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 .
Current Status
This action is responsive to the amended claims of 12/04/2025. Claims 1, 4, 6, 10, 18-23, 25, 27, 29, 31, 39-43, 46, 48-49, 51-54, 57, 63-65, and 68 are pending. Claims 4, 6, 10, 21-23, 25, 27, 29, 31, 39-41, 48-49, 51-54, 57, and 63-65 are withdrawn. Claims 1, 18-20, 42-43, 46, and 68 have been examined on the merits.
Election/Restrictions
The amendments to the claims have overcome the prior art rejections of the previous action (09/04/2025). Thus, the Markush search has been extended to the following species:
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. Note: The Markush search has not been fully extended. See the attached annotated search notes (SEARCH 6).
The extended species reads on claims 1, 18-20, 42-43, 46, and 68.
Claims 4, 6, 10, 21-23, 25, 27, 29, 31, and 39-41 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected species, there being no allowable generic or linking claim. Election was made without traverse in the reply filed on 07/23/2025.
Claims 48-49, 51-54, 57, and 63-65 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention, there being no allowable generic or linking claim.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 07/23/2025.
Please file a RCE to resume Markush searching. Additional Markush Searching will not be conducted in After Final as this constitutes “additional search and/or consideration”.
Priority
The effective filing date remains 08/31/2020.
Information Disclosure Statement
The information disclosure statement (IDS) submitted on 12/04/2025 is in compliance with the provisions of 37 CFR 1.97. Accordingly, the information disclosure statement is being considered by the examiner.
Response to Arguments
Examiner acknowledges receipt of and has reviewed the amendments and remarks of 12/04/2025, no new matter is found.
The objection to claim 18 is withdrawn since Applicant has amended the claim to further recite R2 and Y.
The 112b rejections of claims 1-2, 8, 18-20, 42-43, and 46 are withdrawn due to Applicants amendments:
¶14 Claim 1: Applicant has replaced C1 alkenylene-aryl with C2 alkenylene-aryl; this no longer poses a problem to the valency of C. The dependent claims are similarly no longer rejected. The same amendment has been made to withdrawn claim 22.
¶15 Claim 46: Applicant has struck the phrase “e.g.,” from the claim. The same amendment has been made to withdrawn claim 49.
The 102 rejection of claims 1-2, 8, 18-20, 42-43, and 46 over REN is withdrawn since Applicant has amended claim 1 to recite the negative limitation “(ii) when each of W, X, and Z is independently C(R3) and A is heteroaryl, R1 is not -NRBRC.” This limitation excludes the exemplary compound of REN and REN does not teach any further compounds of instant Formula (I). Support for this negative limitation is found in the original positive disclosure of the instantly recited elements/moieties in claim 1.
Response to Amendment
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, 18-20, 42-43, 46, and 68 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by JANSSEN (WO 2017/050865; provided in IDS of 10/07/2022).
Regarding claims 1 and 18-20, JANSSEN teaches compound
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(Pg. 89 claim 15) wherein:
A is a heterocyclyl substituted with 3 R1 (i.e., one or more) wherein R1 is C3 alkyl and 2 instances of -ORA wherein RA is C1 alkyl;
B is a heteroaryl substituted with 1 R1 wherein R1 is C1 alkyl;
L1 and L2 are both absent;
W, X, and Z are each C(R3) wherein R3 is H;
Y is N wherein the dashed lines in the ring comprising Y are a single and double bond as shown;
R2 is absent; and
R7 is H.
Limitation (i) is met since B is not 1-methylpiperidinyl and limitation (ii) is met since no R1 is -NRBRC. Thus, JANSSEN teaches a compound of Formula (I) and Formulas (I-a), (I-b), and (I-c).
Regarding claim 42, JANSSEN teaches a pharmaceutical composition comprising the compound above (Pg. 89 claim 16) wherein the composition further comprises one or more pharmaceutically acceptable excipients (Pg. 59 lines 20-24).
Regarding claim 43, this claim contains a limitation drawn to properties of the claimed compound, i.e.: the compound has the ability to alter a target nucleic acid.
MPEP 2112.01.II states:
"Products of identical chemical composition cannot have mutually exclusive properties." In re Spada, 911 F.2d 705, 709, 15 USPQ2d 1655, 1658 (Fed. Cir. 1990). A chemical composition and its properties are inseparable. Therefore, if the prior art teaches the identical chemical structure, the properties applicant discloses and/or claims are necessarily present.
Since JANSSEN teaches the above compound of instant claim 1, from which claim 43 depends, it would be expected that the prior art compound would have the same physical/chemical properties as the compound of the instant application, namely that the compound exhibits the ability to alter a target nucleic acid.
Regarding claims 46 and 68, these claims contain limitations drawn to properties of the claimed compound, i.e.: the compound either increases or decreases, respectively, splicing at a splice site on a target nucleic acid between about 0.5-95%. See the MPEP 2112.01.II citation above. Thus, Since JANSSEN teaches a compound of instant claim 1, from which claims 46 and 68 depend, it would be expected that the prior art compound would have the same physical/chemical properties as the compound of the instant application, namely that the compound increases or decreases splicing at a splice site on a target nucleic acid between about 0.5-95%.
Furthermore, the present disclosure defines “about” to mean within the typical ranges of tolerances in the art, e.g., 2 standard deviations, ±10%, or ±5% (see Pg. 20 2nd to last paragraph of the specification). In view of this interpretation, claims 46 and 68 can be broadly understood to encompass compounds that increase or decrease, respectively, splicing by 0-100% (i.e., within ±5% of the recited percentages). Thus, even though JANSSEN is silent as to the above compound’s ability to effect nucleic acid splicing, the broadest reasonable interpretation of the instant claim does not require the compound to have a greater than 0% effect on nucleic acid splicing.
Examiner acknowledges the claims have been amended to use the phrasing “between about 0.5% to about 95%”. The addition of “between”, in view of the interpretation of “about”, does not limit the claim to only values between 0.5% and 95% since these end points are broadly defined by the use of “about”.
Conclusion
Claims 1, 18-20, 42-43, 46, and 68 are rejected.
See the previous action (09/04/2025) ¶20 regarding co-pending application no. 17/917,729 – the claims still do not qualify for double patenting for the same reasons as stated before.
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.
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/S.E.B./Examiner, Art Unit 1625
/JOHN S KENYON/Primary Patent Examiner, Art Unit 1625