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 .
Applicant’s amendment and response to restriction requirement of 12/30/25 are entered.
Claims 1-3, 9-12, 15, 19, 21, 23-24, 31-33, 35-45, 47, 49-50, 52, and 54 are amended.
Claims 13-14 and 34 are canceled.
Claims 1-3, 9-12, 15, 19, 21, 23-24, 31-33, 35-45, 47, 49-50, 52, and 54 are pending.
Election/Restrictions
Applicant’s election without traverse of Group I, as in present claims 1-3, 9-12, 15, 19, 21, 23, 24, 31-33, 49-50, 52, and 54, in the reply filed on 12/30/25 is acknowledged.
Claims 35-45 and 47 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected inventions, there being no allowable generic or linking claim. Election was made without traverse in the reply filed on 12/30/25.
In viewing the elections, it is noted that Applicant has moved GBA coding sequences into the base claim, and so the Examiner has removed the species elections to only require the ORF of the sequences now in claim 1. Such election is the ORF of 386. That ORF is found to be free of the art. Thus, the Examiner has gone to join the ORF of SEQ ID NO: 385, also found in Claim 1, as the next considered species.
Claims 1-3, 9-12, 15, 19, 21, 23, 24, 31-33, 49-50, 52, and 54 are considered with respect to the ORF of SEQ ID NO: 385.
Formalities:
The drawings of 9/22/22 are accepted.
The specification as amended 9/22/22 is accepted.
The IDS filings of 11/4/22; 1/28/25; and 4/10/25 have been considered and are signed off upon, herewith.
Claim Objections
Applicant is advised that should claim 1 be found allowable, claim 12 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). At the very least, the ITRs are necessarily symmetric or not, and thus, Claim 12 is substantially the same scope as Claim 1.
Applicant is advised that should claim 1 be found allowable, claim 15 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). Claim 15 requires at least one ITR to be wild type or not wild type. As such, it has the same scope as Claim 1, despite the slight difference in wording.
Applicant is advised that should claim 1 be found allowable, claim 21 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). Claim 21 requires one ITR to be synthetic. However, the manner of making does not alter the structure, and thus, despite the slight difference in wording, these claims have substantially the same scope.
Applicant is advised that should claim 1 be found allowable, claim 49 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). Claim 49 is drawn to a pharmaceutical composition comprising the composition of Claim 1. However, calling it a pharmaceutical composition, does not change the scope. Thus, despite the slight difference in wording, these claims have substantially the same scope.
Claim Rejections - 35 USC § 103
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.
Claim(s) 1-3, 9-12, 15, 19, 21, 23-24, 31-33, 49-50, and 54 is/are rejected under 35 U.S.C. 103 as being unpatentable over Yang, et al. (2002) “Sustained Expression of Human β-Glucocerebrosidase in Mice Following Administration of AAV”, Molecular Therapy, 5(5), part 2 of 2, pp. s341-s342, ABSTRACT 1050; Li, et al. (2012) “Closed-Ended Linear Duplex AAV (cdldAAV) DNA for Non-Viral Gene Transfer”, Molecular Therapy, 20(Suppl. 1), page S236, ABSTRACT 613; and U.S. Patent Application Publication No. 2011/0217274 to Reld.
Yang teaches the use of AAV vectors carrying GC transgenes, to transfect HepG2 cells, and in B6,129S-Rag-1 mice, via hepatic portal vein administration. (ABSTRACT), however, Yang does not teach the use of cdAAV vectors, nor the use of SEQ ID NO: 385 for expressing the GC transgene.
On the other hand, the Artisan, interested in AAV vectors for transgene delivery, would be aware of Li, for its teaching of AAV vectors for non-viral gene transfer. Li teaches the manufacture of cdldAAV vectors, which are covalently closed-ended DNA, with AAV ITRs, the advantage being that they are devoid of prokaryotic elements and DNA modificatios, and contain no plasmid sequences or bacterial lipoprotein polysaccharides, and they can be used in vitro, ex vivo, or in vivo, for delivery of exogenous DNA sequences to a target for gene therapy. (ABSTRACT) However, Li does not teach the use of a claimed sequence for expressing the GC.
On that point, The Artisan would be aware of Reld for teachings of GC (known in Reld as GBA). Reld teaches SEQ ID NO: 106, which is a match for the GC presently claimed as nucleotides 581-2191 of SEQ ID NO: 385.
