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 .
DETAILED ACTION
Status of Claims
The text of those sections of Title 35, U.S. Code not included in this action can be found in a prior Office action.
This action is in response to the papers filed on February 26, 2026. Pursuant to the amendment filed on November 26, 2025, claims 1, 3, 7, 11-12, 14, 18, 22-23, and 25 have been amended. Claims 2, 4, 13, 15, 24, and 26 are cancelled. Claims 27-36 are newly added.
Therefore, claims 1, 3, 5-12, 14, 16-23, 25, and 27-36 are currently under examination.
Priority
Applicant has submitted an amended Application Data Sheet (ADS) which makes the present application a continuation of the '651 application.
The present application is a CON of 17/778,651, filed May 20, 2022, which is a 35 U.S.C. 371 national stage filing of International Application No. PCT/US2020/062484 filed on November 27, 2020. Applicants' claim for the benefit of a prior-filed application parent provisional applications 63/024,933 filed May 14, 2020, and 62/941,719 filed November 28, 2019, under 35 U.S.C. 119(e) or under 35 U.S.C. 120, 121, or 365(c) is acknowledged.
Thus, the earliest possible priority for the instant application is November 28, 2019.
Withdrawn- Specification Objection
In view of the concurrent submission of a substitute specification, the objections to the specification have been withdrawn.
Withdrawn- Drawings
In view of the concurrent submission of drawing substitute sheets, the objections to the drawings have been withdrawn.
Withdrawn- Claim Objections
In view of Applicant’s amendments, filed February 26, 2026, addressing the informalities, the objections to claims 11 and 22 are moot and have been withdrawn.
Withdrawn- Claim Rejections - 35 USC § 112(b)
In view of Applicants’ cancelation of claim 26, the rejections under 35 U.S.C. 112(b) have been withdrawn.
Withdrawn- Claim Rejection- 35 USC§ 102
In view of Applicants’ amendments to the instant claims, requiring the method of treating a dystrophinopathy and method of delivering a nucleic acid encoding a microdystrophin protein to a cell to comprise a nucleotide sequence encoding a microdystrophin protein comprising an amino acid sequence comprising SEQ ID NO: 79 or at least 97% identity thereto, or an amino acid sequence comprising SEQ ID NO: 1 or at least 97% identity thereto, in combination with an H3 domain and a CT domain comprising an α1-syntrophin binding site, the rejection of claims 1, 5-8, 11-12, 16-19, 22-23, and 25 under 35 U.S.C. 102(a)(1) as being anticipated by Dickson (US 10,647,751 B2, prior published Nov. 10, 2016, WO 2016/177911) has been withdrawn. Applicants’ arguments are moot in view of the withdrawn rejection. A response to Applicant’s arguments pertinent to a new or remaining rejection can be found below.
Withdrawn- Double Patenting
In view of Applicants’ amending the claims to recite additional limitations including, including sequence identity thresholds and structural domain features, the rejection of claims 1-26 on the grounds of nonstatutory double patenting as being unpatentable over claims 51, 54, and 60-79 of copending Application No. 17/778,651, in view of Dickson (US 10,647,751 B2, prior published Nov. 10, 2016, WO 2016/177911) has been withdrawn.
New 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.
Claims 1, 3, 5-12, 14, 16-23, 25, and 27-36 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 51, 54, and 61-67, 69- 110 of copending Application No. 17/778,651, in view of Dickson (US 10,647,751 B2, prior published Nov. 10, 2016, WO 2016/177911), and further in view of Duan (Hum Gene Ther. 2018 Jul 1;29(7):733–736.), Banks et al. (PLoS Genet. 2010 May 20;6(5):e1000958.), and Ramos et al. (Mol Ther. 2019 Mar 6;27(3):623-635. Epub 2019 Feb 1.). This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
The restriction requirement between Group II (rAAV particle) and Group III (method of delivery/therapy), in parent Application 17/778,651, was previously withdrawn. Once the restriction 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.
This is a new rejection necessitated by Applicants’ amendments to the claims in the response filed on February 26, 2026.
Although the claims at issue are not identical, they are not patentably distinct from each other because claims 51, 54, and 61-67, 69- 10 of ‘651 anticipate a method of delivering a nucleic acid encoding a microdystrophin with an rAAV particle for therapeutically effective utility.
Regarding instant claims 1, 3, 5-12, 14, 16-23, 25, 27-36, claims 51, 54, 60-62, 67, 68-70, 74-79 correspond to and anticipate the method of delivery or treatment with the rAAV and nucleic acid sequences SEQ ID NOs: 20 and 81, along with the encoded amino acid sequences SEQ ID NOs: 1 and 79. Likewise, auxiliary genetic elements of instant claims 6-10 and 17-21, such as the SPc5-12 promoter, AAV2 ITR sequence, and capsid proteins are anticipated and correspond to claims 63-66 and 71-75.
