1999
RPS19 identified as the first DBA gene in a Swedish patient.
2002
Gene therapy proof-of-concept demonstrated in RPS19-deficient DBA patient cells.
2014
Gene therapy proof-of-concept demonstrated in a DBA mouse model.
2022
Efficacy and safety demonstrated in animal models, product development initiated.
2022
Ex vivo validation of APR-2020 in patient cells.
2023
Short and long-term toxicity studies initiated.
2024
Commercial scaleup for manufacturing initiated.
2025
IND Filed with FDA.
Safe To Proceed Letter issued to Apriligen to begin APR-2020 trial.
International (PCT) Patent application filed.
2026
Fast Track and Rare Disease Evidence Principles (RDEP) Designations granted from the US FDA.
Clinical Trial Enrollment begins at Boston Children’s Hospital and Stanford Children’s Hospital.
Clinical Trial Enrollment begins at Boston Children’s Hospital and Stanford Children’s Hospital.