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
1. Applicant’s election of the invention of Group I (drawn to a method for treating DMD, in the reply filed on 07/09/2026 is acknowledged. Because applicant did not distinctly and specifically point out the supposed errors in the restriction requirement, the election has been treated as an election without traverse (MPEP § 818.01(a)).
Claims 2, 5-7, 9, 11-14, 18, 20, 27-31 have been cancelled. Claims 1, 4, 15, 16, 17, 19, 22, 24, and 26 have been amended. Claims 32 is new.
Claims 1, 3, 4, 8, 10, 15-17, 19, 21-26, and 32 are pending and under examination.
Nucleotide and/or Amino Acid Sequence Disclosures
Summary of Requirements for Patent Applications Filed On Or After July 1, 2022, That Have Sequence Disclosures
2. 37 CFR 1.831(a) requires that patent applications which contain disclosures of nucleotide and/or amino acid sequences that fall within the definitions of 37 CFR 1.831(b) must contain a “Sequence Listing XML”, as a separate part of the disclosure, which presents the nucleotide and/or amino acid sequences and associated information using the symbols and format in accordance with the requirements of 37 CFR 1.831-1.835. This “Sequence Listing XML” part of the disclosure may be submitted:
1. In accordance with 37 CFR 1.831(a) using the symbols and format requirements of 37 CFR 1.832 through 1.834 via the USPTO patent electronic filing system (see Section I.1 of the Legal Framework for Patent Electronic System (https://www.uspto.gov/PatentLegalFramework), hereinafter “Legal Framework”) in XML format, together with an incorporation by reference statement of the material in the XML file in a separate paragraph of the specification (an incorporation by reference paragraph) as required by 37 CFR 1.835(a)(2) or 1.835(b)(2) identifying:
a. the name of the XML file
b. the date of creation; and
c. the size of the XML file in bytes; or
2. In accordance with 37 CFR 1.831(a) using the symbols and format requirements of 37 CFR 1.832 through 1.834 on read-only optical disc(s) as permitted by 37 CFR 1.52(e)(1)(ii), labeled according to 37 CFR 1.52(e)(5), with an incorporation by reference statement of the material in the XML format according to 37 CFR 1.52(e)(8) and 37 CFR 1.835(a)(2) or 1.835(b)(2) in a separate paragraph of the specification identifying:
a. the name of the XML file;
b. the date of creation; and
c. the size of the XML file in bytes.
SPECIFIC DEFICIENCIES AND THE REQUIRED RESPONSE TO THIS NOTICE ARE AS FOLLOWS:
This application contains sequence disclosures in accordance with the definitions for nucleotide and/or amino acid sequences set forth in 37 CFR 1.831(a) and 1.831(b). However, this application fails to comply with the requirements of 37 CFR 1.831-1.834. The examiner has noted that:
Claim 10 recites that the AAV genome comprises SEQ ID NO: 53. The specification defines SEQ ID NO: 53 is the nucleotide sequence of the artificial AAV8 genome recited in the parent claim 1 (see [0135]; [0145]; [0210]). Thus, it is clear the intent is to claim the sequence of the artificial AAV8 genome. However, SEQ ID NO: 53 included in the Sequence Listing is the nucleotide sequence of a promoter, specifically, the U1a promoter (see the attached Sequence Alignment). Appropriate correction to is required.
Consistent with the specification, claim 10 is interpreted as reciting the nucleotide sequence of the artificial AAV8 genome recited in the parent claim 1. Since claim 10 does not provide a specific nucleotide sequence for the artificial AAV8 genome, any prior art teaching an AAV8 genome comprising an expression cassette as claimed is reasonably interpreted as reading on the claim.
Applicant must provide:
• A replacement “Sequence Listing XML” part of the disclosure, as described above in item 1. or 2., as well as
• A statement that identifies the location of all additions, deletions, or replacements of sequence information in the “Sequence Listing XML” as required by 1.835(b)(3);
• A statement that indicates support for the amendment in the application, as filed, as required by 37 CFR 1.835(b)(4);
• A statement that the “Sequence Listing XML” includes no new matter in accordance with 1.835(b)(5); and
• A substitute specification in compliance with 37 CFR 1.52, 1.121(b)(3), and 1.125 inserting the required incorporation by reference paragraph as required by 37 CFR 1.835(b)(2), consisting of:
o A copy of the previously-submitted specification, with deletions shown with strikethrough or brackets and insertions shown with underlining (marked-up version);
o A copy of the amended specification without markings (clean version); and
A statement that the substitute specification contains no new matter.
