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APR-2020
- Development stage
- Phase 1
- Lead developer
- Apriligen
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
APR-2020 is an autologous CD34+ hematopoietic stem cell gene therapy developed by Apriligen for the treatment of Diamond-Blackfan anemia (DBA) caused by mutations in the RPS19 gene. The therapy involves harvesting a patient's own CD34+ cells and transducing them ex vivo with a proprietary lentiviral vector (CLIN LV EFS coRPS19 PRE*) that delivers a functional, codon-optimized copy of the RPS19 gene. These genetically modified cells are then re-infused into the patient, where they are intended to engraft and restore normal erythropoiesis, thereby reducing or eliminating the need for chronic blood transfusions. APR-2020 is specifically targeted at pediatric and adolescent patients who are transfusion-dependent and steroid-resistant.
- Other names
- CLIN LV EFS coRPS19 PRE*
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