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 .
DETAILED ACTION
This Office Action is a reply to Applicant’s Amendment and Remarks filed June 16, 2026.
Claims 2, 6, 8-12, 18 and 20 have been canceled. New claims 37 and 38 are acknowledged. Claims 1, 2, 13, 15 and 16 have been amended.
Claims 1, 3-5, 7, 13-16, 19 and 21-38 are pending in the instant application.
This application contains claims 21-36 drawn to an invention nonelected without traverse in the reply filed February 2, 2026. A complete reply to the final rejection must include cancellation of nonelected claims or other appropriate action (37 CFR 1.144). See MPEP § 821.01.
Accordingly, claims 1, 3-5, 7, 13-16, 19, 37 and 38 have been examined on the merits as detailed below:
The text of those sections of Title 35, U.S. Code not included in this action can be found in a prior Office action.
Information Disclosure Statement
Applicant’s information disclosure statement (IDS) filed June 16, 2026 is acknowledged. The submission is in compliance with the provisions of 37 CFR §1.97. Accordingly, the Examiner has considered the information disclosure statement, and a signed copy is enclosed herewith.
The listing of references in the specification at pages 24 and 25 is not a proper information disclosure statement. 37 CFR 1.98(b) requires a list of all patents, publications, or other information submitted for consideration by the Office, and MPEP § 609.04(a) states, "the list may not be incorporated into the specification but must be submitted in a separate paper." Therefore, unless the references have been cited by the examiner on form PTO-892, they have not been considered.
Claim Rejections - 35 USC § 102
In the previous Office Action mailed March 24, 2026, claims 1, 2, 8 and 17-20 were rejected under 35 U.S.C. 102(a)(1) as being anticipated by WO 2020/245169 A1 (Kleopa et al.). This rejection is moot against claims 2, 8, 17, 18 and 20 in view of Applicant’s Amendment filed June 16, 2026 to cancel these claims. This rejection is withdrawn against the remaining claims in view of Applicant’s Amendment to the claims filed June 16, 2026.
Claim Rejections - 35 USC § 103
In the previous Office Action mailed March 24, 2026, claims 1, 2, 4, 6, 8, 9, 11, 13 and 15-20 are rejected under 35 U.S.C. 103 as being unpatentable over WO 2020/245169 A1 (Kleopa et al.) in view of Gautier et al. (bioRxiv preprint doi: https://doi.org/10.1101/2020.01.29.924605, posted January 30, 2020). This rejection is moot against claims 2, 6, 8, 9, 11, 18 and 20 in view of Applicant’s Amendment filed June 16, 2026 to cancel these claims. This rejection is withdrawn against the remaining claims in view of Applicant’s Amendment to the claims filed June 16, 2026
Markush Rejection
In the previous Office Action mailed March 24, 2026, claim 14 was rejected on the judicially-created basis that it contains an improper Markush grouping of alternatives. This rejection is withdrawn in view of Applicant’s Remarks filed June 16, 2026.
Applicant’s Amendment and IDS filed June 16, 2026 necessitated a new grounds of rejection as presented below:
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, 3-5, 7, 13-16, 19, 37 and 38 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. This is a new matter rejection.
Claim 1 has been amended and is now drawn to a nucleic acid construct comprising a Schwann cell-specific regulatory element, operably linked to a target gene, wherein the regulatory element comprises a sequence having at least 95% identity to any one of SEQ ID NOs: 1-6.
In Applicant’s Response filed June 16, 2026, Applicants submit that:
Claim 1 has also been amended to recite that the regulatory element comprises a sequence having at least 95% identity to any one of SEQ ID NOs: 1-6. Support for this amendment is found in original claim 2. This amendment further defines the scope of the claimed regulatory elements relative to the previous recitation in claim 2 that "the regulatory element comprises at least a portion of a sequence selected from the group consisting of SEQ ID NOs: 1-6," which the Office interpreted broadly to encompass any dinucleotide or larger oligonucleotide fragment. The recited 95% identity threshold provides a more definite boundary for the claimed sequences.
