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 .
Applicant’s election without traverse of group II and the species ALS and oligonucleotide in the reply filed on 3/20/26 is acknowledged.
Claims 1-4 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention, there being no allowable generic or linking claim.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 3/20/26.
Drawings
The drawings filed on 5/9/23 are objected to because they are not fully legible.
Claim Objections
Claims 5-14 are objected to because of the following informalities: Claim 5 recites “CHMP7” which is an abbreviation. The first recitation of an abbreviation is required to set forth what the abbreviation stands for. Recitation of “Charged Multivesicular Body Protein 7 (CHMP7)” would obviate this objection. Appropriate correction is required.
Improper Markush Rejection
Claims 6, 7, 11, and 12 are rejected on the judicially-created basis that it contains an improper Markush grouping of alternatives. See In re Harnisch, 631 F.2d 716, 721-22 (CCPA 1980) and Ex parte Hozumi, 3 USPQ2d 1059, 1060 (Bd. Pat. App. & Int. 1984). The improper Markush grouping includes species of the claimed invention that do not share both a substantial structural feature and a common use that flows from the substantial structural feature. The members of the improper Markush grouping do not share a substantial feature and/or a common use that flows from the substantial structural feature for the following reasons: The claims are not limited to any specific structure of nucleic acid molecule and encompass a genus of compounds that have different structures and act via different mechanisms. The claims are directed to delivery of small molecules, antibodies, oligonucleotides, antisense oligonucleotides, siRNAs, miRNAs, peptides, or peptidomimetics. Although each are nucleic acid inhibitory therapeutics, each of the agents have a different structure and act via different mechanisms. For example, small molecule drugs are low molecular weight organic compounds designed to interact with specific biological targets to modulate a biological process; antibodies function by recognizing and binding to specific antigens, triggering various immune responses to neutralize or eliminate pathogens; antisense oligonucleotides (ASOs) modulate gene expression by binding to specific RNA sequences, leading to mRNA degradation, translation inhibition, or splicing modulation; siRNAs are incorporated into RISC and act via RNAi; and peptidomimetics can target protein-protein interactions and serve as agonists or antagonists in various biological pathways.
In response to this rejection, Applicant should either amend the claim(s) to recite only individual species or grouping of species that share a substantial structural feature as well as a common use that flows from the substantial structural feature, or present a sufficient showing that the species recited in the alternative of the claims(s) in fact share a substantial structural feature as well as a common use that flows from the substantial structural feature. This is a rejection on the merits and may be appealed to the Board of Patent Appeals and Interferences in accordance with 35 U.S.C. 134 and 37 CFR 41.31(a)(1).
When the Markush grouping is for alternatives of chemical compounds, they shall be regarded as being of a similar nature where the following criteria are fulfilled:
(A) All alternatives have a common property or activity; and
(B) (1) A common structure is present, i.e., a significant structural element is shared by all of the alternatives; or
(B) (2) In cases where the common structure cannot be the unifying criteria, all alternatives belong to a recognized class of chemical compounds in the art to which the invention pertains.
In paragraph (B)(1), above, the words “significant structural element is shared by all of the alternatives” refer to cases where the compounds share a common chemical structure which occupies a large portion of their structures, or in case the compounds have in common only a small portion of their structures, the commonly shared structure constitutes a structurally distinctive portion in view of existing prior art, and the common structure is essential to the common property or activity. The structural element may be a single component or a combination of individual components linked together.
In paragraph (B)(2), above, the words “recognized class of chemical compounds” mean that there is an expectation from the knowledge in the art that members of the class will behave in the same way in the context of the claimed invention. In other words, each member could be substituted one for the other, with the expectation that the same intended result would be achieved.
In order for the members of the Markush group to belong to “recognized class of chemical compounds” there must be an expectation that the members of the class will behave in the same way in the context of the claimed invention. In other words, each member of the class could be substituted one for the other with the expectation that the same intended result would be achieved. In the instant case, activity of any specific type of compound is dependent upon the specific structure and mechanism. There is no expectation that any one of the types of inhibitors as claimed can be substituted for any of the other with a completely different structure and mechanisms with the expectation of the same activity.
