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 of Group I (claims 1-14), SEQ ID NO:11 of Group A, SEQ ID NO:2 of Group B, and SEQ ID NO:27 of Group C in the reply filed on 07/06/2026 is acknowledged. Because applicant did not distinctly and specifically point out the supposed errors in the restriction requirement, the election has been treated as an election without traverse (MPEP § 818.01(a)).
However, the species election requirement of Group B is withdrawn.
Claims 15-16 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention, there being no allowable generic or linking claim. Election was made without traverse in the reply filed on 07/06/2026.
Claims Status
Claims 1-16 is/are currently pending with claims 15-16 withdrawn. Claims 1-14 is/are under examination.
Information Disclosure Statement
The information disclosure statements filed 09/19/2025 and 07/06/2026 fail to comply with 37 CFR 1.98(a)(1), which requires the following: (1) a list of all patents, publications, applications, or other information submitted for consideration by the Office; (2) U.S. patents and U.S. patent application publications listed in a section separately from citations of other documents; (3) the application number of the application in which the information disclosure statement is being submitted on each page of the list; (4) a column that provides a blank space next to each document to be considered, for the examiner’s initials; and (5) a heading that clearly indicates that the list is an information disclosure statement. The information disclosure statement has been placed in the application file, but the information referred to therein has not been considered.
The information disclosure statements filed 09/19/2025 and 07/06/2026 fail to comply with 37 CFR 1.98(a)(3)(i) because they do not include a concise explanation of the relevance, as it is presently understood by the individual designated in 37 CFR 1.56(c) most knowledgeable about the content of the information, of each reference listed that is not in the English language. It has been placed in the application file, but the information referred to therein has not been considered.
A Chinese-language non-patent literature document was provided with the IDS filed 09/19/2025 but was not listed in the IDS, nor was a statement of relevance, English translation, or English abstract provided.
A Japanese-language non-patent literature document was provided with the IDS filed 07/06/2026 but was not listed in the IDS, nor was a statement of relevance, English translation, or English abstract provided.
Specification
The disclosure is objected to because it contains an embedded hyperlink and/or other form of browser-executable code. Applicant is required to delete the embedded hyperlink and/or other form of browser-executable code; references to websites should be limited to the top-level domain name without any prefix such as http:// or other browser-executable code. See MPEP § 608.01. Hyperlinks were found on page 16 line 27. Applicant is advised to review the specification in order to ensure that all hyperlinks are identified and appropriately removed.
Claim Objections
Claim 10 is objected to because of the following informalities: “a sequence encoding capsid protein” should read either “a sequence encoding a capsid protein” or “a sequence encoding capsid proteins”. Appropriate correction is required.
Claim Rejections - 35 USC § 112
112(b):
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, 3, 5-14 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.
Claims 1, 3, 5, and 13 recite limitations preceded by “preferably” or “more preferably”. These terms render unclear whether the subsequent limitations are required or optional limitations (e.g., it is unclear whether the limitation “truncated…to 56-880 base pairs in length” in claim 1 lines 4-7 is required or optional). As such, the metes and bounds of claims 1, 3, 5, and 13 are unclear and the claims are rendered indefinite. Dependent claims 6-14 do not clarify this indefiniteness, and thus are also rendered indefinite.
Claims 9 and 10 recite limitations preceded by the phrase “in particular” (claim 9 line 2, claim 10 line 2). This term renders unclear whether the subsequent limitations are required or optional limitations (e.g., it is unclear whether the limitation “wherein the nucleic acid expression vector is a recombinant AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11 or AAV12 vector” is required or optional). As such, the metes and bounds of claims 9-10 are unclear and the claims are rendered indefinite.
Claim 13 recites that the pharmaceutical agent is “used for treating congenital stationary night blindness (CSBN1) or rod-cone and cone-rod dystrophy, more preferably retinitis pigmentosa and macular degeneration” (lines 6-7). As discussed above, the term “more preferably” renders unclear whether the subsequent limitations are required. The term “more preferably” inherently indicates that the subsequent limitation is a “more preferable” embodiment of the preceding limitation—in other words, that the subsequent limitation is an embodiment of the preceding limitation. However, retinitis pigmentosa and macular degeneration are not the same diseases as or embodiments of CSBN1 or rod-cone and cone-rod dystrophy. It is therefore unclear whether these four diseases are alternatives. As such, the metes and bounds of claim 13 are unclear, and claim 13 is rendered indefinite.
