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 .
Election/Restrictions
Applicant’s election without traverse of Group I, claims 1-16 and 23-26, alongside siRNA in claims 4-5, the combination of all diseases in claims 9 and 11-13, and chemotherapy in claims 16 and 26, in the reply filed on 23 March 2026 is acknowledged. For the purposes of examination, the restriction requirement with respect to claims 17-22 and 27, alongside the species election requirement with respect to the diagnoses present in claims 9 and 11-13 has been withdrawn. Accordingly, claims 1-27 will be examined herein.
Applicant is reminded that upon the cancelation of claims to a non-elected invention, the inventorship must be corrected in compliance with 37 CFR 1.48(a) if one or more of the currently named inventors is no longer an inventor of at least one claim remaining in the application. A request to correct inventorship under 37 CFR 1.48(a) must be accompanied by an application data sheet in accordance with 37 CFR 1.76 that identifies each inventor by his or her legal name and by the processing fee required under 37 CFR 1.17(i).
Claim Rejections - 35 USC § 112(b)
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 3-5 and 20-22 are 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.
Claims 3 and 20 recite “wherein the nucleic acid molecule comprises a dsRNA effective for inhibiting or decreasing the expression of Pacs1.” The grammar of claims 3 and 20 renders them indefinite. There are two mutually exclusive ways to interpret the sentences of claims 3 and 20. The first interpretation is “inhibiting the expression of Pacs1 or decreasing the expression of Pacs1”, which limits the effect of the composition to modulating the expression of the protein. The second interpretation is “inhibiting Pacs1 or decreasing the expression of Pacs1”, which is broader and encompasses a nucleic acid that is effective at inhibiting the activity of the Pacs1 protein. Given that claim 1 includes means of inhibiting Pacs1 expression or protein activity, both of these interpretations are reasonable; however, they are mutually exclusive rendering the claims indefinite.
Claims 4-5 and 21-22 are rejected for depending from claims 3 and 20, respectively, and not remedying the indefiniteness.
If Applicant intends for claims 3-5 and 20-22 to be limited to dsRNA inhibitors whose function is limited to modulating the expression of Pacs1, the following claim language for claims 3 and 20 is suggested: “wherein the nucleic acid molecule comprises a dsRNA that is effective for decreasing the expression of Pacs1.”
Claim Rejections - 35 USC § 112(a)
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 1-27 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.
Claim 1 recites “a method of attenuating or precenting lymphoproliferation in a subject in need thereof […] comprising administering a composition effective for modulating Pacs1, wherein modulating comprises decreasing Pacs1 gene expression, decreasing Pacs1 protein expression, decreasing Pacs1 activity, or any combination thereof”. Claim 23 recites a similar method wherein the administration of the composition of claim 17 (i.e., a claim directed towards “a composition comprising at least one inhibitor of Pacs1 and a pharmaceutically acceptable carrier”) is utilized to treat at least one lymphoproliferation disease.
Accordingly, the claim is interpreted as claiming a genus of compositions that have the
function of being able to treat a lymphoproliferative disease via the inhibition of Pacs1. The claim requires the provision a genus of compositions defined solely by function.
Therefore claims 1, 17, and 23 encompass methods that utilize a composition comprising virtually any chemical/molecule or combination of chemical/molecules that must have the function of inhibiting Pacs1 gene expression, protein expression, or protein activity in order to treat a lymphoproliferation disease. For the reasons described below, Applicant has not sufficiently described the genus of “compositions” that have the recited function.
Size and diversity of genus
Claims 1, 17, and 23 include a genus of compositions comprising virtually any chemical/molecule or combination of chemical/molecules, which can include protein binding partners, antibodies, small molecules, cations, anions, metallic ions, dissolved gasses, nucleic acids, sugars, carbohydrates, lipids and steroids. Each of these sub-genera is extremely diverse. For instance, a genus of short-inhibitory peptides of 20 amino acids would consist of 2020 = 1026 different peptides. Additionally, the composition could include inhibitors of proteins that act “upstream or downstream of the Pacs1 signaling cascade” (Specification [0042]), which includes additional targets of each of the chemicals/molecules. As such, the genus of “compositions” seems to include virtually any chemical or molecule, which is extremely large, possibly limitless, and extremely diverse.