Original claim 2 recited, “wherein the regulatory element comprises at least a portion of a sequence selected from the groups consisting of SEQ ID NO:1-6”. The language recited in original claim 2 does not support the specific limitation, “wherein the regulatory element comprises a sequence having at least 95% identity to any one of SEQ ID NOs: 1-6” as now recited in claim 1.
If Applicants believe the Specification supports, “wherein the regulatory element comprises a sequence having at least 95% identity to any one of SEQ ID NOs: 1-6”, the Examiner urges Applicant to point to, with particularity, where support can be found for the claims as now filed. Otherwise, Applicant is required to cancel the new matter in reply to this Office Action.
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Claims 1, 3-5, 7, 13-16, 19, 37 and 38 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. This is a written description rejection.
The following quotation from section 2163 of the Manual of Patent Examination Procedure is a brief discussion of what is required in a specification to satisfy the 35 U.S.C. 112 written description requirement for a generic claim covering several distinct inventions:
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..., reduction to drawings..., 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 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.
Thus, when a claim covers a genus of inventions, the disclosure must provide written support for the entire scope of the genus. Support for a genus is generally found where the Applicant has provided a number of examples sufficient so that one in the art would recognize from the specification the scope of what is being claimed and that applicant was in possession of the claimed genus.
The instant claims are drawn to a nucleic acid construct comprising a Schwann cell-specific regulatory element, operably linked to a target gene, wherein the regulatory element comprises a sequence having at least 95% identity to any one of SEQ ID NOs: 1-6. The present Specification is explicit in disclosing:
The primary advantage of the construct disclosed herein is that the regulatory elements are “Schwann cell-specific”, meaning they drive gene expression at high levels in Schwann cells, but at minimal levels in other cell types
First, there is insufficient written description of the nucleic acid encompassed by the claims. The present Specification discloses regulatory elements comprising SEQ ID NOs: 1-6, but does not describe sequences with any percent identity, 95% or otherwise that function to drive gene expression at high levels in Schwann cells, but at minimal levels in other cell types as required by the claims.
Second, there is insufficient written description of the nucleic acid encompassed by the claims because the disclosure doesn’t demonstrate possession of the full genus. Again, the claims are drawn to a nucleic acid construct comprising a Schwann cell-specific regulatory element, operably linked to a target gene, wherein the regulatory element comprises a sequence having at least 95% identity to any one of SEQ ID NOs: 1-6, wherein “Schwann cell-specific”, means the nucleic acid constructs of the invention drive gene expression at high levels in Schwann cells, but at minimal levels in other cell types. Given the explicit definition of “Schwann cell-specific”, SEQ ID NO: 1 (Mpz enhancer upstream of Mpz promoter) of the present invention does not lead to higher gene expression in Schwann cells than in oligodendrocytes. See Figure 2.
Regarding SEQ ID NO: 2 (human Mag enhancer) of the instant claims, it appears that the Specification discloses that SEQ ID NO: 2 in combination with either SEQ ID NO: 7 (full length Pmp22 promoter) or SEQ ID NO: 8 (truncated Pmp22 promoter) carries out the functionality as claimed. See Figure 4.
Regarding SEQ ID NO: 3 (mouse Mag enhancer) of Applicant’s invention, the Specification teaches that SEQ ID NO: 3 is a Schwann cell-specific regulatory element. See Figures 2 and 3.
Concerning SEQ ID NO: 4 (rat Mbp enhancer); SEQ ID NO: 5 (human Aatk exon and promoter);and SEQ ID NO: 6 (human minimized Aatk intronic promoter), the Specification does not appear to have any data that reports a nucleic acid construct comprising a Schwann cell-specific regulatory element, operably linked to a target gene, wherein the regulatory element comprises SEQ ID NO: 4 or SEQ ID NO: 5 or SEQ ID NO: 6.
For further explanation, regarding the functional activities of SEQ ID NOs: 1-6 of Applicant’s invention, the Examiner invites Applicant to review the discussion found in the Supplementary Partial European Search Report dated March 12, 2025 filed with the IDS on June 16, 2026.
