Gene therapy could free kids from lifelong blood transfusions
Disease control
Recruiting now
This early-stage trial tests a one-time gene therapy called APR-2020 in 4 children with a severe form of Diamond-Blackfan anemia. The treatment uses the child's own blood stem cells, modified to correct the genetic defect, and given back as an infusion. The goal is to see if it i…
Phase: PHASE1 • Sponsor: Apriligen, Inc. • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC