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 .
Continued Examination Under 37 CFR 1.114
A request for continued examination under 37 CFR 1.114, including the fee set forth in 37 CFR 1.17(e), was filed in this application after final rejection. Since this application is eligible for continued examination under 37 CFR 1.114, and the fee set forth in 37 CFR 1.17(e) has been timely paid, the finality of the previous Office action has been withdrawn pursuant to 37 CFR 1.114. Applicant's submission filed on 09/03/2025 has been entered.
Applicant’s amendments to the claims dated 09/03/2025 are acknowledged. Claims 36, 67-71, 75-98 are pending and subject to prosecution. Claims 36, 67, 78-80 and 83 are amended. Claims 86-98 are new. Claims 72-74 are cancelled.
The 1.130(a) Declaration on behalf of Tristan Marshall, of record 09/03/2025 is acknowledged.
STATUS OF REJECTIONS
The objection of claims 78, 80 and 83 is WITHDRAWN in light of Applicant’s amendments to the claims.
The 112(b) rejection over claim 79 is WITHDRAWN in light of Applicant’s amendment to the claim.
The 112(a) rejection over claims 72-74 is WITHDRAWN in light of the cancellation of these claims.
The 103 rejection over claims 36, 67-69, 75-77, 82 and 84-85 as obvious over Wilson, as evidenced by Genaxxon, taken in view of Wang and Campochiaro, is WITHDRAWN in light of the 1.130(a) Declaration on behalf of Tristan Marshall, of record 09/03/2025. The Declaration is successful in invoking a 102(b)(2)(A) exception in attributing the disclosure of the rAAV composition disclosed in Table 1 of Wilson as having been attained from Marshall:
Marshall makes an "unequivocal" statement that he invented the subject matter of the disclosure of Table 1 of Wilson (page 2, section 9 of Declaration).
Marshall also provides a reasonable explanation of how the subject matter of Table 1 was disclosed to Wilson (sections 11-15 of Declaration):
- Marshall’s employer (REGENXBIO) disclosed a pre-IND briefing book, comprising Marshall’s formulation as Table 9 therein, to the FDA, on or about, 11/17/2017 (earlier than Wilson’s Table 1 and Magnesium Chloride date of 12/18/2017).
-Marshall’s employer (REGENXBIO) emailed the pre-IND briefing book comprising Marshall’s formulation to representatives of the assignee of Wilson (“representatives of the University of Pennsylvania”), on or about, 11/20/2017.
-Wilson filed a provisional Application on 12/18/2017, disclosing Table 1 comprising a formulation that is identical to the formulation invented by Marshall, and that REGENXBIO disclosed to the FDA on 11/17/2017 and University of Pennsylvania on or about 11/20/2017.
Thus, the formal requirements of a declaration are met, and the declaration is timely presented. The Examiner notes that the declaration does not identify the “representatives” of the University of Pennsylvania who were the recipients of the email sent on or about 11/20/2017, nor provide an explanation as to how the information of the email, containing Table 9 of Marshall, went from the “representatives” of the University of Pennsylvania to Wilson. However, there is no evidence to the contrary, nor any evidence to question Marshall’s statement. As such, the Declaration is successful in invoking a 102(b)(2)(A) exception.
It is noted that Wilson is excluded from being applied over the formulation itself. The entirety of the document is not disqualified. Only the work that has been attributed to Marshall.
In light of the above, Wilson’s disclosure relating to the present invention does not qualify as prior art, and the rejection is WITHDRAWN.
The 103 rejection over claims 70-71 as obvious over Wilson, as evidenced by Genaxxon, Wang, Campochiaro, and further in view of Blits is WITHDRAWN in light of the 1.130(a) Declaration on behalf of Tristan Marshall, of record 09/03/2025.
The 103 rejection over claims 81 and 83 as obvious over Wilson, as evidenced by Genaxxon, Wang, Campochiaro, Blits and further in view of UniPro Accession No. P35475 and UniPro Accession No. P22304 is WITHDRAWN in light of the 1.130(a) Declaration on behalf of Tristan Marshall, of record 09/03/2025.
