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 .
DETAILED ACTION
Priority
This application claims the benefit of and priority to U.S. Provisional Patent Application Nos. 62/916,207, filed on October 16, 2019; 62/916,221, filed on October 16, 2019; 63/018,454, filed on April 30, 2020; and 63/055,252, filed on July 22, 2020 that is hereby acknowledged by the Examiner.
Status of the Claims
The amendment dated 01/20/2026 is acknowledged. Claims 1, 3-8, 11, 13, 16, 18-19, 21, 23, 31-35, 37-39, 42, 44-45, 47-48, 52, 58-59, 61-62, 64, 74, 77-78 and 88-90 are pending and under examination.
Information Disclosure Statement
The information disclosure statement (IDS) submitted on 03/28/2022, 06/27/2023, 01/17/2025, 02/05/2025, 03/20/2025, 04/17/2025, 07/28/2025, 08/20/2025, 01/20/2026 and 03/12/2026 are in compliance with the provisions of 37 CFR 1.97. Accordingly, the information disclosure statement(s) is/are being considered by the Examiner.
Drawings
The drawing filed on 03/28/2022 are acknowledged and accepted by the Examiner.
Specification
The disclosure is objected to because it contains an embedded hyperlink and/or other form of browser-executable code (pages 109, 136, 163). Applicant is required to delete the embedded hyperlink and/or other form of browser-executable code; references to websites should be limited to the top-level domain name without any prefix such as http:// or other browser-executable code. See MPEP § 608.01.
Clam Objections
Claim 34 is objected to for the following informalities:
Claim 34 does not contain a period at the end of the sentence.
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.
Claim 61 is rejected under 35 U.S.C. 112, second paragraph, as being indefinite for failing to particularly point out and distinctly claim the subject matter which applicant regards as the invention.
Claim 61 recites the limitation "the vector system, the polypeptide, or the particle of claim 60". Claim 60 has been cancelled. There is insufficient antecedent basis for this limitation in the claim. Accordingly, one of ordinary skill in the art would not know the metes and bounds of the claims.
The following is a quotation 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 35 U.S.C. 112 (pre-AIA ), first paragraph:
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 16 and 59 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 claims contain 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. Applicant does not provide enablement for the claimed composition, wherein the cargo is capable of “preventing” disorders (e.g. cancer and Duchene muscular dystrophy (DMD)).
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 the invention commensurate in scope with these claims. The legal considerations that govern enablement determinations pertaining to undue experimentation have been clearly set forth. “[T]o be enabling, the specification of a patent must teach those skilled in the art how to make and use the full scope of the claimed invention without ‘undue experimentation.' ” Genentech Inc. v. Novo Nordisk 108 F.3d 1361, 1365, 42 USPQ2d 1001, 1004 (Fed. Cir. 1997); In re Wright 999 F.2d 1557, 1561, 27 USPQ2d 1510, 1513 (Fed. Cir. 1993); See also Amgen Inc. v. Chugai Pharm. Co., 927 F.2d 1200, 1212, 18 USPQ2d 1016, 1026 (Fed. Cir. 1991); In re Fisher 427 F.2d 833, 839, 166 USPQ 18, 24 (CCPA 1970). Further, in In re Wands 858 F.2d 731, 737, 8 USPQ2d 1400, 1404 (Fed. Cir. 1988) the court stated: Factors to be considered in determining whether a disclosure would require undue experimentation have been summarized by the board in Ex parte Forman [230 USPQ 546, 547 (BdPatAppInt 1986)]. They include (1) the quantity of experimentation necessary, (2) the amount of direction or guidance presented, (3) the presence or absence of working examples, (4) the nature of the invention, (5) the state of the prior art, (6) the relative skill of those in the art, (7) the predictability or unpredictability of the art, and (8) the breadth of the claims (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).
The breadth of claims. The breadth of the claims encompasses a composition or a vector system of claim 1, wherein the cargo is capable of treating or preventing a muscle disease or disorder, optionally wherein the muscle disease or disorder is a. an auto immune disease; b. a cancer; c. a muscular dystrophy, optionally wherein the muscular dystrophy is Duchene muscular dystrophy, Becker Muscular dystrophy, a Limb-Girdle muscular dystrophy, an Emery Dreifuss muscular dystrophy, a myotonic dystrophy, optionally a Type 1 or a Type 2 myotonic dystrophy, or FSHD; d. a neuro-muscular disease, optionally wherein the neuro-muscular disease is Charcot-Marie-Tooth disease or Friedreich's Ataxia; e. a sugar or glycogen storage disease, optionally wherein the sugar or glycogen storage disease is a MPS type III disease, optionally MPS Type IIIA, IIIB, IIIC, or IIID, or Pompe disease; f. an expanded repeat disease, optionally wherein the expanded repeat disease is Huntington's disease, a myotonic dystrophy, optionally a Type 1 or a Type 2 myotonic dystrophy, or Facioscapulohumeral muscular dystrophy (FSHD); g. a dominant negative disease; h. a cardiomyopathy, optionally wherein the cardiomyopathy is dilated cardiomyopathy, hypertrophic cardiomyopathy, Duchene muscular dystrophy - associated cardiomyopathy, or Dannon disease; i. a viral disease; j. a progeroid disease; or k. any combination thereof.
Nature of the invention. The nature of the invention is directed to muscle targeting compositions including, but not limited to, recombinant adeno-associate virus (AAV) vectors and systems thereof, compositions, and uses thereof. Specifically, the invention is directed to targeting moieties that can be capable of specifically targeting muscle cells and can include an n-mer motif. In some embodiments, the n-mer motif contains an RGD motif.
State of the prior art. The state of the art, citing Grimm et al. “Grimm” (WO2019/207132, IDS of record dated 11/14/2025), Grimm discloses an adeno-associated virus (AA V) capsid polypeptide modified by insertion of a binding peptide. The binding peptide comprises the amino acid sequence RGDX1X2X3X4 (SEQ ID NO: 1), with X1 to X4 being independently selected amino acids, for use in treating and/or preventing a muscular disease and/or in muscle regeneration (page 1, lines 5-9). Grimm also describes insertion of an RGD motif comprising at least one amino acid upstream of the RGD amino acid sequence (see, e.g., Grimm at Fig. 7). Grimm further describes inserting the RGD X1X2X3X4 (SEQ ID NO: 1) peptide into the AAV9 capsid polypeptide after amino acid residues 588 or 589 (pages 7, lines 28-34), where the AA V9 capsid polypeptide can include additional modifications at positions 504 and 505 (page 9, lines 24-34). The modified AAV9 capsid protein improves tropism of AAV particles to muscle tissue and muscle cells, while at the same time exhibiting a strongly reduced liver tropism (page 15, lines 1-5).
Working Examples. There are no working examples that demonstrate the claimed composition or vector system for “preventing” a disease or disorder in a human subject. There are no working examples to demonstrate that the claimed composition can even prevent or treat diseases associated with a targeting moiety comprising an n-mer motif, wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif. The specification does not provide any guidance as to the claimed limitation of wherein the cargo is capable of “preventing” a muscle disease or disorder, wherein the muscle disease or disorder is (e.g. an autoimmune disease, cancer, Duchene muscular dystrophy, Charco-Marie-Tooth disease or Friedreich’s Ataxia).
Guidance in the specification. The applicant’s guidance in the specification provides engineered cells and organisms as well as formulations with pharmaceutical acceptable carriers, whereby the muscle-specific targeting moieties are expressed in the engineered cells (paragraphs [0438] to [0490]); and Methods of use comprising embodiments of therapeutics (e.g. gene modification and treatment of diseases with genetic or epigenetic aspects (paragraphs [0496] to [0526]).
Predictability or unpredictability of the art. Citing Grimm et al. (WO2019/207132, IDS of record dated 11/14/2025), the level of predictability is low since the art demonstrates that “Adeno-associated virus (AAV) vectors have been known to enable transfer and long-term expression of foreign DNA, including therapeutic genes, in a variety of cell types. The different serotypes of AAV have been shown to have different tropisms in cultured cells and within an organism, with AAV9 e.g. having the broadest tropism. Transduction efficiencies and cell specificities obtainable with AAV vectors comprising unmodified capsids are good for some cell types, however, they are far too low to be of experimental or therapeutic use for most other cell types. A further problem in retargeting AAV is that while it may be possible to confer a new specificity to an AAV strain by including amino acid exchanges and/or additional peptides into the capsid protein, frequently a substantial infectivity for the liver, the major target of AAV, remains, which is undesirable for most applications” and “nonetheless, there is still a need in the art for improved AAV vectors targeting these cells” (pages 1-2 of Grimm).
