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 (no argument presented) of species in response to species election in the reply filed on 3/3/2026 is acknowledged.
In light of the Examiner' s search, the requirement for election of a single species from each species group is hereby removed. Examiner has extended the search to include all species. No claims are withdrawn.
Status of Claims
Claims 1-14 are under consideration.
Priority
This application claims priority to provisional application 63/387,733 and 63/364,260. Therefore, it is entitled to the 5th May 2022 priority date of the earliest provisional application.
Nucleotide and/or Amino Acid Sequence Disclosures
Summary of Requirements for Patent Applications Filed On Or After July 1, 2022, That Have Sequence Disclosures
37 CFR 1.831(a) requires that patent applications which contain disclosures of nucleotide and/or amino acid sequences that fall within the definitions of 37 CFR 1.831(b) must contain a “Sequence Listing XML”, as a separate part of the disclosure, which presents the nucleotide and/or amino acid sequences and associated information using the symbols and format in accordance with the requirements of 37 CFR 1.831-1.835. This “Sequence Listing XML” part of the disclosure may be submitted:
1. In accordance with 37 CFR 1.831(a) using the symbols and format requirements of 37 CFR 1.832 through 1.834 via the USPTO patent electronic filing system (see Section I.1 of the Legal Framework for Patent Electronic System (https://www.uspto.gov/PatentLegalFramework), hereinafter “Legal Framework”) in XML format, together with an incorporation by reference statement of the material in the XML file in a separate paragraph of the specification (an incorporation by reference paragraph) as required by 37 CFR 1.835(a)(2) or 1.835(b)(2) identifying:
a. the name of the XML file
b. the date of creation; and
c. the size of the XML file in bytes; or
2. In accordance with 37 CFR 1.831(a) using the symbols and format requirements of 37 CFR 1.832 through 1.834 on read-only optical disc(s) as permitted by 37 CFR 1.52(e)(1)(ii), labeled according to 37 CFR 1.52(e)(5), with an incorporation by reference statement of the material in the XML format according to 37 CFR 1.52(e)(8) and 37 CFR 1.835(a)(2) or 1.835(b)(2) in a separate paragraph of the specification identifying:
a. the name of the XML file;
b. the date of creation; and
c. the size of the XML file in bytes.
SPECIFIC DEFICIENCIES AND THE REQUIRED RESPONSE TO THIS NOTICE ARE AS FOLLOWS:
Specific deficiency - Sequences appearing in the specification are not identified by sequence identifiers (i.e., “SEQ ID NO:X” or the like) in accordance with 37 CFR 1.831(c).
Required response – Applicant must provide:
A substitute specification in compliance with 37 CFR 1.52, 1.121(b)(3), and 1.125 inserting the required sequence identifiers, consisting of:
• A copy of the previously-submitted specification, with deletions shown with strikethrough or brackets and insertions shown with underlining (marked-up version);
• A copy of the amended specification without markings (clean version); and
• A statement that the substitute specification contains no new matter.
Each nucleic acid sequence with 10 or more specifically defined and enumerated nucleotides must have a SEQ ID NO.
The sequences in question are an embodiment of instant invention and disclosed in [0032] as TEG-5'-gguaaguuc*uguc*c*aagc*-3', where c* is 5-methylcytosine, g and c* are LNA nucleotides.
Abstract
Applicant is reminded of the proper content of an abstract of the disclosure.
A patent abstract is a concise statement of the technical disclosure of the patent and should include that which is new in the art to which the invention pertains. The abstract should not refer to purported merits or speculative applications of the invention and should not compare the invention with the prior art.
If the patent is of a basic nature, the entire technical disclosure may be new in the art, and the abstract should be directed to the entire disclosure. If the patent is in the nature of an improvement in an old apparatus, process, product, or composition, the abstract should include the technical disclosure of the improvement. The abstract should also mention by way of example any preferred modifications or alternatives.
Where applicable, the abstract should include the following: (1) if a machine or apparatus, its organization and operation; (2) if an article, its method of making; (3) if a chemical compound, its identity and use; (4) if a mixture, its ingredients; (5) if a process, the steps.