The 103 rejection over claims 78-80 as obvious over Wilson, as evidenced by Genaxxon, Wang, Campochiaro, and further in view of Byrne is WITHDRAWN in light of the 1.130(a) Declaration on behalf of Tristan Marshall, of record 09/03/2025.
PRIORITY
The instant application, filed 5/11/2021, is a 371 National Stage Application of PCT/US2019/061206, filed 11/13/2019, which claims priority to US Provisional Application No. 62/924,060, filed 10/21/2019, and US Provisional Application No. 62/767,410, filed 11/14/2018. Thus, the earliest possible priority for the instant application is 11/14/2018.
Pending claim 36 is drawn to a composition comprising, at least, “sodium phosphate monobasic” and “sodium phosphate dibasic.” Neither US Provisional Application No. 62/924,060 nor 62/767,410 explicitly disclose “sodium phosphate monobasic.” However, both provisional applications recite “NaPi” (see, page 27, line 16 of the ‘410 Application; page 48 of the ‘060 application). While there is no in haec verba requirement, all claims or claim limitations must be supported in the specification through express, implicit, or inherent disclosure. If a skilled artisan would understand that the inventor was in possession of the claim or claimed limitations from the disclosure, the Written Description Burden is considered met. MPEP 2163.02.
In the instant case, a skilled artisan would understand that “NaPi” is “sodium phosphate buffer”, and comprises both sodium phosphate monobasic, and sodium phosphate dibasic, as evidenced by Sodium Phosphate Buffer Powder, Material Safety Data Sheet. Genaxxon Bioscience, 6 pages, 07-27-2018, hereinafter Genaxxon, of record. Genaxxon is cited as evidence to show “NaPi” buffer is “sodium phosphate buffer”, and NaPi buffer (sodium phosphate buffer) necessarily comprises a mixture of sodium phosphate monobasic (sodium dihydrogen) and sodium phosphate dibasic (disodium hydrogen) (page 6 of the PDF):
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CLAIMS
Independent claim 36 is directed to a pharmaceutical composition comprising an rAAV9 virus and 7 additional ingredients (salts, sugars, polymers, etc.). Claim 36 has been amended to remove limitations requiring wherein the composition is formulated for single dosage administration in vials for storage as a 1mL frozen solution. Claim 36 is provided below:
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Independent claim 78 is directed to a method of making a composition comprising an rAAV9 virus and 7 additional ingredients (salts, sugars, polymers, etc.) wherein the composition is formulated for single dosage administration in vials for storage as a 1mL frozen solution.
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Claim Rejections - 35 USC § 112
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.
Claim 90 is 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 90 is a duplicate of claim 89, from which it 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.
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.
This application currently names joint inventors. In considering patentability of the claims the examiner presumes that the subject matter of the various claims was commonly owned as of the effective filing date of the claimed invention(s) absent any evidence to the contrary. Applicant is advised of the obligation under 37 CFR 1.56 to point out the inventor and effective filing dates of each claim that was not commonly owned as of the effective filing date of the later invention in order for the examiner to consider the applicability of 35 U.S.C. 102(b)(2)(C) for any potential 35 U.S.C. 102(a)(2) prior art against the later invention.
Claim36, 67-69, 75-80, and 86-98 are rejected under 35 U.S.C. 103 as being unpatentable over WO20180805688 to Davidson, (of record, cited on IDS dated 09/03/25) further in view of US2015/0252384 to Kaspar; US Patent Application Publication No. 2002/0193335 to Hesson, and US Patent Application No. 2012/0322861 to Byrne, of record.
With regard to claims 36, 78, 80, and 86-87, Davidson discloses pharmaceutical formulations, and their methods of making them, comprising recombinant adeno-associated viral vectors (rAAV) for delivery of therapeutic transgenes to the central nervous system including the brain (abstract, [0078]-[0079], [0096]). Davidson discloses the rAAV vector encodes the transgene TPP1, useful for treating lysosomal storage disorders (paragraphs [0028]-[0030]). Davidson discloses the rAAV vector comprises an AAV9 serotype (paragraphs [0049], [0056]).
