ARPA-H Awards up to $160 Million to Advance Personalized Curative Medicines for Rare Genetic Diseases

Rare Daily Staff

The Advanced Research Projects Agency for Health has awarded up to $160 million in a new effort to accelerate the development of personalized genetic treatments for rare diseases—many of which currently have no cure.

The initiative aims to rethink how therapies for rare genetic conditions are developed, tested, and delivered. The program, called THRIVE (Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicines), will bring together leading research institutions to build faster, more flexible approaches for creating treatments tailored to individual patients.

Rare genetic diseases affect millions of Americans, often appearing in infancy or early childhood. Yet about 95 percent of these conditions still lack approved therapies. One major challenge is that the traditional drug development model—one drug for one disease, tested in its own clinical trial—is too slow and costly for conditions that may affect only a small number of patients.

“By transforming precision genetic medicines through platforms that can test multiple treatments for multiple diseases in a single clinical trial, THRIVE reflects the kind of groundbreaking health innovation needed,” said ARPA-H Director Alicia Jackson.

Instead of starting from scratch for every disease, the program will support platform approaches that reuse key components—such as delivery systems or gene-editing tools—across multiple therapies. This modular strategy could significantly reduce both development time and cost.

A central feature of the program is a new type of umbrella clinical trial. In this model, several different therapies targeting different diseases can be tested within a single trial framework. Researchers hope this will accelerate progress while making it easier for patients—especially children with life-threatening conditions—to access experimental treatments.

Participating teams include researchers from institutions such as the Children’s Hospital of Philadelphia, UC Berkeley, St. Jude Children’s Research Hospital, the Broad Institute, Massachusetts General Hospital, Stanford University, and the biotech company GEMMABio.

Each team will focus on different disease areas and technologies. Some will develop gene-editing therapies for immune disorders or rare metabolic diseases, while others will target conditions such as pediatric epilepsy, bone marrow failure, or severe skin diseases. Several groups are also working to improve how treatments are delivered in the body and how quickly they can be tested.

The program sets aggressive milestones. Within one year, teams must demonstrate gene-editing platforms capable of producing multiple therapies with consistent safety profiles. By year three, they are expected to begin human trials using the umbrella model. By year five, the goal is to expand these trials across additional diseases and validate new approaches to delivering treatments at scale.

ARPA-H says the broader aim is not just to develop individual therapies, but to create a repeatable system that others can use—potentially transforming how rare diseases are treated nationwide.

“This program takes on one of the toughest challenges in medicine,” Jackson said. “It could change the trajectory of genetic disease and expand access to advanced treatments.”

If successful, THRIVE could mark a shift toward faster, more scalable development of curative genetic medicines—offering new hope to families affected by conditions long considered untreatable.