DETAILED ACTION
Claims 1-4 and 6 are currently pending in the instant application and are rejected.
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 .
Response to Amendment and Arguments
Applicant's amendment and arguments filed 4 February 2026 have been fully considered and entered into the instant application. Applicant’s amendment to delete “the entire disclosures of which are incorporated herein by reference” from the cross reference to related applications paragraph of the specification has overcome the objection to the specification. The amendment to claim 1 and the cancelation of claim 5 has overcome the objections to claims 1 and 5. Applicant’s amendment has overcome the 35 USC 102(a)(1) rejection of claims 1-6 as being anticipated by LIU et al. as claim 1 has been amended to limit the nonsense-mediated mRNA decay related disease to muscular dystrophy or beta-thalassemia as LIU et al. discloses the treatment of ADPKD with the compound:
PNG
media_image1.png
292
390
media_image1.png
Greyscale
.
Applicant’s amendment has overcome the 35 USC 102(a)(1) rejection of claims 1-4 and 7 as being anticipated by JIAJING CAI et al. as claim 1 has been amended to limit the nonsense-mediated mRNA decay related disease to muscular dystrophy or beta-thalassemia as JIAJINF CAI et al. discloses the treatment of triple-negative breast cancer with the compound:
PNG
media_image1.png
292
390
media_image1.png
Greyscale
. Additionally, claim 7 has been canceled.
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.
(a)(2) the claimed invention was described in a patent issued under section 151, or in an application for patent published or deemed published under section 122(b), in which the patent or application, as the case may be, names another inventor and was effectively filed before the effective filing date of the claimed invention.
Claim(s) 1-4 and 6 is/are rejected under 35 U.S.C. 102(a)(1) and 35 U.S.C. 102(a)(2) under as being anticipated by LAMMERDING et al. (WO 2020/060779, IDS filed 8/15/2023, Foreign Patent Documents Cite No. 4).
LAMMERDING et al. provides the compound NVP-BEZ235 in paragraph [0083], page 25, as the PIKK is ATR wherein the inhibitor may be an ATR inhibitor including NVP-BEZ235. LAMMERDING et al. provides that Limb-girdle muscular dystrophy is the laminopathy treated in paragraph [0056], page 18 and paragraph [0100] which discloses that the laminopathy is congenital muscular dystrophy or Limb-girdle muscular dystrophy. The claims provides a method of treating a laminopathy by administering an inhibitor wherein the laminopathy is congenital muscular dystrophy and limb-girdle muscular dystrophy (claim 9) and wherein the inhibitor is NVP-BEZ235 (claim 20). The compound NVP-BEZ235 is the compound 2:
PNG
media_image1.png
292
390
media_image1.png
Greyscale
, see page 8 of the instant specification which provides that chemical formula 2 is known as BEZ235 and Dactolisib.
In regards to the claims 2 and 3 which claim that the compound or the pharmaceutically acceptable salt thereof inhibits nonsense-mediated mRNA decay (NMD) (instant claim 2) or inhibits phosphorylation of UPF1 (instant claim 3), a known use of a product in a disease is not rendered novel by a newly discovered mechanism of action as the claimed mechanism of action ,in vivo, i.e. inhibiting NMP or UPF1, is considered to be a characteristic inherent to the compound, even if this characteristic was not known in the prior art. "Products of identical chemical composition cannot have mutually exclusive properties." In re Spada, 911 F.2d 705, 709, 15 USPQ2d 1655, 1658 (Fed. Cir. 1990). A chemical composition and its properties are inseparable. Therefore, if the prior art teaches the identical chemical structure, the properties applicant discloses and/or claims are necessarily present. Additionally, see also In re Papesch, 315 F.2d 381, 391, 137 USPQ 43, 51 (CCPA 1963) ("From the standpoint of patent law, a compound and all its properties are inseparable.").
In regards to claim 4, muscular dystrophy is a genetic disease caused by NMD due to a premature termination codon (PTC) associated with a nonsense mutation or frameshift mutation, see pages 17-19 of the instant specification.
Conclusion
Applicant's amendment necessitated the new ground(s) of rejection presented in this Office action. Accordingly, THIS ACTION IS MADE FINAL. See MPEP § 706.07(a). Applicant is reminded of the extension of time policy as set forth in 37 CFR 1.136(a).
A shortened statutory period for reply to this final action is set to expire THREE MONTHS from the mailing date of this action. In the event a first reply is filed within TWO MONTHS of the mailing date of this final action and the advisory action is not mailed until after the end of the THREE-MONTH shortened statutory period, then the shortened statutory period will expire on the date the advisory action is mailed, and any nonprovisional extension fee (37 CFR 1.17(a)) pursuant to 37 CFR 1.136(a) will be calculated from the mailing date of the advisory action. In no event, however, will the statutory period for reply expire later than SIX MONTHS from the mailing date of this final action.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to REBECCA L ANDERSON whose telephone number is (571)272-0696. The examiner can normally be reached Monday-Friday from 6am-2pm.
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, Andrew Kosar can be reached at 571-272-0913. 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.
/REBECCA L ANDERSON/Primary Examiner, Art Unit 1626 ____________________ 25 February 2026
Rebecca Anderson
Primary Examiner
Art Unit 1626, Group 1620
Technology Center 1600
Prosecution Insights