Appeal No. 1998-0667 Page 8
Applicatiorr No. 081280,306
On the basis of animal studies, and controlled testing in a limited
number of humans (referred to as Phase I testing), the Food and Drug
Administration may authorize Phase II clinical studies. See 21 U.S.C.
5 355(i)(I); 21 C.F.R. § 31 2.23(a)(5), (a)(8) (1 994). Authorization for a
Phase II study means that the drug may be administered to a larger
number of humans, but still under strictly supervised conditions. The
purpose of the Phase II study is to determine primarily the safety of the
drug when administered to a larger human population, as well as its
potential efficacy under different dosage regimes. See 21 C.F.R.
§ 312.21(b).
FDA approval, however, is not a prerequisite for finding a
compound useful within the meaning of the patent laws. Scott, 34 F.3d
1058, 1063, 32 USPQ2d 1 1 15, 1 120. Usefulness in patent law, and in
particular in the context of pharmaceutical inventions, necessarily includes
the expectation of further research and development. The stage at which
an invention in this field becomes useful is well before it is ready to be
administered to humans. Were we to require Phase II testing in order to
prove utility, the associated costs would prevent many companies from
obtaining patent protection on promising new inventions, thereby
eliminating an incentive to pursue, through research and development,
potential cures in many crucial areas such as the treatment of cancer.
While the claims involved in Brana were directed to chemical compounds taught to be
useful in treating cancer, we believe these principles can be applied to the claims at
hand directed to methods of gene therapy, especially in light of the examiner's apparent
holding that gene therapy in general is non-enabled.
The references relied upon by the examiner themselves document various
clinical trials of gene ,therapy techniques. One example is found in Wagner which
states "[cllinical trials are now in progress to evaluate the therapeutic potential of
antisense [oligodeoxynucleotides] in several human diseases . . .." Wagner, page 333,
first full paragraph. Miller states at page 197, right-hand column, "[olf the gene therapy
protocols that have so far entered clinical trials . . .." Marshall sets forth a table at page
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