Written description requirement for claims that recite wherein the regulatory element comprises a sequence having at least 95% identity to any one of SEQ ID NOs: 1-6 is not met because the Specification does not provide any description of any variants or what sequences could be modified (added, deleted or mutated) that retain the function as claimed. Furthermore, the Specification fails to show a representative species correlating to the function recited in the claims since SEQ ID NOs: 1, 2, 4, 5, 7 and 8 as independent nucleic acids appear to be non-working embodiments. Only SEQ ID NO: 3 functions as claimed. Accordingly, the claims encompass using a genus of a nucleic acid construct comprising a Schwann cell-specific regulatory element which are not adequately described. The claims are rejected because there is insufficient description of the genus of the nucleic acid construct comprising a Schwann cell-specific regulatory element encompassed by the claims.
Vas-Cath Inc. v. Mahurkar, 19USPQ2d 1111, clearly states “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). It is noted that conception is not achieved until reduction to practice has occurred 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 compound 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.
To satisfy the written description requirement an applicant must describe the invention is such a way as to convey to one skilled in the art that applicant had the invention in his possession when the application was filed. Ariad Pharms., Inc. v. Eli Lilly & Co., 598 F.3d 1336, 1351 (Fed. Cir. 2010) (en banc). In cases such as the instant application where a genus is claimed, the specification must contain “either a representative number of species falling within the scope of the genus or structural features common to the members of the genus so that one of skill in the art can ‘visualize or recognize’ the members of the genus.” Id. at 1350. However, written description requirement for claims that recite a nucleic acid construct comprising a Schwann cell-specific regulatory element, operably linked to a target gene, wherein the regulatory element comprises a sequence having at least 95% identity to any one of SEQ ID NOs: 1-6 that function as claimed to drive gene expression at high levels in Schwann cells, but at minimal levels in other cell types is not met.
The entire genus of nucleic acid construct comprising a Schwann cell-specific regulatory element as claimed does not exist in the instant application. That is, adequate written description support does not exist to practice the full scope of the invention claimed. The specification nor the art discloses neither a representative number of species nucleic acids nor any structure/function correlation that would enable one of skill to immediately envision the genus nucleic acid construct comprising a Schwann cell-specific regulatory element required to practice the full scope of the invention. Only SEQ ID NO: 3 has been reduced to practice.
The above position is further supported by In re Clarke, 148 USPQ 665, (CCPA 1966), which held that;
“It appears to be well settled that a single species can rarely, if ever, afford support for a generic claim. In re Soll, 25 C.C.P.A. (Patents) 1309, 97 F.2d 623, 38 USPQ 189; In re Wahlforss et al., 28 C.C.P.A. (Patents) 867, 117 F.21 270, 48 USPQ 397. The decisions do not however fix any definite number of species which will establish completion of a generic invention and it seems evident therefrom that such number will vary, depending on the circumstances of particular cases. Thus, in the case of a small genus such as halogens, consisting of four species, a reduction to practice of three, or perhaps even two, might serve to complete the generic invention, while in the case of a genus comprising hundreds of species, a considerably large number of reductions to practice would probably be necessary.”
As stated above, the MPEP states that written description for a genus can be achieved by a representative number of species within a broad generic claim. Given the breadth of the claims, the Specification lacks sufficient variety of species to reflect the variance in the genus.
In conclusion, the Specification as filed does not provide sufficient descriptive support for the myriad of nucleic acid construct comprising a Schwann cell-specific regulatory element embraced by the claims. For the reasons discussed above, the 35 USC § 112 rejection for written description is therefore applicable.
Applicant's amendment necessitated the new ground(s) of rejection presented in this Office action. Accordingly, THIS ACTION IS MADE FINAL. See MPEP § 706.07(a). 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 Terra C. Gibbs whose telephone number is 571-272-0758. The examiner can normally be reached from 8 am - 5 pm M-F.
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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/TERRA C GIBBS/ Primary Examiner, Art Unit 1635
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