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
Applicant’s election of Group I, claims 1-3, 5-7, 10-13, 15, 19-21 in the reply filed on 04/06/2026 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)).
Election of species of enAsCas12a is acknowledged.
Claims 10, 22-27 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected Group and species, there being no allowable generic or linking claim. Election was made without traverse in the reply filed on 04/06/2026.
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.
Claim 7 is 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 the inventor or a joint inventor (or for applications subject to pre-AIA 35 U.S.C. 112, the applicant), regards as the invention.
Claim 7 refers to crRNA3 molecule. Neither disclosure nor claims provide specific sequence for such RNA molecule, therefore it is not clear what exactly such molecule encompass. The only description of the molecule is on Figure 1A pointing out target sequence to which the molecule binds. For the purpose of examination it will be assumed that crRNA3 molecule is a complement to an underlined portion on Figure 1A, but appropriate sequence submission and definition is required.
The following is a quotation of 35 U.S.C. 112(d):
(d) REFERENCE IN DEPENDENT FORMS.—Subject to subsection (e), a claim in dependent form shall contain a reference to a claim previously set forth and then specify a further limitation of the subject matter claimed. A claim in dependent form shall be construed to incorporate by reference all the limitations of the claim to which it refers.
The following is a quotation of pre-AIA 35 U.S.C. 112, fourth paragraph:
Subject to the following paragraph [i.e., the fifth paragraph of pre-AIA 35 U.S.C. 112], a claim in dependent form shall contain a reference to a claim previously set forth and then specify a further limitation of the subject matter claimed. A claim in dependent form shall be construed to incorporate by reference all the limitations of the claim to which it refers.
Claim 11 is rejected under 35 U.S.C. 112(d) or pre-AIA 35 U.S.C. 112, 4th paragraph, as being of improper dependent form for failing to further limit the subject matter of the claim upon which it depends, or for failing to include all the limitations of the claim upon which it depends.
Claim 11 depends on claim 2 and originally on claim 1 and adds a limitation of administering nuclease or base-modifying protein to a cell. Claim 1 recites providing such nuclease or base-modifying protein in order to edit genetic mutation. Claim 2 specifically defines the cell. In order to edit such mutation it is required to administer nuclease or base-modifying protein to the cell, therefore limitation of claim 11 is already present (inherent) in claim 2, meaning that claim 11 does not further limit claim 2.
Applicant may cancel the claim(s), amend the claim(s) to place the claim(s) in proper dependent form, rewrite the claim(s) in independent form, or present a sufficient showing that the dependent claim(s) complies with the statutory requirements.
Claim Rejections - 35 USC § 102
The following is a quotation of the appropriate paragraphs of 35 U.S.C. 102 that form the basis for the rejections under this section made in this Office action:
A person shall be entitled to a patent unless –
(a)(1) the claimed invention was patented, described in a printed publication, or in public use, on sale, or otherwise available to the public before the effective filing date of the claimed invention.
Claim(s) 1, 2, 5, 11-13, 21 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Wolfe et al (WO 2019/213504, November 2019, cited from IDS).
Concerning claims 1 and 21 Wolfe disclose method providing a subject exhibiting symptoms of muscular dystrophy and a genetic mutation, and a nuclease such as Cas9 as an adeno-associated virus encoding such nuclease, such nuclease editing the mutation, reducing at least one symptom of the disease (see bridging paragraph between pages 2 and 3). Such subject can be human (se lines 21-22 on page 5).
Concerning claims 2, 11 and 13 genetic mutation can be in an in vitro human muscle cell (see lines 19-20 on page 31).
Concerning claim 5 a gene to target can be DYSF (see Table 6 on page 47).
Concerning claim 12 cells can be differentiated into muscle tissue (see lines 17-19 on page 32).
Claim(s) 1-3, 5, 11-13, 19-21 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Jones et al (US 2020/0017842, January 2020, cited from IDS) as evidenced by Lemmers et al (Science, 2010, vol.329: 1650-1653, cited from IDS).