MPEP 2173.05(p) states the following:
A single claim which claims both an apparatus and the method steps of using the apparatus is indefinite under 35 U.S.C. 112(b) or pre-AIA 35 U.S.C. 112, second paragraph. See In re Katz Interactive Call Processing Patent Litigation, 639 F.3d 1303, 1318, 97 USPQ2d 1737, 1748-49 (Fed. Cir. 2011). In Katz, a claim directed to "[a] system with an interface means for providing automated voice messages…to certain of said individual callers, wherein said certain of said individual callers digitally enter data" was determined to be indefinite because the italicized claim limitation is not directed to the system, but rather to actions of the individual callers, which creates confusion as to when direct infringement occurs. Katz, 639 F.3d at 1318, 97 USPQ2d at 1749 (citing IPXL Holdings v. Amazon.com, Inc., 430 F.3d 1377, 1384, 77 USPQ2d 1140, 1145 (Fed. Cir. 2005), in which a system claim that recited "an input means" and required a user to use the input means was found to be indefinite because it was unclear "whether infringement … occurs when one creates a system that allows the user [to use the input means], or whether infringement occurs when the user actually uses the input means."); Ex parte Lyell, 17 USPQ2d 1548 (Bd. Pat. App. & Inter. 1990) (claim directed to an automatic transmission workstand and the method of using it held ambiguous and properly rejected under 35 U.S.C. 112, second paragraph).
Claim 14 recites both a pharmaceutical agent and a method step of administering the pharmaceutical agent (see lines 6-10). Claim 14 thus claims both a product and a process, which, as described above, renders the claim indefinite.
112(d):
The following is a quotation of 35 U.S.C. 112(d):
(d) REFERENCE IN DEPENDENT FORMS.—Subject to subsection (e), a claim in dependent form shall contain a reference to a claim previously set forth and then specify a further limitation of the subject matter claimed. A claim in dependent form shall be construed to incorporate by reference all the limitations of the claim to which it refers.
The following is a quotation of pre-AIA 35 U.S.C. 112, fourth paragraph:
Subject to the following paragraph [i.e., the fifth paragraph of pre-AIA 35 U.S.C. 112], a claim in dependent form shall contain a reference to a claim previously set forth and then specify a further limitation of the subject matter claimed. A claim in dependent form shall be construed to incorporate by reference all the limitations of the claim to which it refers.
Claims 2, 4, and 7 are rejected under 35 U.S.C. 112(d) or pre-AIA 35 U.S.C. 112, 4th paragraph, as being of improper dependent form for failing to further limit the subject matter of the claim upon which it depends, or for failing to include all the limitations of the claim upon which it depends. Claim 1 recites sequences at least 60% identical to SEQ ID NOs:4, 35, 6, 37, 9, 10, and 39, while dependent claim 2 recites sequences at least 60% identical to SEQ ID NOs:4-14 and 35-39. As claim 2 recites sequences which are not 100% identical to SEQ ID NOs:4, 35, 6, 37, 9, 10, or 39, the scope of sequences recited in claim 2 which are at least 60% identical to SEQ ID NOs:4-14 and 35-39 differs from and is broader than the scope of sequences recited in claim 1. Likewise, the scope of claim 4 is broader than the scope of claim 3 on which claim 4 depends, and the scope of claim 7 is broader than the scope of claim 5 on which claim 7 depends. Applicant may cancel the claim(s), amend the claim(s) to place the claim(s) in proper dependent form, rewrite the claim(s) in independent form, or present a sufficient showing that the dependent claim(s) complies with the statutory requirements.
112(a):
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 1-14 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.