Davidson discloses the rAAV particles encoding TPP1 are produced in culture from host cells such as HEK293 cells, purified, concentrated, and formulated in pharmaceutically acceptable carriers (paragraphs [0037], [0049], [0053]-[0055], [0089], [0135]). Davidson discloses the pharmaceutically acceptable carriers include artificial cerebral spinal fluid (“artificial CSF”) (paragraphs [0089], [0090]). Davidson discloses the rAAV particles encoding TPP1 are formulated in single unit dosage form, and single dosages comprise about 1-2 mL (paragraphs [0088], [0091]). Prior to use, Davidson thawed viral particles, wherein the particles were administered in PBS180-F69 (0.01M sodium phosphate dibasic (Na2HPO4), 0.18M NaCl, and 0.001% Pluronic F-68) (F-68 is poloxamer 188) (paragraph [0147]). Thus, Davidson had frozen the AAV viruses prior to use. Davisdon discloses the formulations are administered intrathecally (paragraph [0032], [[0080], [0084]).
However, Davidson does not exemplify AAV9, nor disclose wherein the pharmaceutically acceptable carrier comprises sodium chloride (NaCl), magnesium chloride (MgCl2), potassium chloride (KCl), dextrose, poloxamer 188 (F68), sodium phosphate monobasic (NaH2PO4), and sodium phosphate dibasic (Na2HPO2), as required by claims 36 and 78, nor discloses the process of making the viral particles in suspension culture and frozen according to claims 78, 80, and 86-87.
Kaspar exemplifies AAV9 viruses can be successfully delivered to the brain and transduce target tissues in the CNS (abstract, [0004], [0012], [0016]-[0018]; Examples). Kaspar discloses the Aav9 viruses can be used to treat lysosomal storage disorders including MPSI, MPSII, and CLN2/TPP1 disease (paragraph [0021]).
Hesson discloses formulations of artificial cerebrospinal fluid for the delivery of therapeutic agents to the brain, wherein the therapeutic agents are viral vectors, including adeno-associated viral vectors (abstract; [0002], [0012]-[0014], [0062], [0064]). Hesson discloses the aCSF is an appropriate physiological carrier for gene therapy agents administered to the brain ([0002]-[0003], [0006]). Hesson discloses the artificial CSF is formulated such that it is physiologic and can directly contact tissues of the neuraxis for an extended period of time, from hours to days, without causing side effects. Such formulations are buffered, and can comprise nutrients such as amino acids, electrolytes and salts ([0037]).
Hesson discloses aCSF is produced by combining sodium chloride (NaCl), Magnesium Chloride (MgCl2), Potassium Chloride (KCL), Sodium Phosphate -monobasic (NaH2PO4), Sodium Phosphate -dibasic (Na2HPO4), and dextrose (Examples 1 and 3).
Hesson discloses the minimal formulation of aCSF comprises Sodium, Potassium, Chlorine, Calcium and Magnesium ions, and dextrose ([paragraph [0038]). The ions are maintained and balanced to void damage to the cerebrospinal tissue and to provide normal extracellular compositions ([0039], [0040]).
Byrne teaches systems for preparing rAAV9 in either HEK293 cell adherent culture of BHK 67. suspension culture (paragraph [0254]). Byrne teaches that the suspension-based format can be scaled up for large-scale production of rAAV9 and improved rAAV9 yield (paragraph [0256]). Byrne's suspension-cultured rAAV9 vectors are then harvested, clarified by filtration, purified, concentrated and formulated into an appropriate buffer by TFF (paragraph [0256]).
It would have been obvious to combine the AAV formulations of Davidson further with the disclosures Kaspar and Hesson. Davidson teaches AAV9 as an option for CNS delivery, but does not exemplify it. Kaspar shows AAV9 delivered to the CNS is capable of transducing target tissues. In addition, Davidson discloses the AAV vectors can be formulated as pharmaceutical compositions with poloxamer 188 as an excipient, and additionally suggests the use of artificial CSF as a carrier, but does not disclose the constituents. Hesson discloses artificial CSF is an appropriate physiological carrier for gene therapy agents, including AAV vectors, administered to the brain. A skilled artisan would have been motivated to include artificial CSF comprising, sodium chloride (NaCl), Magnesium Chloride (MgCl2), Potassium Chloride (KCL), Sodium Phosphate -monobasic (NaH2PO4), Sodium Phosphate -dibasic (Na2HPO4), and dextrose to formulations of AAV9, because these are the minimal constituents of aCSF, as taught by Hesson. It would have been obvious to include poloxamer 188 because Davidson exemplifies it use when administering an AAV to the CNS.