Extensive mechanical and design details of an apparatus should not be included in the abstract. The abstract should be in narrative form and generally limited to a single paragraph within the range of 50 to 150 words in length.
See MPEP § 608.01(b) for guidelines for the preparation of patent abstracts.
In the instant case:
the abstract sounds like a title.
Is less than 50 words.
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.
Claims 1-14 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.
Regarding claim 1, the term " AON1" in claim 1 renders the claim indefinite because the specification does not disclose what the structure of AON1 is. The specification in Example 1 in [0110] discloses: MW of AON1 is 6517 g/mol. However, the specification does not disclose what the SEQ ID NO of AON1 is. The sequence listing discloses two sequences, SEQ ID 1 and 2. However, it is still unknown if AON1 is either one of these, or any other sequence, or modified versions thereof. Thus, one of ordinary skill in the art would not be reasonably apprised of the scope of the invention. Since the structure of AON1 is indefinite, the claim as a whole is indefinite.
Claim 14 recites various parenthetical expressions. The metes and bounds of claim 14 are rendered vague and indefinite by these various parenthetical recitations; for e.g., “(Sarepta Therapeutics)”. Such parenthetical recitations are indefinite because it is unclear as to whether the limitations are part of the instantly claimed subject matter.
Regarding claim 14 again, the phrase "such as" in “a corticosteroid such as deflazacort,...” renders the claim indefinite because it is unclear whether the limitations following the phrase are part of the claimed invention. See MPEP § 2173.05(d).
Those claims identified in the statement of rejection but not explicitly referenced in the rejection are also rejected for depending from a rejected claim but failing to remedy the indefiniteness therein.
Claim 14 is rejected on the basis that it contains an improper Markush grouping of alternatives. See In re Harnisch, 631 F.2d 716, 721-22 (CCPA 1980) and Ex parte Hozumi, 3 USPQ2d 1059, 1060 (Bd. Pat. App. & Int. 1984). A Markush grouping is proper if the alternatives defined by the Markush group (i.e., alternatives from which a selection is to be made in the context of a combination or process, or alternative chemical compounds as a whole) share a “single structural similarity” and a common use. A Markush grouping meets these requirements in two situations. First, a Markush grouping is proper if the alternatives are all members of the same recognized physical or chemical class or the same art-recognized class, and are disclosed in the specification or known in the art to be functionally equivalent and have a common use. Second, where a Markush grouping describes alternative chemical compounds, whether by words or chemical formulas, and the alternatives do not belong to a recognized class as set forth above, the members of the Markush grouping may be considered to share a “single structural similarity” and common use where the alternatives share both a substantial structural feature and a common use that flows from the substantial structural feature. See MPEP § 2117.
The Markush grouping of eteplirsen, casimersen, golodirsen, viltolarsen, SRP-5051 (Sarepta Therapeutics), a corticosteroid such as deflazacort, a gene therapy (e.g., SRP-9001, GALGT2 or GNT 0004 (Sarepta Therapeutics)), gene editing (e.g., CRISPR/CAS9 (Sarepta Therapeutics)) or a cellular therapy (e.g., CAP- 1002 (Capricor Therapeutics/Nippon Shinyaku Co. Ltd.)) is improper because the alternatives defined by the Markush grouping do not share both a single structural similarity and a common use for the following reasons: The first set of species in the Markush group are modified antisense oligonucleotides, while the latter is not an oligonucleotide at all, rather, as recited, a cell.
To overcome this rejection, Applicant may set forth each alternative (or grouping of patentably indistinct alternatives) within an improper Markush grouping in a series of independent or dependent claims and/or present convincing arguments that the group members recited in the alternative within a single claim in fact share a single structural similarity as well as a common use.
Claim Interpretation
The claims are drawn to a method of treating DMD with AON1. AON1 is not defined in the disclosure. In light of the following descriptions in the disclosure, AON1 is being interpreted as a modified antisense oligonucleotide for treating or delaying the onset of Duchenne muscular dystrophy:
Abstract: Provided herein are methods of treating or delaying the onset of Duchenne muscular dystrophy using modified antisense oligonucleotides.