Claim Objections
3. Claims 4, 22, 34, and 26 should recite: “wherein the nucleotide sequence of the transgene is set forth by SEQ ID NO: 20”.
This is consistent with the parent claims 1, 16, 17, and 19 reciting “ a transgene encoding a microdystrophin protein”, i.e., the encoding transgene consists of the encoding sequence set forth by SEQ ID NO: 20.
4. Applicant is advised that should claims 1 and 4 be found allowable, claims 16, 17, 19, 21, 23, and 25 will be objected to under 37 CFR 1.75 as being a substantial duplicate thereof. When two claims in an application are duplicates or else are so close in content that they both cover the same thing, despite a slight difference in wording, it is proper after allowing one claim to object to the other as being a substantial duplicate of the allowed claim. See MPEP § 608.01(m).
Double Patenting
5. 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.
6. Claims 1, 3, 4, 8, 10, 15-17, 19, 21-26, and 32 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 108-110 of copending Application No. 17/778,651 (reference application), in view of Dickinson et al. (WO 2016/177911; IDS). Although the claims at issue are not identical, they are not patentably distinct from each other because both claim sets are drawn to the same method for treating DMD by administering to a subject a recombinant AAV8 comprising an expression cassette comprising the SPc5-12 promoter operably linked to a transgene encoding the microdystrophin having the amino acid sequence set forth by SEQ ID NO: 1. The application specification discloses that SEQ ID NO: 77 represents the amino acid sequence of AAV8 capsid (see the Sequence Listing). The specific AAV genome set forth by SEQ ID NO: 53 recited in the application claims 109 and 110 anticipates the genus recited in the instant claims.
The application specification discloses that SEQ ID NO: 1 is encoded by SEQ ID NO: 20 (see [0098]). Thus, the AAV8 genome recited in the application claims comprises the transgene set forth by SEQ ID NO: 20. As evidenced by the attached Sequence Alignments, SEQ ID NOs: 1 and 20 are identical to the claimed SEQ ID NOs: 1 and 20. The application specification discloses that the AAV8 dose is 2X1014 vg/kg (see [0199]; [0220]).
With respect to decreasing the inflammation and muscle degeneration (instant claims 16, 17, and 19), Dickinson et al. teach that the absence of dystrophin results in inflammation and muscle degeneration; and that administering microdystrophin alleviates these symptoms (see p. 1, lines 15-25; p. 10, lines 7-12). Thus, one of skill in the art would have reasonably concluded that the method recited in the application claims would also decrease inflammation and muscle degeneration. With respect to the instant claim 32, one of skill in the art would have reasonably expected that the method recited in the application claims would increase the number of microdystrophin-positive fibers.
Thus, the application claims and the instant claims are obvious variants.
This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
7. Claims 1, 3, 4, 8, 10, 15-17, 19, 21-26, and 32 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 12, 14, 16-22, 25, 30-33, and 36-52 of copending Application No. 19/234,245 (reference application), in view of Dickinson et al. Although the claims at issue are not identical, they are not patentably distinct from each other because both claim sets are drawn to the same method for treating DMD by using a recombinant AAV8 comprising the SPc5-12 promoter operably linked to a transgene encoding the microdystrophin having the amino acid sequence set forth by SEQ ID NO: 1. The application specification discloses that SEQ ID NO: 77 represents the amino acid sequence of AAV8 capsid (see [0134]. Table 9).
The application specification discloses that SEQ ID NO: 1 is encoded by SEQ ID NO: 20 (see [0098]). Thus, the AAV8 genome recited in the application claims comprises the transgene set forth by SEQ ID NO: 20. As evidenced by the attached Sequence Alignments, SEQ ID NOs: 1 and 20 are identical to the claimed SEQ ID NOs: 1 and 20. The application specification discloses that the AAV8 dose is 2X1014 vg/kg (see [0199]; [0220]).
The instant specification discloses that SEQ ID NO: 1 comprises a linker between H3 and R24, which is set forth by SEQ ID NO: 12 (see [0084]; [0087], Table 1; [0088]). A comparison between the application SEQ ID NO:12 and the instant SEQ ID NO: 12 revealed that they are identical (Thr Leu Glu).
