DETAILED ACTION
The examiner of record has changed. Please direct all further correspondence to Zachary J. Miknis, who can be reached at (571) 272-7008.
Notice of Pre-AIA or AIA Status
The present application, filed on or after March 16, 2013, is being examined under the first inventor to file provisions of the AIA .
Status of the Application
The claims and election of 14 January 2026 are entered.
Claims 2, 3, 7, 11, 13, 16, 20, 21, 24, 25, and 31-38 have been canceled. Claims 1, 4-6, 8-10, 12, 14, 15, 17-19, 22, 23, and 26-30 are pending and are being examined on the merits.
Election/Restrictions
Applicant’s election without traverse of Syn-TEF3 in the reply filed on 14 January 2026 is acknowledged.
Syn-TEF3 is free of the art. The search has been extended to other members of the genus, rejections for which are provided below.
Claim Rejections - 35 USC § 112
The following is a quotation of the first paragraph of 35 U.S.C. 112(a):
(a) IN GENERAL.—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 or joint inventor of carrying out the invention.
The following is a quotation of the first paragraph of pre-AIA 35 U.S.C. 112:
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.
Claim 30 is rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, because the specification, while being enabling for a method of treating kidney cancer in a subject in need comprising administering to the subject an effective amount of a compound of claim 1 or pharmaceutically acceptable salt thereof, does not reasonably provide enablement for a method of treating a disease or disorder in a subject in need thereof comprising administering an effective amount of a compound of claim 1. The specification does not enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the invention commensurate in scope with these claims.
“[T]o be enabling, the specification of a patent must teach those skilled in the art how to make and use the full scope of the claimed invention without ‘undue experimentation.’” Genentech Inc. v. Novo Nordisk 108 F.3d 1361, 1365, 42 USPQ2d 1001, 1004 (Fed. Cir. 1997); In re Wright 999 F.2d 1557, 1561, 27 USPQ2d 1510, 1513 (Fed. Cir. 1993); See also Amgen Inc. v. Chugai Pharm. Co., 927 F.2d 1200, 1212, 18 USPQ2d 1016, 1026 (Fed. Cir. 1991); In re Fisher 427 F.2d 833, 839, 166 USPQ 18, 24 (CCPA 1970). Further, in In re Wands 858 F.2d 731, 737, 8 USPQ2d 1400, 1404 (Fed. Cir. 1988) the court stated:
Factors to be considered in determining whether a disclosure would require undue experimentation have been summarized by the board in Ex parte Forman [230 USPQ 546, 547 (BdPatAppInt 1986)]. They include (1) the quantity of experimentation necessary, (2) the amount of direction or guidance presented, (3) the presence or absence of working examples, (4) the nature of the invention, (5) the state of the prior art, (6) the relative skill of those in the art, (7) the predictability or unpredict-ability of the art, and (8) the breadth of the claims.
A conclusion of lack of enablement means that, based on the evidence regarding each of the above factors, the specification, at the time the application was filed, would not have taught one skilled in the art how to make and/or use the full scope of the claimed invention without undue experimentation. In re Wright, 999 F.2d 1557,1562, 27 USPQ2d 1510, 1513 (Fed. Cir. 1993).
Nature of the Invention
The invention is drawn to treatment of a disease or disorder in a subject by administration of a compound of claim 1, A-B-C comprising a BET inhibitor as A, a linker B, and a C motif for binding to nucleotide repeats of GAAA.
Breadth of the Claims
The claim is broad with respect to the compound A-B-C of claim 1. The claim is extremely broad with respect to the disease or condition to be treated, as the claim offers no limits on the scope of what may or may not be treated.
State of the Prior Art
As noted below, the prior art recognizes compounds of A-B-C in the treatment of Friedreich’s ataxia (see e.g. US 10,517,877 B2 as discussed below). The compounds A-B-C of ‘877 overlap with the instantly claimed genus, but do have structural differences in the C segment as compared to the specifically claimed compounds of claim 28.
Relative Skill of those in the Art
The relative skill of those in the art is high.
Predictability or Unpredictability of the Art
There is a general lack of predictability in the pharmaceutical art. In re Fisher, 427, F. 2d 833, 166, USPQ 18 (CCPA 1970).
There is no universal treatment for any and all diseases or conditions as claimed.
Amount of Direction or Guidance Given
The specification guides that the compounds utilize repeats of a pyrrole/imidazole polyamide to target cancers having one or more repeats of GAAA. The disease is guided as being generally one with 50 or more GAAA repeats, and focuses more specifically on kidney cancers including carcinomas.
Presence/Absence of Working Examples
Repeat Expansions as targeted by the claimed compositions are noted in Example 2 as being primarily found in prostate and liver cancer databases, and are noted to be largely cancer subtype specific rather than a generically occurring feature of any and all cancers.
