MANUFACTURING
and for the final administration of stemgene treatments to patients. Yet, the stemgene medicine industry currently exists in a state in which only a few companies quantify the tissue stem cell-specific fraction during their biomanufacturing process.
Both history and miseducation help to explain
why tissue stem cell-specific fraction or dosage continues to be a SQA. For most of the more than six-decade history of tissue stem-cell science and medicine, there has been no method available to measure this CQA. Until recently, only one method was available for estimating the specific fraction and dosage of only one type of human tissue stem cells, HSCs. This method, the SCID mouse repopulating cell (SRC) assay, is very expensive because it requires as many as 50 mice for a single count, requires 12 – 16 weeks to complete, and is not reliable. Because of these prohibitive requirements, only a few gene therapy companies have been able to afford using the SRC assay to monitor HSC- specific fraction during the biomanufacturing of genetically engineered HSCs. There is also widespread confusion in the
regenerative medicine industry that other methods are able to quantify tissue stem cells specifically, though they are not. This is a common misconception by non-experts for flow cytometry, because of its high sensitivity, and colony forming unit (CFU) assays, which are
72 | Clinical Trial Supply Handbook
widely used for certifying umbilical cord blood units for HSC transplantation medicine. However, both of these, as well as other mistaken methods, lack sufficient specificity to quantify tissue stem cells distinct from more abundant committed progenitor cells, which are always present in isolated and enriched tissue stem cell-containing preparations. Committed progenitor cells don’t have the long-term curative properties of tissue stem cells. A recently introduced technology, called kinetic stem cell (KSC) counting, solves this specificity problem and has the potential to become a routine solution for measurement of the specific fraction and dosage of stem cells from any human tissue. The supply industry for stemgene clinical trials can accelerate the advance of tissue stem cell-specific fraction and dosage from being SQAs to being powerful new CQAs. As new technologies for specific quantification of therapeutic tissue stem cells are introduced, their rapid adoption by suppliers of treatment products and process services can drive improvements in the development and success of stemgene clinical trials. Stemgene clinical trial suppliers can provide sponsors with stemgene medicinal products, developed with and certified by stem cell-specific fraction and dosage, as well as provide consultant services that help sponsors implement these advantageous new CQAs into their own biomanufacturing processes.
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