With respect to decreasing the inflammation and muscle degeneration (instant claims 16, 17, and 19), Dickinson et al. teach that the absence of dystrophin results in inflammation and muscle degeneration; and that administering microdystrophin alleviates these symptoms (see p. 1, lines 15-25; p. 10, lines 7-12). Thus, one of skill in the art would have reasonably concluded that the method recited in the application claims would also decrease inflammation and muscle degeneration. With respect to the instant claim 32, one of skill in the art would have reasonably expected that the method recited in the application claims would increase the number of microdystrophin-positive fibers.
Thus, the application claims and the instant claims are obvious variants.
This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
Claim Rejections - 35 USC § 103
8. 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.
9. Claims 1, 3, 8, 10, 15-17, 19, 21, 23, 25, and 32 are rejected under 35 U.S.C. 103 as being unpatentable over Dickinson et al. (WO 2016/177911; cited on the IDS filed on 02/03/2026).
Dickinson et al. teach a method for treating DMD in a human subject by intravenously administering to the human subject a pharmaceutical composition comprising a recombinant AAV8 comprising an expression cassette containing the SPc5-12 promoter operably linked to a human-optimized transgene encoding the functional human microdystrophin having the amino acid sequence set forth by SEQ ID NO: 13 (hMD3); the administered dose is 1014 vg/kg. Dickinson et al. teach that the method results in improved skeletal/cardiac muscle function, improved gait, and improved respiratory function; the therapeutic effect lasts for at least one year (claims 1, 15-17, and 19) (see p. 4, lines 3-5; p. 9, line 27-37; p. 11, line 14 through p. 12, line 23; p. 13, lines 30-35; p. 14, lines 5-20; p. 15, line 29 through p. 16, line 4; p. 18, lines 32-35; p. 19, lines 23-26; p. 20, lines 12-15; p. 23, lines 1-4, 17-18, and 28-35; p. 25; p. 30; p. 32, lines 1-5; p. 31, lines 9-25; Fig. 1).
As evidenced by the attached Sequence Alignment, SEQ ID NO: 13 is 96.9% identical to the claimed SEQ ID NO: 1. While 96.9% is not exactly 97% (claims 1, 16, 17, and 19) or 100% (claims 3, 21, 23, and 25), as per MPEP § 716.02, [a]ny differences between the claimed invention and the prior art may be expected to result in some differences in properties. The issue is whether the properties differ to such an extent that the difference is really unexpected. In re Merck & Co., 800 F.2d 1091, 231 USPQ 375 (Fed. Cir. 1986). In this case, there is no evidence that the claimed mycrodystrophins exhibit unexpected properties over the hMD3 taught by Dickinson et al. Similarly, with respect to claim 10, there is no evidence of record showing that the claimed sequence for the artificial AAV8 genome provides unexpected results over the artificial AAV8 genome taught by Dickinson et al.
With respect to decreasing the inflammation and muscle degeneration (claim 16, 17, and 19), Dickinson et al. teach that the absence of dystrophin results in inflammation and muscle degeneration; and that the functional microdystrophin alleviates these symptoms (see p. 1, lines 15-25; p. 10, lines 7-12).
With respect to administration resulting in greater than 50 ng/ml microdystrophin expression (claims 19 and 32), one of skill in the art would have found obvious to use routine experimentation and vary the dose of recombinant AAV8, with the reasonable expectation that doing so would determine the dose resulting in the optimal microdystrophin expression. How the amount of expressed microdystrophin is determined is not patentably because it does not produce a novel feature.
With respect to claim 8, Dickinson et al. teach that the SPc5-12 promoter has a nucleotide sequence identical to the claimed SEQ ID NO: 39 (see p. 32, line 12; see the attached Sequence Alignment).
Thus, the claimed invention was prima facie obvious at the time of its effective filing date.
10. No claim is allowed.
Claims 4, 22, 24, and 26 are free of the prior art of record because the prior art of record does not teach or render obvious the optimized nucleic acid sequence set forth by SEQ ID NO: 20.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to ILEANA POPA whose telephone number is (571)272-5546. The examiner can normally be reached 8:00 am to 4:30 pm.
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If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Christopher Babic can be reached at 571-272-8507. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
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/ILEANA POPA/Primary Examiner, Art Unit 1633
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