$38.9M ARPA-H Award Fuels CHOP’s Push for Personalized Gene Editing Therapies — The Children's Hospital of Philadelphia

Event summary

Children's Hospital of Philadelphia (CHOP) secures up to $38.9M from ARPA-H’s THRIVE program over five years.
Funding supports development of personalized gene-editing therapies for four rare, liver-related genetic disorders.
Project builds on prior success treating baby KJ Muldoon with first-ever personalized gene-editing therapy.
Three core components: refining editing tools, clinical trials, and expanding treatment access.

The big picture

This award positions CHOP at the forefront of personalized gene therapy, addressing critical gaps in treating rare liver-related disorders. The $38.9M investment underscores ARPA-H’s commitment to high-risk, high-reward health innovations, while CHOP’s collaboration with Penn Medicine strengthens its translational research capabilities. Success here could set a precedent for scaling individualized therapies beyond single-case treatments.

What we're watching

Clinical Validation: Whether CHOP can demonstrate safety and efficacy in formal clinical trials for these rare disorders.
Regulatory Pathways: The pace at which regulatory approvals progress for personalized gene-editing therapies.
Scalability Challenges: How CHOP manages manufacturing and access hurdles to bring treatments to community sites.