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 .
Claim Status
Claims 1-4, 6, 11-13, 15-17, 20-22, 25, 27, 29-30, 32 and 34-36 are pending.
Claims 22, 25, 30, 32 and 34-35 are withdrawn.
Claims 1-4, 6, 11-13, 15-17, 20-21, 27, 29 and 36 are under examination.
Rejoinder
The restriction requirement between Group I and claim 27 as set forth in the Lack of Unity mailed on 5th, August, 2025 has been reconsidered in view of Applicant’s amendments.
Applicant has amended claim 27 to depend from claim 1. Claim 27 is now part of elected Group I and is therefore rejoined.
In view of the above noted withdrawal of the restriction and species election requirement, Applicant is advised that if any claim presented in a continuation or divisional application is anticipated by, or includes all the limitations of, a claim that is allowable in the present application, such claim may be subject to provisional statutory and/or nonstatutory double patenting rejections over the claims of the instant application.
Once a restriction requirement or species election requirement is withdrawn, the provisions of 35 U.S.C. 121 are no longer applicable. See In re Ziegler, 443 F.2d 1211, 1215, 170 USPQ 129, 131-32 (CCPA 1971). See also MPEP § 804.01.
Moot Claim Rejections - 35 USC § 112(b)
The rejection of claim 8 under 35 U.S.C. 112(b) or 35 U.S.C. 112 (pre-AIA ), second paragraph, as being indefinite as set forth in the previous office action is moot in view of the cancellation of this claim.
Withdrawn Claim Rejections - 35 USC § 103
The rejection of claims 1-4, 6, 12-13, 15 and 20 under 35 U.S.C. 103 as being unpatentable over Li et al. (Stem Cell Reports. 2015 Jan 13;4(1):143-154. Epub 2014 Nov 26.; henceforth “Li”) as evidenced NCBI_DMD (accessed at: https://www.ncbi.nlm.nih.gov/nuccore/NG_012232.1/) in view of Lee et al. (Elife. 2017 May 2:6:e25312.; henceforth “Lee”), Echigoya et al. (J Pers Med. 2018 Dec 7;8(4):41.; henceforth “Echigoya”) and Hsu et al. (WO-2016/161380-A1; see IDS filed 6th, May, 2022; henceforth “Hsu”) as set forth in the previous office action is withdrawn in view of Applicant’s amendments.
The rejection of claim 11 under 35 U.S.C. 103 as being unpatentable over Li et al. (Stem Cell Reports. 2015 Jan 13;4(1):143-154. Epub 2014 Nov 26.; henceforth “Li”) as evidenced NCBI_DMD (accessed at: https://www.ncbi.nlm.nih.gov/nuccore/NG_012232.1/) in view of Lee et al. (Elife. 2017 May 2:6:e25312.; henceforth “Lee”), Echigoya et al. (J Pers Med. 2018 Dec 7;8(4):41.; henceforth “Echigoya”) and Hsu et al. (WO-2016/161380-A1; see IDS filed 6th, May, 2022; henceforth “Hsu”) as applied to claim 1 above, and in further view of Olson et al. (WO-2019/152609-A1; published 8th, August, 2019 with priority to 31st, January, 2018; henceforth “Olson”) as set forth in the previous office action is withdrawn in view of Applicant’s amendments.
The rejection of claim 16 under 35 U.S.C. 103 as being unpatentable over Li et al. (Stem Cell Reports. 2015 Jan 13;4(1):143-154. Epub 2014 Nov 26.; henceforth “Li”) as evidenced NCBI_DMD (accessed at: https://www.ncbi.nlm.nih.gov/nuccore/NG_012232.1/) in view of Lee et al. (Elife. 2017 May 2:6:e25312.; henceforth “Lee”), Echigoya et al. (J Pers Med. 2018 Dec 7;8(4):41.; henceforth “Echigoya”) and Hsu et al. (WO-2016/161380-A1; see IDS filed 6th, May, 2022; henceforth “Hsu”) as applied to claim 1 above, and in further view of NCBI_DMD (accessed at: https://www.ncbi.nlm.nih.gov/nuccore/NG_012232.1/) as set forth in the previous office action is withdrawn in view of Applicant’s amendments.
