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 .
Election/Restrictions
This action is in response to the papers filed on 03/06/2026. Claims 38-49 are currently pending as per claims filed on 03/06/2026.
Applicant’s election without traverse of Group I, which include claims 38-45, drawn to an RNA targeting nucleic acid, and election of species for Group I, the sequence of SEQ ID NO: 215 in the reply filed on 03/06/2026 is acknowledged.
Claims 46-49 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to non-elected subject matter, there being no allowable generic or linking claim.
The requirement for restriction between Groups I-II is still deemed proper and is therefore made FINAL.
Therefore, claims 38-45 are subject to examination to which the following grounds of rejection are applicable.
Priority
The instant application claims priority to PRO 63/379,983 filed 10/18/2022 and PRO 63/316,659 filed 03/04/2022.
Thus, the earliest possible priority for the instant application is 03/04/2022.Information Disclosure Statement
The information disclosure statements (IDS) submitted on 02/16/2024, 06/23/2025 and 03/06/2026 were filed before the mailing date of the non-final office action. The submission is in compliance with the provisions of 37 CFR 1.97. Accordingly, the information disclosure statement is being considered by the examiner.
Claim Objections
Claim 38 is objected to because of the following informalities: Claim 38 recites “targeting sequence (TS)”, however the abbreviation TS is unnecessary and is not used within the instant claims. Appropriate correction is required.
Claim 45 is objected to under 37 CFR 1.75 as being a duplicate claim of claim 44. It appears the applicant intended for claim 45 to depend from claim 41. For the purpose of compact prosecution, the claim will be examined as such. However, appropriate correction is required.
Claim Rejections - 35 USC § 112
The following is a quotation of 35 U.S.C. 112(b):
(b) CONCLUSION.—The specification shall conclude with one or more claims particularly pointing out and distinctly claiming the subject matter which the inventor or a joint inventor regards as the invention.
The following is a quotation of 35 U.S.C. 112 (pre-AIA ), second paragraph:
The specification shall conclude with one or more claims particularly pointing out and distinctly claiming the subject matter which the applicant regards as his invention.
Claims 44-45 are rejected under 35 U.S.C. 112(b) or 35 U.S.C. 112 (pre-AIA ), second paragraph, as being indefinite for failing to particularly point out and distinctly claim the subject matter which applicant regards as the invention.
Claims 44-45 recite “The AAV of claim 40”. (please see the objection of claim 45 above) There is improper antecedent basis for the “The AAV”. Therefore, the claim is indefinite. For the sake of compact prosecution, the claims will be examined as intending to recite “The AAV vector”. Appropriate correction is required.
Claim Rejections - 35 USC § 103
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 35 U.S.C. 103 which forms the basis for all obviousness rejections set forth in this Office action:
A patent for a claimed invention may not be obtained, notwithstanding that the claimed invention is not identically disclosed as set forth in section 102, if the differences between the claimed invention and the prior art are such that the claimed invention as a whole would have been obvious before the effective filing date of the claimed invention to a person having ordinary skill in the art to which the claimed invention pertains. Patentability shall not be negated by the manner in which the invention was made.
The factual inquiries for establishing a background for determining obviousness under 35 U.S.C. 103 are summarized as follows:
1. Determining the scope and contents of the prior art.
2. Ascertaining the differences between the prior art and the claims at issue.
3. Resolving the level of ordinary skill in the pertinent art.
4. Considering objective evidence present in the application indicating obviousness or nonobviousness.
This application currently names joint inventors. In considering patentability of the claims the examiner presumes that the subject matter of the various claims was commonly owned as of the effective filing date of the claimed invention(s) absent any evidence to the contrary. Applicant is advised of the obligation under 37 CFR 1.56 to point out the inventor and effective filing dates of each claim that was not commonly owned as of the effective filing date of the later invention in order for the examiner to consider the applicability of 35 U.S.C. 102(b)(2)(C) for any potential 35 U.S.C. 102(a)(2) prior art against the later invention.
