We propose to create a world-class cell manufacturing facility in California with an initial task of taking 3,000 donor tissue samples and creating induced pluripotent stem cell (iPSC) lines from the samples. iPSCs are extremely valuable as they are able to self-renew and can be converted into virtually all human tissues. In addition, since they are made from adult cells obtained from a specific donor, they can be used to model the biological profile of that donor, which is especially relevant if the donor has a particular disease. Therefore, iPSCs can be used to create cells or tissues that mimic disease and can be used in drug discovery efforts. To help make these efforts more efficient, we have developed a system that greatly improves the efficiency of making standardized iPSC lines. Typically, researchers generate iPSCs by hand, which limits the cells utility due to researcher variability and an inability to generate large numbers of cells. We seek to circumvent these problems through our system, which is completely automated from receipt of the tissue sample to eventual banking of large stocks of well-defined iPSC lines. We are able to generate very large numbers of cells from many donors, which we believe will facilitate the use of iPSC technology to discover treatments and cures for many diseases. The automated system we have developed is capable of generating all of the cell lines requested by CIRM, as well as many more for other investigators in California.
As a result of CIRM’s creation, California has established itself as a key player in all areas of stem cell research. If awarded this grant, our group will be able to integrate our unique high-throughput induced pluripotent stem cell (iPSC) derivation, characterization, and expansion platform into this rich environment. Based on our previous experience, our efforts to build innovative programs to change the landscape of stem cell research have enabled us to accelerate research and coordinate the stem cell communities at the institutions near our home location, which we hope to replicate in California. The use of iPSCs as a technology for basic biological research, drug discovery, and therapeutic applications has enormous potential, but requires major advances before translation. Typically, researchers generate iPSCs by hand, which limits the cells utility due to researcher variability and an inability to generate large numbers of cells. We seek to address these problems through our platform, which is completely automated from receipt of the tissue sample to eventual banking of large stocks of well-defined iPSC lines. We hope that our proposed program would coordinate activities between our home community and California to build a larger cohort of collaborators. These activities should allow California and its citizens to be among the first communities to see the innovations resulting from this research in the form of medical breakthroughs and commercial opportunities.