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
Response to Amendment
1. Claims 1, 2, 4, 10, 12, 22 and 26 have been amended as requested in the amendment filed on July 09, 2026. Following the amendment, claims 1, 2, 4, 5, 10, 12, 13, 22, 24, 26, 36-40, 48-50, 62 and 72 are pending in the instant application.
2. Claims 36-40, 48-50, 62 and 72 stand withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention(s), there being no allowable generic or linking claim. Election was made without traverse in the reply filed on November 25, 2025.
3. Claims 1, 2, 4, 5, 10, 12, 13, 22, 24 and 26 are under examination.
4. Any objection or rejection of record, which is not expressly repeated in this action has been overcome by Applicant’s response and withdrawn.
5. Applicant’s arguments filed on July 09, 2026 have been fully considered but found to be not persuasive for reasons set forth below.
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.
6. Claims 1, 2, 4, 5, 10, 12, 13, 22, 24 and 26 stand rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, for reasons of record in section 17 of Paper mailed on February 11, 2026.
Applicant traverses the rejection at pp. 4-7 of the Response. Specifically, at p. 5-6, Applicant submits that the claims have been amended to are commensurate with the scope of the disclosure, “[C]laim 1 now recites reducing DYRK1A and/or APP relative to a reference cell. Claim 10 now recites that DYRK1A and/or APP in the subject is increased relative to a reference subject without the disease and that administering the effective amount decreases the level of DYRK1A and/or APP in the subject relative to the level in the subject prior to administration. Claim 26 has been similarly amended in the context of Fragile X syndrome. These amendments align the claims with the disclosure in the Summary at pages 2-4 of the specification, which expressly describes methods of reducing DYRK1A and/or APP by contacting a cell with an expression vector comprising a polynucleotide sequence encoding FMRP, identifies cells having increased DYRK1A and/or APP relative to a normal or non-disease reference, and identifies Fragile X syndrome, Down syndrome, and Alzheimer's disease as relevant developmental or neurodegenerative disorders.” Applicant further argues that, “[T]he working examples reinforce that guidance with concrete experimental protocols and results in relevant human cell systems. Pages 60-61 describe eCLIP-seq studies identifying high-confidence FMRP RNA targets in human pluripotent stem cells and excitatory cortical neurons, and pages 73-74 report enrichment of FMRP targets for genes implicated in developmental disorders, autism, and HSA21-encoded transcripts, including DS-associated targets such as APP and DYRK1A. Pages 78-79 describe CRISPRa experiments in DS patient iPSC cells in which FMRP upregulation significantly decreased DYRK1A protein levels and significantly reduced APP protein expression, providing proof-of-concept that transiently increasing endogenous FMRP in the DS context significantly modulates key HSA21-encoded targets. Pages 81-84 further report that FMRP CRISPRa reversed the directionality of 21% of significant differentially expressed genes at 48 and 120 hours and 43% post-treatment, strengthening the relevance of the molecular connection between DS and FXS.” Applicants’ arguments have been fully considered but found to be not persuasive for reasons that follow.
Evaluation of adequate scope of enabled invention is made as an assessment from the perspective of one of ordinary skill in the art in view of the disclosure and any other evidence of record (e.g., test data, affidavits or declarations from experts in the art, patents or printed publications) that is probative of the Applicant's assertions. Reference to the description of the invention within the Summary pages does not by itself satisfies the enablement requirement of 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph. Claims 1, 2, 4, 5, 10, 12, 13, 22, 24 and 26 encompass methods of administration of FMPR polypeptide or polynucleotide to reduce the level of DYRK1A and/or APP in the cells of a subject with any developmental disorder or any neurodegenerative disorder and so to effectively treat the disorder. The Examiner maintains that the specification fails to provide any meaningful details which are critical to practice the full scope of the invention. In the instant case, the prior art does not teach that all developmental disorders and neurodegenerative disorders have a common etiology directly associated with DYRK1A, APP and FMRP. Granted that a skilled practitioner can practice and discover for himself which developmental or neurodegenerative disorder benefits from clinical administration of FMPR; however, this by itself represents an undue experimentation which is prohibited under the statute of 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph.
For reasons of record fully explained earlier and reasons above, the rejection is maintained.
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.
7. Claim(s) 1, 2, 4, 5, 10, 12, 13 and 26 stand rejected under 35 U.S.C. 102(a) as being anticipated by WO 2019/227219, 2019, the ‘219 document, for reasons of record in section 18 of Paper mailed on February 11, 2026.