As set forth in MPEP2117, “Note that where a Markush group includes only materials from a recognized scientific class of equivalent materials or from an art-recognized class, "the mere existence of such a group in an application tend[s] to prove the equivalence of its members and when one of them [is] anticipated the group [is] therefore rendered unpatentable, in the absence of some convincing evidence of some degree of non-equivalency of one or more of the remaining members." In re Ruff, 256 F.2d 590, 598-99, 118 USPQ 340, 348 (CCPA 1958)("[A]ctual equivalence is not enough to justify refusal of a patent on one member of a group when another member is in the prior art. The equivalence must be disclosed in the prior art or be obvious within the terms of Section 103." Id. at 599, 118 USPQ at 348).”
In the instant case, art against any one type of agent would not be evidence against any of the remaining members that have completely different structures and do not have identical activity.
Claim Rejections - 35 USC § 101
35 U.S.C. 101 reads as follows:
Whoever invents or discovers any new and useful process, machine, manufacture, or composition of matter, or any new and useful improvement thereof, may obtain a patent therefor, subject to the conditions and requirements of this title.
Claims 5, 6, 8-11, 13, and 14 are rejected under 35 U.S.C. 101 because the claimed invention is directed to a judicial exception without significantly more. The claim(s) recite(s) delivery of naturally occurring agents, miRNAs. This judicial exception is not integrated into a practical application because the treatment is recited at a high level of generality. The claim(s) does/do not include additional elements that are sufficient to amount to significantly more than the judicial exception because the claims are not directed to any specific treatment.
The claims recite a judicial exception, delivery of a naturally occurring product. Therefore, the claims are directed to a judicial exception.
Analysis, step 1. Instant claims are directed to a statutory category, i.e., a process.
Analysis, step 2A, the claim is then analyzed to determine whether it is directed to any judicial exception. Claims 5 and 8 are directed to delivery of any agent that inhibits CHMP7, which includes naturally occurring products such as miRNAs (as recited in claims 6 and 11). Thus, the claim is directed to at least one exception.
For example, Dexheimer et al. (Frontiers in Cell and Developmental Biology, 2020, 8, 409, 1-18) teach that miRNAs are short regulatory RNAs that are naturally occurring and have diverse biological roles.
Analysis, step 2B. Next, the claim as a whole is analyzed to determine whether any element, or combination of elements, is sufficient to ensure that the claim amounts to significantly more than the natural law. In instant case, the method steps of delivering the agent via any means consist of well understood, routine, conventional activity already engaged in by the clinical community. The treatment step is recited with such a high level of generality that it encompasses the administration of any known CHMP7 inhibiting agent at the time of the invention. Thus, the claim does not amount to significantly more than the natural law itself.
Therefore, the treatment step amounts only to a generic instruction to apply the exception. Thus, the claims as a whole do not integrate the recited judicial exception into practical application.
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.
Claims 5-14 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 applications subject to pre-AIA 35 U.S.C. 112, the inventor(s), at the time the application was filed, had possession of the claimed invention.
Instant claims 5 and 8 are directed to delivery of any “CHMP7 inhibiting agent”, which is a genus of agents that have not been adequately described in the specification. The specification does not adequately describe the structure required for the function. The agent is not required to have any specific structural relationship with any specific CHMP7 sequence.
With regards to the agents of claims 6, 7, 11, and 12, the specification does not adequately describe the structure requreid for each of the genuses of agents to function as required. The agents are not required to have any specific structural relationship with any specific CHMP7 sequence. The agents in fact can be specific for any other target that has the secondary effect of inhibiting CHMP7, a genus that has not been adequately described in the specification.
The species of ASOs and siRNAs that are fully complementary to a specific portion of a specific CHMP7 target sequence of the specification are not representative of the entire claimed genus of CHMP7 inhibiting agents.
