ARPA-H Launches $160 Million THRIVE Program to Advance Custom Gene Editing for Rare Diseases
核心洞察
ARPA-H (搜索) announced a $160 million program called THRIVE to fund seven research teams developing custom gene editing treatments for rare diseases.
Each awarded team must begin clinical trials by year three, with expanded trials covering multiple patients and mutations by year five.
The program was originally conceived before a landmark case where a baby received a custom gene editing drug for a life-threatening liver disease.
The Advanced Research Projects Agency for Health (ARPA-H (搜索)), the U.S. government's "moonshot" agency for health research, announced Thursday that it will invest up to $160 million to accelerate the development of custom gene editing treatments for a range of rare diseases. The program, called THRIVE, will support seven different research teams pursuing therapies for conditions affecting distinct organ systems.
"How can we build a paradigm that is also sustainable and can treat thousands of babies?" said Daria Fedyukina, the ARPA-H (搜索) program manager for THRIVE.
The initiative comes on the heels of a landmark demonstration of personalized gene editing's potential. Last year, a baby boy named KJ Muldoon received a gene editing drug crafted by a sprawling team of scientists to fix the unique mutation that caused a life-threatening liver disease. While the case was a stunning proof of concept, it was not readily scalable — it required a vast team and a company willing to absorb immense, undisclosed costs.
Although originally conceived before KJ's treatment, the THRIVE program is now one of several efforts aimed at transforming that one-off success into a reliable, repeatable framework for producing gene editing drugs for any patient with a rare or unique mutation.
Seven Teams, Diverse Disease Targets
The seven awardees span leading academic medical centers and biotechnology companies, each focused on a different category of rare disease:
- The Children's Hospital of Philadelphia — where KJ was treated — will develop treatments for rare metabolic disorders and blood disorders.
- University of California, Berkeley, and the Innovative Genomics Institute will target immune disorders.
- St. Jude Children's Research Hospital will focus on bone marrow disorders.
- The Broad Institute, alongside multiple suborganizations, will develop treatments for pediatric epilepsies affecting the brain.
- GemmaBio — the new company led by gene therapy pioneer Jim Wilson — and Profluent Bio will develop AI-based editors for genetic heart disease.
- Massachusetts General Hospital will address rare diseases of the blood vessels.
- Stanford University will work on a group of rare skin diseases known as epidermolysis bullosa.
Clinical Trial Timelines and Manufacturing Standards
Each team faces a deadline of starting clinical trials by year three of the program, though some may begin much sooner. The Children's Hospital of Philadelphia group is hoping to start such a trial this year. By year five, teams are expected to have expanded their trials to cover multiple patients with different mutations and different gene editors.
Groups working on harder-to-reach organs face longer timelines. "Skin would certainly be a big step," said Erik Sontheimer, a gene editing researcher at the UMass Chan Medical School.
Each group will work with a partner — such as a biotech company or contract manufacturer — to ensure they can manufacture the therapies to Food and Drug Administration standards. New rules at the FDA could then provide a pathway for these bespoke editors to gain approval.
A Program Long in the Making
For gene editing researchers, the announcement has been anticipated for years. Word of a sweeping ARPA-H (搜索) program to push gene editing forward began circulating around 2024, generating excitement among academics and advocates just as investors were beginning to pull out of the field. Over 100 groups applied for funding.
The program faced delays, in part due to the change in administrations, according to Mimi Lee, who originally directed the THRIVE program. Funding was approved in early 2025 by former ARPA-H (搜索) director Renee Wegrzyn.
"Shortly thereafter, she was asked to leave and agency program launches were largely put on hold while the administration introduced new approval processes and a new director could be installed," Lee said in an email. "Luckily, we were able to engage productively with HHS leadership who supported releasing THRIVE, one of less than a handful of programs released during the nine-month leadership gap."
Lee left ARPA-H (搜索) last October, citing an opportunity to make a larger impact outside the agency. The program has been scaled down from her original vision, which would have covered a broader suite of genetic technologies, including those that were mostly conceptual. "There was originally some hope that it could really move the field more than just a couple years," said Sontheimer.
As it stands, THRIVE will largely push technology that has already been developed into clinical trials. Lee expressed support for the new focus, saying it will lead to scientific advances and potentially change how regulators approach gene editing.
Fedyukina was measured about the program's evolution. Ideally, she said, in five years, a majority of teams will have demonstrated proof-of-concept in their diseases and shown a path for how to sustainably develop custom treatments. "We are going to maximize the value for the budget that I have," she said. "And the way I'm going to run this program is going to be extremely practical, because I want results."