Specification:
II. AONS FOR USE IN THE METHODS PROVIDED HEREIN
[0031] In one embodiment, the AONs for use in the methods provided herein are reverse complementary to a portion of exon 51 of human dystrophin pre-mRNA. In another embodiment, the AONs for use in the methods provided herein have the sequence 5'gguaaguucuguccaagc- 3' (SEQ ID NO: 1) and contain a modification. In another embodiment, the AONs for use in the methods provided herein have the sequence 5'gguaaguuc* uguc*c*aagc*-3' (SEQ ID NO: 2)
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) 1-13 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Passini (US 20210145852 A1).
Regarding claim 1, Passini teach a method for treating or delaying Duchenne muscular dystrophy (DMD), comprising administering to the patient an effective amount of casimersen (“treating” claim 1; treating or delaying claims 19-21; casimersen is a AON [0007]). An effective amount is generally from about 10-160 mg/kg or 20-160 mg/kg. In some cases, doses of greater than 160 mg/kg may be necessary. In some embodiments, doses for i.v. administration are from about 0.5 mg to 160 mg/kg [0130]. Thus, the recited doses are within the doses recited by Passini.
Regarding claim 2, Passini teach the method of claim 1, wherein the AON is administered for at least 24 weeks, at least 36 weeks, or at least 48 weeks [0129].
Regarding claims 3-5, Passini teach the method of claim 1, wherein the AON is administered for at least 24 weeks, at least 36 weeks, or at least 48 weeks [0129]. This encompasses the recited limitation, QWx24W or QWx25W.
Regarding claims 6-10, Passini teach the method of claim 1, wherein the AON is administered from about 0.5 mg to 160 mg/kg [0130]. This encompasses the recited doses, of 3 mg/kg to 18 mg/kg.
Regarding claims 11-12, Passini teach the method of claim 1, wherein the AON is administered to treat or delay DMD (“treating” claim 1; treating or delaying claims 19-21).
Regarding claim 13, Passini teach the method of claim 1, further comprising a second active agent (non-steroidal anti-inflammatory compound, claim 1; NF- kB inhibitor, claim 2).
Claim(s) 1-14 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Butler (US 2019/0008986 A1, IDS), wherein claim 14 is evidenced by Soblechero-Martín (Soblechero-Martín et al., Neuropathol Appl Neurobiol. 2021;47:711–723).
Regarding claim 1, Butler teach a method for treating or delaying Duchenne muscular dystrophy (DMD), comprising administering to the patient an effective amount of an antisense oligonucleotide (methods for treatment of diseases using provided oligonucleotide compositions, last line of abstract; by effectively skipping exon 51 of DMD to restore the reading frame, pg. 1, R col.[0005]; effective amount to treat or delay [0320]-[0322]). Determining an effective amount is within the skill of the ordinary artisan. See recitation:
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In another example procedure, Butler tests a single s.c. dose of 50 mg/kg [1935]. Thus, the recited doses are within the doses recited by Butler.
Regarding claims 2-5, Butler teach the method of claim 1, wherein the AON is administered over different weekly regimens. See [1620]:
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This encompasses the recited limitations, including QWx24W or QWx25W.
Regarding claims 6-10, Butler teach the method of claim 1, wherein the AON is administered at various doses. See [1622]:
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This encompasses the recited doses, of 3 mg/kg to 18 mg/kg.
Regarding claims 11-12, Butler teach the method of claim 1, wherein the AON is administered to treat or delay DMD (effective amount to treat or delay [0320]-[0322]).
Regarding claim 13, Butler teach the method of claim 1, further comprising a second active agent (combination therapy, [1636]).
Regarding claim 14, Butler teach the method of claim 1, wherein the second agent is a utrophin modulator ([1636] last lines). As evidenced by Soblechero-Martín, there are various strategies for modulation of utrophin. One such strategy is GALGT2 gene delivery as recited (fig. 4).
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.
Claim(s) 1-2 and 6-12 are rejected under 35 U.S.C. 102(a)(1) and 102(a)(2) as being anticipated by De Visser (US 2022/0098586 A1, IDS).
