ARPA-H Commits $160 Million to Rare-Disease Gene Editing as New Center Takes Shape
Updated
Updated · The Boston Globe · Jul 21
ARPA-H Commits $160 Million to Rare-Disease Gene Editing as New Center Takes Shape
3 articles · Updated · The Boston Globe · Jul 21
Summary
$160 million in ARPA-H funding will back custom gene-editing treatments for rare diseases, with an initial push toward precision therapies for children with rare genetic epilepsies.
The Broad Institute, Boston Children’s Hospital and Jackson Laboratory said their new nonprofit Center for Therapeutic Genetics will use that support to build shared treatment platforms, manufacturing processes, safety data and clinical programs.
The model aims to replace disease-by-disease, family-funded development with repeatable clinical procedures that other hospitals can adopt, lowering costs and reducing the need for separate approvals for each patient-specific therapy.
The effort targets a large gap: 1 in 10 Americans has a rare disease, more than 10,000 are known, and fewer than 5% have approved treatments.
The center is still in its founding phase and is not yet taking referrals, underscoring a broader challenge advocates say now matters most—building systems that can deliver fast-moving gene-therapy science at scale.
Can this new center make personalized gene cures a routine medical procedure, not a million-dollar miracle?
With thousands of rare diseases, how will scientists decide which children are first in line for a potential cure?
$160 Million ARPA-H THRIVE Program Launches Umbrella Trials for Scalable Rare Disease Gene Editing
Overview
ARPA-H has launched the THRIVE program, committing up to $160 million over five years to support seven groups working to revolutionize the treatment of rare genetic diseases through gene editing. By adopting a unique, milestone-driven funding model inspired by defense research agencies, THRIVE aims to make gene editing therapies more scalable, accessible, and affordable. The program addresses the critical unmet needs of millions of Americans, especially children, by overcoming traditional inefficiencies and market failures in rare disease drug development. This ambitious initiative hopes to achieve real-world successes and transform the landscape of rare disease care.