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and the fee set forth in 37 CFR 1.17(e) has been timely paid, the finality of the previous Office action has been withdrawn pursuant to 37 CFR 1.114.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 12 March 2026 has been entered.
Election/Restrictions
Applicant’s election without traverse of Group I, claims 1-17, and species election of bipolar cells for the species of retinal cells, Batten disease and subspecies CLN3 for the species of vision-related disorders, CLN polypeptide and subspecies CLN3 for the species transgene, and Anc80 for the species gene therapy vector in the reply filed on 16 December 2024 was previously acknowledged.
Claims 7 and 8 were previously withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected species, there being no allowable generic or linking claim. Election was made without traverse in the reply filed on 16 December 2024.
The species election as it pertains to the election of retinal cell type and the species of gene therapy vector was previously withdrawn in the Official Action mailed 24 January 2025.
Claim Status
Claims 7 and 8 have been withdrawn from consideration, claim 52 is new, claims 1, 5, 11, and 17 have been amended, claims 3, 13, and 18-51 were previously cancelled, claim 16 is newly canceled, and claims 1-2, 4-6, 9-12, 14-15, 17, and 52 have been considered on their merits.
Withdrawn Objections/Rejections
The claim rejections under 35 U.S.C. § 103 have been withdrawn. However, a new ground of rejection is set forth below.
Applicant’s arguments with respect to the rejections of record filed 12 March 2026 have been considered but are moot because the arguments do not apply to the current rejection. In the new ground of rejection, the obviousness is based on the modification of a different method as such the arguments directed to Bosch are moot.
Response to Arguments
In response to Applicant’s assertion of unexpected results on pages 8-9 of the remarks, although Applicant argues unexpected results, the claims are not commensurate in scope with the experimental results cited by the Applicant. MPEP 716.02(d) states that whether the unexpected results are the result of unexpectedly improved results or a property not taught by the prior art, the "objective evidence of nonobviousness must be commensurate in scope with the claims which the evidence is offered to support." In other words, the showing of unexpected results must be reviewed to see if the results occur over the entire claimed range. In re Clemens, 622 F.2d 1029, 1036, 206 USPQ 289, 296 (CCPA 1980). See also In re Peterson, 315 F.3d 1325, 1329-31, 65 USPQ2d 1379, 1382-85 (Fed. Cir. 2003); In re Grasselli, 713 F.2d 731, 741, 218 USPQ 769, 777 (Fed. Cir. 1983). In this case, the claims are directed to a method of delivering a transgene and a method of treating Batten disease in a subject, the efficiency of delivery is not contemplated by the claims. It is not unexpected when the methods are working as intended and the references teach the methods of the claims, thus, would be expected to result in the same outcome.
Additionally, Applicant stated Fig. 3C and paragraph [0087] relate to efficacy of transgene delivery of the intrathecal administration of the claimed AAV vectors, however, neither the figure nor paragraph discuss how these results are unexpected. While Fig. 3C displays results of ICV delivery of a vector compared to a control, this does not provide evidence in how the results are unexpected, rather shows the method works as intended. MPEP 716.02(b) states the evidence relied upon should establish "that the differences in results are in fact unexpected and unobvious and of both statistical and practical significance." Ex parte Gelles, 22 USPQ2d 1318, 1319 (Bd. Pat. App. & Inter. 1992).
Kaspar teaches the method of claims and there is no reasonable explanation or evidence set forth which would suggest the method of Kaspar would not function with the same efficacy as the alleged unexpected results.
The scope of the claims are directed to a method of delivering a transgene and treatment of Batten disease in a subject. The evidence and results presented do not suggest the combination of elements taught by Kaspar and/or Bosch could not be used to accomplish the claimed delivery or treatment. It has been held that a prior art reference must either be in the field of the inventor’s endeavor or, if not, then be reasonably pertinent to the particular problem with which the inventor was concerned, in order to be relied upon as a basis for rejection of the claimed invention. See In re Oetiker, 977 F.2d 1443, 24 USPQ2d 1443 (Fed. Cir. 1992). In this instance Kaspar and Bosch are both in the field of the inventor’s endeavor and are both reasonably pertinent to the particular problem with which the inventor was concerned.
