DETAILED ACTION
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 Application/Amendment/Claims
This Office action is in response to the communications filed on January 8, 2026.
Currently, claims 3, 5, 7-11, and 14 are pending and under examination on the merits in the instant application.
The following rejections are either newly applied or are reiterated and are the only rejections and/or objections presently applied to the instant application.
Information Disclosure Statement
The information disclosure statement (IDS) submitted on January 8, 2026 has been considered by the examiner. Note that Citations Nos. “FP10”, “FP11”, and “FP13” are not considered as they are in non-English language. Note that contrary to the assertion that an English language translation is attached for each of the aforementioned citations as marked in the IDS, no such English language translation is found. Note that Citation No. “FP12” is considered only insofar as the English language title and abstract as submitted by applicant.
Response to Arguments and Amendments
Withdrawn Rejections
Any rejections/objections not repeated in this Office action are hereby withdrawn.
Maintained Rejections
Claim Rejections - Improper Markush Grouping
Claims 3, 5, 7-11, and 14 remain rejected for containing an improper Markush grouping of alternatives for the reasons as set forth in the Office action mailed on July 9, 2025 and for the reasons set forth below.
Applicant's arguments filed on January 8, 2026 have been fully considered but they are not persuasive. Applicant argues the alternatively recited seven SEQ ID NOs constitute a proper Markush group because they all bind to GAA intron 2 pre-mRNA and have the PMO backbone, wherein the “focus of the invention is not the base sequences per se” but the “PMO” compound. In response, applicant’s attention is directed to the fact that applicant’s personal opinion as to what the “focus” of the invention should be is irrelevant to show that all seven recited nucleotide sequences, the essential and claimed element for providing the alleged common use, share a common nucleotide sequence responsible for providing the common function/use. Is applicant arguing that the PMO compound can function without the SEQ ID NOs? In fact, from a scientific viewpoint, the PMO compound allegedly providing the common use of restoring GAA protein can only be feasible because of the nucleotide sequences claimed in the instant application. That is, regardless of whether or not applicant personally deems that the “focus of the invention” should be the PMO structure, not the SEQ ID NOs per se, the objective and scientific fact remains that the function of the claimed compound is imparted by the nucleotide sequences because a nucleotide sequence without the claimed PMO structure (e.g., 2’-O-methyl modification structure) can also function to restore GAA protein. That is, it is the nucleotide sequence that imparts the alleged common use shared by the alternatively recited SEQ ID NOs. In fact, the “compound” itself expressly requires and contains the nucleobase sequences represented by the consecutive “Nu” included in all of “formula (I)” and “formula (IV)” claimed in the instant case, wherein each of the consecutive “Nu” forms “a targeting sequence” of one of the recited SEQ ID NOs. Hence, the alternatively recited SEQ ID NOs in the improper Markush grouping are very much the “focus” of the claimed invention as each “Nu” is claimed to be included in the “compound”, wherein the “Nu” in the “compound” forms each of the claimed SEQ ID NOs. Further, applicant’s personal opinion that the “focus of the invention” should be the PMO structure, not the SEQ ID NOs, is insufficient to demonstrate that all seven alternatively recited SEQ ID NOs do indeed share a significant nucleotide sequence similarity, and furthermore, applicant’s personal opinion does not refute the objective fact that applicant’s elected SEQ ID NO:83 shares only three non-consecutive nucleotides with SEQ ID NO:13 that is also recited in the improper Markush grouping as expressly illustrated in the last Office action.
Applicant incorrectly argues that all SEQ ID NOs share “a targeting sequence”. It is noted that it is scientifically impossible to demonstrate that all SEQ ID NOs share the same targeting sequence when applicant is unable to refute the sequence alignment between SEQ ID NO:83 and SEQ ID NO:13 presented in the last Office action, wherein the two SEQ ID NOs do not whatsoever share any overlapping sequence, much less the entire “targeting sequence”. Furthermore, SEQ ID NO:3 pointed out by applicant is a 616-mer sequence. See the sequence information of SEQ ID NO:3 as reproduced below.
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Applicant did not provide any objective, factual evidence (e.g., sequence alignment) showing that all of the alternatively recited SEQ ID NOs share a significant sequence similarity in view of the 616-mer sequence of SEQ ID NO:3 mentioned by applicant. The examiner is unable to see any relevance between the 616-mer sequence of SEQ ID NO:3 pointed out by applicant and the allegedly shared “targeting sequence” among all of the recited SEQ ID NOs in the improper Markush grouping.
Applicant incorrectly argues that a common use “in and by itself” can support a proper Markush grouping. Contrary to applicant’s argument, alternatively recited members sharing only a common use “in and by itself” does not constitute a proper Markush grouping. The test for a proper Markush grouping is when both a common structure and a common use are met by the alternatively recited members of the group, wherein the grouping is deemed improper when either of the requirements is not satisfied.
“A Markush grouping is proper if the members of a group share a single structural similarity and a common use.” (emphasis added). See MPEP §2117.
“A Markush claim contains an “improper Markush grouping” if: (1) The species of the Markush group do not share a “single structural similarity,” or (2) the species do not share a common use…When an examiner determines that the species of a Markush group do not share a single structural similarity or do not share a common use, then a rejection on the basis that the claim contains an “improper Markush grouping” is appropriate.” (emphasis added). See page 7166, middle column of “Supplementary Examination Guidelines for Determining Compliance with 35 U.S.C. 112 and for Treatment of Related Issues in Patent Applications” in Federal Register, Volume 76, Number 27, published on February 9, 2011.
To reiterate and emphasize the impropriety of the instantly claimed Markush grouping of alternatively recited SEQ ID NOs, the nucleotide sequence alignment between SEQ ID NO:83 and SEQ ID NO:13 as set forth in the last Office action is hereby reproduced below.
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Since applicant did not provide any objective evidence to rebut the sequence dissimilarity between SEQ ID NO:83 and SEQ ID NO:13, and since applicant failed to provide any persuasive arguments pertaining to the expressly claimed different SEQ ID NOs allegedly sharing a significant nucleotide sequence homology, regardless of whether one should deem the PMO structure, not the SEQ ID NOs, as the “focus of the invention”, this rejection is maintained.
Double Patenting
Claims 3, 5, 7-11, and 14 remain provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-21 and 41 of Application No. 18/660,789 for the reasons as set forth in the Office action mailed on July 9, 2025 because applicant did not provide any substantial rebuttal arguments addressing the supposed errors of this rejection.
Conclusion
No claim is allowed.
THIS ACTION IS MADE FINAL. 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 DANA H SHIN whose telephone number is (571)272-8008. The examiner can normally be reached Monday-Thursday: 8am - 6:30pm.
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If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, RAM SHUKLA can be reached at 571-272-0735. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
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/DANA H SHIN/Primary Examiner, Art Unit 1635