Regarding the structural limitations, such as the CT domain component of the microdystrophin, Hinge 3 (H3) domain and recited linkers such as the H3 domain-L4-R24, the ordinary artisan would have recognized the prior art taught these as established elements known to be optimized for treatment of dystrophinopathies including DMD, further in view of the teachings of Duan, Banks, and Ramos.
Duan teaches the design and optimization of microdystrophin constructs, including C-terminal domain composition and hinge regions, for use in systemic rAAV-mediated delivery to treat DMD (pg. 734-735, bridging para.). Duan specifically teaches that Duchenne muscular dystrophy is a systematic disease requiring body-wide delivery and that rAAV microdystrophin constructs are designed for such systematic administration (pg. 733-734). Duan further teaches that, due to AAV packaging constraints, truncated microdystrophin genes retaining key functional domains are engineered, and that numerous known constraints, include hinge regions or domains have been designed and tested to improve therapeutic efficacy (pg. 734-736). Hence, the teaching of Duan demonstrate that the ordinary artisan would have been motivated to optimize structural microdystrophin domain architecture, including hinge domains or regions, with a reasonable expectation of success in achieving improved function for systemic rAAV delivery.
Moreover, the teachings of Banks specifically establish that the H3 domain was known to impart optimized function for such genetically engineered systems. Banks teaches that hinge design and selection directly affects microdystrophin function and specifically that replacing H2 with H3 significantly improved the functional capacity to prevent muscle degeneration, increase muscle fiber area, and maintain the junctions (Fig. 1-4; pg. 5, Section: Discussion). Additionally, the teachings of Ramos establish the design and evaluation of engineered microdystrophin constructs with modified domain architectures to improve functional performance. Ramos specifically teaches that hinge and linker regions are tunable structural elements and describes testing alternative sequences, including linker regions in combination with hinge domains to optimize microdystrophin functionality (Fig.1; pg. 626, column 1, para. 2).
Hence, before the effective filing date of the instant application, a person of ordinary skill in the art would have found it obvious to utilize a recombinant rAAV particle comprising a nucleic acid encoding a microdystrophin protein for treating a dystrophinopathy in a human subject in need thereof in view of the teachings of copending Application No. 17/788,651 and Dickson. One of ordinary skill in the art would have been motivated to modify and optimize the microdystrophin constructs, including selection of H3 hinge region and linker regions, as taught by Duan, Banks, and Ramos, with a reasonable expectation of success in improving functional performance and therapeutic efficacy.
Regarding claims 11 and 22, Dickon discloses the therapeutic efficacious utility for treating a subject with a dystrophic disease included wherein the dystrophinopathy is Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) (claims 12 and 14). Therefore, the ordinary artisan would have recognized the delivery of the pharmaceutical composition comprising the rAAV and nucleic acid sequence encoding the therapeutically effective microdystrophin protein the of ‘651, claims 51, 54, 67, 76, and 77-79, for its therapeutic utility in treating diseases such as Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD).
Response to Applicants' Arguments as they apply to the rejections of claims 1, 3, 5-12, 14, 16-23, 25, and 27-36 on the grounds of nonstatutory double patenting
Applicant's arguments filed February 26, 2026, have been fully considered but they are not persuasive.
At pages 14-15 of the remarks filed February 26, 2026, Applicants essentially argue the
following: In view of the amendments and arguments made herein, as detailed below, Applicant
argues that Dickson does not anticipate the claims and that the claims are patentable over
Dickson.
Applicant’s arguments with respect to claims 1, 3, 5-12, 14, 16-23, 25, and 27-36 have been considered but are moot because the new ground of rejection does not rely on any reference applied in the prior rejection of record for any teaching or matter specifically challenged in the argument. Before the effective filing date of the instant application, the ordinary artisan would have found it obvious to utilize a recombinant rAAV particle comprising a nucleic acid encoding a microdystrophin protein for treating a dystrophinopathy in a human subject in need thereof in view of the teachings of copending Application No. 17/788,651 and Dickson. One of ordinary skill in the art would have been motivated to modify and optimize the microdystrophin constructs, including selection of H3 hinge region and linker regions, as taught by Duan, Banks, and Ramos, with a reasonable expectation of success in improving functional performance and therapeutic efficacy.
Conclusion
Claims 1, 3, 5-12, 14, 16-23, 25, and 27-36 are rejected. No claims are allowed.
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 JOEL D LEVIN whose telephone number is (571)270-0616. The examiner be reached 8:00 am to 5:00 pm, Monday through Friday.
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/J.D.L./Examiner, Art Unit 1633
/CHRISTOPHER M BABIC/Supervisory Patent Examiner, Art Unit 1633