The applied reference has a common Applicant (BioMarin Pharmaceutical Inc.) with the instant application. Based upon the earlier effectively filed date of the reference, it constitutes prior art under 35 U.S.C. 102(a)(2). This rejection under 35 U.S.C. 102(a)(2) might be overcome by: (1) a showing under 37 CFR 1.130(a) that the subject matter disclosed in the reference was obtained directly or indirectly from the inventor or a joint inventor of this application and is thus not prior art in accordance with 35 U.S.C. 102(b)(2)(A); (2) a showing under 37 CFR 1.130(b) of a prior public disclosure under 35 U.S.C. 102(b)(2)(B) if the same invention is not being claimed; or (3) a statement pursuant to 35 U.S.C. 102(b)(2)(C) establishing that, not later than the effective filing date of the claimed invention, the subject matter disclosed in the reference and the claimed invention were either owned by the same person or subject to an obligation of assignment to the same person or subject to a joint research agreement.
Regarding claim 1, De Visser teach a method for treating or delaying Duchenne muscular dystrophy (DMD), comprising administering to the patient antisense oligonucleotides once weekly (abstract; splice-switching hydroxyalkoxylated antisense oligonucleotides [0003]; by effectively skipping exon 51 of DMD, pg. 2, R col.[0017]; various doses [0328]; once weekly [0360]). Some doses are disclosed. See recitation:
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Thus, the recited doses are within the doses recited by De Visser.
Regarding claim 2, De Visser teach the method of claim 1, wherein the AON is administered over different weekly regimens. E.g., for a total of 13 weeks ([0360], Example 2 [0371]-[0376]).
This encompasses the recited limitations re # of weeks.
Regarding claims 6-10, De Visser teach the method of claim 1, wherein the AON is administered at various doses ([0328]).
This encompasses the recited doses of 3 mg/kg to 18 mg/kg.
Regarding claims 11-12, De Visser teach the method of claim 1, wherein the AON is administered to treat or delay DMD ([0326]).
Claim Rejections - 35 USC § 103
In the event the determination of the status of the application as subject to AIA 35 U.S.C. 102 and 103 (or as subject to pre-AIA 35 U.S.C. 102 and 103) is incorrect, any correction of the statutory basis (i.e., changing from AIA to pre-AIA ) for the rejection will not be considered a new ground of rejection if the prior art relied upon, and the rationale supporting the rejection, would be the same under either status.
The following is a quotation of 35 U.S.C. 103 which forms the basis for all obviousness rejections set forth in this Office action:
A patent for a claimed invention may not be obtained, notwithstanding that the claimed invention is not identically disclosed as set forth in section 102, if the differences between the claimed invention and the prior art are such that the claimed invention as a whole would have been obvious before the effective filing date of the claimed invention to a person having ordinary skill in the art to which the claimed invention pertains. Patentability shall not be negated by the manner in which the invention was made.
The text of those sections of Title 35, U.S. Code not included in this action can be found in a prior Office action.
The factual inquiries for establishing a background for determining obviousness under 35 U.S.C. 103 are summarized as follows:
1. Determining the scope and contents of the prior art.
2. Ascertaining the differences between the prior art and the claims at issue.
3. Resolving the level of ordinary skill in the pertinent art.
4. Considering objective evidence present in the application indicating obviousness or nonobviousness.
This application currently names joint inventors. In considering patentability of the claims the examiner presumes that the subject matter of the various claims was commonly owned as of the effective filing date of the claimed invention(s) absent any evidence to the contrary. Applicant is advised of the obligation under 37 CFR 1.56 to point out the inventor and effective filing dates of each claim that was not commonly owned as of the effective filing date of the later invention in order for the examiner to consider the applicability of 35 U.S.C. 102(b)(2)(C) for any potential 35 U.S.C. 102(a)(2) prior art against the later invention.
Claim(s) 3-5 are rejected under 35 U.S.C. 103 as being unpatentable over de Visser (US 2022/0098586 A1, IDS) as applied to claims 1-2 and 6-12 above.