Regarding the arguments directed to Holthaus utilizing a different vector than the claimed AAV9 or Anc80, Holthaus was used as a reference because Holthaus suggests an ocular AAV-mediated treatment of Cln6nclf mice presents a very similar challenge to deficiencies in CLN3 or other transmembrane defects of neuronal ceroid lipofuscinoses (NCL) providing a proof of concept of gene therapy for CLN3 disease, the most common form of NCL caused by a transmembrane protein defect and the treatment taught by Holthaus demonstrated a slowed loss of photoreceptor function. Additionally, the test for obviousness is not whether the features of a secondary reference may be bodily incorporated into the structure of the primary reference; nor is it that the claimed invention must be expressly suggested in any one or all of the references. Rather, the test is what the combined teachings of the references would have suggested to those of ordinary skill in the art. See In re Keller, 642 F.2d 413, 208 USPQ 871 (CCPA 1981).
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.
Claims 1-2, 4-6, and 52 are rejected under 35 U.S.C. 102(a)(1)/(a)(2) as being anticipated by Kaspar et al. (WO 2014/022582, published 02/06/2014, IDS ref.) as evidenced by Tong et al. (Fluids Barriers CNS. 2024 Jan 4;21 (1):1) and Erskine et al. (ASN Neuro, 2014 Dec 3;6(6)).
This is a new rejection.
Claim 1 it is broadly directed toward delivering any transgene to any retinal cell type in any subject by means of administering an AAV9 or Anc80 gene therapy vector encoding any transgene. The limitation directed to the results of expression of the transgene in the retinal cell have not been considered because the limitation does not add structure to the method of delivering a transgene to a retinal cell in a subject it is merely a result of the method. Thus, if the art teach the methods one would expect a similar result.
Regarding the limitation directed the administration of the gene therapy vector to a subject via intrathecal delivery, the gene therapy vector encoding the transgene would inherently be delivered to a retinal cell because the optic nerve, which comprises retinal ganglion cells (RGCs) as evidenced by Erskine et al. (ASN Neuro, 2014 Dec 3;6(6)), has been shown to accumulate large molecules administered to the cerebral spinal fluid, as evidenced by Tong et al. (Fluids Barriers CNS. 2024 Jan 4;21 (1):1; Exhibit 1 of Applicant’s reply filed 12 March 2026). Erskine teaches RGC axons pass across the surface of the retina and are collected in a bundle at the optic disk to leave the eye and form the optic nerve (p. 1, Basic Anatomy of the Mammalian Visual System: From the Eye to the Cortex). Intrathecal delivery involves injecting a substance into the CSF, thus, it is understood that the gene therapy vector would necessarily reach the retinal cells of the optic nerve when administered via intrathecal delivery.
Regarding claims 1-2, 4-6, and 52, Kaspar teaches methods and materials useful for intrathecal delivery of polynucleotides to the central nervous system of a patient using rAAV9 as a vector (para. [0016]). The polynucleotides of Kaspar reads as a transgene. Kaspar teaches in some embodiments, the polynucleotide is delivered to the brain, to include nerve and glial cells, wherein the glial cell is a microglial cell (claim 2) (para. [0020]). Glial and microglial cells read as a retinal cell. Kaspar teaches this rAAV9 delivered polynucleotide can be used to treat Lysosomal storage disorders including Batten-Spielmeyer-Vogt/Juvenile NCL/CLN3 disease (claims 4, 5, and 6) (para. [0021]). Kaspar teaches the transduction of cells is further increased when a vector of the disclosure is administered via intrathecal administration and is used in combination with a contrast agent and when the patient is put in the Trendelenberg position (claim 52) (para. [0051]).
Thus, the reference anticipates the subject matter of claims 1-2, 4-6, and 52.
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.
Claims 1-2, 4-6, 9-12, 14, 17, and 52 are rejected under 35 U.S.C. 103 as being unpatentable over Kaspar et al. (WO 2014/022582, published 02/06/2014, IDS ref.) as evidenced by Tong et al. (Fluids Barriers CNS. 2024 Jan 4;21 (1):1 and Erskine et al. (ASN Neuro, 2014 Dec 3;6(6)) as applied to claims 1-2, 4-6, and 52, and further in view of Bosch et al. (The Journal of Neuroscience, of record).
