DETAILED ACTION
Notice of Pre-AIA or AIA Status
The present application, filed on or after March 16, 2013, is being examined under the first inventor to file provisions of the AIA .
Continued Examination Under 37 CFR 1.114
A request for continued examination under 37 CFR 1.114, including the fee set forth in 37 CFR 1.17(e), was filed in this application after final rejection. Since this application is eligible for continued examination under 37 CFR 1.114, and the fee set forth in 37 CFR 1.17(e) has been timely paid, the finality of the previous Office action has been withdrawn pursuant to 37 CFR 1.114. Applicant's submission filed on 09/18/2026 has been entered.
Restriction/Election
Claims 1-4, 9-10, 25-26, 29, 33-42, 44-66, 69-73, 77 are pending.
Claim 1 is directed to an allowable product. Pursuant to the procedures set forth in MPEP § 821.04(b), claims 35-42, 51, 53-55, directed to the process of making or using the allowable product, previously withdrawn from consideration as a result of a restriction requirement, are hereby rejoined and fully examined for patentability under 37 CFR 1.104.
Claims 48, 50, directed to the invention(s) of method of using the product of cl. 33 require all the limitations of an allowable product claim, and have NOT been rejoined. Here, the claims 48, 50 depend on claim 33, which is rejected, see below.
Claims 71-73, directed to the invention of SEQ ID NO: 2 require all the limitations of allowable product claim, and have NOT been rejoined. Further claims 71-73 are dependent of a canceled claim and SEQ ID NO: 2 does not require all the limitations cl. 1.
Similarly, claims 45, 47, 49, directed to the invention(s) of method of using product of cl. 32 require all the limitations of an allowable product claim, and have NOT been rejoined. Claims 45, 47 and 49 depend on canceled claim 32.
Because a claimed invention previously withdrawn from consideration under 37 CFR 1.142 has been rejoined, the restriction requirement between groups (Group I and Group II) as set forth in the Office action mailed on 09/12/2022 is hereby withdrawn. In view of the withdrawal of the restriction requirement as to the rejoined inventions, applicant(s) are advised that if any claim presented in a divisional application is anticipated by, or includes all the limitations of, a claim that is allowable in the present application, such claim may be subject to provisional statutory and/or nonstatutory double patenting rejections over the claims of the instant application.
Once the restriction requirement is withdrawn for rejoined claims, the provisions of 35 U.S.C. 121 are no longer applicable to those claims . See In re Ziegler, 443 F.2d 1211, 1215, 170 USPQ 129, 131-32 (CCPA 1971). See also MPEP § 804.01.
Thus, claims 45-50, 71-73, 77 stand withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention, there being no allowable generic or linking claim.
Claims 1-4, 9-10, 25-26, 29, 33-42, ,44, 51-66, 69-70 are examined in this Action.
Priority
It is recognized this application claims priority to PCT/US2019/048400 filed on 8/27/2019, which claims benefit of 62/723326, filed on 8/27/2018. All examined claims enjoy the benefit of filing date of ‘326.
Information Disclosure Statement
The information disclosure statement (IDS) submitted on 09/18/2026 was filed after the mailing date of this action. The submission is in compliance with the provisions of 37 CFR 1.97. Accordingly, the information disclosure statement is being considered by the examiner.
The Christiansen reference, cite 93 (title: Relaxed SiGe layers on Si or silicon-on-insulator substrates by ion implantation and thermal annealing), is crossed out. The subject matter of the reference is related to “semiconductor devices” (par. 2). The relevance to the instant subject matter is not provided, thus the reference is not considered.
Claim Objections
Objection of claim 52 is withdrawn, the status identifier is corrected.
Claim 66 objected to because of the following informalities: claim 66 recites “c-kit”, for consistency purposes, it should be recited as “Kit”; . Appropriate correction is required.
