DETAILED ACTION
Election/Restrictions
Applicant’s election without traverse of Group I, claims 11-18, and JM608 in the reply filed on March 17 2026 is acknowledged. Claims 11-28 are pending in the application. Claims 19-28 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention, there being no allowable generic or linking claim.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 March 17 2026. Accordingly, claims 11-18 are being examined on the merits herein.
In light of the discovered prior art, the species election has been expanded to include JM642, DAP as taught by Li et al. and N-[1-[(3-Chlorophenyl)amino]-3-isoquinolinyl]acetamide/R1 substituted versions as taught by Barbosa et al.
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 .
Priority
Receipt is acknowledged of certified copies of papers required by 37 CFR 1.55.
This application is a 371 of PCT/JP2021/018079 (05/12/2021) which claims priority to FOR JAPAN 2020-088499 (05/20/2020).
Information Disclosure Statement
The information disclosure statements (IDS) submitted on November 14 2022, December 14 2022, October 20 2023, May 6 2025, August 13 2025 and October 15 2025 are in compliance with the provisions of 37 CFR 1.97. Accordingly, the information disclosure statement is being considered by the examiner.
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 12 and 14-15 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.
Claim 12 as currently written is vague and indefinite. The claim recites that compound A is capable of binding to a CUG repeat sequence. However, claim 12 depends from claim 11 which recites compound A has a binding response to (CUG)9 which is a specific CUG repeat sequence. Therefore, the scope of compound A as recited in claim 12 is unclear because it isn’t clear if mere binding to any CUG is sufficient or if the binding must be to a (CUG)9 as recited in claim 11.
Claim 12 as currently written is vague and indefinite. Claim 12 depends from claim 11. Claim 11 requires compound A to have a binding response of 10 RU to a (CUG)9 RNA. Claim 12 is a dependent claim which recites compound A is capable of binding a CUG repeat sequence. In order for the claim to be a proper dependent claim it has to further limit the claim from which it depends. It is not clear how a compound which doesn’t bind to a CUG, which is what claim 11 would have to encompass for claim 12 to be a proper dependent claim, and still have a binding response of 10 RU.
The scope of claim 14 as currently written is vague and indefinite. The claim as written recites a structure of formula (1):
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which as indicated by the arrows include what is conventionally represented by a CH3. However, if both ends are CH3 then neither of the specifically taught compounds, specifically JM608 and JM642 would fall within the scope as both of these compounds have additional functional groups other than methyl as shown below:
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Therefore, the scope of the claimed compounds are unclear.
Claim 15 as currently written is vague indefinite. The claim recites a structural unit represented by formula (1A):
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Firstly, for the same reason claim 14 is indefinite, the claim recites a structure which terminates in CH3 groups. This would not allow for the specifically taught compounds JM608 and JM642 as they include additional functional groups. Secondly, the structure is indefinite as it is not clear what the parentheses are being utilized to signify. Typically the parentheses would indicate a repeat group, specifically the “or more structural unit” but as shown in JM642 above, there are a significant number of atoms between the two “structural units”. This creates uncertainty as to the scope of compounds falling within the scope of the claim.
Claim Rejections - 35 USC § 112-Written Description
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.
Claims 11-14 are rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, as failing to comply with the written description requirement. The claim(s) contains subject matter which was not described in the specification in such a way as to reasonably convey to one skilled in the relevant art that the inventor or a joint inventor, or for pre-AIA the inventor(s), at the time the application was filed, had possession of the claimed invention.
The specification discloses chemicals, such as JM608 and JM642 which meet the written description and enablement provisions of 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph. However, claim 11 is directed to any compound A which has a binding response of 10 resonance units (RU) or more at 25 nm to a (CUG)9 RNA immobilized at 401 RU as determined by surface plasmon resonance which only correspond in some undefined way to specifically instantly disclosed chemicals. While claims 12-14 limit the structure of compound A slightly, the claim is still broad with respect to the rest of the molecule. Furthermore, as identified in the 112(b) rejection above, the scope of the claimed compounds None of these compound A meet the written description provision of 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, due to lacking chemical structural information for what they are and chemical structures are highly variant and encompass a myriad of possibilities. The specification provides insufficient written description to support the genus encompassed by the claim. Note: MPEP 2163.