This is a new rejection.
Kaspar anticipates the subject matter of claims 1-2, 4-6, and 52, thus, also render them obvious.
Regarding claims 9-10, Kaspar teaches methods and materials useful for intrathecal delivery of polynucleotides to the central nervous system of a patient using rAAV9 as a vector (para. [0016]). The polynucleotides of Kaspar reads as a transgene. Kaspar teaches this rAAV9 delivered polynucleotide can be used to treat Lysosomal storage disorders including Batten-Spielmeyer-Vogt/Juvenile NCL/CLN3 disease (para. [0021]).
Kaspar is silent to the transgene encodes a CLN polypeptide and wherein the CLN peptide is CLN3.
However, Bosch teaches delivering a transgene to a retinal cell of a subject utilizing a AAV9 vector (p. 9670, Materials and Methods). Bosch teaches gene therapy using two self-complementary adeno-associated virus 9 (scAAV9) constructs, scAAV9/β-actin-hCLN3 or scAAV9/MeCP2-hCLN3, to intravenously administer the transgene CLN3 to Cln3Δex7/8 mice (p. 9670, Material and Methods). Bosch teaches the intravenous injection was administered via the retro-orbital sinus (p. 9670, Materials and Methods). Bosch teaches the systematic administration of scAAV9 successfully crossed the blood brain barrier to transduce neurons and astrocytes throughout the brain, spinal cord, and eye (pp.9671-9672, Systemic administration of scAAV9 successfully crosses the blood–brain barrier to transduce neurons and astrocytes in juvenile Cln3Δex7/8 mice). Bosch teaches both variants of their scAAV9 vector transduced cells in the eye (Bosch, Figure 3A).
Therefore, it would have been obvious to one of ordinary skill in the art to utilize the vector and delivery method of Kaspar to deliver CLN3 of Bosch with a reasonable expectation of success because Kaspar teaches the rAAV9 vector can be utilized to treat disorders of the CNS, to include Batten disease, which is also known as CLN3 disease and Bosch teaches treatment of the same disease with an AAV9 vector comprising CLN3. One would be motivated to utilize the vector and delivery method of Kaspar to deliver CLN3 of Bosch because Bosch teaches treatment of CLN3 disease and Kaspar teaches an alternative method for delivery.
Regarding claims 11, 12, 14, and 17, Kaspar teaches methods and materials useful for intrathecal delivery of polynucleotides to the central nervous system of a patient using rAAV9 as a vector (para. [0016]). Kaspar teaches this rAAV9 delivered polynucleotide can be used to treat Lysosomal storage disorders including Batten-Spielmeyer-Vogt/Juvenile NCL/CLN3 disease (claim 12) (para. [0021]). Kaspar teaches the transduction of cells is further increased when a vector of the disclosure is administered via intrathecal administration and is used in combination with a contrast agent and when the patient is put in the Trendelenberg position (claim 17) (para. [0051]).
Kaspar is silent to the gene therapy vector comprising a polynucleotide encoding a CLN polypeptide.
However, Bosch teaches delivering a transgene to a retinal cell of a subject utilizing a AAV9 vector (p. 9670, Materials and Methods). Bosch teaches gene therapy using two self-complementary adeno-associated virus 9 (scAAV9) constructs, scAAV9/β-actin-hCLN3 or scAAV9/MeCP2-hCLN3, to intravenously administer the transgene CLN3 (claim 14) to Cln3Δex7/8 mice (p. 9670, Material and Methods). Bosch teaches the intravenous injection was administered via the retro-orbital sinus (p. 9670, Materials and Methods). Bosch teaches the systematic administration of scAAV9 successfully crossed the blood brain barrier to transduce neurons and astrocytes throughout the brain, spinal cord, and eye (pp.9671-9672, Systemic administration of scAAV9 successfully crosses the blood–brain barrier to transduce neurons and astrocytes in juvenile Cln3Δex7/8 mice). Bosch teaches both variants of their scAAV9 vector transduced cells in the eye (Bosch, Figure 3A). The administration of the AAV9 vector comprising the CLN3 transgene reads as treating Batten disease in a subject.
