TheTechno Optimist.

Personalized Base/Prime Editing PlatformChildren's Hospital of Philadelphia / Penn Medicine—CHOP secures $38.9M five-year ARPA-H THRIVE award for personalized gene-editing platform

Personalized Base/Prime Editing Platform

by Children's Hospital of Philadelphia / Penn MedicineUS

Enable life-saving personalized gene editing for children with rare genetic liver disorders that currently require lifelong treatment or liver transplant.

PilotStage 3 of 5

ARPA-H grant July 2026; clinical trials for urea cycle, acidemia, clotting, bleeding.

Updated 9 Jul 2026Checked 11 Oct0 updates this week

Milestones

Next · Umbrella trial under FDA plausible-mechanism pathway
  1. Baby KJ receives first personalized gene-editing therapy25 Feb 2025Complete.
  2. KJ discharged from hospital, first personalized CRISPR patient stable3 Jun 2025Complete.
  3. $38.9M ARPA-H THRIVE award for personalized gene-editing platform9 Jul 2026Complete.
  4. Umbrella trial under FDA plausible-mechanism pathwayTarget Q3 2026Current milestone.
  5. FDA regulatory approvals for specific indicationsTarget 2028Not yet reached.
  6. Implementation at community sites and remote hubsTarget 2029Not yet reached.

Most important updates

Upcoming

  1. Q3 2026Umbrella trial under FDA plausible-mechanism pathway (next)
  2. 2027FDA review decisions on first registrational indications
  3. 2028FDA regulatory approvals for specific indications
  4. 2028Expansion to other rare genetic disorders beyond liver
  5. 2029Implementation at community sites and remote hubs
Primer