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 .
Claims 1-25 are pending as of the amendment of 11/16/23 and are considered herein.
Formalities:
The drawings of 11/16/23 are accepted.
The specification of 1/29/24 is accepted.
The IDS filings, and references therein, of 1/29/24 (three filings), 1/30/24; 2/15/24; and 1/10/25, have been considered. A Signed copy of each is provided herewith.
Applicant’s priority is noted to be the following:
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Claim Objections
Claim 1 is objected to because of the following informalities:
Claim 1 provides the first recitation of “MBM”. It is customary to provide the full meaning of meaning of a short hand acyronym upon its first recitation.
Claim 1 provides the first recitation of “MBD”. It is customary to provide the full meaning of meaning of a short hand acyronym upon its first recitation.
Claim 3 recites the first recitation of “CR” without providing the full meaning of such. It is customary to provide, with the first recitation, the full name.
Claim 4 recites the first recitation of “CT” without providing the full meaning of such. It is customary to provide, with the first recitation, the full name.
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.
Claim 5 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 5 recites “at least one domain and at least one domain and at least one other domain”. It is not clear what is meant here. Is this two domains? Are they required to be different. I.e., “at least one other domain” is also unclear. There are only two domains in Claim 1. This is also an antecedent basis problem. I simply do not understand.
Claim Rejections - 35 USC § 112
The following is a quotation of the first paragraph of 35 U.S.C. 112(a):
(a) IN GENERAL.—The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor or joint inventor of carrying out the invention.
The following is a quotation of the first paragraph of pre-AIA 35 U.S.C. 112:
The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor of carrying out his invention.
Claims 1-25 are rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, as failing to comply with the written description requirement. The claim(s) contains subject matter which was not described in the specification in such a way as to reasonably convey to one skilled in the relevant art that the inventor or a joint inventor, or for applications subject to pre-AIA 35 U.S.C. 112, the inventor(s), at the time the application was filed, had possession of the claimed invention.
As seen by depending Claim 12, the claims are generic for tri-recombinant AAVs to permit recombination to form an operable full length micro/mini dystrophin.
The specification provides no antecedent basis for the same, much less description. In fact, the specification is limited in its description to dual AAVs the reconstitute the structure (e.g., p. 4. Penultimate paragraph).
Moreover, the Art did not have these structures in the context of tri-recombinant AAVs. In fact, the purpose of these micro and minidystrophins is intended to allow delivery of a dystrophin gene that can fit in the vector, e.g., AAV. E.g., Chwalena, et al. (2025) “AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects”, Gene Therapy, 32: 447-61, p. 449, col. 2, paragraph 3.
Thus, at best, Applicant’s claims to tri-recombinant AAVs are an extrapolation of the teachings of the specification, and rely on obviousness. Obviousness, however, is not sufficient to demonstrate possession.
Therefore, the Artisan would not have understood Applicant to have been in possession of tri-recombinant AAVs that recombine to form dystrophin.
Claim Rejections - 35 USC § 112
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 6 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 6 contains several recitations of “or a variant thereof”, which is outside the scope of the claim from which it depends: Claim 5. 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 § 112
The following is a quotation of the first paragraph of 35 U.S.C. 112(a):
(a) IN GENERAL.—The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor or joint inventor of carrying out the invention.
The following is a quotation of the first paragraph of pre-AIA 35 U.S.C. 112:
The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor of carrying out his invention.
Claims 1-25 are rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, as failing to comply with the written description requirement. The claim(s) contains subject matter which was not described in the specification in such a way as to reasonably convey to one skilled in the relevant art that the inventor or a joint inventor, or for applications subject to pre-AIA 35 U.S.C. 112, the inventor(s), at the time the application was filed, had possession of the claimed invention.
The claims are generic for a micro/mini dystrophin, comprising the NT and an MBM comprising R11-R12 (Claim 1). Depending claims include R1-R2 (Claim 2), a CR or CT domain (Claims 3-4), hinge regions (Claim 5).
The specification teaches mini/micro dystrophins for reducing damage in DMD, BMD, etc. (e.g., BACKGROUND). Specifically, it is recognized that the joining of the actin cytoskeleton to the ECM with dystrophin and the distroglycans. Figure 1 is an excellent depiction of the normal joining of these regions, bringing the ECM and cytoskeleton together, to protect the membrane.
