ARPA-H Launches Nine-Figure Programs to Advance Personalized Gene Editing Treatments

The Advanced Research Projects Agency for Health (ARPA-H) has unveiled two groundbreaking funding opportunities aimed at revolutionizing the development and delivery of custom gene editing therapies. These programs, representing a significant investment from the Department of Health and Human Services (HHS), seek to build upon recent successes in personalized genetic medicine and expand access to potentially life-saving treatments for patients with rare genetic diseases.

THRIVE and GIVE: Transforming Genetic Medicine

The Treating Hereditary Rare Diseases With In Vivo Precision Genetic Medicines (THRIVE) program will support the development of platforms for creating individualized gene editing therapies. Complementing this initiative, the Genetic Medicines and Individualized Manufacturing for Everyone (GIVE) program aims to enhance the manufacturing capabilities for these personalized treatments, bringing production closer to patients in need.

HHS Secretary Robert F. Kennedy, Jr. emphasized the potential impact of these programs, stating, "THRIVE will speed the design of precision genetic medicines for many rare diseases. GIVE will place compact, automated manufacturing units in hospitals to produce those medicines safely, close to the children who need them."

Building on Recent Breakthroughs

The launch of these programs follows the successful treatment of baby KJ Muldoon earlier this year with a personalized gene editing therapy. This groundbreaking case has served as a catalyst for both regulatory innovation at the FDA and the creation of the new ARPA-H initiatives.

Dr. Kiran Musunuru, a cardiologist at Children's Hospital of Philadelphia and co-leader of KJ's treatment team, highlighted the significance of these developments: "The KJ story has provided a catalyst for both regulatory innovation at the FDA and the launching of the new ARPA-H programs."

Expanding Access and Scaling Production

The ambitious goal of these programs is to transition from isolated success stories to widespread availability of custom gene editing treatments. Secretary Kennedy emphasized this vision, stating, "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."

In parallel with these government initiatives, private sector efforts are also gaining momentum. The Chan Zuckerberg Initiative and Innovative Genomics Institute (IGI) at the University of California, Berkeley, recently launched the $20 million Center for Pediatric CRISPR Cures. Jennifer Doudna, CRISPR pioneer and founder of IGI, expressed the center's mission: "We've already seen the profound impact that an on-demand CRISPR therapy can have for one family. Now we want to ensure that this approach can scale and be made available for more children around the world."

As these programs take shape, the pharmaceutical industry eagerly anticipates the publication of a new regulatory pathway for individualized gene editing therapies from the Food and Drug Administration, expected to appear in the New England Journal of Medicine in the near future.