ARPA-H commits up to $160 million to custom gene editing for rare diseases
ARPA-H, the federal health research agency, is committing up to $160 million to a program called THRIVE that aims to turn custom gene-editing treatments for rare diseases from rare, bespoke efforts into a repeatable process. The money will back seven teams targeting conditions across different organ systems, with each required to reach clinical trials by the third year of the program.
The effort builds on the case of “Baby KJ,” an infant treated last year with a gene-editing therapy designed for his single mutation — an “n-of-1” approach that worked but took an extraordinary, one-time mobilization of researchers and regulators. The bottleneck for such treatments is not usually the biology of editing a given mutation but the cost, time and regulatory groundwork of building each therapy from scratch. THRIVE’s premise is that standardizing the manufacturing, safety testing and trial design could let teams move from a newly identified mutation to a treatment far faster and more cheaply.
The timing aligns with a recent FDA roadmap for expanding individualized gene-editing therapies, suggesting funders and regulators are moving in step to lower the barriers for patients whose diseases are too rare to attract conventional drug development.