Vas-Cath Inc. v. Mahurkar, 19 USPQ2d 1111, (Fed. Cir. 1991), makes clear that "applicant must convey with reasonable clarity to those skilled in the art that, as of the filing date sought, he or she was in possession of the invention. The invention is, for purposes of the 'written description' inquiry, whatever is now claimed." (See page 1117.) The specification does not "clearly allow persons of ordinary skill in the art to recognize that [he or she] invented what is claimed." (See Vas-Cath at page 1116.)
Univ. of Rochester v. G.D. Searle, 69 USPQ2d 1886, 1892 (CAFC 2004), further supports this by stating that:
The appearance of mere indistinct words in a specification or a claim, even an original claim, does not necessarily satisfy that requirement. A description of an anti-inflammatory steroid, i.e., a steroid (a generic structural term) described even in terms of its functioning of lessening inflammation of tissues fails to distinguish any steroid from others having the same activity or function. A description of what a material does, rather than of what it is, usually does not suffice…. The disclosure must allow one skilled in the art to visualize or recognize the identity of the subject matter purportedly described. (Emphasis added).
With the exception of the above specifically disclosed chemical structures, the skilled artisan cannot envision the detailed chemical structure of the encompassed compound A, regardless of the complexity or simplicity of the method of isolation. Adequate written description requires more than a mere statement that it is part of the invention and reference to a potential method for isolating it. The chemical structure itself is required. See Fiers v. Revel, 25 USPQ2d 1601, 1606 (Fed. Circ. 1993) and Amgen Inc. V. Chugai Pharmaceutical Co. Ltd., 18 USPQ2d 1016, (Fed. Cir. 1991). In Fiddes v. Baird, 30 USPQ2d 1481, 1483, (Bd. Pat. App. & Int. 1993), claims directed to mammalian FGF's were found unpatentable due to lack of written description for the broad class. The specification provided only the bovine sequence. Finally, University of California v. Eli Lilly and Co., 43 USPQ2d 1398, 1404, 1405 (Fed. Cir. 1997) held that:
...To fulfill the written description requirement, a patent specification must describe an invention and do so in sufficient detail that one skilled in the art can clearly conclude that "the inventor invented the claimed invention." Lockwood v. American Airlines, Inc., 107 F.3d 1565, 1572, 41 USPQ2d 1961, 1966 (Fed. Cir. 1997); In re Gosteli, 872 F.2d 1008, 1012, 10 USPQ2d 1614, 1618 (Fed. Cir. 1989) (" [T]he description must clearly allow persons of ordinary skill in the art to recognize that [the inventor] invented what is claimed."). Thus, an applicant complies with the written description requirement "by describing the invention, with all its claimed limitations, not that which makes it obvious," and by using "such descriptive means as words, structures, figures, diagrams, formulas, etc., that set forth the claimed invention." Lockwood, 107 F.3d at 1572, 41 USPQ2d at 1966.
Furthermore, to the extent that a functional description can meet the requirement for an adequate written description, it can do so only in accordance with PTO guidelines stating that the requirement can be met by disclosing “sufficiently detailed, relevant identifying characteristics,” including “functional characteristics when coupled with a known or disclosed correlation between function and structure.” Univ. of Rochester v. G.D. Searle, 68 USPQ2d 1424, 1432 (DC WNY 2003).
The instant specification, see for example Fig. 1-2, which show two specific compounds which possess the claimed properties. However, the claims are directed to any compound which possess the claimed properties. These two compounds are not representative of the species of compounds which are capable of binding to a CUG repeat sequence and possess a binding response of 10 RU as required by the claims.
Furthermore, as evidenced by, Li et al. (Chem. Asian, 2016) which is directed to Naphthyridine-Benzoazaquinolone: evaluation of a tricyclic system for the binding to (CAG)n repeat DNA and RNA. Taught is specific compounds NA (napththyridine-azaquinolone):
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(Fig. 1) which as shown in the box encompasses a structure of formula (1) in claim 14. However, as shown in Figure S1 (see supplement information attached to the reference) does not show a SPR (surface plasmon resonance) response to (CUG)9. Compound NBzA (Fig. 2) also does not show a SPR response to (CUG)9 see Figure S2. It is taught that NA- and NbzA- binding to r(CUG)9 was not significant up to 3 µM ligand concentration (page 1974, left column). This shows that merely having a structural unit of formula (1) does not necessarily result in binding to (CUG)9.
