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 without traverse of Group III, claims 28, 29 and 31-34 in the reply filed on 4/28/2026 is acknowledged.
Claims 1, 3-8, 10-13, 18, 20-22, 24, 27 and 30 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention, there being no allowable generic or linking claim. Election was made without traverse in the reply filed on 4/28/2026.
Claims 28, 29 and 31-34 are examined in the instant office action.
Claim Objections
Claims 29 and 31-34 are objected to because of the following informalities: claims 29 and 31-34 are not grammatically correct. The word “Claim” should be replaced with “claim”. Appropriate correction is required.
Claim Rejections - 35 USC § 102
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 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) 28, 29 and 31-34 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Carlo et al. (US 2020/0040362 A1, published 2/6/2020).
Regarding claim 28, Carlo et al. teach a gene editing system, comprising:
a recombinant AAV vector comprising a nucleic acid sequence encoding a repair template, a promoter operably linked to the repair template, and a poly A sequence, wherein the repair template is for the IVS1 variant of the acid alpha-glucosidease (GAA) gene; and a recombinant AAV vector comprising a nucleic acid sequence encoding a guide RNA (gRNA) operably linked to a first promoter and a nucleic acid sequence encoding a Cas9 nuclease operably linked to a second promoter,
wherein the gRNA targets
(i) a PAM sequence at or near the IVS1 variant near the boundary between intron 1 and exon 2 in the GAA gene (parags. 310-319, 780, Example 5 and Fig. 1).
Regarding claim 29, Carlo teaches a pharmaceutical formulation comprising the gene editing system (parag. 323).
Regarding claims 31-33, Carlo teaches the first and second promoters can be CMV (parags. 624-626).
Regarding claim 34, Carlo teaches that the system can further comprise an NLS (parag. 576).
Thus Carlo et al. clearly anticipate the invention of claims 28, 29 and 31-34.
Conclusion
No claims are allowed.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to DAVID A MONTANARI whose telephone number is (571)272-3108. The examiner can normally be reached M-Tr 8-6.
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/DAVID A MONTANARI/Examiner, Art Unit 1632
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