Guidance in the Specification
The Specification generically describes inhibitors of Pacs1 gene expression, protein expression and protein activity as being “biomolecules” including peptides, antibodies, chemicals, compounds, oligos, nucleic acids” ([0042]) and modulators of upstream and downstream proteins/genes ([0043]). The Specification describes sub-genera, for example, dsRNA as a sub-genera of nucleic acid inhibitors ([0047]). However, other than dsRNA that specifically targets the Pacs1Pacs1 RNA, the Specification does not indicate what other genes the dsRNA should target. Likewise, the Specification teaches that CRISPR nucleases can be targeted to DNA for genome editing ([0051]); but other than the Pacs1 gene itself, the Specification does not indicate what other gene to target. The Specification does not actually disclose any means to inhibit Pacs1 other than to create a knockout mouse using CRISPR/Cas9 targeted to exon 4 of the Pacs1 gene ([0026]), but does not disclose the sequence of the guide RNA used to create the frame shifted, Pacs1-null allele. The Specification does not list a single species of any molecule that can modulate the activity of the Pacs1 protein. The Specification also does not provide any protein binding partner other than Wdr37, that can modulate Pacs1 activity. Therefore, other than Wdr37, it is completely unpredictable what other genes may lie “upstream or downstream” that can be targeted modulate the activity or expression of Pacs1.
Therefore, the Specification only describes a single species of a composition, Cas9/gRNA targeted to exon 4 of the Pacs1 gene, that has the function of inhibiting Pacs1 gene expression, protein expression, or protein activity
As a whole, the Specification does not sufficiently describe the genus of compositions that have the claimed function of modulating Pacs1.
Guidance in the Art
Regarding the state of the art, the closest prior art demonstrates that compositions utilized by the claimed method must be of a specific structure in order to perform the claimed function and that the mechanisms by which Pacs1 could be inhibited, or its relationship to the claimed lymphoproliferative diseases, were not well understood.
Thomas (PG Pub No. WO 2020/018647 A1, published 23 January 2020) teaches a method of treating Pacs1 Syndrome in a patient, comprising administering to the patient an amount of an HDAC6 inhibitor effective to treat Pacs1 Syndrome in a patient, or knocking down Pacs1 or HDAC6 expression in the patient (pg. 32; see Claim 1). Thomas teaches the use of a Pacs1 mRNA sequence that comprises a 607C>T missense mutation that was known to occur in a cargo-binding region of the protein and could be targeted with an siRNA ([0003], 0049]). Although Thomas does not disclose the sequence of the siRNA-targeting Pacs1, Thompson does teach that methods to design siRNAs and siRNAs are generally known in the art and that target Pacs1 gene mRNA sequences were known ([0083]-[0084]).
Huttlin ("Architecture of the human interactome defines protein communities and disease networks." Nature 545.7655 (2017): 505-509) teaches that Pacs1 is known to physically interact with a variety of proteins, including Wdr37, Pacs1 and CCT4 (Huttlin et al., Nature (2017), 545: 505-509; Extended data 1).
However, Huttlin does not describe whether these protein interactions are stabilizing, destabilizing, or neither.
Lusk ("Pacs1 neurodevelopmental disorder." (16 July 2020)) is drawn towards a review study concerned with Pacs1 proteins and its association with diseases and teaches that 2 specific missense alleles, 607C>T and 608G>A, in a Pacs1 gene causes a neurodevelopmental disease (Abstract, pg. 12-13; see Table 6). Lusk characterizes Pacs1 as a trans-golgi-membrane traffic regulator and teaches that the two missense mutations “may affect interaction with cargo” (pg. 12). Lusk characterizes the two missense mutations as dominant-negative or gain-of-function disease mechanisms in zebrafish (pg. 13). Lusk teaches that identification of several individuals with the same de novo variant suggests positive selection for the specific missense variant, but that data are insufficient at this time to comment on paternal age effects (pg. 13).
Thus, a study concerned with the claimed inhibited Pacs1 gene supports the conclusion that it was not known what other genes/proteins/pathways to inhibit that would in turn modulate the activity or expression of Pacs1 in order to treat a lymphoproliferative disease.