When a compound or composition claim is limited by a particular use, enablement of that claim should be evaluated based on that limitation. See In re Vaeck, 947 F.2d 488, 495, 20 USPQ2d 1438, 1444 (Fed. Cir. 1991) (claiming a chimeric gene capable of being expressed in any cyanobacterium and thus defining the claimed gene by its use (MPEP 2164.01 (c)). Accordingly, when all the aforementioned factors are considered in toto, it would require undue experimentation for one skilled in the art to practice the claimed invention.
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.
(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.
Claims 1, 3-4, 6-8, 11, 13, 16, 18-19, 21, 23, 31-34, 37-39, 42, 44-45, 47-48, 52, 58-59, 61-62, 64, 74, 77-78, 88-90 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Grimm et al. “Grimm” (WO2019/207132, IDS of record dated 11/14/2025).
The claims are directed to a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety.
Regarding claims 1, 3-4, 6-8, 13, 32-34, Grimm discloses an adeno-associated virus (AA V) capsid polypeptide modified by insertion of a binding peptide. The binding peptide comprises the amino acid sequence RGDX1X2X3X4 (SEQ ID NO: 1), with X1 to X4 being independently selected amino acids, for use in treating and/or preventing a muscular disease and/or in muscle regeneration (page 1, lines 5-9). Grimm also describes insertion of an RGD motif comprising at least one amino acid upstream of the RGD amino acid sequence (see, e.g., Grimm at Fig. 7). Grimm further describes inserting the RGD X1X2X3X4 (SEQ ID NO: 1) peptide into the AAV9 capsid polypeptide after amino acid residues 588 or 589 (pages 7, lines 28-34), where the AA V9 capsid polypeptide can include additional modifications at positions 504 and 505 (page 9, lines 24-34). The modified AAV9 capsid protein improves tropism of AAV particles to muscle tissue and muscle cells, while at the same time exhibiting a strongly reduced liver tropism (page 15, lines 1-5). Grimm at Figure 7 further discloses:
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In describing Figure 7, Grimm explains that it is an "overview for the peptide insertion site of
muscle variants illustrating the surrounding amino acids of the peptide insertion" (page 28, line
33 to page 29, line 1).
Regarding claim 11, Grimm discloses the composition by stating “Preferably, the binding peptide is inserted at a site of the capsid polypeptide exposed to the exterior of the AAV capsid, preferably based on structure predictions and/or experimental data. More preferably, the insertion site of the binding peptide is at a site exposed to the exterior of the AAV capsid in a manner that does not interfere with the activity of said polypeptide in capsid assembly. More preferably, the insertion site of the binding peptide corresponds to amino acid 588 or 589, preferably 588, of the AAV9 capsid polypeptide (page 7, line 28 to page 8 line 10).
Regarding claims 16, Grimm discloses AAV vectors capable of expressing a micro-dystrophin protein in the muscle cell by stating “AAV vectors have been known to enable transfer and long-term expression of foreign DNA, including therapeutic genes in a variety of cell types (page 1 lines 13-14) and “Preferably, the muscular dystrophy is Duchenne muscular dystrophy (gene affected: DMD), Becker muscular dystrophy (gene affected: DMD), Limb girdle muscular dystrophy (Subtypes and affected genes: LGMD1A (Gene: TTID), LGMD1B (Gene: LMNA), LGMD1C (Gene: CAV3), LGMD1D (Gene: DNAJB6), LGMD1E (Gene: DES), LGMD1F (Gene: TNP03), LGMD1G (Gene: HNRPDL), LGMD1H, LGMD2A (Gene: CAPN3), LGMD2B (Gene: DYSF), LGMD2C (Gene: SGCG), LGMD2D (Gene: SGCA), LGMD2E (Gene: SGCB), LGMD2F (Gene: SGCD), LGMD2G (Gene: TCAP), LGMD2H (Gene: TRIM32), LGMD2I (Gene: FKRP), LGMD2J (Gene: TTN), LGMD2K (Gene: POMT1), LGMD2L (Gene: AN05), LGMD2M (Gene: FKTN), LGMD2N (Gene: POMT2), LGMD20 (Gene: POMGNT1), LGMD2Q (Gene: PLEC1)), Congenital muscular dystrophy, Distal muscular dystrophy (Subtypes and affected genes: Miyoshi myopathy (Gene: DYSF), Distal myopathy with anterior tibial onset (Gene: DYSF), Welander distal myopathy (Gene: TIA1), Gowers-Laing distal myopathy (Gene: MYH7), Nonaka distal myopathy, hereditary inclusion-body myositis type 1, distal myopathy with vocal cord and pharyngeal weakness, ZASP-related myopathy), Facioscapulohumeral muscular dystrophy (Subtypes and affected genes: Type 1 (Gene: DUX4), Type 2 (Gene: SMCHD1)), Oculopharyngeal muscular dystrophy (affected gene: PABPN1), and/or myotonic dystrophy (Subtypes and affected genes: DM1 (Gene: DMPK) and DM2 (Gene: ZNF9)” (page 11 lines 20 – page 12 line 3).
Regarding claim 18, Grimm discloses vectors derived from AAV, i.e. gene transfer vehicles using the capsid polypeptide of AAV to mediate the transfer of recombinant polynucleotides into target cells (page 3 lines 29-31). Grimm also discloses “a therapeutically effective does refers to an amount of the polynucleotide to be used in a pharmaceutical composition of the present invention which prevents, ameliorates or treats the symptoms accompanying a disease or condition (page 21, lines 24-26).
Regarding claims 19, 21, Grimm discloses their invention relates to a method for treating and/or preventing a muscular disease and/or for muscle regeneration comprising contacting a subject with an AAV capsid polypeptide according to the present invention, a polynucleotide according to the present invention, a vector according to the present invention, a host cell according to the present invention, and/or an AAV capsid according to the present invention (page 22 line 31 – page 23 lines 1-8). Grimm also discloses said method is capable of expressing a micro-dystrophin protein in the muscle cell more effectively than an AAV without the n-mer motif (page 11 lines 20 – page 12 line 3 and Figures 10-12 and Example 2 Results).
Regarding claim 23, Grimm discloses “Regulatory elements ensuring expression in eukaryotic cells, preferably mammalian cells, are well known in the art. They, preferably, comprise regulatory sequences ensuring initiation of transcription and, optionally, poly-A signals ensuring termination of transcription and stabilization of the transcript. Additional regulatory elements may include transcriptional as well as translational enhancers. Possible regulatory elements permitting expression in prokaryotic host cells comprise, e.g., the lac, trp or tac promoter in E. coli, and examples for regulatory elements permiting expression in eukaryotic host cells are the AOX1 or GAL1 promoter in yeast or the CMV-, SV40-, RSV-promoter (Rous sarcoma virus), CMV-enhancer, SV40-enhancer or a globin intron in mammalian and other animal cells. Moreover, inducble expression control sequences may be used in an expression vector. Such inducible vectors may, preferably, comprise tet or lac operator sequences or sequences inducible by heat shock or other environmental factors (page 18 lines 17-29).
Regarding claim 31, Grimm discloses “Advantageously, it was found in the work underlying the present invention that the AAV capsid polypeptides proposed herein mediate an improved tropism of AAV particles to muscle tissue and muscle cells (page 15, lines 1-3). Grimm also discloses that “Regarding organ specificity (Fig. 1 and Table 2), AAV9_P1 demonstrated a strong muscle tropism with 74% of the virus ending up in the analyzed muscle tissues diaphragm, heart and quadriceps femoris. In contrast, when using the parental virus AAV9 wt, only 10% of corresponding transcripts were found in the muscle tissues, whereas 50% could be observed in the major off-target of AAV, the liver” (page 31 lines 1-5).
Regarding claim 37, Grimm discloses “More preferably, in the vector, the polynucleotide is operatively linked to expression control sequences allowing expression in prokaryotic or eukaryotic cells or isolated fractions thereof. Expression of said polynucleotide comprises transcription of the polynucleotide, preferably into a translatable mRNA. Regulatory elements ensuring expression in eukaryotic cells, preferably mammalian cells, are well known in the art…Preferably, the vector is an AAV vector, more preferably a self-complementary AAV vector, more preferably a self-complementary AAV vector (page 18, line 14 to page 19, line 6).
