1. 1 Demystifying the Examination of Stem Cell-Related Inventions Remy Yucel, Ph.D. Supervisory Patent Examiner Technology Center 1600 United States Patent & Trademark Office (571) 272-0781 irem.yucel@uspto.gov

2. 2 Demystifying the Examination of Stem Cell-Related Inventions Examination of Stem Cell-related inventions is done under the same rules that apply generally to any application

3. 3 Demystifying the Examination of Stem Cell-Related Inventions What kind of stem cell? –embryonic –fetal –adult Does the specification contain a clear definition? Does the definition correlate with the intended use as provided by the specification?

4. 4 Terminology: Defined by Function Totipotency-all cells in the placenta and embryo Pluripotency-all cells in the embryo Multipotency-a subset of cells in a given lineage/tissue

5. 5 Terminology: Defined by Function Cells must fulfill two criteria to be considered “Stem Cells:” must self-renew and must differentiate

6. 6 Types of Claims Cells Methods of making Methods of using (for therapeutic purposes, including screening for therapeutics) Methods of using (making of transgenic animals or cloning)

7. 7 Demystifying the Examination of Stem Cell-Related Inventions EmbryonicTransgenic Animals Fetal Therapeutic Methods Adult

8. 8 Demystifying the Examination of Stem Cell-Related Inventions Claims are analyzed under 35 U.S.C.: –101 –112, first and second paragraphs –102 and 103 (art rejections)

9. 9 35 U.S.C. 101 Is there a specific, substantial, and credible utility asserted? Is the claimed invention directed to statutory subject matter?

10. 10 35 U.S.C. § 112, first paragraph "The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor of carrying out his invention."

11. 11 35 U.S.C. § 112, first paragraph Is the full breadth of the claim adequately described? Is the claimed subject matter enabled? –How to make? –How to use?

12. 12 35 U.S.C. § 112, first paragraph enablement “The test of enablement is whether one reasonably skilled in the art could make or use the invention from the disclosures in the patent coupled with information known in the art without undue experimentation.” –United States v. Telectronics, Inc., 857 F.2d 778, 785, 8 USPQ2d 1217, 1223 (Fed. Cir. 1988) A patent need not teach, and preferably omits, what is well known in the art.

13. 13 Standard for Enablement The standard for determining whether the specification meets the enablement requirement: –Is the experimentation needed to practice the invention undue or unreasonable? Supreme Court decision of Mineral Separation v. Hyde, 242 U.S. 261, 270 (1916) M.P.E.P. 2164.01

14. 14 Standard for Enablement "In making the determination of enablement, the examiner shall consider the original disclosure and all evidence in the record, weighing evidence that supports enablement against evidence that the specification is not enabling.“ Training materials for assessing enablement of chemical and biotech applications www.uspto.gov/web/offices/pac/dapp/1pecba.htm

15. 15 In re Wands, 858 F.2d 731, 8 USPQ2d 1400 (Fed. Cir. 1988) The determination that "undue experimentation" would have been needed to make and use the claimed invention is not a single, simple factual determination.

16. 16 Wands Factors the nature of the invention the state of the prior art the predictability or lack thereof in the art the amount of direction or guidance present the presence or absence of working examples the breadth of the claims the relative skill of those in the art the quantity of experimentation needed

17. 17 Considerations for Treatment Methods using Stem Cells Reproducibility/predictability of obtaining appropriate stem cell population Maintenance of the proper differentiated/de-differentiated state in in- vivo environments Ability to extrapolate from ex vivo to in vivo

18. 18 Considerations for Treatment Methods using Stem Cells Targeting or migration of stem cells to appropriate sites in vivo Viability in vivo Challenges posed by genetic alteration of stem cells

19. 19 References that Demonstrate Unpredictability Regarding Germ Line Transmission (Transgenic Animals) Bradley et al., Modifying the Mouse: Design and Desire. Bio/Technology, Vol. 10, pages 534-539, May 1992. Babinet et al., Genome Engineering via Homologous Recombination in Mouse Embryonic Stem (ES) Cells: an Amazingly Versatile Tool for the Study of Mammalian Biology. An Adad Bras Cienc., Vol. 73, No. 4, pages 577-580, 2001. Moreadith et al., Gene Targeting in Embryonic Stem Cells: The New Physiology and Metabolism. J. Mol. Med., Vol. 75, pages 208-216, 1997.

20. 20 35 USC 102/103 Are the claimed cells novel and non- obvious?

21. 21 Prior Art Issues to Consider Characterization of the claimed cells Distinction between the claimed cells and those of the prior art Method of production Screens using stem cells

22. 22 Demystifying the Examination of Stem Cell-Related Inventions Thank You