BERGISCH GLADBACH, February 5th – Miltenyi Biotec today brings the use of genetically engineered human hematopoietic stem cells (HSCs) in the treatment of inherited red blood cell disorders one step closer. With the global market release of the CliniMACS Prodigy® Hematopoietic Stem Cell (HSC) Engineering System, a new, automated protocol for the world-renowned CliniMACS Prodigy Instrument, researchers and clinicians can automate the manufacturing process for gene-engineered HSCs.
Viral transduction of CD34+ cells is a promising approach for treating many inherited disorders, such as sickle cell disease, ß-thalassemia, or primary immunodeficiencies, including severe combined immunodeficiency (SCID). The HSC Engineering process enables manufacturing of gene-engineered HSCs from human CD34+ cells and boasts efficient transduction at low volume in a GMP-compliant, closed setting.
“The HSC Engineering process provides the complete workflow for manufacturing gene-engineered HSCs by viral transduction from human CD34+ cells,” explains Eleni Papanikolaou, PhD, R&D Manager for Hematopoietic Stem Cell Gene Therapy and Graft Engineering at Miltenyi Biotec. “Because the entire process takes place inside the closed environment of the Prodigy, the final cellular product can be ready for further processing, including cryopreservation or functional testing, within two to three days.”
All parts of the three-step process take place within the CliniMACS Prodigy Cell Manufacturing Platform, and are completely automated. Manufacturing takes place in sterile and single-use tubing sets, ensuring highly reproducible and standardized manufacturing processes. The following basic methodology is used:
Preparation of CD34+ cells as cellular starting material, enriched from mobilized leukapheresis using the CliniMACS Prodigy or the CliniMACS Plus.
Transduction of HSCs with lentiviral vector (repeated, if necessary, to increase transduction efficiency).
Harvest of gene-engineered HSCs in 100 mL buffer
In-process control and quality control (IPC/QC) is enabled by integrated sampling pouches on the CliniMACS Tubing Sets, which allow for controls to be collected at any time during, and at the end of the cell manufacturing process.
For further information on the HSC Engineering process, as well as the CliniMACS Prodigy instrument itself, visit Manufacturing of gene-engineered hematopoietic stem cells.
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