Claim Rejections - 35 USC § 112
Rejection of claims 1-4, 9-10, 25-26, 29, 33-34, 44, 52, 56-65 under 112(a) is withdrawn. Rejection of claims 66, 69-70 is maintained.
The following is a quotation of the first paragraph of 35 U.S.C. 112(a):
(a) IN GENERAL.—The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor or joint inventor of carrying out the invention.
The following is a quotation of the first paragraph of pre-AIA 35 U.S.C. 112:
The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor of carrying out his invention.
Claims 66, 69-70 are rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, as failing to comply with the written description requirement. The claim(s) contains subject matter which was not described in the specification in such a way as to reasonably convey to one skilled in the relevant art that the inventor or a joint inventor, or for applications subject to pre-AIA 35 U.S.C. 112, the inventor(s), at the time the application was filed, had possession of the claimed invention.
Written description issue with claims 66, 69-70.
Claim 66 recites “an antisense RNA molecule comprising a sequence . . . a reverse complement of SEQ ID NO: 28”; it is interpreted that ASO comprising a sequence is a full length of reverse complement of SEQ ID NO: 28, which would be a 50 nt. in length sequence.
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In analyzing whether the written description requirement is met for genus claims, it is first determined whether a representative number of species have been described by their complete structure. In the instant case, only a 25 nt. length SEQ ID NO: 1 and SEQ ID NO: 2 are the only species (oligomers or targeting sequences) disclosed in the specification and demonstrates a modified SEQ ID NO: 1 as a vivo-morpholino oligomer of 25 nt. and hybridizes to a portion of SEQ ID NO: 28.
Prior art of record fails to disclose a morpholino oligonucleotide of 50 nt. in length that targets a splice region of a pre-mRNA. Further, it is known by one skilled in the art that modifying the length of splice switching oligonucleotides affects the efficiency of the oligonucleotide. Further, Summerton (2007, Current Topics in Medicinal Chem., 7, pg. 651-660) indicates that morpholinos “exceptionally high targeting success rate is believed to be a consequence of 2 factors: their extended length (typically 25 subunits) and the inherent affinity of Morpholinos for complementary RNA sequences” (pg. 658). Here the specification does not disclose, i.e. does not possess, a morpholino oligonucleotide of 50 nt. that hybridizes with SEQ ID NO: 28.
Next, then, it is determined whether a representative number of species have been sufficiently described by other relevant identifying characteristics (i.e. other than nucleotide sequence), specific features and functional attributes that would distinguish different members of the claimed genus. In the instant case, the only other identifying characteristic is an ASO’s ability to modulate splicing or hybridize to a Kit pre-mRNA. Such a functional limitation cannot be an identifying characteristic for the claimed 50 nt. morpholino oligonucleotide.
The inventions of dependent claims 69-70 require the use of the inventions of Claim 66 and therefore are likewise rejected under 35 U.S.C. 112, first paragraph, as failing to comply with the written description requirement.
Applicant’s attention is directed to the Guidelines for the Examination of Patent Applications Under the 35 U.S.C. 112(a) or Pre-AIA 35 U.S.C. 112, first paragraph, "Written Description" Requirement (MPEP2163).
In conclusion, Applicant’s failure to disclose a morpholino of 50 nt. in length, of the claimed broad genus is not deemed sufficient to reasonably convey to one skilled in the art that Applicant was in possession of the claimed broad genus at the time the application was filed. Thus, it is concluded that the written description requirement is not satisfied for the claimed genus.
Applicant’s attention is directed to the Guidelines for the Examination of Patent Applications Under the 35 U.S.C. 112(a) or Pre-AIA 35 U.S.C. 112, first paragraph, "Written Description" Requirement (MPEP2163).
Response to Arguments
Applicant's arguments filed 09/18/2026 (“the Remarks”) have been fully considered but they are not persuasive.
Focusing on the Remarks addressing “a reverse complement of SEQ ID NO: 28” of cl. 66, the Remarks indicate that since the “complete nucleotide sequence of that reverse complement is immediately and unambiguously determined from the expressly disclosed target sequence” is sufficient for written description support.