The rejection of claim 17 under 35 U.S.C. 103 as being unpatentable over Li et al. (Stem Cell Reports. 2015 Jan 13;4(1):143-154. Epub 2014 Nov 26.; henceforth “Li”) as evidenced NCBI_DMD (accessed at: https://www.ncbi.nlm.nih.gov/nuccore/NG_012232.1/) in view of Lee et al. (Elife. 2017 May 2:6:e25312.; henceforth “Lee”), Echigoya et al. (J Pers Med. 2018 Dec 7;8(4):41.; henceforth “Echigoya”) and Hsu et al. (WO-2016/161380-A1; see IDS filed 6th, May, 2022; henceforth “Hsu”) as applied to claim 1 above, and in further view of Warnock et al. (Methods Mol Biol. 2011:737:1-25.; henceforth “Warnock”) as set forth in the previous office action is withdrawn in view of Applicant’s amendments.
The rejection of claim 29 under 35 U.S.C. 103 as being unpatentable over Li et al. (Stem Cell Reports. 2015 Jan 13;4(1):143-154. Epub 2014 Nov 26.; henceforth “Li”) as evidenced NCBI_DMD (accessed at: https://www.ncbi.nlm.nih.gov/nuccore/NG_012232.1/) in view of Lee et al. (Elife. 2017 May 2:6:e25312.; henceforth “Lee”), Echigoya et al. (J Pers Med. 2018 Dec 7;8(4):41.; henceforth “Echigoya”) and Hsu et al. (WO-2016/161380-A1; see IDS filed 6th, May, 2022; henceforth “Hsu”) as applied to claim 1 above, and in further view of Ahern et al. (The Scientist Magazine (1995); accessed at https://www.the-scientist.com/technology/biochemical-reagents-kits-offer-scientists-good-return-on-investment-58425) as set forth in the previous office action is withdrawn in view of Applicant’s amendments.
Moot Claim Rejections - 35 USC § 103
The rejection of claim 8 under 35 U.S.C. 103 as being unpatentable over Li et al. (Stem Cell Reports. 2015 Jan 13;4(1):143-154. Epub 2014 Nov 26.; henceforth “Li”) as evidenced NCBI_DMD (accessed at: https://www.ncbi.nlm.nih.gov/nuccore/NG_012232.1/) in view of Lee et al. (Elife. 2017 May 2:6:e25312.; henceforth “Lee”), Echigoya et al. (J Pers Med. 2018 Dec 7;8(4):41.; henceforth “Echigoya”) and Hsu et al. (WO-2016/161380-A1; see IDS filed 6th, May, 2022; henceforth “Hsu”) as set forth in the previous office action is moot in view of the cancellation of this claim.
Moot Double Patenting
Non-Statutory Double Patenting
U.S. Co-pending Application No. 17921316
The provisional rejection of claim 8 on the ground of nonstatutory double patenting as being unpatentable over claims of 1-2, 9, 13, 23, 25, 27-28 and 38 of copending application No. 17/921,316 (claims filed 10th, July, 2023) as set forth in the previous office action is moot in view of the cancellation of this claim.
Double Patenting
The nonstatutory double patenting rejection is based on a judicially created doctrine grounded in public policy (a policy reflected in the statute) so as to prevent the unjustified or improper timewise extension of the “right to exclude” granted by a patent and to prevent possible harassment by multiple assignees. A nonstatutory double patenting rejection is appropriate where the conflicting claims are not identical, but at least one examined application claim is not patentably distinct from the reference claim(s) because the examined application claim is either anticipated by, or would have been obvious over, the reference claim(s). See, e.g., In re Berg, 140 F.3d 1428, 46 USPQ2d 1226 (Fed. Cir. 1998); In re Goodman, 11 F.3d 1046, 29 USPQ2d 2010 (Fed. Cir. 1993); In re Longi, 759 F.2d 887, 225 USPQ 645 (Fed. Cir. 1985); In re Van Ornum, 686 F.2d 937, 214 USPQ 761 (CCPA 1982); In re Vogel, 422 F.2d 438, 164 USPQ 619 (CCPA 1970); In re Thorington, 418 F.2d 528, 163 USPQ 644 (CCPA 1969).
