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 .
Detailed Action
Election/Restrictions
Applicant's election with traverse of Group I, drawn to a liver-specific
promoter selected from i. of claim 1 in the reply filed on 06/26/2025 is
acknowledged.
Election of the following species on the reply filed on 06/26/2025 is
acknowledged: Signal peptide: Seq ID 175; GAA polypeptide: Seq ID No: 182.
Priority
The present application is a 35 U.S.C. 371 national stage filing of International Application No. PCT/US2020/061223, filed 11/19/2020.
Applicant’s claim for the benefit of a prior-filed parent provisional application 63/023,570, filed on 05/12/2020, 62937556 filed 11/19/2019 and 62937583 filed on 11/19/2019 under 35 U.S.C. 119(e) or under 35 U.S.C. 120, 121, or 365(c) is acknowledged.
The earliest priority for the instant application is 11/19/2019.
Applicant’s amendment and response filed on 02/09/2026 has been received and entered into the case.
Claims Status
Claims 71-83 are newly added, claim 68 is cancelled, claim 69 has been withdrawn as being drawn to non-elected subject matter, and claims 1-4, 8, 10-11, 18-19, 21, 24, 26-27, 38-40, 49-51, 55 60-62, 65, 67, and 70-83 have been considered on the merits. All arguments have been considered.
Withdrawn Objections & Rejections
Applicant's response filed 02/25/2026 has been considered. Rejections and/or objections not reiterated from the previous Office action mailed 10/01/2025 are hereby withdrawn.
The amendments to the claims overcome the rejections under 103 and 112(a) and the rejections are withdrawn.
The objections and rejections presented herein represent the full set of objections and rejections currently pending in the application.
Non-compliant Amendment
The reply filed on 02/09/2026 is not fully responsive to the prior Office action because of the following omission(s) or matter(s):
Regarding claims 69-73: The claims status for claims is recited as both “Cancelled” and as “Previously Presented” or “New”.
For purposes of compact prosecution the “Cancelled” status is considered a typing error and the claims are examined accordingly.
For purposes of compact prosecution, the cancelled status is considered a typing error and the claims are examined accordingly.
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.
Claims 1-4, 8, 10-11, 18-19, 21, 24, 26-27, 38-40, 49-51, 55 60-62, 65, 67, and 70-83 are 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.
Regarding claims 1 and 50: The claim recites multiple liver-specific promoters; Seq ID NO: 86, 91 and 92.
It is not clear which of the three promoters is considered the “unmodified” promoter. All three sequences comprise the full sequence of Seq ID NO: 86, thus Seq ID NO: 86 could be considered “unmodified”.
It is not clear which of the three promoters is the “untruncated” promoter. Seq ID NO 92 is the longest promoter sequence (565 bp) and thus could be considered the untruncated promoter compared to the other two sequences.
Note claims 2-4, 8, 10-11, 18-19, 21, 24, 26-27, 38-40, 49, 51, 55 60-62, 65, 67, and 70-83 depend from claims 1 or 50 and fail to cure the deficiency.
Regarding claims 1 and 50: Promoter sequences are identified using multiple identifiers- Applicants terminology (e.g. CRM_SP0412) and a Seq ID NO. Later in the claim sequences are identified only by CRM_SP0412, SP0412 and SP0422.
It is unclear if the parenthetical Seq ID NO: identifies the specific sequence denoted by Applicant’s terminology, or if the Seq ID NO: is representative of the claimed sequence.
Examiner recommends identifying the sequences by Seq ID NOs in place of the Applicants terminology (e.g. CRM_SP0412) to make abundantly clear the sequence that is required.
Note claims 2-4, 8, 10-11, 18-19, 21, 24, 26-27, 38-40, 49, 51, 55 60-62, 65, 67, and 70-83 depend from claims 1 or 50 and fail to cure the deficiency.
Regarding claims 69: As discussed below, claim 69 is withdrawn.
Response to Arguments
The responses are directed to the Arguments filed 02/25/2026, all arguments have been considered.
