Manufacturing of gene-engineered hematopoietic stem cells

Viral transduction of CD34⁺ cells is a promising approach for treating inherited disorders such as sickle cell disease, ß-thalassemia, or primary immunodeficiencies (de Dreuzy E. et al. 2016; Lebensburger, J. and Persons D.A. 2008; Papanikolaou E. and Anagnou N.P. 2010). The CliniMACS Prodigy® HSC Engineering process allows for manufacturing of gene-engineered HSCs from human CD34⁺ cells.

Stepwise procedure to manufacture gene-engineered HSCs

The CliniMACS Prodigy Cell Manufacturing Platform automates the generation of gene-engineered HSCs by viral transduction in a functionally closed system. All steps are automated and manufacturing takes place in sterile, single-use tubing sets, ensuring highly reproducible and standardized manufacturing processes.

The CliniMACS Prodigy HSC Engineering process uses human CD34⁺ cells as cellular starting material. They can be easily enriched from mobilized leukapheresis with the CliniMACS Prodigy or the CliniMACS Plus Instrument. Enriched CD34⁺ cells are transferred to a cell bag, then connected to the CliniMACS Prodigy Tubing Set (TS) 520.

After transduction, the final cell product containing gene-engineered HSCs is washed twice and harvested in a 100 ml buffer of your choice. The gene-engineered HSCs are then ready for further processing, e.g., cryopreservation, or functional testing.
 

In-process and quality control to clinical flow cytometry

In-process and quality control (IPC/QC) are required for consistent cell manufacturing. Integrated sampling pouches on the CliniMACS Tubing Sets allow for controls to be collected at any time throughout the cell manufacturing process. Miltenyi Biotec provides complete solutions with its MACS Flow Cytometers and a wide portfolio of MACS IVD Antibodies that are produced to quality management system ISO 13485.

When using a MACSQuant® Instrument, Express Modes allow you to fully automate and standardize your flow cytometry process. The MACSQuant Instrument and the MACS Flow Cytometry portfolio are for research use only.