The brief
- Baby KJ received the first bespoke personalized CRISPR therapy in Feb 2025 for rare liver disorder; discharged June 2025.
- ARPA-H awarded $38.9M five-year grant (July 2026) to scale base/prime editing platform for rare liver disorders.
- Trials target urea cycle, organic acidemia, blood clotting and bleeding disorders; FDA plausible-mechanism pathway.
Technical approach
Bespoke Design
Personalized therapy for each patient
Each patient's mutation is sequenced; a custom guide RNA corrects that specific variant.
Delivery
Lipid nanoparticle base/prime editors
LNPs deliver base and prime editors to liver cells, making precise edits without double-strand breaks.
Scale
From one patient to a platform
THRIVE turns bespoke design into a repeatable process for four classes of rare liver genetic disorders.
Access
Community sites and remote hubs
The program includes payer engagement and deployment at community centers to broaden treatment access.