Without further description of the structure required for the function, one would not be able to readily envision which antibodies or antigen-binding fragments, oligonucleotides, antisense oligonucleotides, siRNAs, miRNAs, peptides, or peptidomimetics necessarily have the structure to inhibit any CHMP7.
The MPEP states that for a generic claim, the genus can be adequately described if the disclosure presents a sufficient number of representative species that encompass the genus. See MPEP § 2163. If the genus has a substantial variance, the disclosure must describe a sufficient variety of species to reflect the variation within that genus. See MPEP § 2163. Although the MPEP does not define what constitute a sufficient number of representative species, the courts have indicated what do not constitute a representative number of species to adequately describe a broad genus. In Gostelli, the courts determined that the disclosure of two chemical compounds within a subgenus did not describe that subgenus. In re Gostelli, 872, F.2d at 1012, 10 USPQ2d at 1618. Additionally, in Carnegie Mellon University v. Hoffman-La Roche Inc., Nos. 07-1266, -1267 (Fed. Cir. Sept. 8, 2008), the Federal Circuit affirmed that a claim to a genus described in functional terms was not supported by the specification’s disclosure of species that were not representative of the entire genus. Furthermore, for a broad generic claim, the specification must provide adequate written description to identify the genus of the claim. In Regents of the University of California v. Eli Lilly & Co. the court stated:
"A written description of an invention involving a chemical genus, like a description of a chemical species, 'requires a precise definition, such as by structure, formula, [or] chemical name,' of the claimed subject matter sufficient to distinguish it from other materials." Fiers, 984 F.2d at 1171, 25 USPQ2d 1601; In re Smythe, 480 F.2d 1376, 1383, 178 USPQ 279, 284985 (CCPA 1973) ("In other cases, particularly but not necessarily, chemical cases, where there is unpredictability in performance of certain species or subcombinations other than those specifically enumerated, one skilled in the art may be found not to have been placed in possession of a genus ...") Regents of the University of California v. Eli Lilly & Co., 43 USPQ2d 1398.
The Guidelines for Examination of Patent Applications under the 35 USC § 112, first paragraph, “Written Description” Requirement”, published at Federal Register, Vol. 66, No. 4, pp. 1099-1111 outline the method of analysis of claims to determine whether adequate written description is present. The first step is to determine what the claim as a whole covers, i.e., discussion of the full scope of the claim. Second, the application should be fully reviewed to understand how applicant provides support for the claimed invention including each element and/or step, i.e., compare the scope of the claim with the scope of the description. Third, determine whether the applicant was in possession of the claimed invention as a whole at the time of filing.
With regards to siRNAs, a single type of agent, Elbashir et al. (The EMBO Journal, Vol. 20, No. 23, pages 6877-6888, 2001) teaches that duplexes of 21-23 nt RNAs are the sequence specific mediators of RNAi and that even single mismatches between the siRNA duplex and the target mRNA abolish interference (abstract and page 6888).
With regards to antisense oligonucleotides, a single type of agent, Nedorezova et al. (Theranostics, 2022, Vol. 12, Issue 16, 7132-7157) teach that to ensure tight binding, a typical oligonucleotide gene therapy agent hybridizes to the stretch of 15-25 nucleotides of a unique targeted sequence. However, hybrids of such lengths tolerate one or more mismatches under physiological conditions, the problem known as the affinity/specificity dilemma (abstract). The instant antisense oligomers are not required to have any specific structural relationship with any specific target sequence.
Thus, having analyzed the claims with regard to the Written Description guidelines, it is clear that the specification does not disclose a representative number of species for CHMP7 inhibiting agents within the instant enormous genus that are inhibitory of the target as claimed. Thus, one skilled in the art would be led to conclude that Applicant was not in possession of the claimed invention at the time the application was filed.