With regard to the requirements of claims 78 and 80, it would have been obvious to further combine the disclosures of Davidson, Kaspar and Hesson rendering obvious an rAAV9 virus in a simplified aCSF – NaCl, MgCl2, KCL, NaH2PO4, Na2HPO4, dextrose, further comprising poloxamer 188, formulation further with the disclosure of Byrne. Davidson discloses the pharmaceutical compositions comprising AAV viruses are produced in vitro from HEK293 cells, formulated as single unit dosage form, and single dosages comprise about 1-2 mL, and that the formulations had been frozen. It would have been obvious for the person of ordinary skill in the art to substitute Byrne’s BHK suspension culture for Davidson’s HEK293 adherent culture because Byrne teaches that the BHK suspension system was known for preparing rAAV9 formulations. The skilled artisan would have been motivated to make the substitution in order to achieve large-scale rAAV9 production and to improve viral yield, as taught by Byrne.
With regard to claim 67, Davidson, Kaspar, Hesson nor Byrne disclose providing the pharmaceutical composition as a kit comprising one or more containers and instructions for use, wherein at least of the one or more containers comprises the pharmaceutical composition, using kits for pharmaceutical compositions was routine in the prior art at the time of the invention, and that, for convenience, it would have been obvious to assemble components and instructions into a kit.
With regard to claims 68-69, Davidson discloses the vector genome comprises:(i) an AAV 5' inverted terminal repeat (ITR) sequence;(ii) a promoter;(iii) a coding sequence of human tripeptidyl-peptidase-1 (TPP1) and(iv) an AAV 3' ITR (paragraph [0042], [0065], [0135]).
With regard to claims 75-77 Davidson discloses the viruses are used in methods of treating CLN2 Batten disease in a human subject comprising administering a therapeutically effective amount of the viruses to the human subject (paragraphs [0006], [0028]-[0029], [0031], [0086], [0093], claims 48-51).
With regard to claim 88, Hesson disclose minimal aCSF comprises Calcium ions, and are sourced from calcium chloride (paragraph [00380, Examples 1, 3). Thus, this claim is obvous for the same reasons as stated above for claims 36 and 78.
With regard to claims 79, 89, 90, wherein the formulation comprises concentrations of NaCl at about 150 mM, MgCl2 at about 1.2 mM, KCl about 3 mM, CaCl2 at about 1.4 mM, dextrose at about 4.4 mM, and poloxamer 188 at about 0.001% (volume/volume), and sodium phosphate at a concentration of about 1 mM, these concentrations are obvious from the cited art. Davidson discloses the poloxamer is at 0.001% in the formulation. In addition, Hesson provides the preferred concentration ranges of the ions in the simplified aCSF at paragraph [0038]:
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The working examples show use of the claimed ionic sources (sodium chloride, magnesium chloride, potassium chlorides, etc). It would be within the preview of the skilled artisan to arrive the claimed concentrations.
With regard to claims 91, 92, 93, and 98 Hesson discloses the pH of aCSF is close to physiological range, such as a pH of 7.3 (paragraph [0036]).
With regard to claim 94, the pharmaceutical composition of claim 68, wherein the AAV 5' ITR and/or the AAV 3' ITR is from AAV2 (paragraph [0008], [0056], [0061]). In addition, Kaspar discloses the rAAV9 virus comprises AAV2 ITRS (paragraph [0057]).
With regard to claim 95, Davidson discloses wherein the promoter is a a chicken beta actin (CBA) promoter and/or a CBA promoter hybrid with CMV (paragraph [0104], [0135]).
With regard to claim 96, Davidson discloses wherein the vector genome further comprises a polyA, and/or an intron; and/or an enhancer (paragraph [0044]).
With regard to claim 97, Davidson discloses wherein the intron is from CBA and the enhancer is a CMV enhancer (paragraph [0135]; SEQ ID NO:3.