Santa Cruz Biotechnology lists 3 small molecule inhibitors as Pacs-1a inhibitors (Pacs-1a inhibitors, https://www.scbt.com/browse/Pacs-1a-inhibitors, [retrieved 14 April, 2026]).
However, it is noted that the claim is not limited to Pacs-1a inhibitors and that Pacs-1a is a splice variant of the larger Pacs1 gene, as described by Omim (PHOSPHOFURIN ACIDIC CLUSTER SORTING PROTEIN 1, https://omim.org/entry/607492, [retrieved 14 April, 2026]). It does not appear that any of the listed compounds directly inhibit the claimed Pacs1 gene, and it is unverified if the Pacs1 gene is actually a component of the listed pathways.
Finally, various companies sell polyclonal antibodies targeted to the Pacs1 protein (see e.g., Millipore Sigma, Anti-Pacs1 antibody produced in rabbit, https://www.sigmaaldrich.com/US/en/product/sigma/sab3501285, [retrieved 14 April, 2026])).
However, in each case the Pacs1 antibody has only been verified for immunohistochemistry, immunofluorescence, and/or immunoblot applications. There is no evidence in the art that any of the antibodies are functional in vitro or in vivo at inhibiting the activity of the Pacs1 protein.
Thus, the art teaches that there was only a single species of compositions that could be utilized to inhibit Pacs1 mRNA and that the mechanisms that Pacs1 interacts with other proteins, or has its expression levels modulated by the other proteins, or the relationship that Pacs1 has with lymphoproliferative diseases, was unpredictable at the effective filing date of the claimed invention.
Conclusion
Given the extremely vast and diverse genus of the claimed compositions, a lack of working examples in the specification of compositions that can modulate Pacs1 expression and activity, the lack of Pacs1-modulating compositions in the art, and the general lack of understanding in the art about the interaction that Pacs1 has with other proteins or the relationship it has to lymphoproliferative diseases, the skilled artisan would reasonably conclude that Applicant did not possess the genus of “compositions effective for modulating Pacs1” as claimed.
Claims 2, 6-16, 18-19, and 24-27 do not limit the genus of “compositions” in a manner sufficient to overcome the 35 USC 112(a) rejection above that have the claimed function, and therefore are not sufficiently described for the reasons recited above for claims 1, 17, and 23.
Claims 3-5 and 20-22 require that inhibitor of Pacs1 comprises a nucleic acid molecule comprising dsRNA, which include the known RNAi modalities shRNAs and siRNAs. However, the claims still encompass targeting the inhibitory dsRNAs to genes other than Pacs1. Because the function and regulation of Pacs1 is largely unknown and it is not predictable which genes, other than Pacs1 itself, the skilled artisan should target to modulate the expression and/or activity of Pacs1, the genus of dsRNA-based inhibitors is still insufficiently described.
Claims 4 and 21 recite that the dsRNA is a small temporal RNA (stRNA), small nuclear RNA (snRNA) and small nucleolar RNA (snoRNA). StRNAs appear to be a subset of naturally occurring miRNAs that are temporarily activated during animal development (See e.g., Sokol, Current Opinion in Genetics and Development (2012), 22: 368-373). Thus, although stRNAs are dsRNA species, it is completely unknown how to design a dsRNA to a target such that it would be considered an stRNA and not generally a miRNA. SnRNAs are short, noncoding RNAs of ~ 150 nucleotides that associate with proteins to form snRNPs, which function primarily in pre-mRNA splicing (see Blog post: Small nuclear RNAs: biogenesis, function, and NGS-based study techniques, https://www.revvity.com/blog/small-nuclear-rnas-biogenesis-function-and-ngs-based-study-techniques, [retrieved February 23, 2026]). Although the Pacs1 pre-mRNA has introns, it is not clear how administering a snRNA would in any way change the expression level of Pacs1. Administering snRNAs to affect pre-mRNA splicing is not a typical means for regulating genes at the transcriptional or post-transcriptional level. SnoRNAs are non-coding RNAs involved in rRNA and snRNA processing (See e.g., Bratkovič and Rogelj, Cellular and Molecular Life Sciences (2011), 68: 3843-3851). It is not clear how administering a snoRNA would in any way change the expression level of Pacs1 since snoRNAs do not directly impact Pacs1 RNA metabolism. Thus, it is not understood how the skilled artisan would design an stRNA, snRNA or snoRNA such that it would modulate Pacs1 expression and/or function.