Regarding claims 3-5, the method of claim 1 is discussed above. de Visser further teach their disclosure is not limited by the embodiments disclosed…various modifications are apparent to those skilled in the art [0417]. Specifically, de Visser teach administering QWx8W and QWx13W [0372]. Further, de Visser teach complement activation with each dose of their oligonucleotide. However, with each repeated dose there was a decrease complement activation [0382].
de Visser lacks a teaching on administered QWx24W or QWx25W.
It would be prima facie obvious to add either a second and/or third cycle or a week of the antisense oligonucleotides in a method to treat and delay DMD as taught by de Visser. One would be motivated to do so to sustain the benefits seen in the first cycle and gain the further advantage of reduced complement activation. One skilled in the art would have a reasonable expectation of success because the second and/or third cycle would merely be a repeat of the first, already tested in the method and would be performing the same function as already seen in the method of de Visser. Such a repeat cycle would result in a schedule of antisense oligonucleotides administered QWx24W or QWx25W. Generally, it is prima facie obvious to duplicate a step, such as repeat a cycle, based on its recognized suitability for its intended use. See MPEP 2144.04 VI. B. Legal Precedent as Source of Supporting Rationale, Duplication of Parts.
Claim(s) 13-14 are rejected under 35 U.S.C. 103 as being unpatentable over de Visser (US 2022/0098586 A1, IDS) as applied to claims 3-5 above, and further in view of Soblechero-Martín (Soblechero-Martín et al., Neuropathol Appl Neurobiol. 2021;47:711–723).
Regarding claims 13 and 14, the method of de Visser is discussed above.
de Visser lacks a teaching on further comprising a second active agent.
However, before the effective filing date of instant invention, Soblechero-Martín had taught dystrophin restoration therapies could be used in combination with other therapies; for e.g., modulation of utrophin (abstract). Soblechero-Martín teach utrophin overexpression acts as a surrogate, compensating for the lack of dystrophin (abstract) as preclinical studies indicate an inverse correlation between utrophin expression and disease severity in DMD (pg. 713, R col, 1st para, pg. 717, R col, 1st para). Soblechero-Martín teach there are various strategies for modulation of utrophin, one such strategy is GALGT2 gene delivery as recited (fig. 4).
It would have been prima facie obvious to one of ordinary skill in the art before the filing date of the instant application to have incorporated administering a second active agent into the method taught by de Visser for the advantage of gaining a protecting effect imparted by a surrogate protein in the patient. The ordinary skilled artisan, would have been motivated to make this combination because of 1) the known benefit of the second agent as taught by Soblechero-Martín and 2) the reduction to practice by means of clinical trials of such combinations as taught by Soblechero-Martín. The combination of prior art elements according to known methods to yield predictable results supports a conclusion of obviousness. Given the teachings of the cited references and the level of skill of the ordinary skilled artisan, and de Visser assertion that various embodiments are within the scope of the ordinary artisan at the time of applicants’ invention, it must be considered, absent evidence to the contrary, that the ordinary skilled artisan would have had a reasonable expectation of success in practicing the claimed invention reached when combining the cited references. See MPEP 2144 II and 2143 I.(A) and (G).
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 Longi, 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. Please visit www.uspto.gov/patent/patents-forms. The actual filing date of the application in which the form is filed determines what form (e.g., PTO/SB/25, PTO/SB/26, PTO/AIA /25, or PTO/AIA /26) should be used. A web-based eTerminal Disclaimer may be filled out completely online using web-screens. An eTerminal Disclaimer that meets all requirements is auto-processed and approved immediately upon submission. For more information about eTerminal Disclaimers, refer to www.uspto.gov/patents/apply/applying-online/eterminal-disclaimer.
Claims 1-14 are rejected on the ground of nonstatutory double patenting as being unpatentable over claim 18 of copending Application No. 17/449290 (reference application) in view of de Visser (US 2022/0098586 A1, IDS). Claims 13-14 are further rejected in view of Soblechero-Martín (Soblechero-Martín et al., Neuropathol Appl Neurobiol. 2021;47:711–723).
