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MDGN201 (TARGTEPO) is an autologous ex vivo gene therapy designed to treat anemia associated with chronic kidney disease (CKD) and end-stage renal disease (ESRD). Developed using the proprietary TARGT (Transduced Autologous Regenerative Gene Therapy) platform, the process involves taking a small biopsy of the patient's dermal tissue and transducing it with an adenoviral vector carrying the human erythropoietin (EPO) gene. The modified tissue, referred to as a "biopump," is then re-implanted subcutaneously into the patient. This approach aims to provide sustained, physiological levels of erythropoietin, potentially reducing the hemoglobin fluctuations and the need for frequent injections associated with conventional recombinant EPO therapy. Development was primarily led by Medgenics, which later became Aevi Genomic Medicine and was subsequently acquired by Cerecor (now Avalo Therapeutics). Clinical development was halted following corporate transitions and strategic shifts.
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