The search for stem cell therapies includes research on embryonic stem cells, adult stem cells and induced pluripotent stem (iPS) cells. The idea behind iPS cells is this: through various cell engineering techniques, in what is now a very rapidly developing field, a multipotent adult stem cell -- or already differentiated adult (somatic) cell -- can be returned to a pluripotent, embryonic stem cell-like state and can then be coaxed into becoming any cell in the body as part of a stem cell therapy approach.
Fate Therapeutics, a privately owned company on the cutting edge of iPS cell technology, has received the first notice of allowance issues by the U.S. patent office on iPS technology. The patent allowance is entitled “Methods for Reprogramming Somatic Cells.”
Upon issuance, the patent will cover foundational induced pluripotent stem cell (iPSC) technology for identifying agents that enable the reprogramming of human somatic cells, including pluripotency genes, small molecules and biologics. The invention by Rudolf Jaenisch, M.D., founding member of the Whitehead Institute for Biomedical Research and scientific founder of Fate Therapeutics, has a priority date of November 26, 2003 and is believed to be the earliest art that describes broad methods and key agents to reprogram human somatic cells to a pluripotent state. Fate Therapeutics holds an exclusive license to the application in commercial fields, including for drug discovery and therapeutic purposes.
We recently reported that Stanford researchers have converted adipose (fat) stem cells directly into iPS cells using only minicircles - rings of DNA about one-half the size of those usually used to reprogram cells - to induce pluripotency. As this technique is pursued, and if it is as efficient as suggested, questions about whether it falls within the realm of the Fate Therapeutics patent will surely arise.
The possibility that somatic cells could be directly converted to multipotent or pluripotent, therapeutically usable stem cells without first becoming iPS cells would also presumably provide an end run around this patent. Such a possibility was suggested recently other Stanford researchers who succeeded in transforming mouse skin cells in a laboratory dish directly into functional nerve cells with the application of just three genes. The cells make this transition without first becoming a pluripotent type of stem cell.
The Notice of Allowance issued to Fate Therapeutics represents the earliest allowed claims in the U.S. for iPSC technology. In this 2003 application (20080280362), Dr. Jaenisch first describes the groundbreaking potential to generate human pluripotent cells from somatic cells without using embryos, oocytes and/or nuclear transfer technology and how reprogrammed somatic cells can enable autologous cell therapy, including (in theory) the treatment, prevention or stabilization of neurological diseases such as Alzheimer’s, Parkinson’s or ALS.
In addition, the application covers compositions used in screening for agents to generate these pluripotent cells and further describes specific agents that can be used to reprogram human somatic cells, including certain genes, classes of small molecules and pluripotency proteins. Fate Therapeutics also holds an exclusive license to other inventions of Dr. Jaenisch relating to iPSC technology including PCT/US2008/004516 with a priority date of April 7, 2007, which describes the reprogramming of human somatic cells using one or more pluripotency factors, including Oct3/4, Sox2 and/or Klf4, and combinations thereof.
Because iPSCs have been shown to behave similarly to embryonic stem cells with the ability to differentiate into various cell types, such as cardiomyocytes, hepatocytes, neurons and pancreatic cells, and can be created from any adult somatic cell, like a skin cell, iPSC technology has significant commercial and medical value. For example, iPSCs can be used to assess drug toxicity across diverse genetic backgrounds, enable the development of disease model systems for basic research and drug discovery and may ultimately result in personalized (autologous) cell therapies.
Adapted from the Fate Therapeutics announcement.
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