The working examples only show usage of the Syn-TEF3 compound in renal cell carcinoma cell lines Caki-1 and 786-o (see e.g. Example 3). Syn-TEF1 and Syn-TEF2 are disclosed, as well as Syn-TEF4 targeting GGAA repeats, but only Syn-TEF3 is tested for activity.
Quantity of Experimentation Necessary
The level of experimentation associated for only compounds of A-B-C might merely be routine. But the scope associated with “disease or disorder” as claimed essentially makes it such that the skilled artisan must test the claimed compounds against any and all known diseases or disorders. Such a level of experimentation is undue, since it is impossible to test for a “silver bullet” against all known diseases or disorders.
In view of the Wands factors as discussed above, it is the Examiner’s opinion that the claims are not fully enabled and one of skill in the art would have to engage in undue experimentation to practice the invention as claimed herein, without a reasonable assurance of success.
Claim Rejections - 35 USC § 102
In the event the determination of the status of the application as subject to AIA 35 U.S.C. 102 and 103 (or as subject to pre-AIA 35 U.S.C. 102 and 103) is incorrect, any correction of the statutory basis (i.e., changing from AIA to pre-AIA ) for the rejection will not be considered a new ground of rejection if the prior art relied upon, and the rationale supporting the rejection, would be the same under either status.
The following is a quotation of the appropriate paragraphs of 35 U.S.C. 102 that form the basis for the rejections under this section made in this Office action:
A person shall be entitled to a patent unless –
(a)(1) the claimed invention was patented, described in a printed publication, or in public use, on sale, or otherwise available to the public before the effective filing date of the claimed invention.
Claims 1, 8-10, 12, 14, 15, 17-19, 22, 23, 26, 27, 29, and 30 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Ansari et al. (US 10,517,877 B2, published 31 December 2019, hereafter referred to as ‘877).
The ‘877 patent discloses a compound
PNG
media_image1.png
262
1236
media_image1.png
Greyscale
. This comprises a compound A-B-C, where A is a BET inhibitor, B is a linker, and C is a formula as claimed where Z1 is selected from the three options, w is 7, and Z2 is
PNG
media_image2.png
144
90
media_image2.png
Greyscale
. This anticipates claim 1.
With respect to claims 8-10, 12, 14, 15, 17-19, 22, and 23, the A of ‘877 reads upon the claimed compounds.
With respect to claims 26 and 27, the linker of ‘877 reads upon the B as claimed.
With respect to claim 29, the compounds of ‘877 are disclosed to be prepared also as pharmaceutical compositions with a carrier (see e.g. Col.24 lines 17-22).
With respect to claim 30, the compounds of ‘877 are utilized to treat Freidreich’s ataxia, which is a disease or disorder (see e.g. Col.24 lines 17-22, Example 3, claim 27).
Allowable Subject Matter
Claims 4-6 and 28 are objected to as being dependent upon a rejected base claim, but would be allowable if rewritten in independent form including all of the limitations of the base claim and any intervening claims.
The following is a statement of reasons for the indication of allowable subject matter: The prior art fails to recognize or suggest compounds of formula A-B-C where C is the subformula as found in claims 4-6 or the specific compounds as found in claim 28. As noted above, US 10,517,877 B2 discloses similar compounds of formula A-B-C, but fails to suggest the specific C substructures as claimed. The claimed compounds are novel and unobvious pending consideration of any amendments applicants may make in response to this action.
Conclusion
Any inquiry concerning this communication or earlier communications from the examiner should be directed to ZACHARY J MIKNIS whose telephone number is (571)272-7008. The examiner can normally be reached M-F 9-5.
Examiner interviews are available via telephone, in-person, and video conferencing using a USPTO supplied web-based collaboration tool. To schedule an interview, applicant is encouraged to use the USPTO Automated Interview Request (AIR) at http://www.uspto.gov/interviewpractice.
If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Melissa Fisher can be reached at (571) 270-7430. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
Information regarding the status of published or unpublished applications may be obtained from Patent Center. Unpublished application information in Patent Center is available to registered users. To file and manage patent submissions in Patent Center, visit: https://patentcenter.uspto.gov. Visit https://www.uspto.gov/patents/apply/patent-center for more information about Patent Center and https://www.uspto.gov/patents/docx for information about filing in DOCX format. For additional questions, contact the Electronic Business Center (EBC) at 866-217-9197 (toll-free). If you would like assistance from a USPTO Customer Service Representative, call 800-786-9199 (IN USA OR CANADA) or 571-272-1000.
/ZACHARY J MIKNIS/Patent Examiner, Art Unit 1658