Regarding claims 38-39, Grimm discloses a self-complementary AAV genome was used carrying a CMV promoter…During AAV production, HEK293T cells are transfected by an Adenoviral helper plasmid delivering essential genes for the replication of the AAV. Additionally, a plasmid for the expression of rep2 (rep gene of AAV2) and the respective cap gene is needed as well as a plasmid carrying the ITR-flanked (inverted terminal repeats), barcoded AAV genome production, a packaging of a defined barcoded genome into a synthetic capsid was allowed” (page 30, lines 2-10).
Regarding claims 42 and 44-45, Grimm discloses an adeno-associated virus (AA V) capsid polypeptide modified by insertion of a binding peptide. The binding peptide comprises the amino acid sequence RGDX1X2X3X4 (SEQ ID NO: 1), with X1 to X4 being independently selected amino acids, for use in treating and/or preventing a muscular disease and/or in muscle regeneration (page 1, lines 5-9). Grimm also describes insertion of an RGD motif comprising at least one amino acid upstream of the RGD amino acid sequence (see, e.g., Grimm at Fig. 7). Grimm further describes inserting the RGD X1X2X3X4 (SEQ ID NO: 1) peptide into the AAV9 capsid polypeptide after amino acid residues 588 or 589 (pages 7, lines 28-34 and page 8 lines 1-10), where the AA V9 capsid polypeptide can include additional modifications at positions 504 and 505 (page 9, lines 24-34). The modified AAV9 capsid protein improves tropism of AAV particles to muscle tissue and muscle cells, while at the same time exhibiting a strongly reduced liver tropism (page 15, lines 1-5).
Regarding claims 47-48, Grimm discloses “During AAV production, HEK293T cells are transfected by an Adenoviral helper plasmid delivering essential genes for the replication of the AAV. Additionally, a plasmid for the expression of rep2 (rep gene of AAV2) and the respective cap gene is needed as well as plasmid carrying the ITR-flanked (inverted terminal repeats), barcoded AAV genome production, a packaging of a defined barcoded genome into a synthetic capsid was allowed” (page 30, lines 5-10).
Regarding claim 52, Grimm discloses “During AAV production, HEK293T cells are transfected by an Adenoviral helper plasmid delivering essential genes for the replication of the AAV. Additionally, a plasmid for the expression of rep2 (rep gene of AAV2) and the respective cap gene is needed as well as plasmid carrying the ITR-flanked (inverted terminal repeats), barcoded AAV genome production, a packaging of a defined barcoded genome into a synthetic capsid was allowed” (page 30, lines 5-10). Grimm also discloses “The present invention also relates to a host cell comprising the AAV capsid polypeptide according to the present invention, the polynucleotide according to the present invention, and/or the vector according to the present invention for use in treating and/or preventing a muscular disease and/or in muscle regeneration” (page 19, lines 7-10).
Regarding claim 58, Grimm discloses “the AAV capsid polypeptides proposed herein mediate an improved tropism of AAV particles to muscle tissue and muscle cells” (page 15, lines 1-3).
Regarding claim 59, Grimm discloses an adeno-associated virus (AA V) capsid polypeptide modified by insertion of a binding peptide. The binding peptide comprises the amino acid sequence RGDX1X2X3X4 (SEQ ID NO: 1), with X1 to X4 being independently selected amino acids, for use in treating and/or preventing a muscular disease and/or in muscle regeneration (page 1, lines 5-9). Grimm also describes insertion of an RGD motif comprising at least one amino acid upstream of the RGD amino acid sequence (see, e.g., Grimm at Fig. 7). Grimm further describes inserting the RGD X1X2X3X4 (SEQ ID NO: 1) peptide into the AAV9 capsid polypeptide after amino acid residues 588 or 589 (pages 7, lines 28-34), where the AA V9 capsid polypeptide can include additional modifications at positions 504 and 505 (page 9, lines 24-34). The modified AAV9 capsid protein improves tropism of AAV particles to muscle tissue and muscle cells, while at the same time exhibiting a strongly reduced liver tropism (page 15, lines 1-5). Grimm also discloses “the term “muscular disease” is a muscular dystrophy, a cardiomyopathy, a myotonia, a muscular atrophy, a myoclonus dystonia (affected gene: SGCE), a mitochondrial myopathy, a rhabdomyolysis, a fibromyalgia and/or a myofascial pain syndrome”) (page 11, lines 13-18).
Regarding claim 61, Grimm discloses vectors derived from AAV, i.e. gene transfer vehicles using the capsid polypeptide of AAV to mediate the transfer of recombinant polynucleotides into target cells (page 3 lines 29-31). Grimm also discloses “a therapeutically effective does refers to an amount of the polynucleotide to be used in a pharmaceutical composition of the present invention which prevents, ameliorates or treats the symptoms accompanying a disease or condition (page 21, lines 24-26).
Regarding claims 62 and 64, Grimm discloses AAV vectors capable of expressing a micro-dystrophin protein in the muscle cell by stating “AAV vectors have been known to enable transfer and long-term expression of foreign DNA, including therapeutic genes in a variety of cell types (page 1 lines 13-14) and “Preferably, the muscular dystrophy is Duchenne muscular dystrophy (gene affected: DMD), Becker muscular dystrophy (gene affected: DMD), Limb girdle muscular dystrophy (Subtypes and affected genes: LGMD1A (Gene: TTID), LGMD1B (Gene: LMNA), LGMD1C (Gene: CAV3), LGMD1D (Gene: DNAJB6), LGMD1E (Gene: DES), LGMD1F (Gene: TNP03), LGMD1G (Gene: HNRPDL), LGMD1H, LGMD2A (Gene: CAPN3), LGMD2B (Gene: DYSF), LGMD2C (Gene: SGCG), LGMD2D (Gene: SGCA), LGMD2E (Gene: SGCB), LGMD2F (Gene: SGCD), LGMD2G (Gene: TCAP), LGMD2H (Gene: TRIM32), LGMD2I (Gene: FKRP), LGMD2J (Gene: TTN), LGMD2K (Gene: POMT1), LGMD2L (Gene: AN05), LGMD2M (Gene: FKTN), LGMD2N (Gene: POMT2), LGMD20 (Gene: POMGNT1), LGMD2Q (Gene: PLEC1)), Congenital muscular dystrophy, Distal muscular dystrophy (Subtypes and affected genes: Miyoshi myopathy (Gene: DYSF), Distal myopathy with anterior tibial onset (Gene: DYSF), Welander distal myopathy (Gene: TIA1), Gowers-Laing distal myopathy (Gene: MYH7), Nonaka distal myopathy, hereditary inclusion-body myositis type 1, distal myopathy with vocal cord and pharyngeal weakness, ZASP-related myopathy), Facioscapulohumeral muscular dystrophy (Subtypes and affected genes: Type 1 (Gene: DUX4), Type 2 (Gene: SMCHD1)), Oculopharyngeal muscular dystrophy (affected gene: PABPN1), and/or myotonic dystrophy (Subtypes and affected genes: DM1 (Gene: DMPK) and DM2 (Gene: ZNF9)” (page 11 lines 20 – page 12 line 3).
Regarding claims 74, 77 and 78, Grimm discloses “Also describe are polynucleotides, host cells, addeno-asscoiated virus (AAV) capsids, pharmaceutical compositions, uses, and methods related to the AAV capsid polypeptide” (Abstract). Grimm discloses “The composition according to the present specification is a pharmaceutical composition, thus, preferably, the carrier is a pharmaceutically acceptable carrier” (page 21, lines 5-11). Grimm discloses “the pharmaceutical compositions are, preferably, administered topically or systemically” (page 21, lines 5-9) and “a therapeutically effective does refers to an amount of the polynucleotide to be used in a pharmaceutical composition of the present invention which prevents, ameliorates or treats the symptoms accompanying a disease or condition (page 21, lines 24-26). Grimm also discloses “the term “muscular disease” is a muscular dystrophy, a cardiomyopathy, a myotonia, a muscular atrophy, a myoclonus dystonia (affected gene: SGCE), a mitochondrial myopathy, a rhabdomyolysis, a fibromyalgia and/or a myofascial pain syndrome”) (page 11, lines 13-18).
Regarding claim 88, Grimm discloses “During AAV production, HEK293T cells are transfected by an Adenoviral helper plasmid delivering essential genes for the replication of the AAV. Additionally, a plasmid for the expression of rep2 (rep gene of AAV2) and the respective cap gene is needed as well as plasmid carrying the ITR-flanked (inverted terminal repeats), barcoded AAV genome production, a packaging of a defined barcoded genome into a synthetic capsid was allowed” (page 30, lines 5-10).