Thus, at the time of invention, it would have been obvious to make the vectors claimed. One would do so for expressing the GBA in cells in vitro, or in mice, as taught by Yang. The Artisan would expect success, as the components are utilized for Art-recognized purposes.
With regard to a promoter (Claim 3), it is asserted such is essential, as it is required to be expressed, and it is well known to the Artisan to utilize a promoter for expression. However, it is also noted that Reld teaches the use of promoters for expression of the transgene (e.g., paragraphs 12-13).
With regard to the coding sequence being cDNA (Claim 9), Applicant’s own specification teaches that SEQ ID NO: 385 is a cDNA, and thus Reld’s disclosed SEQ ID NO: 106, having the same sequence, is necessarily of the structure of a cDNA.
With regard to the trs and rep sites (Claim 10), they are necessarily present, as Li teaches that they can be rescued and amplified (ABSTRACT).
With regard to the ITRs being from an adenovirus (Claim 11), Li teaches the use of adenovirus ITRs (ABSTRACT).
With regard to symmetry (Claim 12): the ITRs are symmetric, or asymmetric, as those are the only choices, and considering they function identically, they are also substantially symmetric.
With regard to the ITRs (Claim 15), each are either wild type or not wild-type, and each are from AAV2, in Li (ABSTRACT).
With regard to Claim 19, as each type of ITR is distinct and structure affects function, these ITRs being AAV2 ITRs, can be considered to be mutated from another ITR serotype, and would necessarily have distinct 3D structure.
With regard to Claim 21: the ITRs have the structure of an ITR that could be made synthetically, and thus, the claim is met.
With regard to Claim 23: each of the ITRs are mutated forms of the other serotypes, and thus, because the regions in the ITRs are not all identical, they have the structure that infringes each embodiment.
With regard to Claim 24, because each serotype ITR is distinct, and has distinct stem loop structure, the ITRs have the structure claimed, in that one is shorter than another in one region or another.
With regard to Claim 31: the ITRs have distinct structure, but the AAV ITR has overall 3D symmetry, and thus, it may be viewed as mutated from another serotype ITR.
Claim 32: SEQ ID NOs: 1 and 2 are derived of AAV2 and thus, assumed to be present as Li utilized AAV2 ITRs.
Claim 33: Reld teaches the us of TET-regualted promoters for controlled expression (e.g., paragraph 147).
Claim 49: the vector, to be injected must be in a buffer/water and is necessarily then a pharmaceutical composition.
Claim 50: the cells obtained would meet the claim.
Claim 53: the kit is met by the ceDNA, as the instructions do not change the composition of the vector.
Claim(s) 1-3, 9-12, 15, 19, 21, 23-24, 31-33, 49-50, 52, and 54 is/are rejected under 35 U.S.C. 103 as being unpatentable over Yang, et al. (2002) “Sustained Expression of Human β-Glucocerebrosidase in Mice Following Administration of AAV”, Molecular Therapy, 5(5), part 2 of 2, pp. s341-s342, ABSTRACT 1050; Li, et al. (2012) “Closed-Ended Linear Duplex AAV (cdldAAV) DNA for Non-Viral Gene Transfer”, Molecular Therapy, 20(Suppl. 1), page S236, ABSTRACT 613; and U.S. Patent Application Publication No. 2011/0217274 to Reld, as applied to claims 1-3, 9-12, 15, 19, 21, 23-24, 31-33, 49-50, and 54, above, and further in view of Zhao, et al. (2014) “Lipid Nanoparticles for Gene Delivery”, Advances in Genetics, 88: 13-36, provided as an HHS Public Access Author Manuscript, 21 pages long.
As shown above, the base claims are obvious over the base art, however the aspect of utilizing a lipid nanoparticle for the delivery of the vector is not taught or obvious from the base art.
On the other hand, the Artisan, interested in delivery DNAs would be aware of Zhao for its teaching of gene delivery. Zhao teaches the use of lipid nanoparticles for delivery of DNA (e.g., ABSTRACT, INTRODUCTION).
Thus, it would have been obvious to further modify the invention to utilize lipid nanoparticles for delivery. The Artisan would do so to deliver the DNA and expect success, as the components are utilized for their art-recognized purposes.
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