Claims 1-16 and 23-26 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 enablement requirement. The claim(s) contains subject matter which was not described in the specification in such a way as to enable one skilled in the art to which it pertains, or with which it is most nearly connected, to make and/or use the invention.
Exemplary factors to be considered in determining whether undue experimentation is required are summarized in In re Wands, 858 F.2d 731, 737, 8 U.S.P.Q.2d 1400, 1404 (Fed. Cir. 1988) (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. See MPEP 2164.01(a). All of these factors were considered, along with others, and a sufficient number are addressed below so as to create a prima facie case.
Nature of the Invention and Breadth of Claims
Claims 1 and 23 are drawn to methods for attenuating, treating or preventing at least one lymphoproliferative disease [or at least one lymphoid malignancy] by administering to the subject a composition effective for inhibiting Pacs1 expression and/or activity. As indicated above in the rejection for lack of written description, the genus of “compositions effective for modulating or inhibiting Pacs1 expression and/or activity” is not sufficiently described. For the purposes of this enablement rejection, the analysis will focus on the predictability of treating and preventing lymphoproliferative diseases and lymphoid malignancies using compositions comprising 1) dsRNA inhibitors targeted to Pacs1 coding region and 2) a gene editing system such as Cas9/gRNA targeted to the Pacs1 locus, for which there is sufficient written description.
The genus of lymphoproliferative diseases and/or lymphoid malignancies includes those diseases listed in claims 9 and 13 and include proliferation of any lymphoid cells or tissues including, T cell lineages, B cell lineages, Natural Killer cells, and Innate Lymphoid cells (ILCs). “Prevention” is interpreted as preventing any genetic, metabolic or cellular change that leads to the formation lymphoid proliferation diseases including chromosomal translocations, chromosomal amplifications and dysregulated signaling pathways. See e.g., Shaffer et al., Annu. Rev. Immunol. (2012), 30: 565-610. “Attenuation” and “treating” is interpreted as decreasing the proliferative capacity of lymphoid cells and/or reducing the symptoms associated with the diseases.
Accordingly, enablement of the method requires one skilled in the art to be able to prevent genetic modifications and/or signaling dysregulation leading to the genus of lymphoid proliferative diseases and decreasing the proliferative capacity of lymphoid cells and/or reducing the symptoms associated with the diseases using a Pacs1-targeted RNAi inhibitor or gene editing system.
State of the Prior Art/Unpredictability in the Art
As indicated above in the rejections for lack of written description, Pacs1 is a relatively uncharacterized protein and that although the generation of siRNA molecules targeted to a specific mutation present in Pacs1 mRNA was known, the result of the protein’s inhibition, and the relationship of that inhibition to lymphoproliferative diseases, was unknown.
As discussed above, Thomas (PG Pub No. WO 2020/018647 A1, published 23 January 2020) teaches a method of treating Pacs1 Syndrome in a patient, comprising administering to the patient an amount of an HDAC6 inhibitor effective to treat Pacs1 Syndrome in a patient, or knocking down Pacs1 or HDAC6 expression in the patient (pg. 32; see Claim 1). Thomas teaches the use of a Pacs1 mRNA sequence that comprises a 607C>T missense mutation that was known to occur in a cargo-binding region of the protein and could be targeted with an siRNA ([0003], 0049]).
Thus, the generation of specific siRNA molecule that could target and knockdown a specific Pacs1 mRNA mutation was known.
Brasacchio ("Epigenetic control of mitochondrial cell death through Pacs1-mediated regulation of BAX/BAK oligomerization." Cell Death & Differentiation 24.6 (2017): 961-970) is drawn towards a study concerned with how PCAF and ADA3 regulate the expression of Pacs1 (Abstract). Notably, Brasacchio teaches that cells with decreased Pacs1 expression were refractory to cell death mediated by a variety of stimuli that operate through the mitochondrial pathway (Abstract).
