// VIKTORIOUS RESEARCH TERMINAL

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Gene therapy, gene editing and the trials that could one day cure Diamond-Blackfan anemia. We scan the research every week and add what matters.

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[CLINICAL TRIAL] · 2026-03-17 · Apriligen, Inc.

Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

The first clinical trial of lentiviral RPS19 gene addition for DBA. Children's own blood stem cells are collected, given a working copy of the RPS19 gene and infused back once. The Phase 1 study checks safety and whether the corrected cells cut or end the need for transfusions. It started in April 2026.

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