DETAILED ACTION
The present application, filed on or after March 16, 2013, is being examined under the first inventor to file provisions of the AIA .
This Office action is in response to the communication filed 6-10-26.
Claims 1-12, 15 and 16 are pending in the instant application.
Specification
The amendments to the specification filed 6-10-26 are hereby approved.
Withdrawn Objections/Rejections
Any objections or rejections not repeated in this Office action are hereby withdrawn.
Maintained Rejections
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 1-12, 15 and 16 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 for the reasons of record set forth in the Office action mailed 3-18-26 and for the reasons set forth below.
Applicant’s Arguments
Applicant’s response clarifies that “N-x” means “N minus x.”
Nevertheless, claim 1 broadly recites “N-x” and “x” modifications with regard to SEQ ID No. 1. However, on page 21, Table 2 of the specification lists SEQ ID Nos. 1-6. All of the sequences listed in Table 2 comprise the 15-mer oligonucleotide sequence listed in claim 1 and a 5’ terminal palmitoyl moiety, but differ in the positions of 2’-O-methyl and tricyclonucleotide modifications. These specific modifications are described on pages 11-12 of the specification.
It is confusing that SEQ ID No. 1 recited in claim 1 differs from SEQ ID No. 1 listed in Table 2. SEQ ID No. 1 listed in Table 2 recites different and specific modifications than those recited in claim 1.
Appropriate correction/clarification is required.
New Rejections Necessitated by Amendments
Claim Rejections - 35 USC § 112
The following is a quotation of the first paragraph of 35 U.S.C. 112(a):
(a) IN GENERAL.—The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor or joint inventor of carrying out the invention.
The following is a quotation of the first paragraph of pre-AIA 35 U.S.C. 112:
The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor of carrying out his invention.
Claims 1-12, 15 and 16 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 pre-AIA the inventor(s), at the time the application was filed, had possession of the claimed invention.
The breadth of the claims:
The claims are drawn to compositions and methods of restoring the function of any mutated type VII collagen comprising preventing splicing of exon 73 of the COL7A1 gene encoding the amino acid sequence of any type VII collagen involved in dysfunction. The claims are also drawn to methods for treating a patient suffering from Dystrophic Epidermolysis Bullosa (DEB) comprising the administration of a nucleic acid consisting of N consecutive nucleotides of the nucleic acid sequence of SEQ ID NO. 1 GCCCGCGTTCTCCAG, wherein N is an integer ranging from 13 to 15, N-x nucleotides of the N consecutive nucleotides are tricyclonucleotides, with x being an integer equal to 1 or 2, x nucleotides are 2-O-methyl-ribonucleotides, and each nucleotide is linked to an adjacent nucleotide through a phosphodiester internucleoside linkage.
Teachings in the specification:
On page 21, Table 2 of the specification lists SEQ ID Nos. 1-6. All of the sequences listed in Table 2 comprise the full length 15-mer oligonucleotide sequence listed in claim 1 and a 5’ terminal palmitoyl moiety, and SEQ ID Nos. 1-6 of Table 2 differ in the positions of 2’-O-methyl and tricyclonucleotide modifications. These specific modifications are described on pages 11-12 of the specification.
Preferred nucleic acids according to the present disclosure are those for which (i) the integer N is 15, (ii) the integer X means 1, (iii) all internucleoside linkages consist of phosphodiester linkages and (iv) the said nucleic acid comprises one nucleotide consisting of 2'-O-methyl- ribonucleotide (i.e. the sole nucleotide that does not consist of a tricyclonucleotide).
As illustrated herein, optimal nucleic acids according to the present disclosure are those for which the single 2'-O-methyl deoxyribonucleotide is located at a nucleotide position selected in the group consisting of nucleotide positions 4 (C), 6 (C) and 7 (G) of SEQ ID NO. 1, according to a nucleotide numbering sense from 5'-end to 3'-end.
The nucleic acid of SEQ ID NO. 1 comprising a single 2'-O-methyl deoxyribonucleotide at position 4 (C) is also referenced as the nucleic acid of SEQ ID NO. 2 herein.