Thus, this study suggests that the reduction of Pacs1 in a cell results in the cell’s ability to survive various apoptotic stimuli. Although Brasacchio does not comment on the knockout cells’ proliferation capacity, based on the teachings of Brasacchio one of ordinary skill in the art would have expected the knockout of Pacs1 to result in increased proliferation of the cells in the presence of the apoptotic stimuli compared to control cells because the cells had a higher survival rate in the presence of the stimuli.
Pacs1 is known to physically interact with a variety of proteins (Huttlin et al., Nature (2017), 545: 505-509; Extended data 1), a few of which are known to be associated with lymphomas. For instance, increased TCP-1 expression is correlated with increased incidence of lymphoma lymphatic metastasis (Jiang et al., Oncotarget (2015), 6: 42105-42117).
However, Huttlin does not describe whether these protein interactions are stabilizing, destabilizing or neither. Thus, it is not clear from Huttlin if inhibiting Pacs1 would likewise inhibit TCP-1 expression or function.
Lusk ("Pacs1 neurodevelopmental disorder." (16 July 2020)) is drawn towards a review study concerned with Pacs1 proteins and its association with diseases and teaches that 2 specific missense alleles, 607C>T and 608G>A, in a Pacs1 gene causes a neurodevelopmental disease (Abstract, pg. 12-13; see Table 6). Lusk characterizes Pacs1 as a trans-golgi-membrane traffic regulator and teaches that the two missense mutations “may affect interaction with cargo” (pg. 12). Lusk teaches that identification of several individuals with the same de novo variant suggests positive selection for the specific missense variant, but that data are insufficient at this time to comment on paternal age effects (pg. 13).
Thus, a review study concerned with the inhibition of the claimed Pacs1 gene supports the conclusion that it was not known what other genes/proteins/pathways to inhibit that would in turn modulate the activity or expression of Pacs1 in order to treat a lymphoproliferative disease and that the understanding of the reduced expression of Pacs1 and an associated therapeutic benefit with regard to a lymphoproliferative disease was relatively unknown.
A thorough search of the prior art found no teaching of delivering Pacs1-targeted RNAi agents or gene editing systems specifically to lymphoid cells or cells of a lymphoproliferation diseases to determine the effects of downregulating Pacs1 in those tissues. As such, it is unknown what the effect of inhibiting the expression of Pacs1 would have on its many protein binding partners or cell-survival pathways. Finally, there are no teachings in the prior art that Pacs1 has any function in the nucleus where it might influence DNA repair networks such that inhibiting the protein would be predicted to prevent lymphoproliferation diseases from occurring in the first place.
Thus, in view of the prior art, it is highly unpredictable if or how one skilled in the art could administer a Pacs1 inhibiting RNAi or gene editing system to prevent the claimed diseases or ameliorate symptoms of the diseases.
Guidance in the Specification/Prescence of Working Examples
Applicant provides a set of studies that progresses the understanding of Pacs1 and Wdr37 in the Calcium-dependent signaling during lymphocyte activation. Applicant shows that Pacs1 knockout mice have a deficiency in circulating B and T cells, demonstrating the requirement for Pacs1 in lymphocyte development ([0108]), likely through regulating Ca++ efflux from subcellular compartments ([0112]-[0115]). Pacs1 deletion causes increased Ca++ leakage from the ER and reduced stimulated ER Ca++ release ([0133]-[0135]). Applicant also shows that the Wdr37 protein binds to and stabilizes Pacs1 and that Wdr37 null mice had reduced numbers of lymphocytes ([0120]-[0124]). Applicant demonstrates that Pacs1-null B cells have normal proliferative capacity in vitro, are functional in vivo, but have reduced life upon activation in some environments ([0137]-[0141]). Finally, Applicant shows that Pacs1-null cells have reduced proliferative capacity in mouse models of lymphoproliferation ([0142]-[0146]). Specifically, eliminating Pacs1 protein expression in mice that either overexpress the anti-apoptotic protein, Bcl2, or are deficient in Fas pro-apoptotic signaling showed higher sensitivity to cell death stimuli ([0142]-[0146]), suggesting that disrupting Pacs1 stabilization in vivo could suppress lymphoproliferative diseases in B and T cell lineages ([0145]).
