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 .
Continued Examination Under 37 CFR 1.114
A request for continued examination under 37 CFR 1.114, including the fee set forth in 37 CFR 1.17(e), was filed in this application after final rejection. Since this application is eligible for continued examination under 37 CFR 1.114, and the fee set forth in 37 CFR 1.17(e) has been timely paid, the finality of the previous Office action has been withdrawn pursuant to 37 CFR 1.114. Applicant's submission filed on 8/12/2026 has been entered.
Claim Status
Claims 1, 2, 4, 20, 25, 97-100, and 103. Claims 2, 20, 98-100, and 103 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected species.
Therefore, Claims 1, 4, 25 and 97 are under examination.
Election/Restrictions
Applicant elected BI-9564 as the bromodomain 9 (BRD9) inhibitor species without traverse during an interview on 9/3/2025.
Claims 2, 20, 98-100, and 103 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected species.
Priority
This application is a 371 national stage application of PCT/US2021/026223 filed April 07, 2021, which claims the priority benefit of U.S. provisional application no. 63/007,161, filed April 8, 2020.
Information Disclosure Statement
No Information Disclosure Statement was filed with Applicant’s recent remarks.
Treatment of HD with the elected species is free of the prior art
The use of the elected BRD9 inhibitor species (BI-9564) for treating Huntington’s disease (HD) is free of the prior art. Therefore, the search and consideration has been expanded to other BDR9 inhibitor species, which were also found to be free of the prior art for treating Huntington’s disease.
Withdrawn Objections/Rejections
Claims 1, 4, and 25 were rejected under 35 U.S.C. 103 as being unpatentable over Olzscha et al. (Cell Chemical Biology (2017); 24:9–23 in view of ATM inhibitor (“ATMi”) (“Bromosporine.” ATM inhibitor (6/7/2017)) and Sdelci et al. (US 20190117641 A1). Applicant’s amendment and corresponding reply pertaining to the newly added limitation have overcome the rejection made of record in the previous Office Action, specifically, the addition/removal of the limitation to claim 1 that requires the BRD9 inhibitor to be a pyridinone compound. Therefore, the rejection is hereby withdrawn.
Claims 1, 4, 8, 25, and 97 were rejected under 35 U.S.C. 112(b) or 35 U.S.C. 112 (pre-AIA ), second paragraph, as being indefinite. Applicant’s amendment and corresponding reply pertaining to the newly added limitation have overcome the rejection made of record in the previous Office Action by removing the limitations that elicited the rejection.
Claim Objections
Claim 1 is objected to because of the following informalities: the term “HD partially” at line 6 should be changed to “partially”. Appropriate correction is required.
Claim Rejections - 35 USC § 112 – Scope of Enablement
New Rejection, modified to address addition of the limitation: “completely reversing HD neuroids to wildtype configuration”
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 1, 4, 25, and 97 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 alleviating Huntington’s Diseases (HD) in a subject suffering from HD,
does not reasonably provide enablement for treating HD as defined by the specification or completely reversing HD neuroids to a wildtype configuration.
The specification does not enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to use the invention commensurate in scope with these claims.
The test of enablement requires a determination of whether the disclosure, when filed, contained sufficient information regarding the subject matter of the claims as to enable one skilled in the art of pertinent art to make and use the claimed invention. That standard is still the one to be applied. In re Wands, 858 F.2d 731, 737, 8 USPQ2d 1400, 1404 (Fed. Cir. 1988). Accordingly, even though the statute does not use the term “undue experimentation,” it has been interpreted to require that the claimed invention be enabled so that any person skilled in the art can make and use the invention without undue experimentation. In re Wands, 858 F.2d at 737, 8 USPQ2d at 1404 (Fed. Cir. 1988). Keeping that in mind, the Wands factors are relevant to the instant fact situation for the following reasons:
1. The nature of the invention, the breadth of the claims, state and predictability of the art, and relative skill level:
The invention is based on inhibiting bromodomain 9 (BRD9) in a subject afflicted with Huntington’s disease (HD) by administering a BRD9 inhibitor in effective amount to “completely reverse HD neuroids”. The specification defines the terms "treatment", "treat", and "treating" as referring to, inter alia, reversing a disease described therein, including HD. See 0031-0032. Given this broad definition, the breadth of the invention is broad enough to encompass “reversing HD” which is further exacerbated by the recitation of “completely reversing HD neuroids to wildtype”, which is not predictable. The specification does not enable reversal of Huntington’s disease (HD) in a subject, which falls within the scope of the claimed method because the specification defines “treating” to include reversal. In this regard, it is unpredictable whether a drug can be used to reverse HD because the state of the art after the filing of the current patent application is such that HD cannot be reversed. For example, with regard to reversing HD, the more recent art states the following:
eMentalHealth.ca (“Dementia.” The eMentalHealth.ca Team and Editorial Board, Dr. Tim Lau, Geriatric Psychiatrist, Royal Ottawa Mental Health Centre, and Assistant Professor, University of Ottawa (2016). https://primarycare.ementalhealth.ca/Simcoe-County/La-demence/index.php?m=article&ID=8921&r=ns-western) teaches “[h]untington's disease cannot be reversed; thus, the person with these conditions will gradually lose more and more mental functions.” See p. 3.