A timely filed terminal disclaimer in compliance with 37 CFR 1.321(c) or 1.321(d) may be used to overcome an actual or provisional rejection based on nonstatutory double patenting provided the reference application or patent either is shown to be commonly owned with the examined application, or claims an invention made as a result of activities undertaken within the scope of a joint research agreement. See MPEP § 717.02 for applications subject to examination under the first inventor to file provisions of the AIA as explained in MPEP § 2159. See MPEP § 2146 et seq. for applications not subject to examination under the first inventor to file provisions of the AIA . A terminal disclaimer must be signed in compliance with 37 CFR 1.321(b).
The filing of a terminal disclaimer by itself is not a complete reply to a nonstatutory double patenting (NSDP) rejection. A complete reply requires that the terminal disclaimer be accompanied by a reply requesting reconsideration of the prior Office action. Even where the NSDP rejection is provisional the reply must be complete. See MPEP § 804, subsection I.B.1. For a reply to a non-final Office action, see 37 CFR 1.111(a). For a reply to final Office action, see 37 CFR 1.113(c). A request for reconsideration while not provided for in 37 CFR 1.113(c) may be filed after final for consideration. See MPEP §§ 706.07(e) and 714.13.
The USPTO Internet website contains terminal disclaimer forms which may be used. Please visit www.uspto.gov/patent/patents-forms. The actual filing date of the application in which the form is filed determines what form (e.g., PTO/SB/25, PTO/SB/26, PTO/AIA /25, or PTO/AIA /26) should be used. A web-based eTerminal Disclaimer may be filled out completely online using web-screens. An eTerminal Disclaimer that meets all requirements is auto-processed and approved immediately upon submission. For more information about eTerminal Disclaimers, refer to www.uspto.gov/patents/apply/applying-online/eterminal-disclaimer.
Examiner’s Remark
U.S. Co-pending Application No. 17921316 has had a notice of allowance mailed on 11th, June, 2026. When the Patent issue, the Non-Statutory Double Patenting below will no longer be provisional.
Non-Statutory Double Patenting
U.S. Co-pending Application No. 17921316
Claims 1-4, 6, 11-13, 15-17, 21, and 29 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims of 1-2, 9, 13, 23, 25, 27-28 and 38 of copending application No. 17/921,316 (claims filed 11th, May, 2026).
This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented. U.S. Co-pending Application No. 17921316 has had a notice of allowance mailed on 11th, June, 2026. When the Patent issues, the Non-Statutory Double Patenting rejections below will no longer be provisional.
The subject matter claimed in the instant application is disclosed in the referenced application as follows: the CRISPR/Cas-based genome editing system anticipates or makes obvious the CRISPR/Cas-based genome editing system of instant application.
Although the claims at issue are not identical, they are not patentably distinct for the reasons stated below.
Regarding claims 1-2, U.S. Co-pending App ‘316 claims a CRISPR/Cas-based genome editing system comprising one or more vectors encoding a composition, the composition comprising:
(a) a guide RNA (gRNA) targeting an intron (intron 51) that is in junction with an exon of a mutant dystrophin gene;
(b) a Cas protein or a fusion protein comprises comprising the Cas protein; and
(c) a donor sequence comprising exon 52 of the wild-type dystrophin gene (claim 1).
Regarding claims 1-2, Co-pending claims 1-2 of U.S. Co-pending App ‘316 specifies hybridizing to a target sequence within intron 51, which is an anticipatory species of the instantly claimed “intron that is in junction with an exon,” and thereby make it obvious.
Regarding claim 1, one of ordinary skill would have at once envisaged the gRNA and the donor sequence are encoded by one vector from the limited genus of vector configurations claimed by U.S. Co-pending App ‘316.
Regarding claims 1-2, 12 and 15, further to the discussion of claims 1 and 3 above, as stated above, U.S. Co-pending App ‘316 claims a target of intron 51.