Claims 38-45 are rejected under 35 U.S.C. 103 as being unpatentable over Byrne et al. (WO 2021/216853 Al; IDS filed 02/13/2024 as Shape Therapeutics INC.) and in view of Echigoya et al. (Echigoya Y et al., PLoS One., 2015) as evidenced by Uniprot (UniProt: the Universal Protein Knowledgebase in 2025, Nucleic Acids Res. 53:D609–D617 (2025))
Regarding claims 38-39, Byrne et al. teaches RNA-targeting nucleic acid molecules comprising targeting sequences that hybridize to target DNA (Abstract). Moreover, Byrne et al. teaches “the present disclosure provides compositions and methods of use thereof of guide RNAs having snRNA sequences and snRNA hairpins (e.g., SmOPT sequences and U7 hairpins) that are capable of facilitating RNA editing of a Duchenne muscular dystrophy (DMD) gene. In some embodiments, a guide RNA of the present disclosure having snRNA sequences and snRNA hairpins (e.g., SmOPT sequences and U7 hairpins) can target an exon of a DMD gene, such as exon 51…” (Pg. 97, Paragraph [00237]).
Byrne et al. does not teach the specific sequence of SEQ ID NO: 215 (elected sequence). However, the DMD sequences, including transcript variants and isoforms are well known as evidenced by Uniprot which details this information and provides an extensive citation list from 1988 onward with the respective DMD sequence publications (exact citation information, including relevant web link provided with this office action submission).
Furthermore, the teachings of Echigoya et al. cure the deficiencies of Byrne et al. as they demonstrate design for oligonucleotides to target and induce exon skipping was well known in the art prior to the instant application. Echigoya et al. teaches “The use of antisense ‘splice-switching’ oligonucleotides to induce exon skipping represents a potential therapeutic approach to various human genetic diseases. It has achieved greatest maturity in exon skipping of the dystrophin transcript in Duchenne muscular dystrophy (DMD), for which several clinical trials are completed or ongoing, and a large body of data exists describing tested oligonucleotides and their efficacy. The rational design of an exon skipping oligonucleotide involves the choice of an antisense sequence, usually between 15 and 32 nucleotides, targeting the exon that is to be skipped.” (Abstract). Echigoya et al. teaches several design guidelines well described in the art (Pg. 2, Paragraph 4) and further sets forth their design strategy using their predictive computational modeling pipeline and set parameters (Materials and Methods entirety) for in silico pre-screening, predictive optimization for DMD exon-skipping oligonucleotides. This pipeline includes the following: selection of antisense sequences targeting the exon to be skipped, evaluating the parameters of target-site energetics, splice-factor binding propensity, splice-site characteristics, GC content, and RNA secondary structure accessibility (Table 2; Pg. 4, additional descriptors). Of the parameters, Echigoya teaches “We confirmed (1) the binding energetics of the oligonucleotide to the RNA, and (2) the distance in bases of the target site from the splice acceptor site, as the two most predictive parameters… (Abstract).
It would have been prima facie obvious to a person having ordinary skill in the art at the time of the instant application to have modified the snRNA that are capable of facilitating RNA editing of a known sequence such as a Duchenne muscular dystrophy (DMD) gene as taught by Byrne et al. by incorporating the computational rationale design strategies for oligo nucleotides as taught by Echigoya et al. to arrive at the claimed sequence of SEQ ID NO:215.
One would have been motivated to combine these teaching as both references described sequence-directed modulation of dystrophin pre-mRNA and the use of Echigoya’s teaching would allow for optimization of the RNA-targeting platform of Byrne et al. by identifying favorable antisense target positions within DMD exons to improve exon-skipping efficacy.
There would have been reasonable expectations of success in combining these teachings as one of ordinary skill in the art would recognize to combine known elements in the art to give predictable results. In this case, the combination would merely use a known design tool for selecting effective target-complementary sequences for DMD exon skipping in an established RNA-targeting platform.
Regarding Claims 40-45, Byrne et al. teaches the use of an AAV vector (for instant claims 40-41), including a self-complimentary AAV (for instant claims 42-43), and that the AAV can be one of AAV6 or AAV9 (Pg. 100-101, Paragraphs [00245] – [00246]).
Conclusion
Claims 38-45 are rejected.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to KODYE LEE ABBOTT whose telephone number is (703)756-1111. The examiner can normally be reached M-F 8-5.
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/KODYE LEE ABBOTT/Examiner, Art Unit 1634
/MARIA G LEAVITT/Supervisory Patent Examiner, Art Unit 1634
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