Therefore, only the above chemically structurally defined chemicals, but not the full breadth of the claim(s) meet the written description provision of 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph. The species specifically disclosed are not representative of the genus because the genus is highly variant. Applicant is reminded that Vas-Cath makes clear that the written description provision of 35 USC § 112 is severable from its enablement provision. (See page 1115.)
Claim Rejections - 35 USC § 102
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) 11-18 is/are rejected under 35 U.S.C. 102(a)(2) as being anticipated by Matsumoto et al. (Chem. Eur. J., 2020, cited on PTO Form 1449, also attached to this OA to make supplemental information of record).
The applied reference has a common inventor/author with the instant application. Based upon the earlier effectively filed date of the reference, it constitutes prior art under 35 U.S.C. 102(a)(2). This rejection under 35 U.S.C. 102(a)(2) might be overcome by: (1) a showing under 37 CFR 1.130(a) that the subject matter disclosed in the reference was obtained directly or indirectly from the inventor or a joint inventor of this application and is thus not prior art in accordance with 35 U.S.C. 102(b)(2)(A); (2) a showing under 37 CFR 1.130(b) of a prior public disclosure under 35 U.S.C. 102(b)(2)(B) if the same invention is not being claimed; or (3) a statement pursuant to 35 U.S.C. 102(b)(2)(C) establishing that, not later than the effective filing date of the claimed invention, the subject matter disclosed in the reference and the claimed invention were either owned by the same person or subject to an obligation of assignment to the same person or subject to a joint research agreement.
Applicant cannot rely upon the certified copy of the foreign priority application to overcome this rejection because a translation of said application has not been made of record in accordance with 37 CFR 1.55. When an English language translation of a non-English language foreign application is required, the translation must be that of the certified copy (of the foreign application as filed) submitted together with a statement that the translation of the certified copy is accurate. See MPEP §§ 215 and 216.
The instant application claims a compound A having a binding response of 10 resonance units (RU) or more at 25 nM to a (CUG)9 RNA immobilized at 401 RU as determined by surface plasmon resonance. As elected the compound is JM608. In light of the elected species the recitation “having” is interpreted as open claim language allowing for additional functionality to be present. Note: MPEP 2111.03 IV.
Matsumoto et al. is directed to the dimeric form of 1,3-diaminoisoquinoline derivative rescued the mis-splicing of Atp2a1 and Clcn1 genes in myotonic dystrophy type 1 mouse model. Shown in Figure 1 are compound JM608 and JM642 which are the same as those taught in the instant specification (see page 18 of the instant specification).
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. Dissolution of the ligand in a HBS-EP buffer at different concentrations is taught (supplement content page 9).
Regarding claims 11-16, JM608 taught by Matsumoto et al. is the elected species.
Regarding claims 17-18, Matsumoto et al. teaches the dissolution in HBS-EP buffer which reads on a composition comprising the compound and a pharmaceutically acceptable additive.
Claim(s) 11-13 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Li et al. (Chemistry-A European Journal, 2016, cited on PTO Form 1449).
Li et al. is directed to a ligand that targets CUG trinucleotide repeats. One compound taught is DAP (fig. 1)
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which is shown as binding, via hydrogen bonding, to U (figure 2) reading on claim 13. As shown in figure 3 at a concentration of 0.25 µM ligand was 10 RU in SPR (surface plasmon resonance) reading on claims 11-12.
Claim(s) 15 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Barbosa et al. (WO2008089307, cited in the Office Action mailed on 12/17/25).
The instant application claims a compound having one or more structural units represented by formula 1A wherein R1 represents a substituent. In light of the indefiniteness above, the claim scope is interpreted as including any additional functional groups outside of formula IA.
Barbosa et al. is directed to methods and compositions for the treatment of pain, inflammation and cancer.
A specific compound taught is shown below (claim 116; page 85 line 24).
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CAS Name: N-[1-[(3-Chlorophenyl)amino]-3-isoquinolinyl]acetamide
This compound contains a structural unit that encompasses 1A wherein the substituent is H.
Claim Rejections - 35 USC § 103
The following is a quotation of 35 U.S.C. 103 which forms the basis for all obviousness rejections set forth in this Office action:
A patent for a claimed invention may not be obtained, notwithstanding that the claimed invention is not identically disclosed as set forth in section 102 of this title, if the differences between the claimed invention and the prior art are such that the claimed invention as a whole would have been obvious before the effective filing date of the claimed invention to a person having ordinary skill in the art to which the claimed invention pertains. Patentability shall not be negated by the manner in which the invention was made.
