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APR-2020

Development stage: Phase 1
Lead developer: Apriligen
Modality: Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration: Intravenous

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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