Claims 8-14 are rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, because the specification, while being enabling for a method of inhibiting CHMP7 with specific siRNAs or antisense oligonucleotides of the specification, does not reasonably provide enablement for a method of treating any neurodegenerative disease via broad systemic delivery of any CHMP7 inhibiting agent. The specification does not enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make a the invention commensurate in scope with these claims.
Factors to be considered in a determination of lack of enablement include, but are not limited to:
(A) The breadth of the claims;
(B) The nature of the invention;
(C) The state of the prior art;
(D) The level of one of ordinary skill;
(E) The level of predictability in the art;
(F) The amount of direction provided by the inventor;
(G) The existence of working examples; and
(H) The quantity of experimentation needed to make or use the invention based on the content of the disclosure.
In re Wands, 858 F.2d 731, 737, 8 USPQ2d 1400, 1404 (Fed. Cir. 1988)
The claims are directed to a method for treating any possible neurodegenerative disease via delivery of any CHMP7 inhibiting agent via any mode of delivery in vivo.
The specification demonstrates in vitro transfection of iPSC derived neurons with antisense oligonucleotides targeting a specific region of a specific CHMP7 target sequence with a resultant inhibition of CHMP7, which is not enabling for in vivo broad systemic delivery (i.e. oral) of any agent that inhibits CHMP7 at any level (i.e. 10%) with a predictable outcome of treating any possible neurodegenerative disease, which encompasses an enormous possible genus of diseases that have not been shown to be reliant upon CHMP7 expression alone.
The specification does not draw an adequate nexus between delivery of any possible inhibitor of CHMP7 and the predictable outcome of treating any possible neurodegenerative disease.
The specification does not draw an adequate nexus between any level of inhibition of CHMP7 and the predictable outcome of treating any possible neurodegenerative disease.
Additionally, there is no guidance in the specification as filed that teaches how to deliver the instantly recited genus of inhibitors, each having different delivery challenges, and predictably treat any possible neurodegenerative disease in vivo.
With regards to siRNAs, a single species of the instant inhibitors, the instant siRNAs are not required to have any specific structural relationship with any specific target sequence. The siRNAs of the specification that resulted in the recited outcome in vitro are targeted to specific regions of a specific CHMP7 target sequence. However, the instant claims not only encompass targeting any target that has the secondary effect of inhibiting CHMP7, but also encompasses siRNAs targeted to any possible region of any CHMP7 sequence. It is known in the siRNA field that not any siRNA to any target sequence will result in inhibitory effect. Even from those that are inhibitory to CHMP7, not all of this pool would predictably result in treatment effects of any possible neurodegenerative disease in vivo.
Although applicant has demonstrated RNA interference in vitro via specific siRNAs that are targeted to specific regions of a specific CHMP7 target sequence, applicant is not enabled for mediating RNA interference in vivo by the broadly recited methods, as delivery and effective action therein is known in the art to be unpredictable with regards to dsRNA duplexes. Activity in vitro is not predictable of the in vivo therapeutic effect in the in vivo complex environment.
For example, Elbashir et al. (The EMBO Journal, Vol. 20, No. 23, pages 6877-6888, 2001) teaches that duplexes of 21-23 nt RNAs are the sequence specific mediators of RNAi and that even single mismatches between the siRNA duplex and the target mRNA abolish interference (abstract and page 6888).
Fujita et al. (Int. J. Mol. Sci. 2015, 16, 5254-5270) teach that two types of small RNA molecules, small interfering RNAs (siRNAs) and microRNAs (miRNAs), have a central function in RNAi technology. The success of RNAi-based therapeutic delivery may be dependent upon uncovering a delivery route, sophisticated delivery carriers, and nucleic acid modifications (page 5254). Fujita et al. teach that the success of an RNAi-based therapy in clinical trials rests on careful selection of target genes and miRNAs. Moreover, we suggest that a delivery route, sophisticated delivery carriers, chemical modification, and modified RNAi platforms are needed to enhance RNAi effects in cancer cells (pages 5262-5263).
