Cellular Dynamics International has already situated itself as an early leader—if not the leader—in large-scale manufacturing of human cells from induced pluripotent stem cells. Today the company says it has strengthened its market position with three new patents related to making stem cells from human blood samples.
Madison, WI-based Cellular Dynamics (NASDAQ: ICEL) says it has accumulated a portfolio of more than 800 issued and pending patents since the company was founded in 2004 by University of Wisconsin-Madison biologist James Thomson, who derived the first human embryonic stem cell line in 1998.
Thomson and his research team are among a small handful of scientists around the world who have worked to reprogram adult human cells into an embryonic-like state, creating what’s called induced pluripotent stem cells (iPS). The iPS cells can then be coaxed into various types of functioning cells, including neurons, heart and liver cells, and the cells that line blood vessels.
The field has moved fast. Cellular Dynamics has turned that science into a business by honing a manufacturing process that allows it to produce cells in industrial quantities and with industrial consistency. The company markets its cells mainly for drug companies to use in their research and development: for example, to test the toxicity of a drug candidate in human heart or liver cells before getting clearance to do real human testing. CDI also markets its cells for testing in chemical safety, food nutrition research, and to create stem cell banks.
Eventually, the company wants to partner with biopharmaceutical companies to develop cells that can be used as therapies themselves, a more uncertain proposition for several reasons.
Cellular Dynamics has been making iPS cells from both skin and blood samples, but the recently issued patents protect the company’s intellectual property around reprogramming cells from blood—the preferred tissue because of the “standardized and relatively non-invasive nature of blood collection,” Cellular Dynamics says. Its method needs only small amounts of blood, on par with a typical doctor’s office blood draw. Meanwhile, skin punch biopsies can be painful, require a local anesthetic, and could leave scars, the company says.
CEO Bob Palay also says the patents boost the company’s profile in the “large-scale manufacturing of iPS lines and the downstream cells,” and they give its customers “freedom to operate.”
But a law professor who follows stem cell research and biotech patents says the additions to the portfolio probably aren’t “earth-shaking.” “I don’t think it gives them a big market advantage,” says Hank Greely, director of Stanford University’s Center for Law and the Biosciences.
The first patent covers the conversion of hematopoietic progenitor cells from human blood into iPS cells using Cellular Dynamics’ previously patented episomal reprogramming method. That method involves introducing episomal vectors, or circular pieces of DNA, into an adult cell, which revert it to stem cell form before the vectors depart the cell. That’s an improvement over the original viral reprogramming method that involves inserting four genes into the adult cell’s DNA, Cellular Dynamics says. There are concerns over the potential side effects of that infusion of foreign DNA into the cell’s genome, including errors that could cause tumors.
The other two patents cover the production of iPS cells from freshly collected and banked blood samples. (For the hardcore scientists and patent attorneys out there, the exact U.S. patent numbers are 8,691,574, 8,741,648, and 8,765,470.)
Greely says the patent covering episomal reprogramming of cells from blood samples is likely the most important of the three. “But for it to turn out to be important, first, iPSCs have to turn out to be really important,” he says. “I think we expect they will, but nobody knows for sure.”
And since the first two patents cover one type of cell, there’s always the risk that a competitor will come up with a different method that achieves the same result without violating the patent, Greely says.
“Even if the cell type has some advantages over using other cell types, the market power is limited. If you charge too much, somebody else could make iPSCs from skin cells or some other cell type that doesn’t infringe their patent,” Greely says. “[The blood sourcing is] only really important if their route is powerfully the best. And there are lots of different routes.”