Regarding claims 89 and 90, Grimm discloses “Also describe are polynucleotides, host cells, addeno-asscoiated virus (AAV) capsids, pharmaceutical compositions, uses, and methods related to the AAV capsid polypeptide” (Abstract). Grimm discloses “The composition according to the present specification is a pharmaceutical composition, thus, preferably, the carrier is a pharmaceutically acceptable carrier” (page 21, lines 5-11). Grimm discloses “the pharmaceutical compositions are, preferably, administered topically or systemically” (page 21, lines 5-9) and “a therapeutically effective does refers to an amount of the polynucleotide to be used in a pharmaceutical composition of the present invention which prevents, ameliorates or treats the symptoms accompanying a disease or condition (page 21, lines 24-26). Grimm also discloses “the term “muscular disease” is a muscular dystrophy, a cardiomyopathy, a myotonia, a muscular atrophy, a myoclonus dystonia (affected gene: SGCE), a mitochondrial myopathy, a rhabdomyolysis, a fibromyalgia and/or a myofascial pain syndrome”) (page 11, lines 13-18).
Therefore, the cited prior art anticipates the claimed invention.
Double Patenting
The nonstatutory double patenting rejection is based on a judicially created doctrine
grounded in public policy (a policy reflected in the statute) so as to prevent the unjustified or
improper timewise extension of the "right to exclude" granted by a patent and to prevent
possible harassment by multiple assignees. A nonstatutory double patenting rejection is
appropriate where the conflicting claims are not identical, but at least one examined
application claim is not patentably distinct from the reference claim(s) because the examined
application claim is either anticipated by, or would have been obvious over, the reference
claim(s). See, e.g., In re Berg, 140 F.3d 1428, 46 USPQ2d 1226 (Fed. Cir. 1998); In re Goodman,
11 F.3d 1046, 29 USPQ2d 2010 (Fed. Cir. 1993); In re Langi, 759 F.2d 887, 225 USPQ 645 (Fed.
Cir. 1985); In re Van Ornum, 686 F.2d 937, 214 USPQ 761 (CCPA 1982); In re Vogel, 422 F.2d
438, 164 USPQ 619 (CCPA 1970); In re Thorington, 418 F.2d 528, 163 USPQ 644 (CCPA 1969).
A timely filed terminal disclaimer in compliance with 37 CFR 1.321(c) or 1.321(d) may be
used to overcome an actual or provisional rejection based on nonstatutory double patenting
provided the reference application or patent either is shown to be commonly owned with the
examined application, or claims an invention made as a result of activities undertaken within
the scope of a joint research agreement. See MPEP § 717.02 for applications subject to examination under the first inventor to file provisions of the AIA as explained in MPEP § 2159.
See MPEP § 2146 et seq. for applications not subject to examination under the first inventor to
file provisions of the AIA . A terminal disclaimer must be signed in compliance with 37 CFR
1.321(b).
The filing of a terminal disclaimer by itself is not a complete reply to a nonstatutory
double patenting (NSDP) rejection. A complete reply requires that the terminal disclaimer be
accompanied by a reply requesting reconsideration of the prior Office action. Even where the
NSDP rejection is provisional the reply must be complete. See MPEP § 804, subsection I.B.1. For
a reply to a non-final Office action, see 37 CFR 1.111(a). For a reply to final Office action, see 37
CFR 1.113(c). A request for reconsideration while not provided for in 37 CFR 1.113(c) may be
filed after final for consideration. See MPEP §§ 706.07(e) and 714.13.
The USPTO Internet website contains terminal disclaimer forms which may be used.
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Claims 1, 3-4, 6-8, 11, 13, 21, 23, 78 and 90 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1, 3, 5, 7, 15-17, 19-20 and 27 of copending Application No. 19/390,032.
Although the claims at issue are not identical, they are not patentably distinct from each other because the claims are coextensive in scope and species with one another. This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
The claims of the present invention are directed to a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety.
The copending claims are directed to:
1. A composition comprising an adeno-associated virus (AAV) which comprises a targeting moiety effective to target a muscle cell and a polynucleotide encoding a cargo polynucleotide wherein the targeting moiety comprises a viral capsid protein comprising an n-mer motif, which comprises XmRGDXn, wherein X is any amino acid, m is 3, and n is 4, and wherein the n-mer motif is (a) inserted between any two native amino acids of the viral capsid protein, (b) replaces one or more native viral capsid protein amino acids, or both (a) and (b) (instant claims 1, 3-4, 6-8, 13).
3. The composition of claim 1, wherein the n-mer motif is inserted in the N or C terminus of any amino acid in amino acids 581 to 593 corresponding to the wild type AAV9 capsid protein (instant claim 11).
5. The composition of claim 1, wherein the n-mer motif is located in the N or C terminus of any amino acid in amino acids 452 and 460 corresponding to the wild type AAV9 capsid protein (instant claim 11).
7. The composition of claim 6, wherein the viral capsid protein comprises an AAVRh74 capsid protein with one, two, or three additional amino acid substitution, insertion, or deletion (instant claim 13).
15. The composition of claim 1, wherein the cargo comprises a micro-dystrophin protein (instant claim 21).
16. The composition of claim 13, wherein the cargo polynucleotide is operably linked to a promoter (instant claim 23).
17. The composition of claim 16, wherein the promoter comprises a muscle-specific promoter (instant claim 23).
19. A method of targeting a muscle cell in a subject in need thereof having a muscular dystrophy comprising administering the composition of claim 1 to the subject, wherein the cargo is expressed in the muscle cell (instant claim 78).
20. A method of targeting a muscle cell in a subject in need thereof having a muscular dystrophy comprising administering the composition of claim 6 to the subject, wherein the cargo is expressed in the muscle cell (instant claim 78).
27. The method of claim 19, wherein the composition is capable of expressing a micro- dystrophin protein in the muscle cell (instant claim 90).
There is no patentable difference between the claimed compositions and methods and the copending claims in application no. 19/390,032. Application no. 19/390,032 discloses A composition comprising an adeno-associated virus (AAV) which comprises a targeting moiety effective to target a muscle cell and a polynucleotide encoding a cargo polynucleotide wherein the targeting moiety comprises a viral capsid protein comprising an n-mer motif, which comprises XmRGDXn, wherein X is any amino acid, m is 3, and n is 4, and wherein the n-mer motif is (a) inserted between any two native amino acids of the viral capsid protein, (b) replaces one or more native viral capsid protein amino acids, or both (a) and (b). Moreover, The MPEP states “where the claimed and prior art products are identical or substantially identical in structure or composition, or are produced by identical or substantially identical processes, a prima facie case of either anticipation or obviousness has been established. In re Best, 562 F.2d 1252, 1255, 195 USPQ 430, 433 (CCPA 1977). This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
Claims 1-8, 13, 23, 32, 35, 37-38, 42, 45, 74, 77-78, 88 and 89-90 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-6, 9, 12-17, 22, 27 and 30 of U.S. Patent No. 11920150 (application no. 17/707,940). Although the claims at issue are not identical, they are not patentably distinct from each other because the claims are coextensive in scope and species with one another.
The claims of the present invention are directed to a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety.
The patented claims are directed to:
1. A composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, wherein the n-mer motif is an RGD motif, wherein the RGD motif has a formula of XmRGDXn, wherein each instance of X is independently selected from any amino acid, m is 0-4 amino acids and n is 1-15 amino acids or m is 1-4 amino acids and n is 0-15 amino acids, wherein the targeting moiety comprises a viral capsid protein, wherein the n-mer motif is (a) inserted between any two contiguous amino acids of the viral capsid protein, (b) replaces one or more native viral capsid protein amino acids, or both (a) and (b); and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety (instant claims 1-4).
2. The composition of claim 1, wherein n is 4 or 5 amino acids (instant claim 1-4).
3. The composition of claim 1, wherein the n-mer motif is any one of SEQ ID NO: 13-50, 1277-2493, 3737-4979, 6647-8313, 8314-8502, or 8692-8889 (instant claims 1-5).
4. The composition of claim 1, wherein the viral capsid protein is an adeno associated virus (AAV) capsid protein (instant claim 1-8).
