Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children. The main questions it aims to answer are: * Is APR-2020 safe and well tolerated? * Does APR-2020 modify or correct an underlying genetic condition which causes DBA? * Does APR-2020 reduce or eliminate the need for blood transfusions and/or restore certain blood counts affected by DBA? Participants will: * Take the drug one time as an infusion. * Undergo two rounds of a cellular harvest procedure in which their own cells will be used in the manufacturing of their own participant-specific product. * Initially return to the clinic for two years of follow up at increasingly sparse intervals.
Age range
2 Years – 25 Years
Sex
ALL
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Incidence of protocol-defined dose limiting toxicities (DLTs) for APR-2020
Timeframe: 30 Days
Incidence and severity of treatment emergent adverse events (TEAEs), assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE)
Timeframe: 24 Months
Monitoring laboratory parameters, frequency and severity of TEAEs, assessed by the NCI CTCAE
Timeframe: 24 Months
Fraction of reticulocytes greater than the baseline value where reticulocyte increases are sustained over 3 consecutive measurements within 4 weeks
Timeframe: 24 Months
Proportion of subjects with a hemoglobin level of ≥ 8 g/dL starting 90 days after the last RBC transfusion, sustained over 2 consecutive measurements that are approximately 1 month apart
Timeframe: 24 Months