Regarding claims status:
Regarding claims 4, 65, 67: The claims are pending and were examined on the merits in the previous action, however the claims were omitted from the claims summary coversheet of the previous action due to a typing error.
The claims were rejected in the previous action filed 10/01/2025 under 112(a) and 103 (p16 and p26 of the previous action).
The claims status has been corrected in the instant action.
Regarding claim 69: Applicant requests clarification of the claim status for the claim because claim 69 is listed as rejected in the previous action. The “rejected” status of claim 69 is a typing error, the claim is withdrawn.
As stated in the claims status of the instant action, claim 69 is withdrawn as drawn to unelected subject matter. This is consistent with the statement of Applicant in the Remarks filed 06/26/2025, that claim 69 is not encompassed by the species election “GAA sequence Seq ID NO: 182”.
The claims status of claim 69 has been corrected in the instant action.
Regarding the Restriction/Election:
Applicant amended claim 1 to include Seq ID NO: 92 in the alternative for a liver-specific promoter. The claims are examined accordingly.
Regarding Arguments directed to 35 USC § 112(a):
The claims have been amended which overcome the rejection as written.
Specifically, claims 1 and 50 were rejected over the recitation of “at least 60% promoter activity”.
The claims have has been amended to recite “a functional fragment of SP0412 that comprises a contiguous portion of the unmodified promoter that has at least 75% of the untruncated promoter, truncated from the 3' end, and retains liver specific promoter activity of at least of CRM SP0412, or a functional fragment of SP0422 that comprises a contiguous portion of the unmodified promoter that has at least 35 % of the untruncated promoter, truncated from the 3' end, and retains liver-specific promoter activity of at least of CRM SP0412”.
Claim 38 was rejected over the recitation of “at least about 85% sequence homology”. The claim has been amended to recite “at least about 95% sequence homology”. Applicant submits that at least 95% sequence homology results in no more than 9 amino acid substitutions. This is persuasive and the rejection is withdrawn.
Claim 21 was rejected due to a lack of antecedent basis for which the amendments to the claims overcome.
The rejection is withdrawn.
Regarding Arguments directed to 35 USC § 103:
Regarding claims 1 and 50: The amendments to the claims overcome the rejection as written.
Specifically, Rouet et al was relied upon to teach the liver regulatory sequence of Seq ID NO: 86, or a functional variant or functional fragment thereof.
The amended claims require the full sequence of Seq ID NO: 86 (in contrast to a functional fragment thereof).
Thus the claim amendments overcome the rejection as written and the rejection is withdrawn.
Regarding claim 2-4, 8, 10-11, 18-19, 21, 24, 26-27, 38-40, 49, 51, 55, 60-62, 65, 67, 70-83: The claims depend from claims 1 or 50 for which the amendments to the claims overcome the rejection. Thus rejection over the dependent claims are also withdrawn.
Conclusion
No claims are allowed.
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 ANDREA LYNNE MORRIS SPENCER whose telephone number is (571)272-3328. The examiner can normally be reached Monday-Friday 9:00-5:00.
Examiner interviews are available via telephone, in-person, and video conferencing using a USPTO supplied web-based collaboration tool. To schedule an interview, applicant is encouraged to use the USPTO Automated Interview Request (AIR) at http://www.uspto.gov/interviewpractice.
If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, James (Doug) Schultz can be reached at 571-272-0763. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
Information regarding the status of published or unpublished applications may be obtained from Patent Center. Unpublished application information in Patent Center is available to registered users. To file and manage patent submissions in Patent Center, visit: https://patentcenter.uspto.gov. Visit https://www.uspto.gov/patents/apply/patent-center for more information about Patent Center and https://www.uspto.gov/patents/docx for information about filing in DOCX format. For additional questions, contact the Electronic Business Center (EBC) at 866-217-9197 (toll-free). If you would like assistance from a USPTO Customer Service Representative, call 800-786-9199 (IN USA OR CANADA) or 571-272-1000.
/ANDREA LYNNE MORRIS SPENCER/Examiner, Art Unit 1631
/TAEYOON KIM/Primary Examiner, Art Unit 1631
Prosecution Insights