Vector Claims in Gene Therapy Applications: In vivo vs. In vitro Utilities

1. Vector Claims in Gene Therapy Applications: In vivo vs. In vitro Utilities Deborah Reynolds SPE GAU 1632 703-305-4051

2. 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."

3. How to Make

4. Analyze the scope of the claim • Are all materials/components known? • Is there sufficient guidance to put the components together to arrive at the claimed vector?

5. How to Use Analyzing the function of the vector

6. How does the specification disclose to use the vector? • If the specification discloses a specific, substantial, and credible utility for the claimed vector, and the skilled artisan could use the claimed vector without undue experimentation, then the claim complies with the how-to-use prong of the enablement requirement.

7. Scope of Enablement • “the specification must teach those skilled in the art how to make and use the full scope of the claimed invention without ‘undue experimentation’.” In re Wright, 999 F.2d 1557, 1561, 27 USPQ2d 1510, 1513 (Fed. Cir. 1993).

8. Scope of Enablement • “That claims are interpreted in light of the specification does not mean that everything in the specification must be read into the claims.” Raytheon Co. v. Roper Corp., 724 F.2d 951, 957, 220 USPQ 592, 597 (Fed. Cir. 1983), cert. Denied, 469 U.S. 835 (1984).

9. Intended Use Limitation • When a compound or composition is limited by a particular use, enablement of that claim should be evaluated based on that limitation. See In re Vaeck, 947 F.2d 488, 495, 20 USPQ2d 1438, 1444 (Fed. Cir. 1991).

10. No Limitation of Use • When a product claim is not limited by a recited use, any enabled use that would reasonably correlate with the entire scope of that claim is sufficient to preclude a rejection based upon a lack of enablement. See 35 U.S.C. 112, 1st paragraph Enablement Training Manual, August 1996, Overview, page 20.

11. Example G 35 U.S.C. 112, 1st paragraph Enablement Training Manual, August 1996 • 1. A viral vector comprising: a virus comprising a cell binding receptor on the surface thereof and a gene of interest, not normally present in the virus, inserted within the DNA of the virus; and algernin complexed to the cell binding receptor of the virus. • 2. A pharmaceutical composition comprising a therapeutically effective amount of the complex of claim 1 and a pharmaceutically acceptable carrier. • 3. A method for introducing a gene of interest into a cell comprising contacting said cell with the viral vector of claim 1.

12. Example G cont. • The specification discloses an in vitro use for the viral vector of claim 1 and clearly discloses how to make and use the viral vector in the in vitro environment. Since claim 1 does not recite any environment of use, only one enabled use covering the scope of the claim is needed to enable the claim. Therefore, the disclosure with respect to the in vitro use of the viral vector is sufficient to enable claim 1 and it would be inappropriate to include claim 1 in a rejection under 35 U.S.C. 112, first paragraph. (Emphasis added).

13. Example-1 • 1. A viral vector for use in gene therapy comprising: a virus comprising a cell binding receptor on the surface thereof and a gene of interest, not normally present in the virus, inserted within the DNA of the virus.

14. Example-2 • 1. A viral vector comprising: a virus comprising a cell binding receptor on the surface thereof and a therapeutic gene of interest, not normally present in the virus, inserted within the DNA of the virus.

15. Example-3 • 1. A viral vector for delivering a gene of interest to a cell comprising: a virus comprising a cell binding receptor on the surface thereof and a gene of interest, not normally present in the virus, inserted within the DNA of the virus.

16. Example-4 • 1. A viral vector comprising: a virus comprising a cell binding receptor on the surface thereof and a gene of interest, not normally present in the virus, inserted within the DNA of the virus; wherein, when delivered to a cell, the gene of interest is expressed at a therapeutic level.

17. Example-5 • 1. A viral vector comprising: a virus comprising a cell binding receptor on the surface thereof and a nucleic acid sequence encoding CFTR, inserted within the DNA of the virus.

18. Example-6 • 1. A method of delivering a viral vector to a mammal comprising: contacting cells of said mammal with a virus comprising a cell binding receptor on the surface thereof and a gene of interest, not normally present in the virus, inserted within the DNA of the virus.

19. Example-7 • 1. A method of delivering a viral vector to a mammal comprising: contacting cells of said mammal with a virus comprising a cell binding receptor on the surface thereof and a nucleic acid sequence encoding CFTR, inserted within the DNA of the virus.

20. Thank You! • Questions?