Applicant argues at pp. 7-8 of the Response that, “[T]he Office Action relies on '219 for treatment of Fragile X syndrome by administration of an AAV-FMRP vector and acknowledges that '219 does not teach increased levels of DYRK1A and/or APP in cells of subjects with Fragile X syndrome and does not teach that administration of an FMRP vector reduces DYRK1A and/or APP levels. […] Those limitations remain material claim limitations. Claim 1 requires reducing and decreasing DYRK1A and/or APP relative to a reference cell, claim 10 requires both an increased level of DYRK1A and/or APP relative to a reference subject without the disease and a decrease in DYRK1A and/or APP relative to the level in the subject prior to administration, and claim 26 includes corresponding limitations for a subject having or having a propensity to develop Fragile X syndrome.” Applicant further submits that, “inherency is not established because the cited reference does not necessarily and inevitably disclose the claimed DYRK1A/APP modulation. The present application describes the discovery that increasing FMRP is sufficient to significantly reduce APP and DYRK1A expression and provides examples in which acute upregulation of endogenous FMRP in Down syndrome patient cells significantly reduced DYRK1A and APP.” Applicant’s arguments have been fully considered but found to be not persuasive for the following reasons.
MPEP § 2111 Claim Interpretation; Broadest Reasonable Interpretation, states,
During patent examination, the pending claims must be “given their broadest reasonable interpretation consistent with the specification.” (The Federal Circuit’s en banc decision in Phillips v. AWH Corp., 415 F.3d 1303, 75 USPQ2d 1321 (Fed. Cir. 2005) expressly recognized that the USPTO employs the “broadest reasonable interpretation” standard:
The Patent and Trademark Office (“PTO”) determines the scope of claims in patent
applications not solely on the basis of the claim language, but upon giving claims their
broadest reasonable construction “in light of the specification as it would be interpreted
by one of ordinary skill in the art.” In re Am. Acad. of Sci. Tech. Ctr., 367 F.3d
1359, 1364[, 70 USPQ2d 1827] (Fed. Cir. 2004)). See also, In re Hyatt, 211 F.3d 1367, 1372, 54 USPQ2d 1664, 1667 (Fed. Cir. 2000). Applicant always has the opportunity to
amend the claims during prosecution, and broad interpretation by the examiner reduces
the possibility that the claim, once issued, will be interpreted more broadly than is
justified. In re Prater, 415 F.2d 1393, 1404-05, 162 USPQ 541, 550-51 (CCPA
1969).
Further, MPEP § 2111.01 Plain Meaning, states
“[Although] claims of issued patents are interpreted in light of the specification, prosecution history, prior art and other claims, this is not the mode of claim interpretation to be applied during examination. During examination, the claims must be interpreted as broadly as their terms reasonably allow. In re American Academy of Science Tech Center, 367 F.3d 1359, 1369, 70 USPQ2d 1827, 1834 (Fed. Cir. 2004).
As fully explained earlier, claims 1, 2, 4, 5, 10, 12, 13 and 26, by broadest reasonable interpretation and consistent with the specification as filed, encompass a method of treating Fragile X syndrome by administration of FMRP polynucleotide. The ‘219 document teaches treatment of Fragile X syndrome by administration of an AAV-FMRP vector, see abstract, [0010], claim 19 and the whole text of the document. Thus, the step of administration of FMRP vector provides for a contact with the cells, such as any cell, within the body of the subject under treatment. Further, the instant specification specifically points out that Fragile X pathology is associated with the increased cellular level of DYRK1A and APP, which provides for the inventive concept to treat Fragile X so to reduce the pathological levels of DYRK1A and APP. Thus, it is reasonable to assert that a subject suffering from Fragile X and administered FMRP would benefit from the same results, regardless of knowing the mechanism or physiological significance of the process. Therefore, the Examiner maintains that the ‘219 fully anticipates the instant claimed invention.
Conclusion
8. No claim is allowed.
9. THIS ACTION IS MADE FINAL. 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 OLGA N CHERNYSHEV whose telephone number is (571)272-0870. The examiner can normally be reached 9AM to 5:30PM, Monday to Friday.
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, Jeffrey Stucker can be reached at (571)272-0911. 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.
/OLGA N CHERNYSHEV/ Primary Examiner, Art Unit 1675
July 29, 2026
Prosecution Insights