To satisfy the written description requirement, a patent specification must describe the claimed invention in sufficient detail that one skilled in the art can reasonably conclude that the inventor had possession of the claimed invention. See, e.g., Moba, B.V, v. Diamond Automation, Inc., 325 F.3d 1306, 1319, 66 USPQ2d 1429, 1438 (Fed. Cir. 2003); Vas-Cath, Inc. v. Mahurkar, 935 F.2d at 1563, 19 USPQ2d at 1116. Possession may be shown in a variety of ways including description of an actual reduction to practice, or by showing that the invention was "ready for patenting" such as by the disclosure of drawings or structural chemical formulas that show that the invention was complete, or by describing distinguishing identifying characteristics sufficient to show that the applicant was in possession of the claimed invention. See, e.g., Pfaff v. Wells Eiees., Inc., 525 U.S. 55, 68, 119 S.Ct. 304, 312, 48 USPQ2d 1641,1647 (1998); Eli Lilly, 119 F.3d at 1568, 43 USPQ2d at 1406; Amgen, Inc. v. Chugai Pharm., 927 F. 2d 1200, 1206, 18 USPQ2d 1016, 1021 (Fed. Cir. 1991) (one must define a compound by "whatever characteristics sufficiently distinguish it”).
According to the MPEP § 2163, "The written description requirement for a claimed genus may be satisfied through sufficient description of a representative number of species by actual reduction to practice (see i)(A) above), reduction to drawings (see i)(B) above), or by disclosure of relevant, identifying characteristics, i.e., structure or other physical and/or chemical properties, by functional characteristics coupled with a known or disclosed correlation between function and structure, or by a combination of such identifying characteristics, sufficient to show the applicant was in possession of the claimed genus (see i)(C) above). See Eli Lilly, 119 F.3d at 1568, 43 USPQ2d at 1406. A "representative number of species" means that the species which are adequately described are representative of the entire genus. Thus, when there is substantial variation within the genus, one must describe a sufficient variety of species to reflect the variation within the genus. See AbbVie Deutsch land GmbH & Co., KG v. Janssen Biotech, Inc., 759 F.3d 1285, 1300, 111 USPQ2d 1780, 1790 (Fed. Cir. 2014) (Claims directed to a functionally defined genus of antibodies were not supported by a disclosure that "only describe[d] one type of structurally similar antibodies" that "are not representative of the full variety or scope of the genus.")."
Claims 1-5 and 7 recite sequences at least 60% identical to SEQ ID NOs: 4, 35, 6, 37, 9, 10, and 39 (claim 1), 4-14 and 35-39 (claim 2), 1 and 33 (claims 3 and 5), 1-3 and 33-34 (claim 4), and 15-31 and 40-42 (claim 7).
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, SEQ ID NOs:1-42 are the only species whose complete structures are disclosed. While the genus encompasses a large number of variants and molecules that have the same activity as transcriptional regulators (enhancers and promoters) and the genus encompasses a large number of variants and molecules that have a different structure, the specification does not describe the complete structure of a representative number of species of the large genus of enhancers or promoters at least 60% identical to instant SEQ ID NOs:1-42 or functional equivalents thereof. Additionally, the specification does not describe the complete structure of a representative number of species of the large genus of modified enhancers or promoters at least 60% identical to SEQ ID NOs:1-42.
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 that the sequence “has expression specificity and sufficient expression activity in retinal ON-bipolar cells, and can be efficiently expressed in human retinal organoids, and has the potential to be used to treat human ophthalmology retinal related diseases, especially thoses retinal macular region-related diseases” (page 3 lines 3-6). Such a functional limitation cannot be an identifying characteristic for the claimed diverse genus of molecules since by Applicant’s definition of these enhancers and promoters or functional equivalent thereof, all members of the claimed genus will have that characteristic. Further, no identifying characteristics of the modified enhancers or promoters are disclosed.
The inventions of claims 6 and 8-14 require the use of the inventions of claims 1-5 and 7 and therefore are likewise rejected under 35 U.S.C. 112, first paragraph, as failing to comply with the written description requirement.
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) 3-9, 12-14 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Koike (JP2015062387A, of record).
Regarding claims 3-4, Koike teaches a promoter sequence comprising a sequence 100% identical to instant SEQ ID NO:2 (see alignment below) and instant SEQ ID NO:1 (see alignment below) (pages 7-8).
Koike SEQ ID NO:3 and instant SEQ ID NO:2:
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Koike SEQ ID NO:3 and instant SEQ ID NO:1:
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Regarding claims 5-6, Koike teaches an isolated nucleic acid molecule consisting of a promoter of SEQ ID NO:3 (claim 1, see page 16; pages 7-8).
