When baby KJ Muldoon was treated with a personalized gene editing treatment back in February, scientists behind the effort envisioned a world where all patients born with genetic diseases could have access to similar custom CRISPR therapies. Now, the Advanced Research Projects Agency for Health (ARPA-H) has unveiled two new funding opportunities meant to support those trying to make that happen.
The Treating Hereditary Rare Diseases With In Vivo Precision Genetic Medicines (THRIVE) program aims to fund those building platforms that allow for the creation of custom gene editing therapies unique to individual patients, while the Genetic Medicines and Individualized Manufacturing for Everyone (GIVE) program intends to boost manufacturing of these personalized medicines close to where patients need them.
“THRIVE will speed the design of precision genetic medicines for many rare diseases,” Department of Health and Human Services Secretary Robert F. Kennedy Jr., said in a Sept. 25 post on X. “GIVE will place compact, automated manufacturing units in hospitals to produce those medicines safely, close to the children who need them.”
“Together, these programs represent a nine-figure HHS investment—helping us go from saving one baby like KJ to saving thousands, because every family deserves access to lifesaving treatment,” RFK Jr. added.
The programs are being introduced in advance of a new regulatory pathway for individualized gene editing therapies from the FDA, which is expected to be published in The New England Journal of Medicine soon.
“The KJ story has provided a catalyst for both regulatory innovation at the FDA and the launching of the new ARPA-H programs,” Kiran Musunuru, M.D., Ph.D., a cardiologist at Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, who co-led KJ’s groundbreaking treatment, told Fierce Biotech on Sept. 25.
Musunuru and fellow KJ team leader Rebecca Ahrens-Nicklas, M.D., Ph.D., also of CHOP and Penn Medicine, have been in discussions with both the FDA and ARPA-H about creating a framework for custom gene editing platforms to be approved by regulators, the duo said.
The successful treatment of KJ’s extremely rare metabolic disease also spurred others from the team to take action that could bring similar life-saving therapies to children. In July, the Chan Zuckerberg Initiative and Innovative Genomics Institute (IGI) at the University of California, Berkeley, teamed up to launch the $20 million Center for Pediatric CRISPR Cures.
“We've already seen the profound impact that an on-demand CRISPR therapy can have for one family,” Jennifer Doudna, Ph.D., the CRISPR pioneer and 2020 Nobel laureate who founded IGI, said at the time. “Now we want to ensure that this approach can scale and be made available for more children around the world.”
Another KJ team member, Benjamin Kleinstiver, Ph.D., of Mass General Hospital and Harvard Medical School, recently co-led a different custom gene editing approach that successfully fixed a deadly disease-causing mutation in mice.