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 .
Applicant’s arguments and amendments filed on 7/16/2026 have been entered.
Claims 77, 81-85 and 89 have been amended.
Claims 73-76 and 78-80 have been cancelled.
Claims 77 and 81-89 are examined in the instant application.
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 77 and 81-89 remain 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 for reasons set forth in the Non-Final Office Action mailed on 6/17/2026 (and repeated below as amended). 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.
When the claims are analyzed in light of the specification, the instant invention encompasses a virus particle comprising a capsid polypeptide which is a VP1 capsid polypeptide comprising the amino acid sequence of SEQ ID NO: 12, a VP2 capsid polypeptide comprising the amino acids 138 to 736 of SEQ ID NO: 12 or a VP3 capsid polypeptide comprising the amino acids 203 to 736 of SEQ ID NO: 12.
Regarding function, the claims recite that a nucleic acid will encode a capsid polypeptide and the structural limitation of the claims is that the encoded polypeptide will form and function as a viral capsid which permits the formation of a virus particle as claimed.
The specification teaches that a combination of nine mutations were observed after performing high-throughput AAV9 screening in a variety of tissues (see Example 2 starting on pg. 219). Specifically, the specification teaches:
“AAV capsid variant V1 showed an increase in biodistribution to skeletal and cardiac
muscle tissues and a decrease in biodistribution to the liver relative to biodistribution associated with wildtype AAV9 in these tissues (Table 7B). AAV capsid variant V1 also displayed increased transduction in skeletal and cardiac muscle tissues, relative to transduction associated with wildtype AAV9” (parag. 313 lines 1-4); and
“The results show that many variants containing one or more of mutations of V1 target liver less than WT AAV9, target heart (e.g., primate heart or murine heart) more than WT AAV9, target muscle (e.g., primate muscle or murine muscle) more than WT AAV9, and/or target spleen (e.g., primate spleen or murine spleen) less than WT AAV9 with respect to biodistribution and/or transduction. Some evaluated mutations, e.g., H584K, Q588Y, and/or W595A are important for in vivo muscle targeting. The results further show that some evaluated mutations, e.g., Q579T, are important for liver detargeting. The results further show that some evaluated mutations, e.g., R550H, S576A, and/or I601V, are important for capsid productivity. The results further suggest that some evaluated mutations, e.g., V596L and/or N598S, are important for in vitro muscle targeting of relevant human muscle cell lines (FIGS. 2A-20, 3A-30, 4A-40, and 5A-5O). Furthermore, for some evaluated properties, variant capsids with up to an edit distance of 2, 3, 4, 5, 6, 7, or 8 amino acids showed comparable or improved function relative to WT AAV9 (FIGS. 6A-60).” (parag. 315).
However, while the specification contemplates VP1, VP2 or VP3 capsid proteins comprising a variety of mutations, the specification does not provide any support for any sequence other than an amino acid sequence which consist of the amino acid sequence of SEQ ID NO: 12.
For example, the specification teaches on page 7 parags. 36 and 37 that the VP1, VP2 or VP3 capsids (set forth in SEQ ID NO: 12) can comprise additional mutations (substitutions, deletion or insertion) within the amino acid sequence that will form the capsid. As the claims require the formation of a virus particle comprising the capsid polypeptide, these mutation would encompass an array of substitutions, deletions or insertions that would inhibit the formation of a capsid and thus a virus particle. In this regard the art (Naumer et al., 2012, J. Virology, Vol. 86(23) pgs. 13038-13048) teaches that dependoviruses such as AAV are particularly sensitive to mutations in the capsid protein and leading to a failure to assemble a viral capsid.
In agreement with the teachings of Naumer are the teachings of Choi that that mutations in a capsid can prevent the capsid from forming in an AAV and alter the function of an AAV (see Choi et al, 2005, Curr. Gene Ther., Vol. 5(3), pgs. 299-310, specifically pg. 12 parag. 4).
The skilled artisan could not rely upon the disclosure in the specification such that the specification would sufficiently describe that Applicant was in possession of a virus particle comprising a capsid polypeptide which comprises any number or combination of mutations at the time of filing.
Applicants' attention is directed to the decision in Vas-Cath Inc. v. Mahurkar, 19USPQ2d 1111, which clearly states that "applicant must convey with reasonable clarity to those skilled in the art that, as of the filing date sought, he or she was in possession of the invention. The invention is, for purposes of the ‘written description’ inquiry, whatever is now claimed." (See page 1117.) The specification does not "clearly allow persons of ordinary skill in the art to recognize that [he or she] invented what is claimed." (See Vas-Cath at page 1116).
With the exception of the sequences referred to above, the skilled artisan cannot envision the detailed chemical structure of the encompassed polynucleotides, and therefore conception is not achieve regardless of the complexity or simplicity of the method of isolation. Adequate written description requires more than a mere statement that it is part of the invention and reference to a potential method of isolating it. The nucleic acid itself is required. See Fiers v. Revel, 25 USPQ2d 1601 at 1606 (CAFC 1993) and Amgen Inc. v. Chugai Pharmaceutical Co. Ltd., 18 USPQ2d 1016.
One cannot describe what one has not conceived. See Fiddes v. Baird, 30 USPQ2d 1481 at 1483. In Fiddes, claims directed to mammalian FGF’s were found to be unpatentable due to lack of written description for that broad class. The specification provided only the bovine sequence.
Therefore, only an amino acid sequence which consist of the amino acid sequence of SEQ ID NO: 12 meets the written description provision of 35 U.S.C. §112, first paragraph. Applicant is reminded that Vas-Cath makes clear that the written description provision of 35 U.S.C. §112 is severable from its enablement provision (see page 1115).
The claimed invention as a whole is not adequately described if the claims require essential or critical elements that are not adequately described in the specification and that is not conventional in the art as of applicants effective filing date. Possession may be shown by actual reduction to practice, clear depiction of the invention in a detailed drawing, or by describing the invention with sufficient relevant identifying characteristics such that a person skilled in the art would recognize that the inventor had possession of the claimed invention. Pfaff v. Wells Electronics, Inc., 48 USPQ2d 1641,1646 (1998).
In conclusion, this limited information is not deemed sufficient to reasonably convey to one skilled in the art that applicant is in possession of the genus of capsid polypeptides thereof as embraced by the claims.
Response to Arguments
While Applicant’s arguments have been fully considered they are not found persuasive. While Applicants have amended the claims to a capsid polypeptide which is a VP1 capsid polypeptide comprising the amino acid sequence of SEQ ID NO: 12, a VP2 capsid polypeptide comprising the amino acids 138 to 736 of SEQ ID NO: 12 or a VP3 capsid polypeptide comprising the amino acids 203 to 736 of SEQ ID NO: 12 and Table 1 teaches a collection of mutations that can occur in the capsid polypeptides. However, the claims encompass a capsid polypeptide comprising the amino acid sequence of SEQ ID NO: 12, thus the capsid polypeptide is no limited to only the disclosed mutations in Table 1, but any number and combination of mutations in the capsid polypeptide. As taught in the art above, any number mutations can result in the failure of the capsid to form and thus obtaining a viral particle as claimed not possible.
Thus for the reasons above and of record the rejection is maintained.
Conclusion
No claims are allowed. The claims are free of the prior art.
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.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to DAVID A MONTANARI whose telephone number is (571)272-3108. The examiner can normally be reached M-Tr 8-6.
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DAVID A. MONTANARI
Examiner
Art Unit 1632
/ANOOP K SINGH/ Primary Examiner, Art Unit 1632
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