5. The composition of claim 4, wherein the n-mer motif is located between two amino acids of the viral capsid protein such that the n-mer motif is external to a viral capsid of which the viral capsid protein is part, wherein the n-mer motif is inserted between any two contiguous amino acids between amino acids 262-269, 327-332, 382-386, 452-460, 488-505, 527-539, 545-558, 581-593, 704-714, or any combination thereof, in an AAV9 capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, or AAV rh.10 capsid polypeptide, or is inserted between amino acids 588 and 589 in an AAV9 capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, or AAV rh.10 capsid polypeptide (instant claim 11).
6. The composition of claim 1, wherein the composition is an engineered viral particle, or an engineered viral capsid, optionally an engineered AAV capsid and/or engineered AAV particle, wherein the optionally engineered AAV capsid and/or engineered AAV particle is optionally an engineered AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV rh.74, or AAV rh.10 viral particle or capsid (instant claim 13).
9. The composition of claim 1, wherein the cargo is operably coupled to a muscle specific promoter (instant claim 23).
12. A vector system comprising:
a polynucleotide encoding the composition of claim 1;
optionally a cargo; and
optionally one or more regulatory elements operatively coupled to the polynucleotide encoding a targeting moiety, the cargo, or both (instant claim 32).
13. The vector system of claim 12, wherein n is 4 or 5, the n-mer motif is any one of SEQ ID NO: 13-50, 1277-2493, 3737-4979, 6647-8313, 8314-8502, or 8692-8889, or both (instant claim 35).
14. The vector system of claim 12, wherein the cargo is a cargo polynucleotide, is coupled to one or more of the one or more polynucleotides encoding the targeting moiety, or both (instant claim 37).
15. The vector system of claim 12, wherein the vector system is a viral vector system, optionally an AAV vector system, and is capable of producing virus particles, optionally AAV particles, comprising a viral capsid, optionally an AAV capsid, comprising the targeting moiety and that contain the optional cargo when present (instant claim 38).
16. The vector system of any of claim 15, wherein the AAV particles and/or AAV capsid are engineered AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV rh.74, or AAV rh.10 viral particle (instant claim 42).
17. The vector system of claim 12, wherein at least one of the one or more polynucleotides encoding the n-mer motif(s) is inserted between two codons corresponding to two amino acids of the viral protein such that at least one of the n-mer motifs is external to the viral capsid, optionally wherein the two codons correspond to any two contiguous amino acids between amino acids 262-269, 327-332, 382-386, 452-460, 488-505, 527-539, 545-558, 581-593, 704-714, or any combination thereof, optionally between amino acids 588 and 589, in an AAV9 capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, or AAV rh.10 capsid polypeptide (instant claims 45 and 47).
22. A cell, pharmaceutical formulation, or viral particle, optionally an adeno associated virus (AAV) particle, comprising: the composition of claim 1 (instant claim 74, 77, 88 and 89).
27. A method comprising: administering, to a subject in need thereof, the composition of claim 1 or a pharmaceutical formulation thereof, wherein the wherein the cargo is capable of treating or preventing a muscle disease or disorder, optionally wherein the muscle disease or disorder is an auto immune disease; a cancer; a muscular dystrophy; a neuro-muscular disease; a sugar or glycogen storage disease; an expanded repeat disease; a dominant negative disease; a cardiomyopathy; a viral disease; a progeroid disease; or any combination thereof, and wherein the cargo is optionally a morpholino; a peptide-linked morpholino; an antisense oligonucleotide; a PMO, a therapeutic transgene; a polynucleotide encoding a therapeutic polypeptide or peptide; a PPMO; one or more peptides or polypeptides; one or more polynucleotides encoding a CRISPR-Cas protein, a guide RNA, or both; a ribonucleoprotein, wherein the ribonucleoprotein comprises a CRISPR-Cas system molecule; a therapeutic transgene RNA, or other gene modifying or therapeutic RNA and/or protein; or any combination thereof (instant claims 78 and 90).
30. A method comprising: administering, to a subject in need thereof, a cell, pharmaceutical formulation, or viral particle of claim 22 (instant claims 78 and 90).
There is no patentable difference between the claimed compositions and methods and the patented claims in U.S. Patent no. 11920150. The U.S. Patent no. 11920150 discloses a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety and methods of treating said composition. Moreover, The MPEP states “where the claimed and prior art products are identical or substantially identical in structure or composition, or are produced by identical or substantially identical processes, a prima facie case of either anticipation or obviousness has been established. In re Best, 562 F.2d 1252, 1255, 195 USPQ 430, 433 (CCPA 1977).
Claims 1, 3-5, 7, 13, 15, 32, 78 and 90 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-2, 5, 7-10, 15-16, 19-20 and 22 of copending Application No. 17/707,951. Although the claims at issue are not identical, they are not patentably distinct from each other because the claims are coextensive in scope and species with one another. This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
The claims of the present invention are directed to a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety.
The copending claims are directed to:
1. An engineered adeno-associated virus (AAV) capsid polypeptide comprising: an n-mer motif inserted between amino acids 588-589 of a wild-type AAV-9 capsid polypeptide, or an equivalent position in a wild-type AAV capsid polypeptide of a different serotype, wherein then-mer motif comprises any one of SEQ ID NO: 13-17, 19, 22, 47, 49, 1290-1303, 1305-1312, 1314-1350, 1352-1408, 1410-1472, 1474-1491, 1493-1591, 1593- 1636, 1638-1639, 1641-1668, 1670-1673, 1675-1729, 1731-1755, 1757-1918, 1920-1936, 1938-2015,2017-2033,2035-2109,2111-2128,2130-2134,2136-2178,2180-2304,2306-2432,2434-2436,2438-2493,3749,3451,3753-3760,3762-3764,3766-3803,3805-3815, 3817-3822,3825-3831,3833-3843,3845-3856,3858-3864,3866-3879,3881-3893,3895-3946,3950-3960,3962-3985,3987-4007,4009-4011,4013,4015-4025,4028-4029,4031-4047,4049-4051,4053-4054,4056-4058,4061-4063,4065-4070,4072-4080,4082-4092, 4094-4097,4099-4128,4130,4132-4139,4141-4154,4156-4158,4160,4162-4173,4175-4177,4179,4181-4182,4184,4186-4187,4189-4204,4206-4209,4212-4213,4215-4216, 4220-4224,4226-4256,4258-4259,4262-4270,4272-4276,4278-4289,4291-4299,4301-
4303, 4305-4310, 4312-4315, 4317-4328, 4330-4331, 4333-4339, 4341-4345, 4347-4378,
4380-4420,4423-4430,4433-4434,4436-4437,4439-4442,4444-4450,4452-4455,4457,
4459-4466,4468-4474,4476-4484,4486-4504,4506-4508,4510-4522,4524-4534,4538-
4569, 4571-4596, 4598-4601, 4603-4608, 4610-4637, 4639-4649, 4651-4652, 4654-4703,
4706-4729,4731-4733,4735-4738,4740-4749,4751,4753,4755-4762,4764-4776,4778,
4780-4801,4803-4821,4823-4848,4850-4878,4880-4901,4904-4918,4920-4922,4924-
4928,4930-4944,4946-4947,4949-4974,4976-4979,6824,6826-6827,6829,6835,6838,
6849, 6854, 6860, 6865, 6875, 6889, 6921, 6923-6924, 6952, 6973, 7012, 7020, 7035,
7062, 7094, 7100-7101, 7109, 7118, 7124-7125, 7134, 7143, 7152, 7180, 7184, 7187- 7188, 7194-7195, 7198, 7213, 7217, 7220, 7229, 7238, 7240, 7245, 726~ 7276, 7277,7292, 7306, 7323-7324, 7332, 7337, 7339, 7352, 7367, 7371, 7375, 7387, 7390, 7395, 7397, 7412, 7414, 7422, 7452-7453, 7455, 7459, 7464, 7471, 7477, 7479, 7482, 7484, 7490, 7496, 7507, 7520, 7526, 7530, 7532, 7539, 7549, 7553, 7567, 7572, 7585, 7594, 7603, 7608, 7620-7622, 7627, 7634-7635, 7641, 7650, 7654, 7656, 7664, 7678, 7683,
7695, 7697, 7701, 7721, 7725-7726, 7728, 7741, 7785, 7795, 7809, 7823-7824, 7826- 7827, 7829-7830, 7837-7838, 7848, 7850, 7860, 7869, 7877, 7885-7886, 7889, 7909, 7915, 7919, 7921-7922, 7927, 7931-7932, 7936, 7938-7940, 7947-7948, 7953, 7958, 7967, 7972, 7981, 7988, 7993, 7996-7999, 8004, 8007, 8016, 8018-8019, 8023-8024, 8028, 8034,8037,8039,8046,8048,8052-8053,8056,8063-8064,8066-8067,8075-8076,8079, 8086, 8089-8092, 8095, 8098-8099, 8105, 8108-8109, 8118, 8120, 8124, 8126, 8130, 8134, 8141, 8144, 8148-8151, 8154-8155, 8167-8168, 8173, 8176, 8178, 8182, 8185, 8189, 8190, 8192, 8197-8199, 8203, 8206, 8208, 8210-8211, 8217, 8220, 8223, 8225, 8227-8228, 8231, 8233-8235, 8240, 8242, 8248, 8253-8255, 8257, 8259, 8264, 8266, 8268, 8271, 8275, 8277, 8279, 8283-8288, 8294, 8300, 8302-8307, 8310-8312,or 8313 (instant claims 1 and 3-5).
