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 6-8-23.
Claims 1-12, 15 and 16 are pending in the instant application.
Specification
The disclosure is objected to because of the following informalities: The specification on page 1 recites the acronyms “DEB,” RDEB,” and “C7”, but provides no definitions for these acronyms.
Appropriate correction is required.
Claim Objections
Claim 2 is objected to because of the following informalities: the claim recites “a tricylonucleotide” but should recite “the tricylonucleotide,” since this term was recited in claim 1. Appropriate correction is required.
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 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.
Claim 1 recites “N-x” and “-x” with regard to SEQ ID No. 1, but it is unclear how these denotations apply to SEQ ID No. 1.
Appropriate correction/clarification are required.
Claim 6 recites the terms “cytosyl” and “ribocytosyl.” It is unclear what these terms mean.
Appropriate correction/clarification are required.
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 15 and 16 are rejected under 35 U.S.C. 112, first paragraph, because the specification, while being enabling for the in vitro synthesis of SEQ ID No. 1 with various modifications, an in vitro dimerization assay, and in vitro exon 73 skipping comprising the administration of SEQ ID No. 1 comprising palmitoyl modifications and 2’-O-methyl modifications, does not reasonably enable methods for restoring the function of any mutated type VII collagen in vivo or treating any subject suffering from Dystrophic Epidermolysis Bullosa.
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 and/or use the invention commensurate in scope with these claims.
The following factors have been considered in determining that the specification does not enable the skilled artisan to make and/or use the invention over the broad scope claimed.
The breadth of the claims:
The claims are drawn to a 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 SEQ ID No. 1 comprising 2’-O-methyl ribonucleotides and tricyclonucleotides.
Teachings in the specification:
The specification teaches the synthesis of SEQ ID No. 1 with various modifications (see Table 2 on page 21). The specification teaches a dimerization assay of some DNA molecules, reporting that the modified forms of SEQ ID No. 1, SY-0871_MK986, SY-0874_MK989, induce some dimerization and are “thus not optimal for an in vivo use.” (See the text on page 20, lines 15-16, and Table 2 on page 21). And the specification teaches the in vitro exon 73 skipping comprising the administration of SEQ ID No. 1 comprising palmitoyl modifications and 2’O-methyl modifications (see Table 2 on page 21 of the specification).
The examples provided in the instant specification, of the in vitro testing of exon 73 skipping efficiency upon administration of SEQ ID No. 1 and some modified forms of SEQ ID No. 1, and the in vitro testing of dimerization of SEQ ID No. 1 and some modified forms of SEQ ID No. 1, are not representative or correlative of the ability to restore function of mutated type VII collagen or treat any subject suffering from DEB.
In light of the teachings in the art and the specification, one skilled in the art would not accept on its face the examples provided in the instant disclosure as being correlative or representative of the ability to provide exon skipping and/or treatment in a subject comprising the administration of SEQ ID No. 1 or modified forms thereof..
Since the specification fails to provide the requisite guidance for treatment or restoration of function in a subject, and since determination of the factors required for accomplishing this in any subject is highly unpredictable, it would require undue experimentation to practice the invention over the broad scope claimed.
For these reasons, the instant rejection for lacking enablement over the full scope claimed is proper.
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.
(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) 15 is/are rejected under 35 U.S.C. 102(a)(1) and 102(a)(2) as being anticipated by Haisma et al (WO 2016/142538).
Haisma et al (WO 2016/142538) teach methods of restoring function of a mutated type VII collagen (C7) comprising preventing splicing of exon 73 of the COL7A1 gene comprising a dysfunctional, mutated type VII collagen (see esp. the abstract, pages 1-3, Table 1 on page 5, 10, Tables 2 and 3 on pages 20-21, 24, 25).
Claim(s) 15 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Turczynski et al (J. Investigative Dermatology, Vol. 136, pages 2387-2395 (2016)).
Turczynski et al (J. Investigative Dermatology, Vol. 136, pages 2387-2395 (2016)) teach methods of restoring function of a mutated type VII collagen (C7) comprising preventing splicing of exon 73 of the COL7A1 gene comprising a dysfunctional, mutated type VII collagen (see esp. the abstract on page 2387, Results on pages 2388-2389).
Allowable Subject Matter
SEQ ID No. 1 appears free of the prior art searched and of record..
Conclusion
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
2-27-26
/JANE J ZARA/Primary Examiner, Art Unit 1637