Claims 82, 84 and 85 are rejected under 35 U.S.C. 103 as being unpatentable over WO20180805688 to Davidson, (of record, cited on IDS dated 09/03/25) further in view of US2015/0252384 to Kaspar; US Patent Application Publication No. 2002/0193335 to Hesson, and US Patent Application No. 2012/0322861 to Byrne, of record, as applied to claims 36, 67-69, 75-80, and 86-98 above, further in view of WO2018/2029205 to Wilson, of record.
Claims 82, 84 and 85 encompass embodiments directed to SEQ ID NOs encoding TPP1. Davidson, Kaspar, Hesson, and Byrne are applied as in the 103 rejection above, the content of which is incorporated herein in its entirety.
Davidson discloses the amino acid and nucleic acid sequences of TPP1, but the disclosed portions are an amino acid shorter than those disclosed by the pending claims.
Wilson discloses the rAAV-CLN2 viruses are formulated as pharmaceutical compositions comprising carriers, buffers, diluents, preservatives and/or surfactants (page 24, lines 8-31. Such formulations include sodium chloride, NaPi (sodium phosphate), potassium chloride, dibasic sodium phosphate (page 24, lines 24-31). The formulations can further comprise surfactants, including poloxamers, including poloxamer 188 (Pluronic F68) (page 24, lines 24-31; page 33, lines 1-15).
Wilson discloses the rAAV-CLN2 particles are administered in a therapeutically effective amount (page 27, lines 1-5, and 27-34).
Claim 82 is directed to wherein the human TPP1 comprises the amino acid sequence of SEQ ID NO: 1. Wilson’s SEQ ID NO: 1 is 100% identical to presently claimed SEQ ID NO:1.
Claim 84 is directed to wherein the coding sequence comprises the nucleotide sequence SEQ ID NO:3. Wilson’s SEQ ID NO: 3 is 100% identical to presently claimed SEQ ID NO:3.
Claim 85 is directed to wherein the coding sequence comprises the nucleotide sequence SEQ ID NO: 2. Wilson’s SEQ ID NO: 2 is 100% identical to presently claimed SEQ ID NO:2.
It would have been obvious to combine the rAAV vectors encoding human TPP1 Davidson, Kaspar, Hesson, and Byrne further with the disclosure of Wilson on rAAV vectors encoding human TPP1. It would have been obvious to utilize the known human amino acid and nucleic acid sequences of human TPP1 in the prior art. A skilled artisan would have had a reasonable expectation of success in practicing the claimed invention as use of known TPP1 sequences in AAV vectors was known in the art at the time of the invention.
Claims 70-71 are rejected under 35 U.S.C. 103 as being unpatentable over WO20180805688 to Davidson, (of record, cited on IDS dated 09/03/25) further in view of US2015/0252384 to Kaspar; US Patent Application Publication No. 2002/0193335 to Hesson, and US Patent Application No. 2012/0322861 to Byrne, of record, as applied to claims 36, 67-69, 75-80, and 86-98 above, further in view of US Patent Application Publication No. 2016/0243260 to Blits, of record, cited on Applicant’s IDS dated 12/13/2021. Claims 70 and 71 require wherein the rAAV vector encodes human iduronate 2-sulfatase (claim 70) or human alpha-L-iduronidase (claim 71).
The disclosures of Davidson, Kaspar, Hesson, and Byrne are applied as in the 103 rejection above, the content of which is incorporated herein in its entirety. Davidson discloses a method of treating the lysosomal storage disease Batten disease comprising administering an AAV virus encoding human TPP1. However, none of Kaspar, Hesson, or Byrne disclose wherein the AAV9 virus encodes human iduronate 2-sulfatase or human alpha-L-iduronidase, as required by claims 70 and 71, respectively.
Blits discloses adeno-associated viruses (rAAV) encoding therapeutic peptides to treat lysosomal storage diseases in humans, including Batten disease, by administering the rAAV vectors to the central nervous system via different routes (Abstract, paragraphs [0006]-[0010], [0014], [0035]-[0036], [0051]-[0058], [0080]). Blits discloses the rAAV can encode tripeptidyl peptidase to treat Batten Disease (paragraph [0061], [0063]-[0064], Table 1). Blits discloses the rAAV can encode Iduronate 2-sulfatase (IDS) to treat the lysosomal storage disease Hunter (Table 1; paragraphs [0061], [0063]-[0064], [0067]). Blits discloses the rAAV can encode alpha-L-iduronidase (IDUA) to treat the lysosomal storage disease Hurler-Scheie (Table 1, paragraphs [0061], [0063]-[0064]).
