COMPANY

Apriligen

Our first clinical gene therapy program, APR-2020, is a purpose-built lentiviral vector to treat RPS19 deficient Diamond Blackfan Anemia (“DBA”). From company website Source · Oct 12, 2026

Keywords: gene therapy lentiviral anemia rare disease rare diseases APR-2020

Profile checked Oct 10, 2026

1Trials (company is sponsor)

Timeline

Apr 16, 2026 Trial start Phase 1: Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia source ↗

Upcoming milestones

2027-04 (ESTIMATED) · Phase 1 · Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia · NCT07476183

Estimated primary completion per ClinicalTrials.gov. Dates move.

Clinical trials (lead sponsor) 1

Record	Title	Phase	Status	Start	Primary completion
NCT07476183	Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia	Phase 1	Recruiting	Apr 16, 2026	2027-04 (estimated)