The Advanced Research Projects Agency for Health (ARPA-H) has awarded a new tranche of funds for companies and institutions developing personalized genetic medicines, with the recipient list dominated by high-profile players in the buzzy field.
The commitment comes from the agency's THRIVE program, which stands for treating hereditary rare diseases with in vivo precision genetic medicines. In total, the agency is distributing up to $160 million over a five-year period.
The program’s aim is to support the development of new platforms for creating personalized therapies, especially for the treatment of rare pediatric diseases. By year three of their projects, recipients need to have begun first-in-human trials, with at least one trial fitting an umbrella model that includes multiple different products individualized to different patients.
“Our current system is built to develop one drug for one disease tested in clinical trials designed specifically for that drug and that disease,” THRIVE program manager Daria Fedyukina, Ph.D., said in a July 9 release. “Patients with rare genetic diseases, especially children, cannot wait for this legacy approach.”
“This innovative umbrella clinical trial model will engage patients and their families to help shape clinical trials and make sure these treatments can rapidly and affordably reach the people who need them,” Fedyukina added.
The most notable name on the grantee list is the Children’s Hospital of Philadelphia, which will net as much as $38.9 million for the team that last year captured the world’s attention by treating baby KJ Muldoon with a custom-built gene therapy designed specifically to fix the genetic mutation causing his rare metabolic disease.
CHOP’s effort will center on building platform infrastructure, running clinical trials and seeking regulatory approval for personalized gene therapies for four rare genetic liver diseases, the hospital said in its own release.
The work will be led by Rebecca Ahrens-Nicklas, M.D., Ph.D., one of the leaders of baby KJ’s treatment, and Lindsey George, M.D, CHOP’s director of clinical in vivo gene therapy. Kiran Musunuru, M.D., Ph.D., who co-led the creation of KJ’s treatment alongside Ahrens-Nicklas, will also contribute to the project.
Another key player in baby KJ’s treatment, the Innovative Genomics Institute at the University of California, Berkeley, will also receive some ARPA-H funds to advance therapies for genetic immune diseases.
Another beneficiary is Gemma Biotherapeutics, a start-up launched by gene therapy pioneer James Wilson, M.D., Ph.D., in 2024 following his departure from the University of Pennsylvania. The biotech will work with AI outfit Profluent Bio to use machine learning for the design of new base-editing medicines for rare liver diseases.
“We believe our work with Profluent will lead to a novel and powerful base-editing approach that can be applied across diverse mutations and genetic diseases,” Wilson said in a July 9 release. “We believe these attributes may permit significant cost reductions and have the potential to increase access around the world.”
Other major health centers besides CHOP are also awardees. St. Jude Children’s Research Hospital will work on bone marrow failure disorders, according to the ARPA-H release, while Massachusetts General Hospital will develop treatments for rare blood vessel diseases.
The Broad Institute has been awarded up to $34.5 million to lead a team working on custom treatments for rare pediatric epilepsy, alongside partners like The Jackson Laboratory and CHOP.
The final recipient of the ARPA-H boon is Stanford University, which will focus on a rare group of genetic skin diseases called epidermolysis bullosa, which cause fragile skin that frequently blisters, among other complications.
“This ARPA-H program takes on one of the toughest challenges in medicine,” ARPA-H Director Alicia Jackson, Ph.D., said in the agency’s release, “and could change the trajectory of genetic disease, expand access to advanced treatments and reinforce U.S. leadership in the future of medicine.”Â