The argument is not persuasive. Neither prior art of record nor the specification provide a 50-nt. in length morpholino that is a reverse complement and that binds to pre-mRNA target, in vitro or in vivo.
New Matter
Rejection of claims 1, 2-4, 6-10, 12-15, 19-29, 32, 34, 43-44, 52, 56, 57-60 is withdrawn under 112(a), the claim is amended to recite antisense RNA molecule.
35 U.S.C. 112(b):
Rejection of claim 25 is withdrawn under 112(b) as it recites complementary. Rejection of claim 29 and 52 is maintained, although the claims amend to “target sequence,” claim 1 has been amended to “target region.” Thus, still lacking antecedent basis.
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 29, 52, 33, 35, 36, 38, 41, 42, 44, 51, 61-64, 66, 69-70 are rejected under 35 U.S.C. 112(b) or 35 U.S.C. 112 (pre-AIA ), second paragraph, as being indefinite for failing to particularly point out and distinctly claim the subject matter which the inventor or a joint inventor (or for applications subject to pre-AIA 35 U.S.C. 112, the applicant), regards as the invention.
Claims 29, 52 recite the limitation "the target sequence", while the amendment of claim 1 has ”target region”, line 7. There is insufficient antecedent basis for this limitation in the claim. The use of target sequence and target region in the claims creates confusion whether target sequence and target region are the same or are distinct.
In the interest of compact prosecution and for consistency, for claims 29 and 52, the claims will be interpreted as “the target region.”
Claims 33 and 66 recites “wherein the RNA molecule is a morpholino RNA molecule.” Both RNA and morpholino are distinct backbone of an antisense oligonucleotide. The claim is indefinite since RNA cannot be a morpholino or vice-versa. Should the claim be interpreted that antisense RNA molecule is a hybrid morpholino backbone and a ribose backbone? The specification does not provide support for a hybrid RNA and morpholino molecule. The dependent claims 44, 61-64, 69-70 fail to overcome the indefiniteness.
For cl. 33 and 66, the language “the RNA molecule is an antisense morpholino oligonucleotide” would be remedial and has support in the specification (pg. 44, line 20).
Further cl. 66 recites “the antisense RNA molecule is a morpholino RNA molecule” (line 4), but then later recites “the antisense RNA molecule comprises a modification . . . a nucleotide modification,. . . a sugar modification” (line 7-9). The claim is confusing, since it is a morpholino oligonucleotide and morpholino is not a sugar, so does the claim mean to say that RNA molecule comprises both a ribose monomer and a morpholino monomer, with ribose sugar being further modified?
Claims 35, 36, 38 recite the limitation "the antisense oligomer". There is insufficient antecedent basis for this limitation in the claim.
Claim 38 also recites a “method for treating a disease, disorder, or condition associated with Kit expression.” The term “associated” is a relative term and fails to provide a clear scope of the metes and bounds of the claim. Not any disease, disorder or condition associated with Kit expression has a causal link with Kit expression and thus administration with oligomer of SEQ ID NO: 1 would not necessarily treat a disease “associated” with Kit expression. Claims 41, 42 and 51 depend on claim 38 and do not overcome the indefiniteness.
Claim Rejections - 35 USC § 102
Rejection of claims 1-4, 9-10, 23, 25-27, 29-30, 34, 52, 56 in view of Khvorova under 102 is withdrawn due to amendment of claim 1.
Allowable Subject Matter
Claims 1-4, 9, 10, 25-26, 34, 37, 39, 40, 53-60, and 65 are allowed.
Conclusion
Any inquiry concerning this communication or earlier communications from the examiner should be directed to KEYUR A. VYAS whose telephone number is (571)272-0924. The examiner can normally be reached M-F 9am - 4 pm (EST).
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/KEYUR A VYAS/Examiner, Art Unit 1637 /Soren Harward/Primary Examiner, TC 1600