Regarding claims 1-2, 12-13 and 15, U.S. Co-pending App ‘316 claims one or vectors comprising SEQ ID NO: 60 or 130 of U.S. Co-pending App ‘316 (claim 27). The claimed SEQ ID NO: 60 or 130 of U.S. Co-pending App ‘316 comprises a sequence 100% identical to instant SEQ ID NO: 17 as well as sequence 100% identical to instant SEQ ID NO: 19 which encodes a gRNA 100% identical to instant SEQ ID NO: 25. The gRNA 100% identical to instant SEQ ID NO: 25 “targets and binds the polynucleotide sequence of SEQ ID NO: 17” (instant claims 1-2) as claimed.
Regarding claim 3, further to the discussion of claim 1 above, U.S. Co-pending App ‘316 claims the fragment of the functional dystrophin gene (exon 52 of the wild-type dystrophin gene) is flanked by two gRNA spacers and/or PAM sequences (claim 9).
Regarding claim 3, U.S. Co-pending App ‘316 claims the donor sequence is flanked on both sides by a gRNA spacer and/or a PAM sequence (claim 9), the fragment being flanked on both sides is an anticipatory species of “flanked by two gRNA spacers and/or PAM sequences,” and thereby makes it obvious.
Regarding claim 4, further to the discussion of claim 1 above, U.S. Co-pending App ‘316 claims the gRNA targets an intron that is in junction with an exon of the mutant dystrophin gene (intron 51; claim 1).
Regarding claim 4, U.S. Co-pending App ‘316 claims the gRNA hybridizes to a target sequence within intron 51, which is an anticipatory species of the instantly claimed “an intron that is in junction with an exon of the mutant dystrophin gene,” and thereby make it obvious.
Regarding claim 6, further to the discussion of claims 1 and 4 above, U.S. Co-pending App ‘316 claims the exon (Exon 52) of the mutant dystrophin gene is mutated or at least partially deleted from the dystrophin gene (claim 13).
Regarding claim 6, U.S. Co-pending App ‘316 claims “exon 52 of the mutant dystrophin gene is mutated or at least partially deleted from the dystrophin gene” which is an anticipatory species of the instantly claimed “the exon of the mutant dystrophin gene is mutated or at least partially deleted from the dystrophin gene,” and thereby makes it obvious.
Regarding claim 7, further to the discussion of claims 1 and 4 above, U.S. Co-pending App ‘316 claims the exon is exon 52 (Exon 52; claim 1).
Regarding claim 11, further to the discussion of claim 1 above, U.S. Co-pending App ‘316 claims the Cas protein comprises an amino acid sequence of SEQ ID NO: 2 (SEQ ID NO: 19 of U.S. Co-pending App ‘316 is a 100% match to instant SEQ ID NO: 2) (claim 23).
Regarding claim 13, further to the discussion of claims 1 and 12 above, although U.S. Co-pending App ‘316 does not claim a literal recitation of two gRNA spacers are identical, the vectors claimed by U.S. Co-pending App ‘316 are double stranded, and therefore comprise two of the identical gRNA spacers that flank the donor sequence, one on each strand.
Regarding claim 16, further to the discussion of claim 1 above, U.S. Co-pending App ‘316 claims the donor sequence comprises the polynucleotide of SEQ ID NO: 21 (claimed SEQ ID NO: 56, 60 and 130 vector of U.S. Co-pending App ‘316 comprises the donor sequence of SEQ ID NO: 21) (claim 27).
Regarding claim 17, further to the discussion of claim 1 above, U.S. Co-pending App ‘316 claims the vector is a viral vector (claim 25).
Regarding claim 17, U.S. Co-pending App ‘316 claims the vector is an “AAV vector” (claim 25), which is an anticipatory species of the instantly claimed “viral vector,” and thereby makes it obvious.
Regarding claim 21, further to the discussion of claim 1 above, U.S. Co-pending App ‘316 claims the molar ratio between gRNA and donor sequence is 1: 1, or from 5: 1 to 1: 10, or from 1: 1 to 1 :5 (claim 28).
