The Approach
HSCs can be used as allogeneic 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 gene-modified ex vivo with a vector containing the gene of interest before transplantation to provide corrected gene sequences in all of the downstream prodigy. This potential is being heavily studied for the treatment of monogenic gene disorders such as sickle cell anemia and has recently led to two new approved therapies for use in beta-thalassemia.
The Challenge
Isolating a large number of a homogeneous population of HSCs is challenging. There are multiple potential sources, but all have their issues. HSCs are relatively rare in the peripheral blood, cord blood volume is very limited, and while the cells are more frequent in bone marrow, large bone marrow collections are invasive and very taxing on the donor. Post isolation, maintaining the HSCs in an undifferentiated state while expanding the cells to sufficient quantities to be clinically applicable is an additional challenge faced by companies developing such replacement therapies.
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
GMP-compliant cell culture facilities with capabilities to isolate, expand, and cryopreserve 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.