Regarding claim 7, Koike teaches a sequence of SEQ ID NO:3, which comprises a sequence 65.4% identical to instant SEQ ID NO:27 (see alignment below).
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Regarding claim 8, Koike teaches an expression vector comprising the isolated nucleic acid (claim 6, see page 16).
Regarding claims 9 and 11, Koike teaches that the vector is an AAV vector (claim 7).
Regarding claim 12, Koike teaches a pharmaceutical agent—an AAV vector comprising the isolated nucleic acid molecule—used as a medicament (pages 12-13).
Regarding claim 13, Koike teaches that the pharmaceutical agent may be used in the treatment of congenital arrest night blindness, retinitis pigmentosa, or macular degeneration (page 11).
Regarding claim 14, Koike teaches that the pharmaceutical agent may be formulated for subretinal or intravitreal injection (page 13).
Claim(s) 1-2 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Olek (WO2003078657A1, of record).
Regarding claims 1-2, Olek teaches a nucleic acid comprising SEQ ID NO:1 (SEQ ID NO:1 comprises a sequence 100% identical to instant SEQ ID NO:4, see alignment below).
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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 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.
Claim(s) 3-14 is/are rejected under 35 U.S.C. 103 as being unpatentable over Koike (JP2015062387A, of record), in view of Aponte-Ubillus (2018).
Regarding claims 3-4, Koike teaches a promoter sequence comprising a sequence 100% identical to instant SEQ ID NO:2 (see alignment below) and instant SEQ ID NO:1 (see alignment below) (pages 7-8).
Koike SEQ ID NO:3 and instant SEQ ID NO:2:
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Koike SEQ ID NO:3 and instant SEQ ID NO:1:
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Regarding claims 5-6, Koike teaches an isolated nucleic acid molecule consisting of a promoter of SEQ ID NO:3 (claim 1, see page 16; pages 7-8).
Regarding claim 7, Koike teaches a sequence of SEQ ID NO:3, which comprises a sequence 65.4% identical to instant SEQ ID NO:27 (see alignment below).
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Regarding claim 8, Koike teaches an expression vector comprising the isolated nucleic acid (claim 6, see page 16).
Regarding claims 9 and 11, Koike teaches that the vector is an AAV vector (claim 7).
Regarding claim 10, Koike teaches that the vector comprises a transgene, wherein the transgene is a photosensitive protein gene (claims 9-10).
Regarding claim 12, Koike teaches a pharmaceutical agent—an AAV vector comprising the isolated nucleic acid molecule—used as a medicament (pages 12-13; claims 10-12).
Regarding claim 13, Koike teaches that the pharmaceutical agent may be used in the treatment of congenital arrest night blindness, retinitis pigmentosa, or macular degeneration (page 11; claims 10-12).
Regarding claim 14, Koike teaches that the pharmaceutical agent may be formulated for subretinal or intravitreal injection (page 13).
However, Koike does not teach that the expression vector further comprises a sequence encoding a capsid protein, as required by instant claim 10.
Aponte-Ubillus teaches AAV viral capsid proteins and AAV viral vector design.
Regarding claim 10, Aponte-Ubillus teaches that in order to create the AAV viral particles of Koike, the viral expression vector must necessarily comprise sequences encoding capsid proteins (see Fig. 1). Aponte-Ubillus teaches that they may be present on separate nucleic acid molecules, or integrated into the same nucleic acid molecule (Fig. 1; page 1048, Rep/Cap and vector sequences are integrated into a genome, and thus into the same nucleic acid molecule). It would have been obvious to an artisan at the time of filing that the AAV vector of Koike should be designed based on what was known regarding AAV vectors in the art. As such, it would have been obvious to said artisan that sequences encoding viral capsid proteins would be necessary, and that these sequences encoding viral capsid proteins should be comprised in the overall vector.
Conclusion
Any inquiry concerning this communication or earlier communications from the examiner should be directed to AFRICA M MCLEOD whose telephone number is (703)756-1907. The examiner can normally be reached Mon-Fri 9:00AM-6:00PM EST.
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If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Ram Shukla can be reached on (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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/AFRICA M MCLEOD/Examiner, Art Unit 1635
/KIMBERLY CHONG/Primary Examiner, Art Unit 1636
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