Friedrich et al. (BioDrugs (2022) 36:549–571) teach that still, the development of siRNA therapeutics faces several challenges and issues, including the definition of optimal siRNAs in terms of target, sequence, and chemical modifications, siRNA delivery to its intended site of action, and the absence of unspecific off-target effects (Abstract). Friedrich et al. teach that the use of short siRNA is preferred because longer siRNAs can provoke an inflammatory antiviral immune response (page 551).
Friedrich et al. teach: As a basic parameter, the GC content of an siRNA is addressed by algorithms and its range should be between ~30 and 60%. Too low GC content can lead to weak or unspecific binding, whereas too high GC content may impede unwinding by helicase and incorporation in the RISC complex. Between nucleotides 9 and 14, however, low GC content is important to increase RISC function during mRNA cleavage. Sequences that could lead to secondary structures in the sense or antisense stand must be avoided (e.g., internal repeats, palindromes, CCC or GGG sequences). A proper duplex formation is essential for functional siRNA. Additionally, sequences that contain single nucleotide polymorphisms, miRNA seed matches, and known toxic motifs must be avoided (page 552).
Friedrich et al. teach: The 5′-untranslated region (and 3′-untranslated region) of mRNA as well as sequences close to the start codon are not recommended as siRNA targets, as the binding of regulatory proteins in this area may impede RISC binding and thus the silencing effect. Rather, selecting regions in the open reading frame about 50–100 nucleotides downstream of the start codon is recommended. Furthermore, siRNAs closer to the start codon seem to be more efficient than those further downstream (page 552). Friedrich et al. is evidence as to the delivery challenges, as well as the fact that not any siRNA inhibitor of a target would result in the desired therapeutic effect.
As outlined above, it is well known that there is a high level of unpredictability in the RNAi art (a single species of the instant inhibitors) for therapeutic in vivo applications and design. The scope of the claims in view of the specification as filed together do not reconcile the unpredictability in the art to enable one of skill in the art to make and/or use the claimed invention, namely a broad method of treating any possible neurodegenerative disease via broad systemic delivery of a broad genus of agents that act via different mechanisms and have different levels of activity encompassing in vivo effects.
MPEP 2164.01
Any analysis of whether a particular claim is supported by the disclosure in an application requires a determination of whether that disclosure, when filed, contained sufficient information regarding the subject matter of the claims as to enable one skilled in the pertinent art to make and use the claimed invention.
Also, MPEP 2164.01(a)
A conclusion of lack of enablement means that, based on the evidence regarding each of the above factors, the specification, at the time the application was filed, would not have taught one skilled in the art how to make and/or use the full scope of the claimed invention without undue experimentation. In re Wright, 999 F.2d 1557,1562, 27 USPQ2d 1510, 1513 (Fed. Cir. 1993).
Given the teachings of the specification as discussed above, one skilled in the art could not predict a priori whether introduction of any CHMP7 inhibitor of the instant genus in vivo by the broadly disclosed methodologies of the instantly claimed invention, would result in successful treatment of any possible neurodegenerative disease. To practice the claimed invention, one of skill in the art would have to de novo determine; the stability of the molecule in vivo, delivery of the molecule to the whole organism, specificity to the target tissue in vivo, dosage and toxicity in vivo, and entry of the molecule into the cell in vivo and the effective action therein. Without further guidance, one of skill in the art would have to practice a substantial amount of trial and error experimentation, an amount considered undue and not routine, to practice the instantly claimed invention.
A conclusion of lack of enablement means that, based on the evidence regarding each of the above factors, the specification, at the time the application was filed, would not have taught one skilled in the art how to make and/or use the full scope of the claimed invention without undue experimentation (see MPEP 2164.01(a)).
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)(2) the claimed invention was described in a patent issued under section 151, or in an application for patent published or deemed published under section 122(b), in which the patent or application, as the case may be, names another inventor and was effectively filed before the effective filing date of the claimed invention.
