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 without traverse of claims 1-9 in the reply filed on December 10, 2025 is acknowledged.
Status of Claims
Claims 1-18 are currently pending in the instant application. Claims 10-18 are withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected invention, there being no allowable generic or linking claim. Accordingly, claims 1-9 are under examination on the merits in the instant application.
Specification
The abstract of the disclosure is objected to because the single sentence “Methods and compositions for improved gene therapy are disclosed.” is not a concise statement of the technical disclosure of the patent, nor does it include what is new in the art to which the invention pertains, wherein the statement is generally limited to a single paragraph within the range of 50 to 150 words in length. Where applicable, the abstract should include the following: (1) if a machine or apparatus, its organization and operation; (2) if an article, its method of making; (3) if a chemical compound, its identity and use; (4) if a mixture, its ingredients; (5) if a process, the steps.
A corrected abstract of the disclosure is required and must be presented on a separate sheet, apart from any other text. See MPEP § 608.01(b).
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.
Claim 7 is 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 7 recites the limitation "the subject" in line 3. There is insufficient antecedent basis for this limitation in the claim.
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 1-9 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.
The instant claims are drawn to a method for increasing AAV vector gene transfer to non-liver cells and a method for decreasing AAV liver toxicity comprising administering to a subject an inhibitor of AAVR prior to or at the same time as the administration of the AAV vector comprising CD47, further comprising administering plerixafor.
It is noted that the claimed method that administers an AAV vector comprising CD47 to cells other than the liver by blocking AAVR in the liver and further administering an HSC mobilization agent (plerixafor) is merely generically disclosed in a prophetic manner by disclosing each of the method steps in separate embodiments.
Although a method of reducing AAV delivery to target cells including liver cells by inhibiting AAVR in the target cells by administering an inhibitor (e.g., RNAi agent) targeting AAVR was known in the relevant prior art as evidenced by Pillay et al. (WO 2017/083423 A1, applicant’s citation), the combination method that administers an inhibitor of AAVR and plerixafor with a simultaneous or subsequent administration of an AAV vector comprising CD47 was not known in the prior art. As such, the instant specification must adequately describe the claimed method in sufficient detail in order to comply with the written description requirement as there is no relevant knowledge pertaining to the methods that encompass and require administration of an AAVR inhibitor, plerixafor, and an AAV vector comprising CD47. It is noted that plerixafor was recognized as an immunostimulant useful for mobilization of stem cells including hematopoietic stem cells (HSC), wherein plerixafor was suggested to be useful in “mobilizing stem cells (e.g., from the bone marrow) into circulation”. See paragraphs 0003-0007 of Cohen et al. (US 2012/0225028 A1). However, there is no teaching in the prior art that plerixafor or an HSC mobilization agent provides the function “for increasing adeno-associated virus (AAV) vector gene transfer to cells other than the liver and/or decreasing AAV liver toxicity”, nor is it adequately supported by the instant specification’s disclosure. In fact, there does not appear to be any prior art pertaining to the use of plerixafor in relation to an AAV vector delivery, regardless of the type of target cells. Similarly, an AAV vector comprising CD47 in providing the aforementioned function recited in the claims was not known in the prior art, nor is it described in the specification. As such, the instant specification fails to provide sufficient written description support for the requisite structure-function correlation between plerixafor and CD47 and their function in non-liver cell delivery of an AAV vector and reduced AAV liver toxicity.
In view of the foregoing, the method of claims 7-9, which is fully encompassed by claims 1-6, is not adequately described by the instant specification in the manner to reasonably convey to one skilled in the relevant art that the instant co-inventors had possession of the claimed method as of the filing date sought in the instant application.
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.
Claims 1-6 are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Pillay et al. (WO 2017/083423 A1, applicant’s citation).
Pillay discloses a method of reducing AAV infection in target cells or reducing delivery of a “gene therapy” AAV particle/vector “that includes a nucleic acid of interest” to target cells in vivo in an individual comprising contacting the target cell with an agent that inhibits AAVR that binds to AAV, wherein the target cells are “liver cells” and the agent administration is followed by contacting the target cell with the AAV particle, wherein the agent is “an anti-AAVR RNAi agent (i.e., an RNAi agent such as an shRNA, an siRNA, or a microRNA that specifically targets AAVR)”, wherein “cells with decreased levels of AAVR are less permissive to AAV infection.” See pages 1, 5, 10, 23, 71-72, 75-80; claims 53-66.
Accordingly, claims 1-6 are described by Pillay et al.
Conclusion
No claim is allowed.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to DANA H SHIN whose telephone number is (571)272-8008. The examiner can normally be reached Monday-Thursday: 8am - 6:30pm.
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/DANA H SHIN/Primary Examiner, Art Unit 1635