Therefore, it would have been obvious to one of ordinary skill in the art to utilize the vector and delivery method of Kaspar to deliver CLN3 of Bosch thereby treating Batten disease in a subject with a reasonable expectation of success because Kaspar teaches the rAAV9 vector can be utilized to treat disorders of the CNS, to include Batten disease, which is also known as CLN3 disease and Bosch teaches treatment of the same disease with an AAV9 vector comprising CLN3. One would be motivated to utilize the vector and delivery method of Kaspar to deliver CLN3 of Bosch because Bosch teaches treatment of CLN3 disease and Kaspar teaches an alternative method for delivery.
Therefore, the invention as a whole would have been prima facie obvious to a person of ordinary skill before the effective filing date of the claimed invention.
Claim 15 is rejected under 35 U.S.C. 103 as being unpatentable over Kaspar et al. (WO 2014/022582, published 02/06/2014, IDS ref.) as evidenced by Tong et al. (Fluids Barriers CNS. 2024 Jan 4;21 (1):1 and Erskine et al. (ASN Neuro, 2014 Dec 3;6(6)) in view of Bosch et al. (The Journal of Neuroscience, of record) as applied to claims 1-2, 4-6, 9-12, 14, 17, and 52, and further in view of Holthaus et al. (Molecular Therapy, Vol. 26 No 5, of record).
This is a new rejection.
Kaspar anticipates the subject matter of claims 1-2, 4-6, and 52, thus, also render them obvious.
Regarding claim 15, Kaspar is silent to reducing symptoms of Batten disease.
Bosch teaches the neuron-targeted promoter (MeCP2) used to drive human CLN3 expression restored motor function and decreased glial activation and lysosomal pathology in the ClnΔex7/8 brain (treating Batten disease in a subject) (p. 9680, 1st column, last para.). Therefore, Bosch teaches reducing one or more symptoms of Batten disease. Thus, Kaspar in view of Bosch teach reducing one or more symptoms of Batten disease.
Kaspar in view of Bosch are silent to where the treatment slowed the loss of vision.
However, Holthaus teaches rescued photoreceptor degeneration (treating a vision related disorder) in Cln6nclf mice (a subject), a naturally occurring model of CLN6 disease (Batten disease), via intravitreal injections with 7m8.hCLN6 vectors (gene therapy vector encoding a transgene) (p. 1344, Introduction and p. 1346, 7m8-Mediated Supplementation of CLN6 in Bipolar Cells Specifically Rescued the Photoreceptor Degeneration in Cln6nclf mice). Holthaus teaches the production of two 7m8.hCLN6 vectors, one carrying the weak PCP2 promoter to drive expression in rod bipolar cells (retinal cells) and one carrying the 4x enhanced Grm6 promoter to drive expression in all ON bipolar cells (p. 1346, 7m8-Mediated Supplementation of CLN6 in Bipolar Cells Specifically Rescued the Photoreceptor Degeneration in Cln6nclf mice). The slowed loss of photoreceptor function reads as slowing a symptom of loss of vision.
Therefore, it would have been obvious to one of ordinary skill in the art to utilize the treatment taught by Kaspar in view of Bosch to slow vision loss in a subject as taught by Holthaus because Kaspar in view of Bosch teaches reducing multiple symptoms of Batten disease. There would have been a reasonable expectation of success for the treatment taught by Kaspar in view of Bosch to slow vision loss in a subject as taught by Holthaus because Holthaus suggests an ocular AAV-mediated treatment of Cln6nclf mice presents a very similar challenge to deficiencies in CLN3 or other transmembrane defects of neuronal ceroid lipofuscinoses (NCL) providing a proof of concept of gene therapy for CLN3 disease, the most common form of NCL caused by a transmembrane protein defect (Holthaus, p. 1350, Discussion). One would have been motivated to treat vision loss with the treatment taught by Kaspar in view of Bosch to slow vision loss in a subject as taught by Holthaus because Bosch teaches both variants of their scAAV9 vector transduced cells in the eye (Bosch, Figure 3A).
