Gene therapy may free kids with rare anemia from lifelong blood transfusions
Disease control
Recruiting now
This early-stage trial tests a gene therapy called APR-2020 in children and teens with a specific type of Diamond-Blackfan anemia (DBA). The treatment uses the patient's own blood stem cells, modified to correct the genetic defect, and given as a one-time infusion. The goal is to…
Phase: PHASE1 • Sponsor: Apriligen, Inc. • Aim: Disease control
Last updated May 01, 2026 15:57 UTC