Concerning claims 1, 3, 11 and 19 Jones disclose methods of treatment of facioscapulohumeral muscular dystrophy (see Abstract) comprising administering to a subject in need thereof such as mammal or human, such subject comprising genetic mutation (see paragraphs [0016-0017]), a nucleic acid encoding Cas nuclease and gRNA, targeting gene DUX4 comprising genetic mutation (see paragraphs [0006-0011]). Lemmers disclose that subjects with facioscapulohumeral muscular dystrophy comprise mutation in 4qA locus with ATTAAA sequence (see pages 1650 and 1652).
Concerning claims 2 and 13 the cell to administer the nucleic acid is an in vitro human muscle cell (see paragraphs [0015, 0019]).
Concerning claim 12 the cells can be further differentiated into muscle tissue (see paragraph [0054]).
Concerning claim 20 nuclease can be administered using nanoparticles (see paragraph [0099]).
Concerning claim 21 nuclease can be delivered using associated adenovirus (see paragraph [0011]).
Claim Rejections - 35 USC § 103
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.
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.
Claim(s) 1-3, 5-7, 11-13, 15, 19-21 is/are rejected under 35 U.S.C. 103 as being unpatentable over Jones, above, as applied to claims 1-3, 5, 11-13, 19-21, and in further view of Kleinstiver et al (Nature Biotechnology, 2019, 37: 276-282, cited from IDS) and Dumonceaux et al (US 2018/0016577, January 2018).
Teachings of Jones are discussed above.
Jones do not teach using enAsCas12a nuclease, which can bind to crRNA3 molecule, or usage of immortalized FSHD myoblast cell lines.
Kleinstiver teach an enhanced variant of Cas nuclease, enAsCas12a, with expanded targeting range (see Abstract). Kleinstiver teach that enAsCas12a binds a variety of crRNAs and describes design of such crRNAs (see page 278, Methods section).
Dumonceaux teach use of immortalized FSHD myoblast cell lines for treatment of facioscapulohumeral dystrophy (see Abstract, paragraph [0087]).
It would have been obvious to one of the ordinary skill in the art before the effective filing date of the claimed invention to utilize enAsCas12a nuclease taught by Kleinstiver and immortalized FSHD myoblast cell lines taught by Dumonceaux in methods taught by Jones, arriving at instant invention. One of the ordinary skill in the art would be motivated to do so, because Kleinstiver suggest enhanced variant of Cas nuclease, enAsCas12a, which can be used in the methods of Jones. Kleinstiver also teach design of crRNAs, providing basis for designing molecules such as instant crRNA3. Further Dumonceaux teach that immortalized FSHD myoblast cell lines can be used in the treatment of the same disease as in methods taught by Jones, therefore such cell lines can be used in methods taught by Jones for treatment of facioscapulohumeral muscular dystrophy.
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, 5-7, 11-13, 15, 19-21 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-13 of U.S. Patent No. 11,566,237 in view of Kleinstiver and Dumonceaux, as above. Claims from ‘237 teach nucleases which can be used for correction of mutations in DUX4 gene. Further, Example 2 of specification specifically teach application of such nucleases for treatment of facioscapulohumeral muscular dystrophy, same as in instant invention. Teachings of Kleinstiver and Dumonceaux are discussed above. It would have been obvious to modify methods from ‘237 with teachings of Kleinstiver and Dumonceaux, arriving at instant invention.
Claims 1-3, 5-7, 11-13, 15, 19-21 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 21-22, 24-25, 28-33, 38-39 of copending Application No. 18/065,255 (reference application) in view of Kleinstiver and Dumonceaux, as above. Claims from ‘255 teach treatment of facioscapulohumeral muscular dystrophy by administration of Cas nucleases. Teachings of Kleinstiver and Dumonceaux are discussed above. It would have been obvious to modify methods from ‘255 with teachings of Kleinstiver and Dumonceaux, arriving at instant invention.
This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
Conclusion
Any inquiry concerning this communication or earlier communications from the examiner should be directed to EKATERINA POLIAKOVA whose telephone number is (571)270-5257. The examiner can normally be reached Mon-Fri 8-5.
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/EKATERINA POLIAKOVA-GEORGANTAS/Primary Examiner, Art Unit 1637
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