The claims, however, only require R11 and R12, which are shown in the specification to bind the membrane in experiments (e.g., Examples) and the N-terminal domain binds to the cytoskeleton. While these do appear to join the membrane to the cytoskeleton, it is also admitted by Applicant that this does not mean it will join it with the ECM (e.g., Figure 1). Additionally, the components are not in context with the hinge regions that require furether studies to see if it will work with or without such hinge regions (e.g., p. 34, paragraph 2).
It is admitted Applicant’s own studies are the first in the field localizing a membrane-binding region to R11-R12, however, even in Applicant’s own non-patent publication of the same results, it is admitted that it is not known if these regions will work in proper context (e.g., Zhao, et al. (2016) “Dystrophin contains multiple independent membrane-binding domains” Human Molecular Genetics, 25(17): 3647-53, pp. 3651-52, paragraph bridging).
Thus, without the presence of the DGC localizing units, the Artisan would not have understood Applicant to have been in possession of a mini/micro dystrophin with R11-R12 only. Nor would the Artisan have understood Applicant to have been in possession of the same, in the context of hinge regions.
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 1-11 and 13-25 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-24 of U.S. Patent No. 11,202,840. Although the claims at issue are not identical, they are not patentably distinct from each other because:
Claim 1: Patent Claim 1 recites a nucleic acid encoding a micro/mini dystrophin (non full length), comprising a NT and an MBM comprising R11 and R12, and a corresponding cysteine residue of the S-palmitoylation site peptide of SEQ ID NO: 57.
Claim 2: Patent Claim 1 recites “an MBM of an R1-R2 membrane binding domain (MBD), wherein the MBM of R1-R2 comprises a cysteine residue corresponding to the conserved cysteine residue of the S-palmitoylation site peptide of SEQ ID NO: 54”.
Claim 3: Claim 1 recites “an MBM of a CR membrane binding domain”.
Claim 4: Claim 1 recites “an MBM of a CT membrane binding domain”.
Claim 5-6: Claim 6 recites the various components of the hinge region.
Claim 7: Claim 7 contains the options of size and elements.
Claim 8: Claim 8 requires the integration in the endogenous dystrophin gene locus of the X chromosome.
Claim 9: Claim 9 requires the same lentiviral vector with cassette, LTRs and psi sequence.
Claim 10: Claim 10 is the AAV vector with ITRs and expression cassette.
Claim 11: Claim 11 is to the same dual AAV vector system.
Claim 12:
Claim 13: Claim 12 is mixed with the pharmaceutically acceptable carrier.
Claim 14: Claim 13 requires the LTRs and psi, or ITRs in an AAV.
Claim 15: Claim 14 requires the isolated host cell.
Claim 16: Claim 15 requires integration in the endogenous dystrophin gene locus.
Claim 17: Claim 16 requires the LTRs and cassette, or AAV ITRs and cassette.
Claim 18: Claim 17 requires a myogenic stem cell.
Claim 19: Claim 18 is to the same treating or ameliorating DMD, BMD, or XLDC symptoms, in a subject, with the same administration.
Claim 20: Claim 19 is to systemic delivery.
Claim 21: Claim 20 teaches the same stem or myogenic stem cell.
Claim 22: Claim 21 teaches autologous host cell administration.
Claim 23: Claim 22 teaches the host cell gene/portion is edited to provide the NA within the host cell X chromosome.
Claim 24: Claim 23 teaches the lentiviral vector, with the same cassette, LTRs and psi sequence.
Claim 25: Claim 24 teaches a single AAV with cassette and ITRs.
Thus, in light of the patent the invention is obvious. The Artisan would make the invention and expect success, as it is claimed subject matter.
Conclusion
No claim is allowed.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to ROBERT M KELLY whose telephone number is (571)272-0729. The examiner can normally be reached M-F: 8a-5p.
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ROBERT M. KELLY
Examiner
Art Unit 1638
/ROBERT M KELLY/Primary Examiner, Art Unit 1638
Prosecution Insights