Brain Research UK (“Huntington’s disease.” Brain Research UK (2020);
https://web.archive.org/web/20200806215510/https://www.brainresearchuk.org.uk/neurological-conditions/huntingtons-disease) teaches that there is no cure for Huntington's and its progress cannot be reversed. See p. 2.
Reilmann et al. (“Pridopidine in early-stage manifest Huntington’s disease: a phase 3 trial.” Nat Med (2025). https://doi.org/10.1038/s41591-025-03920-3) teaches that available therapies are only symptomatic or investigational and no clinical trial has demonstrated restoration of loss of neuronal function. This Phase 3 PROOF-HD trial confirmed that “only symptomatic treatments are available” for HD. See abstract.
NIH (“Huntington’s disease – Neurological condition.” https://www.ninds.nih.gov/health-information/disorders/huntingtons-disease. Accessed 9/30/2026) teaches HD neuroloids cannot be fully reversed, but specific genetic and structural defects within these cellular models have been partially rescued in laboratory research using experimental drug treatments. In humans, Huntington's Disease (HD) remains a progressive, incurable neurodegenerative disorder with no existing therapies capable of stopping or reversing its course.
Therefore, the current state of the art contradicts the validity of the claim of reversing HD or HD neuroids. As a result of such difficulties, HD “treatment” as broadly defined by the instant specification is unpredictable.
2. The amount of direction or guidance provided and the presence or absence of working examples:
The specification provides guidance for in vitro testing and results using organoid assays for HD. See Examples 1-8. No working examples demonstrating reversal of HD in a subject have been provided.
3. The quantity of experimentation necessary:
Because of the known unpredictability of the art, and in the absence of sufficient experimental evidence, no one skilled in the art would accept the assertion that the instantly claimed agents could be predictably used to reverse (i.e., “treat” as defined) HD or completely reverse neuroloid as inferred by the claims and contemplated by the specification. Accordingly, the instant claims do not comply with the enablement requirement of §112, since to practice the invention claimed in the patent a person of ordinary skill in the art would have to engage in undue experimentation, with no assurance of success.
Response to arguments
Applicant's arguments have been fully considered but have not been found to be persuasive.
Applicant argues that the disclosed neuroid and gastruloid models correlate with Huntington’s disease and that phenotypic rescue observed in those models therefore provides a working example supporting the claimed reversal of HD. However, the rejection concerns whether the disclosure enables the substantially broader claimed result partially and completely reversing HD or neuroloid to wildtype configuration.
The state of the art references provides evidence of a reasonable basis for questioning that conclusion. As set forth in the rejection, multiple references characterize HD as progressive and irreversible and indicate that available therapies do not reverse its course. Particularly, the NIH reference expressly distinguishes partial rescue of specific genetic or structural defects in cellular HD models from the disease in humans, which remains a progressive, incurable neurodegenerative disorder for which no existing therapy is capable of stopping or reversing its course. Thus, the in vivo results of rescue relied upon by Applicant was already known to occur without demonstrating reversal of HD in a subject.
Therefore, Applicant’s showing that BRD9 inhibitors reduce PAX6+ area, SOX17+ ring formation or shift an in nitro HD phenotype toward wildtype phenotype does not establish the claimed method of reversing HD in a subject without undue experimentation.
Conclusion
No claims are allowed.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to CHRIS E SIMMONS whose telephone number is (571)272-9065. The examiner can normally be reached M-F: 9:30-6:00p.
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CHRIS E. SIMMONS
Examiner
Art Unit 1622
/CHRIS E SIMMONS/Examiner, Art Unit 1622
/JAMES H ALSTRUM-ACEVEDO/Supervisory Patent Examiner, Art Unit 1622