2. The engineered AAV capsid polypeptide of claim 1, wherein the n-mer motif comprises one of SEQ ID NO: 13-17, 19, 22, 47, 49, 1291, 1301, 1354, 1363, 1375, 1427, 1435, 1593, 1657, 1673, 1749, 1761, 1791, 1915, 3748, 3766, 3788, 3806, 4013, 4083,4213, 4245, 7414, or 7620 (instant claim 5).
5. The engineered AAV capsid polypeptide of claim 1, wherein the wild-type AAV capsid polypeptide is selected from the group consisting of an AAV-1, AAV-2, AAV-3, AAV-4, AAV-5, AAV-6, AAV-7, AAV-8, AAV-9, AAV rh.74, and an AAV rh.10 capsid polypeptide (instant claim 13).
7. The engineered AAV capsid polypeptide of claim 1, wherein the engineered capsid polypeptide is capable of conferring muscle tropism to a capsid or a viral particle (instant claims 1 and 7).
8. An engineered viral capsid comprising one or more engineered AAV capsid polypeptides of claim 1 (instant claim 1).
9. An engineered viral particle comprising the engineered viral capsid of claim 8 (instant claim 1).
10. The engineered viral particle of claim 9, wherein the n-mer motif comprises any one of SEQ ID NO: 13-17, 19, 22, 47, 49, 1291, 1301, 1354, 1363, 1375, 1427, 1435, 1593, 1657, 1673, 1749, 1761, 1791, 1915, 3748, 3766, 3788, 3806, 4013, 4083, 4213, 4245, 7414, or 7620 (instant claims 1-5).
15. The engineered viral particle of claim 9, wherein the engineered viral particle has muscle tropism (instant claims 1 and 15).
16. The engineered viral particle of claim 9, wherein the engineered virus particle further comprises a cargo, wherein the cargo comprises a polynucleotide, a polypeptide, or both (instant claim 1).
19. A polynucleotide encoding the engineered AAV capsid polypeptide of claim 1 (instant claims 1 and 7).
20. A vector system comprising one or more vectors, wherein at least one of the one or more vectors comprises the polynucleotide of claim 19 (instant claim 32).
22. A method of delivering a therapeutic or preventative treatment to a subject, comprising administering to the subject the engineered viral particle of claim 16 to the subject (instant claims 78 and 90).
There is no patentable difference between the claimed compositions and methods and the patented claims in copending Application no. 17/707,951. The copending Application no. 17/707,951 discloses a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety and methods of treating said composition. Moreover, The MPEP states “where the claimed and prior art products are identical or substantially identical in structure or composition, or are produced by identical or substantially identical processes, a prima facie case of either anticipation or obviousness has been established. In re Best, 562 F.2d 1252, 1255, 195 USPQ 430, 433 (CCPA 1977).
Claims 1, 3-4, 6-8, 11, 13, 32-34, 37, 42, 44-45, 52 and 78 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1, 3-4, 7, 10, 44, 47-49, 56, 78 and 104-105 of copending Application No. 18/294,594. Although the claims at issue are not identical, they are not patentably distinct from each other because the claims are coextensive in scope and species with one another. This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
The claims of the present invention are directed to a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety.
The copending claims are directed to:
1. A composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises one or more n-mer motif, wherein at least one n-mer motif of the one or more n-mer motifs comprises or consists of XmRGDXn, wherein Xm and Xn are each independently selected from any amino acid, wherein n is 1, 2, 3, 4, 5, 6, 7, 8, or 9, and wherein m is 1-4, optionally wherein then-mer motif is 3-15 amino acids; and optionally a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety (instant claims 1 and 3-4).
3. The composition of claim 1, wherein the targeting moiety comprises a
polypeptide, a polynucleotide, a lipid, a polymer, a sugar, or a combination thereof (instant claims 1 and 6).
4. The composition of claim 1, wherein the targeting moiety comprises a viral protein, optionally wherein the viral protein is a capsid protein, wherein the viral protein is an adeno-associated virus (AAV) protein, or both (instant claims 1 and 7-8).
7. The composition of claim 4, wherein then-mer motif is located between two amino acids of the viral protein such that then-mer motif is external to a viral capsid, optionally wherein the n-mer motif is inserted between any two contiguous amino acids between amino acids 262-269, 327-332, 382-386, 452-460, 488-505, 527-539, 545-558, 581-593, 704-714, or any combination thereof in an AAV9 capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, AAV rh.10 capsid polypeptide; or optionally wherein then-mer motif is inserted between amino acids 588 and 589 in an AAV9 capsid polypeptide or in an analogous position in an AAVI, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAVrh.74, AAVrh.10 capsid polypeptide (instant claim 11).
10. The composition of claim 1, wherein the composition 1s an engineered viral particle, optionally wherein the engineered viral particle is an engineered AAV viral particle, or optionally wherein the engineered viral particle is an engineered AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV rh.74, or AAVrh. l 0 viral particle (instant claim 13).
44. A vector system comprising: a vector comprising: one or more polynucleotides each encoding all or part of one or more targeting moieties effective to target a muscle cell, wherein each targeting moiety comprises one or more n-mer motifs, wherein at least one n-mer motif of the one or more n-mer motifs comprises or consists of XmRGDXn, wherein Xm and Xn are each independently selected
from any amino acid, wherein n is 1, 2, 3, 4, 5, 6, 7, 8, or 9, and wherein mis 1-4, and wherein at least one of the one or more polynucleotides at least encodes the at least one n-mer motif; and optionally, a regulatory element operatively coupled to one or more of the polynucleotide(s) (instant claim 32-34).
47. The vector system of claim 46, wherein the cargo is a cargo polynucleotide and is optionally coupled to one or more of the one or more polynucleotides encoding the targeting moiety, optionally wherein the cargo polynucleotide is present on the same vector or a different vector than the one or more polynucleotides encoding the targeting moiety (instant claims 32 and 37).
49. The vector system of claim 44, wherein the vector system is capable of producing (a) virus particles that optionally contain a cargo when present; (b) a viral polypeptide comprising one or more of the targeting moieties, optionally wherein the viral polypeptide is a capsid polypeptide or an AAV capsid polypeptide, optionally wherein the capsid polypeptide or AAV capsid polypeptide is an engineered AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh.74, AAVrh.10 capsid polypeptide; (c) AA V virus particles, optionally wherein the AAV viral particles are engineered AAV1, AAV2 AAV3 AAV4 AAV5 AAV6 AAV7 AAV8 AAV9 AAVrh.74 or AAVrh.10 viral particles or (d) any combination of (a)-(c) (instant claim 42).
56. The vector system of claim 49, wherein the one or more polynucleotides encoding one n-mer motifs is inserted between two codons corresponding to two amino acids of the viral polypeptide such that the n-mer motif(s) is external to the viral capsid, optionally wherein the one or more polynucleotides encoding one or more n-mer motifs is/are inserted between two codons corresponding to any two contiguous amino
acids between amino acids 262-269, 327-332, 382-386, 452-460, 488-505, 527-539, 545- 558, 581-593, 704-714, or any combination thereof in an AAV9 capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, AAV rh.10 capsid polypeptide; or
optionally wherein the one or more polynucleotides encoding one or more n-mer motifs is/are inserted between the codons corresponding to amino acid 588 and 589 in the AAV9 capsid polynucleotide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, AAV rh.10 capsid polypeptide (instant claims 44-45).
78. A polypeptide encoded by and/or produced by a vector system of claim 44, optionally wherein the polypeptide is a viral polypeptide or an AAV polypeptide, and optionally wherein the polypeptide has increased muscle cell potency, muscle cell specificity, reduced immunogenicity, or any combination thereof (instant claim 52).
