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 .
Election/Restrictions
Applicant’s election without traverse of
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in the reply filed on 9/11/2025 is acknowledged.
A search for the elected species did not find prior art.
Claims 1-3, 13, 18-24, 26, 35, 40-41, 43-44, 53, 58-60, 70 and 75 read on the elected species.
Claims 5, 27, 45, and 62 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected species, there being no allowable generic or linking claim.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 9/11/2025.
Priority
This application is a national stage entry of PCT/US2021/013517 and also claims foreign priority to US 62/961191.
The instant claims find support from US 62/961191. Therefore, the effective filing date is 1/14/2020.
Information Disclosure Statement
The information disclosure statements (IDS), submitted on 9/9/2025, 10/07/2022, and 07/13/2022, are in compliance with the provisions of 37 CFR 1.97. Accordingly, the information disclosure statement is being considered by the examiner.
Claim Objections
Claims 13, 35, 53 and 70 are objected to because of the following informalities: the structures of these claims have poor resolution. Appropriate correction is required.
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 –
z
(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) 1-3, 13, 18-24, 26, 35, 40-41, 43-44, 53, 58-60, and 70 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by TAZI (WO 2009/087238).
TAZI anticipates pharmaceutically acceptable compositions (page 1 lines 5-10) containing compound with CAS #56149-30-5 and structure of
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(MB260 on page 71), which is the second compound of claim 13. TAZI anticipates using this compound in treating a disease in a subject such as Duchenne muscular dystrophy DMD) (reference claim 34 on page 146).
There is no requirement that a person of ordinary skill in the art would have recognized the inherent disclosure at the time of invention, but only that the subject matter is in fact inherent in the prior art reference. Chemical properties are inherent to their compounds. A chemical composition containing compound CAS#56149-30-5 and its properties, increasing expression of utrophin, are inseparable. See MPEP 2112.01 (II).
Applicants are reminded that the office does not have the facilities and resources to provide the factual evidence needed in order to establish that the product of the prior art does not possess the same material, structural and functional characteristics of the claimed product. In the absence of evidence to the contrary, the burden is on the applicant to prove that the claimed product is different from those taught by the prior art and to establish patentable differences. See In re Best 562F.2d 1252, 195 USPQ 430 (CCPA 1977) and Ex parte Gray 10 USPQ 2d 1922 (PTO Bd. Pat. App. & Int. 1989).
This anticipates claims 1-3 and 13.
TAZI anticipates treating Duchenne muscular dystrophy (DMD) (ref claim 34). TAZI discloses that DMD is a serious illness resulting from mutations in the dystrophin gene (page 2 lines 28-35). This anticipates claims 58-60 and 70.
TAZI anticipates that Duchenne muscular dystrophy results from mutations in the dystrophin gene, leading to the absence of its expression or to the expression of truncated proteins (page 113 lines 10-15). TAZI tests an animal model of Duchenne muscular dystrophy (page 113 lines 25-34) by administering the inventive compounds (page 113 lines 25-30) including the elected species of MB260 on page 116, which is the second compound of claim 13. Examiner understands this mouse model as a subject with loss (page 113 line 30) of dystrophin protein expression of claim 19. This anticipates claims 18-19, 35, and 40-41. This also anticipates a method for treating muscle wasting caused by a lack or reduced expression of dystrophin protein of claims 43-44, and 53.
There is no requirement that a person of ordinary skill in the art would have recognized the inherent disclosure at the time of invention, but only that the subject matter is in fact inherent in the prior art reference. Chemical properties are inherent to their compounds. A chemical composition containing MB260 and its properties, improving muscle function/performance and muscle structure, are inseparable. See MPEP 2112.01 (II). Additionally, MB260 increased the percentage of luciferase, showing it as a potent therapeutic agent for DMD treatment. This anticipates claims 20-24 and 26.
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, 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 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.
Claim 75 is rejected under 35 U.S.C. 103 as being unpatentable over MOORWOOD (Moorwood et al., “A cell-based High-throughput screening assay for posttranscriptional utrophin upregulation”, J Biomol Screen., April 2013), cited by applicants, and as evidenced by PROMEGA (“Technical Manual for Bright-Glo Luciferase Assay System”, Promega, Revised 3/24).
MOORWOOD teaches a method of high-throughput screening to identify post-transcriptional utrophin upregulator compounds of claim 75 (Abstract, “This assay can be used to study utrophin regulatory mechanisms or to screen chemical libraries for compounds that upregulate utrophin posttranscriptionally via its UTRs). However, MOORWOOD does not test a whole library of compounds.
MOORWOOD teaches stable report cell line with CMV-promoted luciferase flanked by human UTRs (C2C12 cell line expressing luciferase coding sequence flanked by human utrophin 5’ and 3’ UTRs) (Materials and Methods, also see Abstract) of step a.
MOORWOOD teaches administering a plurality of compounds in multi-wells (“Compounds identified via this assay, used alone or in a synergistic combination with utrophin promoter-targeting molecules, would be predicted to have therapeutic potential for DMD” in Abstract and Assay Validation) of step ai.
MOORWOOD teaches “Luciferase assays were done using either the BrightGlo Assay (Promega) for DMSO testing in 96-well plate format or the Dual Luciferase Assay (Promega) for 24-well format experiments, following the manufacturer’s instructions” (Luciferase Assay).
MOORWOOD teaches the administering a positive control compound (“Renilla luciferase endogenous control”) to the cell line in a second plurality of microplates and administering a negative control compound (“firefly luciferase luminescence”) to the cell in a third plurality of microplates of step aii (Luciferase Assays). MOORWOOD teaches incubating these cell lines of step aiii (Cell Culture).
MOORWOOD teaches measuring luminescence to assay for luciferase expression (Luciferase Assays). MOORWOOD uses PROMEGA’s instructions. PROMEGA is relied upon for the beneficial teaching that “Because the luminescent signal is affected by assay conditions, results should be compared only between samples measured using the same medium/serum combination… By this method, luminescence measurements of each plate can be normalized to the control contained within the same plate” (page 3). MOORWOOD as evidenced by PROMEGA teaches step a iv.
MOORWOOD states that “ Our assay uses the cytomegalovirus (CMV) promoter to produce a reporter mRNA that consists of the coding sequence of luciferase flanked by the utrophin UTRs” (page 401). MOORWOOD continues and teaches counter-screening of step b (“Any tested substance that alters mRNA stability, rate of translation, or targeting by miRNAs would result in a change in luciferase activity. We have validated the assay based on the known regulation of utrophin by miR-206”) (page 401).
MOORWOOD teaches that a limitation of the assay is that compounds affecting cell growth and compounds that activate the CMV promotor may produce a change in luciferase readout (page 404). MOORWOOD further teaches that it would be possible to counter screen using a cell line stably transfected with the parent construct pGL4:50, which does not contain the utrophin UTRs (page 404).
While MOORWOOD teaches step a, it does not explicitly do step b (counter screening).
The artisan would, however, find it obvious to add the counter screening step as MOORWOOD suggests a way to counter screen using C2C12 cell line stably transfected with the parent construct pGL4:50, which does not contain the utrophin UTRs (page 404). The artisan would be motivated to do the same steps i-iv (in step a) at the same time or to replicate steps i-iv (in step a) in order to follow the same lab procedure.
Conclusion
No claims are allowed.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to GILLIAN A HUTTER whose telephone number is (571)272-6323. The examiner can normally be reached M-F 7:30-5.
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/G.A.H./Examiner, Art Unit 1625 /Andrew D Kosar/Supervisory Patent Examiner, Art Unit 1625