However, the working examples in the Specification do not actually administer any Pacs1 inhibitor or Wdr37 inhibitor to the mice models having lymphoproliferative diseases. The specification also doesn’t disclose inhibiting Pacs1 in any manner (knockout, RNAi, or otherwise) in the mice models having lymphoproliferative diseases. The specification also provides no guidance as to how one would prevent lymphoid diseases from occurring, especially those that are caused by chromosomal translocations and amplifications.
Accordingly, in light of the specification, it is highly unpredictable if one skilled in the art could administer a Pacs1 inhibiting RNAi or gene editing system to prevent the claimed diseases or ameliorate symptoms of the diseases.
Experimentation Required
In order to practice the invention, one skilled in the art would need to design Pacs1 RNAi and/or gene editing systems to reduce expression in target cells to determine the effect of Pacs1 inhibition. Then the inhibitor studies would need to be recapitulated in mouse and primate models of each disease, and finally clinical trials. However, given the great unpredictability in the art regarding the role of the Pacs1 in apoptotic or cell-survival signaling networks, it is highly unpredictable whether such inhibitors would have any effect.
Conclusion
Taking into consideration the factors outlined above, including the nature of the invention, the breadth of the claims, the state of the art, the guidance provided by the applicant, and the lack of working examples of inhibiting expression of Pacs1 in a model of lymphoproliferative diseases, it is the conclusion that undue experimentation would be required to make and use the invention.
Claims 2-14 and 24 do not limit the lymphoproliferative disease, do not recite a disease in which the function of Pacs1 is characterized, or do not recite a disease in which the effect of inhibiting Pacs1 is known. Thus, for the reasons recited above for claims 1 and 23, it is the conclusion that an undue experimentation would be required to make and use the invention.
Claims 15-16 recite the patients are undergoing therapy or have already undergone therapy for treatment of a lymphoproliferation diseases. These claims also lack enablement because there is no evidence in the art that the treatments recited in claim 16 can prevent the disease in the first place. Additionally, the claims do not recite administering the therapy and include instances where the subject has already undergone the therapy. Thus, there is no requirement for administration of an already proven therapy.
Claims 25-26 recite administering to the subjects an effective amount of a therapy for lymphoproliferation. These claims also lack enablement because there is no evidence in the art that the treatments recited in claim 26 can prevent the disease in the first place.
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.
Claim(s) 17-22 and 27 is/are rejected under 35 U.S.C. 102(a)(1) and 102(a)(2) as being anticipated by Thomas (PG Pub No. WO 2020/018647 A1, published 23 January 2020).
Regarding claims 17-19 and 27, Thomas teaches a method of treating Pacs1 Syndrome in a patient, comprising administering to the patient an amount of an HDAC6 inhibitor effective to treat Pacs1 Syndrome in a patient, or knocking down Pacs1 or HDAC6 expression in the patient ([0006]). Thomas teaches the use of an siRNA molecule (i.e., a nucleic acid) that could target and knockdown Pacs1 mRNA (i.e., a composition comprising at least one inhibitor of Pacs1 that could inhibit Pacs1 activity through the knockdown of the Pacs1 mRNA) ([0099]-[00100]; see Figures 8A-8B; see Claims 1 and 6). Thomas teaches that the nucleic acids for inhibiting Pacs1 may be administered alongside a pharmaceutically acceptable carrier or excipient ([0035]). Thomas teaches that dosages of the Pacs1 inhibitor may be present within containers (i.e., within a kit) ([0033], [0037]).
Regarding claims 20-22, Thompson teaches that RNAi agents also may be used to knock down Pacs1 mRNA expression ([0053]). Thompson teaches that the RNA interference agents may be siRNA, shRNA, a miRNA ([0057]), or a double-stranded RNA interfering nucleic acid ([0082]). Although Thomas does not disclose the sequence of the siRNA-targeting Pacs1, Thompson does teach that methods to design siRNAs and siRNAs are generally known in the art and that target Pacs1 gene mRNA sequences were known ([0083]-[0084]).
Conclusion
Any inquiry concerning this communication or earlier communications from the examiner should be directed to KYLE T REGA whose telephone number is (571)272-2073. The examiner can normally be reached M-R 8:30-4:30, every other F 8:30-4:30 (EDT/EST).
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/KYLE T REGA/Examiner, Art Unit 1636
/NEIL P HAMMELL/Supervisory Patent Examiner, Art Unit 1636
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