104. A method comprising: administering, to a subject in need thereof, a
a. a composition as in claim 1;
b. a vector system encoding the composition or capable of producing the composition;
c. a polypeptide encoded by or produced by the vector system;
d. a particle comprising the composition, the vector system, the polypeptide or any
combination thereof or produced by the vector system;
e. a cell comprising (a), (b), (c), (d), or any combination thereof;
f. pharmaceutical formulation comprising (a), (b), (c), (d), (e), or any combination
thereof and a pharmaceutically acceptable carrier; or
g. any combination thereof (instant claim 78).
105. The method of claim 104, wherein the subject has a muscle disease or disorder, optionally wherein the muscle disease or disorder is (a) an auto immune disease; (b) a cancer; (c) a muscular dystrophy; (d) a neuro-muscular disease; (e) a sugar or glycogen storage disease; (f) an expanded repeat disease; (g) a dominant negative disease; (h) a cardiomyopathy; (i) a viral disease; (j) a progeroid disease; or any
combination thereof (instant claim 78).
There is no patentable difference between the claimed compositions and methods and the patented claims in copending Application no. 18/294,594. The copending Application no. 18/294,594 discloses a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety and methods of treating said composition. Moreover, The MPEP states “where the claimed and prior art products are identical or substantially identical in structure or composition, or are produced by identical or substantially identical processes, a prima facie case of either anticipation or obviousness has been established. In re Best, 562 F.2d 1252, 1255, 195 USPQ 430, 433 (CCPA 1977).
Claims 1, 3, 6-8, 11, 13, 32-33, 37, 39, 42, 44-45, 52, 59, 78 and 90 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1, 7, 10-11, 13-14, 16, 32, 48, 54, 57, 59-61, 63, 66-67, 89, 98 and 116-117 of copending Application No. 18/689897. Although the claims at issue are not identical, they are not patentably distinct from each other because the claims are coextensive in scope and species with one another. This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
The claims of the present invention are directed to a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety.
The copending claims are directed to:
1. A composition comprising: a targeting moiety effective to target a muscle cell or both a muscle cell and a central nervous system (CNS) cell, wherein the targeting moiety comprises one or more n-mer inserts each comprising: one or more P-motifs, wherein at least one P-motif comprises or consists of the amino acid sequence XmPX1QGTX2RXn (SEQ ID NO: 1699), wherein X1, X2, Xm, and Xn are each independently selected from any amino acid, wherein m is 0, 1, 2, or 3, and wherein n is 0, 1, 2, 3, 4, 5, 6, or 7; or one or more RGD motifs, wherein at least one of the RGD motifs comprises or
consists of XmRGDXn, wherein m is 0-4 amino acids, wherein n is 0-15 amino acids, and wherein Xm, and Xn are each independently selected from any amino acid; or both, and optionally a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety; and optionally wherein the targeting moiety is effective target (a) a skeletal muscle ell, (b) a cardiac muscle cell, (c) a skeletal muscle cell and a CNS cell, or (d) a cardiac muscle cell and a CNS cell (instant claim 1).
7. The composition of claim 1, wherein (a) the one or more n-mer inserts are each 3-25 or 3-15 amino acids in length; (b) (i) X1 is S, T, or A, (ii) X2 is L, V, F, or I, or (iii) both (i) and (ii); (c) the one or more RGD motifs and/or one or more P-motifs is/are immediately preceded by AQ or DG in the targeting moiety; or (d) any combination of (a)-(c) (instant claim 3).
10. The composition of claim 1, wherein the targeting moiety comprises a polypeptide, a polynucleotide, a lipid, a polymer, a sugar, or a combination thereof (instant claim 6).
11. The composition of claim 1, wherein the targeting moiety comprises a viral protein, optionally wherein the viral protein is a capsid protein. (instant claims 7-8).
13. The composition of claim 11, wherein one or more of the n-mer inserts are incorporated into the viral protein such that at least one of the one or more RGD motifs, at least one of the one or more P motifs, or both is/are located between two amino acids of the viral protein such that at least one of the one or more RGD motifs and/or one or more P-motifs is external to a viral capsid (instant claim 8).
14. The composition of claim 11, wherein the viral protein is an adeno-associated virus (AAV) protein, optionally wherein the AAV protein is an AAV capsid protein (instant claim 7).
16. The composition of claim 14, wherein one or more of the one or more n-mer inserts are incorporated into the AAV protein such that at least one or more of the one more RGD motifs and/or at least one or more of the one or more P motifs are each inserted between any two contiguous amino acids independently selected from amino acids 262-269, 327-332, 382-386, 452-460, 488-505, 527-539, 545-
558, 581-593, 598-599, 704-714, or any combination thereof in an AAV9 capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, AAV rh.10 capsid polypeptide; or wherein at least one of the one or more n-mer inserts is incorporated into the AA V
protein such that at least one of the one more RGD motifs and/or at least one of the one or
more P motifs is inserted between amino acids 588 and 589 in an AA V9 capsid polypeptide
or in an analogous position in an AAV1, AA V2, AA V3, AA V 4, AA VS, AA V6, AA V7,
AAV8, AAV rh.74, AAV rh.10 capsid polypeptide (instant claim 11).
32. The composition of claim 1, wherein the composition is an engineered viral particle, optionally wherein the engineered viral particle is an engineered AAV viral particle, optionally an engineered AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV rh.74, or AAV rh.10 viral particle (instant claim 13).
48. A vector system comprising: a vector comprising: one or more polynucleotides, wherein at least one of the one or more polynucleotides encodes all or part of a targeting moiety effective to target a muscle cell or both a muscle cell and a central nervous system (CNS) cell, wherein the targeting moiety comprises one or more n-mer inserts comprising: one or more P-motifs, wherein at least one P-motif comprises or consists of the amino acid sequence XmPX1QGTX2RXn (SEQ ID NO: 1699), wherein X1, X2, Xm, and Xn are each independently selected from any amino acid, wherein mis 0, 1, 2, or 3, and wherein n is 0, 1, 2, 3, 4, 5, 6, or 7; or one or more RGD motifs, wherein at least one of the RGD motifs comprises or consists of XmRGDXn, wherein m is 0-4 amino acids, wherein n is 0-15 amino acids, and wherein Xm, and Xn are each independently selected from any amino acid; or
both, wherein at least one of the one or more polynucleotides encodes the at least one RGD
motif, at least one P-motif, or both; and optionally, a regulatory element operatively coupled to one or more of the one or more polynucleotides; and optionally wherein the targeting moiety is effective target (a) a skeletal muscle ell, (b) a cardiac muscle cell, (c) a skeletal muscle cell and a CNS cell, or (d) a cardiac muscle cell and a CNS cell (instant claim 32).
54. The vector system of claim 48, wherein (a) the one or more n-mer inserts are each 3-25 or 3-15 amino acids in length; (b) (i) X1 is S, T, or A, (ii) X2 is L, V, F, or I, or (iii) both (i) and (ii); (c) the one or more RGD motifs and/or one or more P-motifs is/are immediately preceded by AO or DG in the targeting moiety; or (d) any combination of (a)-(c) (instant claim 33).
57. The vector system of claim 48, further comprising a cargo, optionally wherein the cargo is a cargo polynucleotide and is optionally operatively coupled to one or more of the one or more polynucleotides encoding all or part of the targeting moiety (instant claim 37).
59. The vector system of claim 48, wherein the vector system is capable of producing virus particles, virus particles that contain a cargo, or both, optionally wherein the virus particles are adeno-associated virus (AAV) particles, optionally engineered AAV1, AAV2, AAV3, AAV 4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV rh.74, or AAV rh.10 particles (instant claims 39 and 42).
60. The vector system of claim 48, wherein the vector system is capable of producing a polypeptide comprising one or more of the targeting moieties (instant claim 39).
61. The vector system of claim 60, wherein the polypeptide is a viral
polypeptide, optionally wherein the viral polypeptide is a capsid polypeptide, optionally an
adeno associated virus (AAV) capsid polypeptide (instant claims 39 and 42).
63. The vector system of claim 61, wherein the capsid polypeptide is an engineered AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh.74, or AAV rh.10 polypeptide (instant claim 42).
66. The vector system of claim 61, wherein one or more of the one or more n-mer inserts are incorporated in the targeting moiety such that at least one of the one or more RGD motifs, at least one of the one or more P motifs, or both is/are located between two amino acids of the viral protein such that at least one of
the one or more RGD motifs and/or at least one or more P-motifs is external to a viral
capsid of the virus particles (instant claim 44).
