The high failure rate of compounds in clinical development is a major problem for the pharmaceutical industry. Some 90% of compounds fail in clinical trials, mainly due to low efficacy and safety issues—costing the industry hundreds of millions of dollars. The biochemical assays and animal models used in the current drug development process need to be improved—and new tools based on cell lines generated by genetically reprogrammed cells may offer a solution. Promising markets are beginning to form around these new tools, but, as with any technology, companies should be aware of both the risks and benefits.
Induced pluripotent stem cells (iPSC) are somatic cells that have been reprogrammed to have embryonic stem cell-like properties, specifically the ability to differentiate into other cell types. Using iPSC methods, a researcher can harvest a person’s skin cells, induce pluripotency, and then encourage those cells to differentiate into another type of cell, like a neuron or cardiomyocyte. One major benefit of this method is that the induced stem cells are derived from nonembryonic tissue, alleviating many ethical concerns.
Since the introduction of this technique in 2006, researchers have successfully created both iPSC-derived healthy state and disease-specific cell lines that have potential to replace current tools used in toxicity and drug efficacy screening, respectively. Within only a few short years of their invention, a commercial market is forming around the use of iPSC as life science tools.
A number of commercial opportunities exist all along the iPSC value chain, from the reagents used to generate iPSCs to the cell lines that are derived from iPSCs (Figure).