Claim(s) 5-7 is/are rejected under 35 U.S.C. 102(a)(2) as being anticipated by Bui (WO 2022/072787 A1) (supported in 63/086,576 10/1/20).
Bui teach a method of inhibiting ChmP7 in a cell or a subject comprising delivery of a Chmp7 antisense oligonucleotide (abstract and claims) (instant claims 1-3). Delivery to a human meets the instant limitation of inhibiting CHMP7 in a population of cells.
Therefore, the claims are anticipated by Bui.
Claim Rejections - 35 USC § 103
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 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.
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(s) 8-14 is/are rejected under 35 U.S.C. 103 as being unpatentable over Bui (WO 2022/072787 A1) (supported in 63/086,576 10/1/20) as applied to claims 5-7 above, and further in view of Malik et al. (Scientific Reports, 2019, 9, 3539, 1-16) and Rusten et al. (Cell Cycle 7:9, 1166-1172; 1 May 2008).
Bui teach a method of inhibiting ChmP7 in a cell or a subject comprising delivery of a Chmp7 antisense oligonucleotide (abstract and claims) (instant claims 1-3). Delivery to a human meets the instant limitation of inhibiting CHMP7 in a population of cells.
Bui teaches: Provided are compounds and pharmaceutical compositions for reducing the amount or activity of Charged Multivesicular Body Protein 7 (CHMP7) RNA in a cell or subject, and in certain instances reducing the amount of CHMP7 protein in a cell or subject. Such compounds and pharmaceutical compositions are useful to ameliorate diseases or conditions associated with aberrant activation of Endosomal Sorting Complexes Required for Transport-Ill proteins (abstract).
Bui teach dose dependent inhibition of human Chmp7 with modified antisense oligonucleotides (example 2). The doses of example 2 fall into the instantly recited dose range of instant claim 9 (i.e. 62.5 nM is approximately 58.7 mg/kg).
Bui do not teach treatment of a neurodegenerative disease.
However, it would have been obvious to treat a neurodegenerative disease with the antisense oligonucleotide composition of Bui because Malik et al. offer motivation to target Chmp7 for the treatment of SBMA.
Malik et al. teach that Chmp7 is a potential therapeutic target in spinal and bulbular muscular atrophy (SBMA) (abstract). SBMA is a neurodegenerative disease (page 1). Malik et al. teach expression profiling in SBMA embryonic motor neurons (page 3).
Malik et al. teach that Chmp7 is dysregulated in SBMA mice and human iPSC-derived pMNs (page 5). Malik et al. teach that there is a key role of this gene in the early dysfunction of motor neurons (page 5).
Therefore, in view of Malik et al., it would have been obvious to inhibit/target Chmp7 in neuron cells to inhibit Chmp7 or to treat SBMA in a human (instant claims 5, 8, and 10).
It would have been obvious as a matter of design choice to utilize an antisense oligonucleotide targeting Chmp7 as taught by Bui for the agent targeting Chmp7 in the method of treating SBMA (instant claims 6, 7, 11, and 12).
Additionally, although Bui teaches that the compounds and pharmaceutical compositions are useful to ameliorate diseases or conditions associated with aberrant activation of Endosomal Sorting Complexes Required for Transport-Ill proteins (abstract), Bui does not teach that the compound can be used in a method of treating ALS.
However, mutations in the endosomal sorting complexes required for transport (ESCRT)-III subunit CHMP2B are associated with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), both human neurodegenerative diseases characterized by accumulation of ubiquitinated proteins aggregates in affected neurons, as taught by Rusten et al. (abstract). Therefore, it would have been obvious to treat these conditions via delivery of the composition of Bui (instant claims 13 and 14).
Conclusion
Any inquiry concerning this communication or earlier communications from the examiner should be directed to Amy R Hudson whose telephone number is (571)272-0755. The examiner can normally be reached M-F 8:00am-6:00pm.
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If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Neil Hammell can be reached at 571-270-5919. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
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/AMY ROSE HUDSON/Primary Examiner, Art Unit 1636
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