Although the claims at issue are not identical, they are not patentably distinct from each other because they are directed to overlapping subject matter. The claims of the aforementioned reference are also drawn to a method of treating DMD using a modified ASO.
Recitations of the method claims:
Instant Claim 1
Reference Claim 18
A method of treating a subject having DMD or delaying the onset of DMD in a subject comprising
A method of inducing skipping of exon 51 of dystrophin pre-mRNA, comprising
administering to the subject AON1 at a dose of 0.4 mg/kg, 0.6 mg/kg, 0.8 mg/kg, 1.5 mg/kg, 3 mg/kg, 6 mg/kg, 9 mg/kg, 12 mg/kg or 18 mg/kg, wherein the AON1 is administered once per week (QW).
contacting the dystrophin premRNA with the hydroxyalkoxylated AON of claim 1.
Instant claim 1 requires AON1 while reference claim 18 makes reference to a hydroxyalkoxylated AON of claim 1 consisting of SEQ ID NO: 22333. One of ordinary skill in the art wouldn’t know what the recited SEQ ID NO of the reference application is so they would go to the seq listing of the two applications and find that the sequences are 100% identical. Therefore, the SEQ ID NO of the ref app could replace the AON1 of instant.
Instant claim 1 further requires specific doses of the AON. Reference claim does not recite doses.
However, the de Visser discloses various doses and schedules as discussed in the de Visser 102/103 rejections above. The doses are effective at exon skipping. See Fig. 1 for e.g. Further the reference application states, “various modifications are apparent to those skilled in the art [0417]”.
It would be prima facie obvious to utilize the doses of the antisense oligonucleotides disclosed in the reference app in a method to treat and delay DMD using AON1. One would be motivated to do so because the reference application shows that the doses utilized are effective at exon skipping. One skilled in the art would have a reasonable expectation of success in utilizing the doses disclosed in the ref. app. because these doses, already tested in the method, would be performing the same function as already seen in the method of ref app. Generally, it is prima facie obvious to utilize a composition for a given purpose based on its recognized suitability for its intended use. See MPEP 2144.07.
Dependent Claims 2-12:
Instant dependent claims require particular schedules which are not recited in reference claims. However, de Visser teaches that doses and schedules of hydroxyalkoxylated AON administration are within all of the recited doses and schedules and are feasible and therefore obvious to a person of ordinary skill in the art as per legal precedent. Specifically, the reference application discloses administering QWx8W and QWx13W [0372].
It would be prima facie obvious to add either a second and/or third cycle or a week of the antisense oligonucleotides in a method to treat and delay DMD as taught by the reference application. See the complete analysis in the de Visser 103 rejection above. See MPEP 2144.04 VI. B. Legal Precedent as Source of Supporting Rationale, Duplication of Parts.
Dependent Claims 13-14:
Instant dependent claims require combination of AON with another active agent, which are not recited in reference claims.
However Soblechero-Martín teaches combination therapies and therefore provide the teachings to combine with the reference application to a person of ordinary skill in the art. See full analysis in the 103 discussion above.
This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
Relevant Prior Art
Uno (US 20210261963 A1) disclose an antisense oligonucleotide composition and a method to treat Duchenne muscular dystrophy (title, abstract). Uno specifically disclose administration of the pharmaceutical composition for 24 weeks [0036].
Conclusion
No claims are allowed.
Correspondence
Any inquiry concerning this communication or earlier communications from the examiner should be directed to SHABANA MEYERING, Ph.D. whose telephone number is (703)756-4603. The examiner can normally be reached M - F: 9am to 5pm EST.
Examiner interviews are available via telephone, in-person, and video conferencing using a USPTO supplied web-based collaboration tool. To schedule an interview, applicant is encouraged to use the USPTO Automated Interview Request (AIR) at http://www.uspto.gov/interviewpractice.
If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Ram Shukla can be reached at (571) 272-0735. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
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SHABANA S. MEYERING, Ph.D.
Examiner
Art Unit 1635
/SHABANA S MEYERING/Examiner, Art Unit 1635
/CATHERINE KONOPKA/Primary Examiner, Art Unit 1635