Therefore, the invention as a whole would have been prima facie obvious to a person of ordinary skill before the effective filing date of the claimed invention.
Double Patenting
The nonstatutory double patenting rejection is based on a judicially created doctrine grounded in public policy (a policy reflected in the statute) so as to prevent the unjustified or improper timewise extension of the “right to exclude” granted by a patent and to prevent possible harassment by multiple assignees. A nonstatutory double patenting rejection is appropriate where the conflicting claims are not identical, but at least one examined application claim is not patentably distinct from the reference claim(s) because the examined application claim is either anticipated by, or would have been obvious over, the reference claim(s). See, e.g., In re Berg, 140 F.3d 1428, 46 USPQ2d 1226 (Fed. Cir. 1998); In re Goodman, 11 F.3d 1046, 29 USPQ2d 2010 (Fed. Cir. 1993); In re Longi, 759 F.2d 887, 225 USPQ 645 (Fed. Cir. 1985); In re Van Ornum, 686 F.2d 937, 214 USPQ 761 (CCPA 1982); In re Vogel, 422 F.2d 438, 164 USPQ 619 (CCPA 1970); In re Thorington, 418 F.2d 528, 163 USPQ 644 (CCPA 1969).
A timely filed terminal disclaimer in compliance with 37 CFR 1.321(c) or 1.321(d) may be used to overcome an actual or provisional rejection based on nonstatutory double patenting provided the reference application or patent either is shown to be commonly owned with the examined application, or claims an invention made as a result of activities undertaken within the scope of a joint research agreement. See MPEP § 717.02 for applications subject to examination under the first inventor to file provisions of the AIA as explained in MPEP § 2159. See MPEP § 2146 et seq. for applications not subject to examination under the first inventor to file provisions of the AIA . A terminal disclaimer must be signed in compliance with 37 CFR 1.321(b).
The filing of a terminal disclaimer by itself is not a complete reply to a nonstatutory double patenting (NSDP) rejection. A complete reply requires that the terminal disclaimer be accompanied by a reply requesting reconsideration of the prior Office action. Even where the NSDP rejection is provisional the reply must be complete. See MPEP § 804, subsection I.B.1. For a reply to a non-final Office action, see 37 CFR 1.111(a). For a reply to final Office action, see 37 CFR 1.113(c). A request for reconsideration while not provided for in 37 CFR 1.113(c) may be filed after final for consideration. See MPEP §§ 706.07(e) and 714.13.
The USPTO Internet website contains terminal disclaimer forms which may be used. Please visit www.uspto.gov/patent/patents-forms. The actual filing date of the application in which the form is filed determines what form (e.g., PTO/SB/25, PTO/SB/26, PTO/AIA /25, or PTO/AIA /26) should be used. A web-based eTerminal Disclaimer may be filled out completely online using web-screens. An eTerminal Disclaimer that meets all requirements is auto-processed and approved immediately upon submission. For more information about eTerminal Disclaimers, refer to www.uspto.gov/patents/apply/applying-online/eterminal-disclaimer.
Claims 1, 4-6, 9-12, and 14 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-16 and 18-20 of copending Application No. 18/880,819 (reference application).
This is a new rejection.
Although the claims at issue are not identical, they are not patentably distinct from each other because the reference application anticipates the instant claims.
Regarding claims 1, 4-6, 9-12, and 14, reference claim 20 recites a method of treating Batten disease in a subject comprising administering to the subject the rAAV composition of reference claim 2. Reference claim 2 which depends from reference claim 1 recites an rAAV that expresses a therapeutic protein comprising a CLN3 protein. Reference claim 9 recites the rAAV comprises an AAV9 capsid, thus, reads as administering an AAV9 gene therapy vector comprising a polynucleotide encoding a CLN3 protein. Reference claim 14 recites the rAAV composition is formulated for intrathecal delivery.