The factual inquiries set forth in Graham v. John Deere Co., 383 U.S. 1, 148 USPQ 459 (1966), that are applied for establishing a background for determining obviousness under 35 U.S.C. 103 are summarized as follows:
1. Determining the scope and contents of the prior art.
2. Ascertaining the differences between the prior art and the claims at issue.
3. Resolving the level of ordinary skill in the pertinent art.
4. Considering objective evidence present in the application indicating obviousness or nonobviousness.
This application currently names joint inventors. In considering patentability of the claims the examiner presumes that the subject matter of the various claims was commonly owned as of the effective filing date of the claimed invention(s) absent any evidence to the contrary. Applicant is advised of the obligation under 37 CFR 1.56 to point out the inventor and effective filing dates of each claim that was not commonly owned as of the effective filing date of the later invention in order for the examiner to consider the applicability of 35 U.S.C. 102(b)(2)(C) for any potential 35 U.S.C. 102(a)(2) prior art against the later invention.
Claim 16-18 are rejected under 35 U.S.C. 103 as being unpatentable over Barbosa et al. as applied to claim 15 above.
Applicant Claims
The instant application claims the R1 is an aromatic ring group.
The instant application claims a composition comprising the compound and a pharmaceutically acceptable additive.
Determination of the Scope and Content of the Prior Art
(MPEP §2141.01)
The teachings of Barbosa et al. are set forth above. Barbosa et al. teaches
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suggesting a substituent on the N-[1-[(3-Chlorophenyl)amino]-3-isoquinolinyl]acetamide. R1 can be an optionally substituted aryl, alkylaryl, arylalkyl, heterocycle, alkyl heterocycle or heterocycle alkyl (claim 106; page 76). Pharmaceutical composition comprising the inhibitor and a pharmaceutically acceptable excipient or diluent is claimed (claim 117).
Ascertainment of the Difference Between Scope the Prior Art and the Claims
(MPEP §2141.02)
While Barbosa et al. teaches R1 can be a variety of different aryl (aka aromatic) substituents, Barbosa et al. does not exemplify an isoquinolinyl aceteamide with a R1 substituent other than H. While Barbosa et al. suggests compositions with an excipient or diluent, Barbosa et al. does not exemplify the isoquinolinyl acetamide with an excipient or diluent.
Finding of Prima Facie Obviousness Rationale and Motivation
(MPEP §2142-2143)
It would have been obvious to one of ordinary skill in the art before the effective filing date of the claimed invention to replace the R1 H of N-[1-[(3-Chlorophenyl)amino]-3-isoquinolinyl]acetamide with an aryl or heteroaryl. It would have been obvious to one of ordinary skill in the art to try any of the specifically taught R1 substituents as a person with ordinary skill has good reason to pursue known options within his or her technical grasp. Note: MPEP 2141 KSR International CO. v. Teleflex Inc. 82 USPQ 2d 1385 (Supreme Court 2007).
It would have been obvious to one of ordinary skill in the art before the effective filing date of the claimed invention to utilize an excipient or diluent with the N-[1-[(3-Chlorophenyl)amino]-3-isoquinolinyl]acetamide or the aryl/heteroaryl substituted acetamide to form a pharmaceutical composition. One skilled in the art would have been motivated to utilize a pharmaceutical composition with an excipient or diluent as Barbosa et al. expressly teaches combining the compounds of the invention with an excipient or diluent. Therefore, all of the claimed elements were known in the prior art and one skilled in the art could have combined the elements as claimed by known methods with no change in their respective functions and the combination would have yielded predictable results to one of ordinary skill in the art at the time of the invention. Note: MPEP 2143 KSR International Co. v. Teleflex Inc., 550 US 398, 82 USPQ 2d 1385 (2007). Either excipient or diluent is an additive as claimed.
Conclusion
Any inquiry concerning this communication or earlier communications from the examiner should be directed to ABIGAIL VANHORN whose telephone number is (571)270-3502. The examiner can normally be reached M-Th 6 am-4 pm EST.
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/ABIGAIL VANHORN/Primary Examiner, Art Unit 1636
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