67. The vector system of claim 61, wherein one or more of the one or more n-mer inserts are incorporated into the AAV protein capsid polypeptide such that at least one or more of the one more RGD motifs and/or at least one or more of the one or more P motifs are each inserted between any two contiguous amino acids independently selected from amino acids 262-269, 327-332, 382-386, 452-460, 488-505, 527-539, 545-558, 581-593, 598-599, 704-714, or any combination thereof in an AAV9 capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, AAV rh.10 capsid polypeptide; or wherein at least one of the one or more n-mer inserts is incorporated into the AA V capsid polypeptide such that at least one of the one more RGD motifs and/or at least one of the one or more P motifs is inserted between amino acids 588 and 589 in the AA V9 capsid polynucleotide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, AAV rh.10 capsid polypeptide (instant claim 45).
89. A polypeptide encoded, produced, or both by a vector system or a particle produced by a vector system as in claim 48, optionally wherein the polypeptide is a viral polypeptide, optionally an AAV polypeptide, optionally wherein the polypeptide is coupled to or otherwise associated with a cargo, optionally wherein the particle is a viral particle optionally wherein the viral particle is an AAV particle, a
lentiviral particle, or a retroviral particle, and optionally wherein the particle comprises the polypeptide, the cargo, or both, optionally wherein the viral particle has a muscle tropism, or a muscle and central nervous system (CNS) tropism, and optionally wherein the polypeptide, the particle, or both have increased muscle cell potency, muscle cell specificity, reduced immunogenicity, or any combination thereof (instant claim 52).
98. The vector system of claim 48, the polypeptide encoded, produced, or both by the vector system, or a particle produced by the vector system optionally wherein the polypeptide is a viral polypeptide, optionally an AAV polypeptide, optionally wherein the polypeptide is coupled to or otherwise associated with a cargo, optionally wherein the particle is a viral particle optionally wherein the viral particle is an AA V particle, a lentiviral particle, or a retroviral particle, and optionally wherein the particle comprises the polypeptide, the cargo, or both, and optionally wherein the viral particle has a muscle tropism, or a muscle and central nervous system (CNS) tropism, wherein the cargo is capable or preventing a CNS disease or, a muscle disease or disorder, or both a CNS and muscle disease or disorder (instant claim 59).
116. A method of treating a muscle disease, disorder, or a symptom thereof, or both a muscle and a central nervous system disease, disorder, or a symptom thereof comprising: administering, to the subject in need thereof, (a) a composition as in claim 1; (b) a polynucleotide encoding the composition;
(c) a vector system comprising the polynucleotide; (d) a polypeptide encoded, produced, or both by the polynucleotide, the vector system, or both; (e) a particle produced by the vector system, comprising the polypeptide or both; (f) a cell comprising (a), (b), (c), (d), (e), or any combination thereof; or (g) a pharmaceutical formulation comprising (a), (b), (c), (d), (e), (f) or any combination thereof and a pharmaceutically acceptable carrier; or (h) any combination thereof; and a pharmaceutical acceptable carrier (instant claim 59).
117. (Currently Amended) The method of claim 116, wherein (a) the central nervous system disease or disorder comprises a secondary muscle disease, disorder, or symptom thereof (b) the central nervous system disease or disorder is Friedreich's Ataxia, Dravet Syndrome, Spinocerebellar Ataxia Type 3, Niemann Pick Type C, Huntington's Disease, Pompe Disease, Myotonic Dystrophy Type 1, Glutl Deficiency Syndrome (De Vivo Syndrome), Tay-Sachs, Spinal Muscular Atrophy, Alzheimer's disease, Amyotrophic lateral sclerosis (ALS), Danon disease, Rett Syndrome, Angleman Syndrome, or a
combination thereof; (c) the CNS or muscle disease or disorder is an auto immune disease; a cancer; a
muscular dystrophy; a neuro-muscular disease; a sugar or glycogen storage disease; an
expanded repeat disease; a dominant negative disease; a cardiomyopathy; a viral disease;
a progeroid disease; or any combination thereof, optionally wherein (i) the expanded repeat
disease is Huntington's disease, a Myotonic Dystrophy, or Facioscapulohumeral muscular
dystrophy (FSHD); (ii) the muscular dystrophy is Duchene muscular dystrophy, Becker
Muscular dystrophy, a Limb-Girdle muscular dystrophy, an Emery-Dreifuss muscular
dystrophy, a myotonic dystrophy, or FSHD, optionally wherein the myotonic dystrophy is
Type 1 or Type 2;(iii) the cardiomyopathy is dilated cardiomyopathy, hypertrophic
cardiomyopathy, DMD-associated cardiomyopathy, or Dannon disease; (iv) the sugar or
glycogen storage disease is a MPS type III disease or Pompe disease, optionally wherein
the MPS type III disease, is MPS Type IIIA, IIIB, IIIC, or IIID; (v) the neuro-muscular
disease is Charcot-Marie-Tooth disease or Friedreich's Ataxia; or (vi) any combination of
(i)-(v); or (d) any combination of (a)-(c) (instant claim 78 and 90).
There is no patentable difference between the claimed compositions and methods and the patented claims in copending Application no. 18/689,897. The copending Application no. 18/689,897 discloses a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety and methods of treating said composition. Moreover, The MPEP states “where the claimed and prior art products are identical or substantially identical in structure or composition, or are produced by identical or substantially identical processes, a prima facie case of either anticipation or obviousness has been established. In re Best, 562 F.2d 1252, 1255, 195 USPQ 430, 433 (CCPA 1977).
Claims 1, 3, 6-8, 11, 13, 18, 21, 23, 32-34, 52, 58-59, 61, 62, 64 and 78 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1, 7, 9, 16-18 and 20 of copending Application No. 19/390,070. Although the claims at issue are not identical, they are not patentably distinct from each other because the claims are coextensive in scope and species with one another. This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
The claims of the present invention are directed to a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety.
The copending claims are directed to:
1. A composition comprising an adeno-associated virus (AAV) which comprises a targeting moiety effective to target a muscle cell and a polynucleotide encoding a cargo (cargo polynucleotide), wherein the targeting moiety comprises a viral capsid protein comprising an n-mer motif; which comprises XmRGDXn, wherein X is any amino acid, m is 1 or 2, and n is 4, and wherein the n-mer motif is (a) inserted between any two amino acids of the viral capsid protein, (b) replaces one or more native viral capsid protein amino acids, or both (a) and (b) (instant claims 1, 3, 6-8, 32-34, 52, 58-59, 61).
7. The composition of claim 1, wherein the n-mer motif is located at the N or C terminal of any amino acid in amino acids 452 and 460 corresponding to the wild type AAV9 capsid protein (instant claim 11).
9. The composition of claim 4, wherein the viral capsid protein comprises an AAVRh74 capsid protein with one, two, or three amino acid substitution, insertion, or deletion (instant claim 13).
16. The composition of claim 1, wherein the cargo comprises a micro-dystrophin protein (instant claims 18, 21, 62, 64).
17. The composition of claim 16, wherein the cargo polynucleotide is operably linked to a promoter (instant claim 23).
18. The composition of claim 17, wherein the promoter comprises a muscle-specific promoter (instant claim 23).
20. A method of targeting a muscle cell in a subject in need thereof having a muscular dystrophy comprising administering the composition of claim 1 to the subject, wherein the cargo is expressed in the muscle cell (instant claim 78).
There is no patentable difference between the claimed compositions and methods and the patented claims in copending Application no. 19/390,070. The copending Application no. 19/390,070 discloses a composition comprising: a targeting moiety effective to target a muscle cell, wherein the targeting moiety comprises an n-mer motif, optionally wherein the n-mer motif comprises an RGD motif or a non-RGD n-mer motif; and a cargo, wherein the cargo is coupled to or is otherwise associated with the targeting moiety and methods of treating said composition. Moreover, The MPEP states “where the claimed and prior art products are identical or substantially identical in structure or composition, or are produced by identical or substantially identical processes, a prima facie case of either anticipation or obviousness has been established. In re Best, 562 F.2d 1252, 1255, 195 USPQ 430, 433 (CCPA 1977).
Conclusion
No claims are allowed.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to Barry Chestnut whose telephone number is (571)270-3546. The examiner can normally be reached on M-Th 8:00 to 4:00.
If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Thomas Visone can be reached on 571-270-0684. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
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/BARRY A CHESTNUT/Primary Examiner, Art Unit 1672
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