The nucleic acid of SEQ ID NO. 1 comprising a single 2'-O-methyl deoxyribonucleotide at position 6 (C) is also referenced as the nucleic acid of SEQ ID NO. 4 herein.
The nucleic acid of SEQ ID NO. 1 comprising a single 2'-O-methyl deoxyribonucleotide at position 7 (G) is also referenced as the nucleic acid of SEQ ID NO. 5 herein.
Most preferred nucleic acids according to the present disclosure consist of the oligonucleotide of SEQ ID NO. 1, wherein all internucleoside linkages consist of phosphodiester linkages, all the nucleotides consist of tricyclonucleotides, excepted one nucleotide which consists of a 2'-O-methyl-ribonucleotide selected in the group consisting of (i) the cytosyl nucleotide at position 4, (ii) the cytosyl nucleotide at position 6 and (iii) the guanyl nucleotide at position 7, in the sense from 5'-end to 3'-end. –
As already previously mentioned herein, most preferred tricyclonucleotides are those of formula (I) wherein R1 means O, and R1, R2 and "Base" are as defined for formula (I). Thus, at least in the above-described nucleic acids for which the location of the single 2'-O- methyl nucleotide is specified, the tricyclonucleotides comprised therein are tricyclonucleotides of formula (I) wherein (i) R1 is 0 and (ii) each of R2 and R3 means a phosphodiester linkage and (iii) "Base" means a nucleobase.
The specification fails to provide the requisite guidance for using the genus of therapeutic agents instantly claimed, and further whereby treatment is provided in any subject. The specification teaches SEQ ID Nos. 1-6 of Table 2 and their ability to skip exon 73 in vitro (see Table 2 on page 21 of the specification). These examples do not provide a representative number of species for the genus of therapeutics encompassed by the claims.
Since the disclosure fails to describe the common attributes and characteristics concisely identifying members of the proposed genus of therapeutic agents and vectors, and because the claimed genus is highly variant, the description provided is insufficient. One of skill in the art would reasonably conclude that the disclosure fails to provide a representative number of species to describe the broad genus of therapeutic vectors and agents instantly claimed and further whereby therapeutic effects are provided in a subject.
Thus, Applicant was not in possession of the broadly claimed genus.
Allowable Subject Matter
SEQ ID No. 1 appears free of the prior art searched and of record..
Conclusion
THIS ACTION IS MADE FINAL. Applicant is reminded of the extension of time policy as set forth in 37 CFR 1.136(a).
A shortened statutory period for reply to this final action is set to expire THREE MONTHS from the mailing date of this action. In the event a first reply is filed within TWO MONTHS of the mailing date of this final action and the advisory action is not mailed until after the end of the THREE-MONTH shortened statutory period, then the shortened statutory period will expire on the date the advisory action is mailed, and any nonprovisional extension fee (37 CFR 1.17(a)) pursuant to 37 CFR 1.136(a) will be calculated from the mailing date of the advisory action. In no event, however, will the statutory period for reply expire later than SIX MONTHS from the mailing date of this final action.
Certain papers related to this application may be submitted to Art Unit 1637 by facsimile transmission. The faxing of such papers must conform with the notices published in the Official Gazette, 1156 OG 61 (November 16, 1993) and 1157 OG 94 (December 28, 1993) (see 37 C.F.R. ' 1.6(d)). The official fax telephone number for the Group is 571-273-8300. NOTE: If Applicant does submit a paper by fax, the original signed copy should be retained by applicant or applicant's representative. NO DUPLICATE COPIES SHOULD BE SUBMITTED so as to avoid the processing of duplicate papers in the Office.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to Jane Zara whose telephone number is (571) 272-0765. The examiner’s office hours are generally Monday-Friday, 10:30am - 7pm. If attempts to reach the examiner by telephone are unsuccessful, the examiner's supervisor, Jennifer Dunston, can be reached on (571)-272-2916. Any inquiry of a general nature or relating to the status of this application should be directed to the Group receptionist whose telephone number is (703) 308-0196.
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Jane Zara
8-5-26
/JANE J ZARA/Primary Examiner, Art Unit 1637
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