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 .
This action is in response to the amendment, filed 12/10/2025, in which claims 8-20 were cancelled; claims 1-2, 4 were amended and claims 21-29 were added. Applicant’s arguments have been thoroughly reviewed, but are not persuasive for the reasons that follow. Any rejections and objections not reiterated in this action have been withdrawn. This action is FINAL.
Withdrawn Rejections
The rejection of claims 1, 3-7 under 35 U.S 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, has been withdrawn in view of Applicant’s amendments to the claims in the reply in the reply filed 12/10/2025.
The rejection of claims 8-13 under 35 U.S 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, is moot in view of Applicant’s cancellation of the claims in the reply in the reply filed 12/10/2025.
The rejection of claims 1-7 under 35 U.S 102(a)(1) and 35 U.S.C. 102(a)(2) as being anticipated by Davidson et al., has been withdrawn in view of Applicant’s amendments to the claims in the reply in the reply filed 12/10/2025.
The rejection of claims 8-20 under 35 U.S 102(a)(1) and 35 U.S.C. 102(a)(2), is moot in view of Applicant’s cancellation of the claims in the reply in the reply filed 12/10/2025.
Terminal Disclaimer
The electronic terminal disclaimer filed on December 10, 2025 disclaiming the terminal portion of any patent granted on this application which would extend beyond the expiration dates of U.S. Patent No. 11,473,084 has been reviewed and is accepted. The terminal disclaimer has been recorded.
These rejections are made to address the amendment to the claims in the reply filed 12/10/2025.
Claim Objections
Claim 1 is objected to because of the following informalities:
- In line 3, claim 1 recites “said method comprising” should be “said method comprises”.
- In line 9, claim 1 recites “encoded for mutant protein” should be “encoded mutant protein”.
Claim 2 is objected to because of the following informalities: Claim 2 recites “encoded for mutant protein” should be “encoded mutant protein”.
Claim 5, recites “wherein said guide RNA are administered as a nucleic acid molecule encoding said guide RNA” should be “administering (i) a nucleic acid molecule encoding Cas 9 and (ii) at least two guide RNA,”
Claim 23, recites “wherein said guide RNA are delivered as a nucleic acid molecule encoding said guide RNA”, should be “delivering (i) a nucleic acid molecule encoding Cas9 and (ii) at least two guide RNA.”
Claims 28 and 29 are objected to because of the following informalities: Claims recite “of gene” should be “of the gene”.
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.
Claims 1-7, 21-29 are rejected under 35 U.S.C. 102(a)(1) and 35 U.S.C. 102(a)(2) as being anticipated by Deglon et al. (“Deglon”, WO 2016/020399 A1, cited as reference 6 on IDS filed 03/09/2023).
Regarding claim 1-3, 21, 28-29, Deglon teaches treatment of Huntington's disease (HD) using the CRISPR system. This technology offers the possibility to design a small RNA (sgRNA), which is incorporated into a CRISPR- associated protein (Cas9) to recognize and induce DNA double-strand breaks at a specific target location. In the context of HD, this allows to block the expression of the mutant huntingtin or repair the CAG expansion causing the disease (e.g., abstract). Deglon teaches a kit for the treatment of Huntington's disease (HD) for allele or non-allele-specific huntingtin (HTT) gene editing or repair comprising: a gene delivery vector consisting of at least one viral vector selected among adeno-associated vector serotypes (AAV) and/or lentiviral vectors (LV); a Cas9 being human codon-optimized at least one artificial single guide RNA (sgRNA) recognizing the sequence of the HTT gene around the expanded CAG repeat mutation (e.g., lines 10-30, page 4). Deglon teaches a second strategy for the allele-specific gene editing of mutant HTT gene, use two sgRNAs, one targeting a SNP located close to the TSS/ATG (It reads on targeting the 5’ UTR/ promoter region of HTT gene, sgHTT4 in Fig. 2B) and HD mutation of the HTT gene and a second sgRNA targeting the disease isoform of heterozygous single-nucleotide polymorphisms (SNP) located after the CAG expansion and preferentially in an intron. This leads to the complete deletion of exon 1 containing the TSS/ATG codon and expanded CAG mutation of the HTT. (e.g., lines 8-23, page 32; Fig. 2B-C [see below]).
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Regarding claims 4, 22, 26-27, Deglon teaches a kit for the treatment of Huntington's disease, for use in a method for non-allele-specific HTT gene repair based on HR with a DNA template containing a WT HTT sequence (e.g., line 3, page 5).
Regarding claim 5-7, 23-25, Deglon teaches a kit for the treatment of Huntington's disease (HD) for allele or non-allele-specific huntingtin (HTT) gene editing or repair comprising: a gene delivery vector consisting of at least one viral vector selected among adeno-associated vector serotypes (AAV) and/or lentiviral vectors (LV); a Cas9 being human codon-optimized at least one artificial single guide RNA (sgRNA) recognizing the sequence of the HTT gene around the expanded CAG repeat mutation (e.g., lines 10-30, page 4).
Conclusion
Applicant's amendment necessitated the new ground(s) of rejection presented in this Office action. Accordingly, THIS ACTION IS MADE FINAL. See MPEP § 706.07(a). Applicant is reminded of the extension of time policy as set forth in 37 CFR 1.136(a).
A shortened statutory period for reply to this final action is set to expire THREE MONTHS from the mailing date of this action. In the event a first reply is filed within TWO MONTHS of the mailing date of this final action and the advisory action is not mailed until after the end of the THREE-MONTH shortened statutory period, then the shortened statutory period will expire on the date the advisory action is mailed, and any nonprovisional extension fee (37 CFR 1.17(a)) pursuant to 37 CFR 1.136(a) will be calculated from the mailing date of the advisory action. In no event, however, will the statutory period for reply expire later than SIX MONTHS from the mailing date of this final action.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to JULIO GOMEZ RODRIGUEZ whose telephone number is (571)270-0991. The examiner can normally be reached Monday - Friday 8:00 am - 5:00 pm.
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/JULIO WASHINGTON GOMEZ RODRIGUEZ/Examiner, Art Unit 1637
/Jennifer Dunston/Supervisory Patent Examiner, Art Unit 1637