.
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