The Approach
HSCs can be used as allogenic or autologous transplant material for blood diseases, immune disorders, and some oncology indications as a result of their unique ability to differentiate into all types of blood cells. The genome of HSCs can be edited by transfecting the cells ex vivo with a viral vector containing the gene of interest before transplantation to implement a more robust therapeutic response.
The Challenge
Isolating a homogeneous population of HSCs is challenging, primarily because they are generally collected from patients or donors with different progenitor cells. Maintaining the HSCs in an undifferentiated state while scaling up cultures for clinical applications can be challenging as well.
Theragent’s Pharma 4.0-enabled GMP facility is fully equipped to support the successful scale-up of HSCs.
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Data Integration Solutions
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Manufacturing Transparency
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GMP Ready Facilities
HSCs have been FDA approved for use in patients with blood cancers. Additionally, ongoing clinical trials are investigating the use of HSCs and genetically modified HSCs in immune disorders, ß-thalassemia, and sickle cell disease, among others.
When developing HSCs, it is important to identify the appropriate cell markers to use for sorting and isolating the desired subset of non-differentiated HSCs. GMP-compliant cell culture facilities with capabilities to isolate, expand, and bank HSCs can minimize the challenges associated with HSC manufacturing. Theragent’s Pharma 4.0-enabled GMP facility is fully equipped to support the successful scale-up of HSCs. Contact us to learn more about our experience with stem cells.