Regarding claim 29, further to the discussion of claim 1 above, U.S. Co-pending App ‘316 claims a kit comprising the system of claim 1 (claim 38).
Since the instant application claims are anticipated by or obvious over cited application claims, said claims are not patentably distinct.
Response to Arguments
Applicant’s arguments, filed 6th, April, 2026, have been fully considered but are not found persuasive.
Applicant argues the “the present application has the earlier patent term filing date, and according to MPEP § 804(I)(B)(1)(b)(i)” (pg. 10).
In response, U.S. Co-pending Application No. 17921316 has had a notice of allowance mailed on 11th, June, 2026. When the Patents issue, the Non-Statutory Double Patenting will no longer be provisional. U.S. Co-pending Application No. 17921316 is expected to be issued before the instant application and therefore the rejection cannot be withdrawn.
New Non-Statutory Double Patenting
U.S. Co-pending Application No. 17921316
Claim 27 is provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims of 1-2, 9, 13, 23, 25, 27-28 and 38 of copending application No. 17/921,316 (claims filed 11th, May, 2026) as applied to claim 1 above, and in further view of Li et al. (Stem Cell Reports. 2015 Jan 13;4(1):143-154. Epub 2014 Nov 26.; henceforth “Li”). The teachings of copending application No. 17/921,316 above are incorporated herein in their entirety.
This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented. U.S. Co-pending Application No. 17921316 has had a notice of allowance mailed on 11th, June, 2026. When the Patent issues, the Non-Statutory Double Patenting below will no longer be provisional.
Regarding claim 27, further to the discussion of claim 1 above, U.S. Co-pending Application No. ‘316 does not claim an isolated cell comprising the system.
Nevertheless, regarding claim 27, Li teaches an isolated cell (DMD-patient-derived iPSC; summary; see also pg. 152 “Generation of Integration-Free DMD iPSCs”) comprising a CRISPR/Cas-based genome editing system comprising multiple vectors (donor vector and Cas9 and sgRNA vector; “TALEN- or CRISPR-Mediated Exon 44 Knockin” pg. 125 col. 1 last para.) encoding a composition, the composition comprising:
(a) a guide RNA (gRNA) (sgRNA) targeting an intron that is in junction with an exon of a mutant dystrophin gene (intron 45 Figure S2);
(b) a Cas protein; and
(c) a donor sequence comprising Exon 44 of a wild-type dystrophin gene (exon 44 “knockin of the missing exon 44” pg. 144 col. 1; Figure s2A, 4; pg. 147 “Correction of the Full-Length Dystrophin Protein with a Donor Template”)(see also Figures 2 and S2). Li teaches contacting the isolated cell with the composition to restore the dystrophin protein, differentiate the corrected iPSCs toward skeletal muscle cells with the expression of full-length dystrophin protein, and to develop iPSC-based gene therapy for genetic disorders (summary).
Therefore, regarding claim 27, it would have been obvious to a person of ordinary skill in the art before the effective filing date of the claimed invention to prepare the CRISPR/Cas based editing composition as claimed by U.S. Co-pending Application No. ‘316, and combine the known prior art element of contacting a cell with the composition of Li to obtain the predictable result of a cell comprising a CRISPR/Cas based editing composition. One of ordinary skill would have been motivated to do so a taught by Li to restore the dystrophin protein, differentiate the corrected iPSCs toward skeletal muscle cells with the expression of full-length dystrophin protein, and to develop iPSC-based gene therapy for genetic disorders (summary).
Since the instant application claims are obvious over cited application claims, in view of Li, said claims are not patentably distinct.
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.
Claims 1-4, 6, 11-13, 15-17, 21 and 29 are rejected.
Claims 20 and 36 are allowed.
Correspondence
Any inquiry concerning this communication or earlier communications from the examiner should be directed to BRIANA N EBBINGHAUS whose telephone number is (703)756-4548. The examiner can normally be reached M-F 9:30 AM to 5:30 PM ET.
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/BRIANA N EBBINGHAUS/Examiner, Art Unit 1632
/PETER PARAS JR/Supervisory Patent Examiner, Art Unit 1632
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