It would have been obvious to substitute the TPP1 gene encoded in the pharmaceutical compositions comprising AAV9 capsids of Davidson with the iduronate 2-sulfatase gene or alpha-L-iduronidase gene of Blits. A person of ordinary skill in the art would have had a reasonable expectation of success in substituting iduronate 2-sulfatase gene or alpha-L-iduronidase gene for human TPP1 because all three genes are explicitly taught as being useful for treating lysosomal storage disorders when encoded in adeno-associated viral vectors. Therefore, these compositions are functional equivalents in the art, and substituting one for the other would have been obvious at the time of the invention. “When a patent ‘simply arranges old elements with each performing the same function it had been known to perform’ and yields no more than one would expect from such an arrangement, the combination is obvious.” See KSR International Co. v. Teleflex Inc., 82 USPQ2d 1385 (U.S. 2007) at 1395-1396, quoting Sakraida v. AG Pro, Inc., 425 U.S. 273 (1976) and In re Fout, 675 F.2d 297, 301 (CCPA 1982) (“Express suggestion to substitute one equivalent for another need not be present to render such substitution obvious”). It would have been obvious to select a human iduronate 2-sulfatase gene or human alpha-L-iduronidase gene because Blits discloses the method is used to treat humans suffering from the lysosomal storage disorders ([0080]). A skilled artisan would have had a reasonable expectation of success in practicing the claimed invention because the iduronate 2-sulfatase gene, alpha-L-iduronidase gene, and TPP1 gene were known to be encoded in recombinant AAV viruses at the time of the invention.
Claims 70-71 are rejected under 35 U.S.C. 103 as being unpatentable over WO20180805688 to Davidson, (of record, cited on IDS dated 09/03/25) further in view of US2015/0252384 to Kaspar; US Patent Application Publication No. 2002/0193335 to Hesson, US Patent Application No. 2012/0322861 to Byrne, of record, US Patent Application Publication No. 2016/0243260 to Blits, of record, as applied to claims 36, 67-71, 75-80, and 86-98 above, and further in view of UniProt Accession No. P35475, sequence dated 4-04-2006, 12 pages downloaded from https://www.uniprot.org/uniprotkb/P35475/entry on 2/22/25, and UniProt Accession No P22304, sequence dated 8-01-1991, 10 pages, downloaded from https://www.uniprot.org/uniprotkb/P22304/entry on 2/22/25.
The disclosures of Davidson, Kaspar, Hesson, and Byrne and Blits are applied as in the 103 rejection above, the content of which is incorporated herein in its entirety. Davidson in view of Blits discloses a method of treating the lysosomal storage diseases comprising administering an AAV virus encoding human alpha-L-iduronidase (IDUA) or human iduronate 2-sulfatase (IDS). However, none of Davidson, Kaspar, Hesson, and Byrne nor Blits disclose the sequence of human IDUA or IDS.
With regard to claim 81, UniProt Accession No. P35475 encoding human alpha-L-iduronidase is 100% identical to SEQ ID NO: 9.
With regard to claim 83, UniProt accession no. P22304 human iduronate 2-sulfonase encoding human iduronate 2-sulfonase is 100% identical to SEQ ID NO:10.
It would have been obvious to include amino acid sequences SEQ ID NO: 9 and 10 in the rAAV of Davidson, Kaspar, Hesson, and Byrne and Blits because UniProt accession no. P35475 evidences that SEQ ID NO: 9 is wild-type human alpha-L-iduronidase while UniProt accession no. P22304 evidences that SEQ ID NO:10 is wild-type human iduronate 2-sulfonase. The person of ordinary skill in the art would have recognized that providing wild-type alpha-L-iduronidase or iduronate 2-sulfonase would be therapeutic for lysosomal storage disorders, as suggested by Blits.
Conclusion
No claims are allowed.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to KIMBERLY A ARON whose telephone number is (571)272-2789. The examiner can normally be reached Monday-Friday 9AM-5PM.
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KAA
/CHRISTOPHER M BABIC/Supervisory Patent Examiner, Art Unit 1633