The reference claims do not specifically state the vector delivers the transgene to a retinal cell, however, as discussed in the art rejections above, claim 1 it is broadly directed toward delivering any transgene to any retinal cell type in any subject by means of administering an AAV9 or Anc80 gene therapy vector encoding any transgene. The limitation directed to the results of expression of the transgene in the retinal cell have not been considered because the limitation does not add structure to the method of delivering a transgene to a retinal cell in a subject it is merely a result of the method. Thus, if the art teach the methods one would expect a similar result.
Regarding the limitation directed the administration of the gene therapy vector to a subject via intrathecal delivery, the gene therapy vector encoding the transgene would inherently be delivered to a retinal cell because the optic nerve, which comprises retinal ganglion cells (RGCs) as evidenced by Erskine et al. (ASN Neuro, 2014 Dec 3;6(6)), has been shown to accumulate large molecules administered to the cerebral spinal fluid, as evidenced by Tong et al. (Fluids Barriers CNS. 2024 Jan 4;21 (1):1; Exhibit 1 of Applicant’s reply filed 12 March 2026). Erskine teaches RGC axons pass across the surface of the retina and are collected in a bundle at the optic disk to leave the eye and form the optic nerve (p. 1, Basic Anatomy of the Mammalian Visual System: From the Eye to the Cortex). Intrathecal delivery involves injecting a substance into the CSF, thus, it is understood that the gene therapy vector would necessarily reach the retinal cells of the optic nerve when administered via intrathecal delivery.
Therefore, since the reference claims teach administering the AAV9 vector comprising the transgene and administer via intrathecal delivery, the vector would necessarily be delivered to a retinal cell. Thus, the reference claims read on the instant claims.
This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
Claims 1, 4-6, 9, 11, 17, and 52 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1, 7, 11-14, 16-24, and 28-29 of copending Application No. 18/880,004 (reference application).
This is a new rejection.
Although the claims at issue are not identical, they are not patentably distinct from each other because the reference application anticipates the instant claims.
Regarding claims 1, 4-6, 9, 11, 17, and 52, reference claims 1, 13-14, 23-24, and 29 recite a method of treating CLN1-Batten Disease comprising administering a composition via an intrathecal route comprising an rAAV vector, wherein the vector is of the serotype AAV9 or Anc80, which comprises a polynucleotide encoding the CLN1 polypeptide, further comprising placing the subject in the Trendeleburg position after administration of the rAAV.
The reference claims do not specifically state the vector delivers the transgene to a retinal cell, however, as discussed in the art rejections above, claim 1 it is broadly directed toward delivering any transgene to any retinal cell type in any subject by means of administering an AAV9 or Anc80 gene therapy vector encoding any transgene. The limitation directed to the results of expression of the transgene in the retinal cell have not been considered because the limitation does not add structure to the method of delivering a transgene to a retinal cell in a subject it is merely a result of the method. Thus, if the art teach the methods one would expect a similar result.
Regarding the limitation directed the administration of the gene therapy vector to a subject via intrathecal delivery, the gene therapy vector encoding the transgene would inherently be delivered to a retinal cell because the optic nerve, which comprises retinal ganglion cells (RGCs) as evidenced by Erskine et al. (ASN Neuro, 2014 Dec 3;6(6)), has been shown to accumulate large molecules administered to the cerebral spinal fluid, as evidenced by Tong et al. (Fluids Barriers CNS. 2024 Jan 4;21 (1):1; Exhibit 1 of Applicant’s reply filed 12 March 2026). Erskine teaches RGC axons pass across the surface of the retina and are collected in a bundle at the optic disk to leave the eye and form the optic nerve (p. 1, Basic Anatomy of the Mammalian Visual System: From the Eye to the Cortex). Intrathecal delivery involves injecting a substance into the CSF, thus, it is understood that the gene therapy vector would necessarily reach the retinal cells of the optic nerve when administered via intrathecal delivery.
Therefore, since the reference claims teach administering the AAV9 vector comprising the transgene and administer via intrathecal delivery, the vector would necessarily be delivered to a retinal cell. Thus, the reference claims read on the instant claims.
This is a provisional nonstatutory double patenting rejection because the patentably indistinct claims have not in fact been patented.
Conclusion
No claims are allowed.
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/N.A.H./Examiner, Art Unit 1631
/LAURA SCHUBERG/Primary Examiner, Art Unit 1631
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