Prosecution Insights
Last updated: April 19, 2026
Application No. 18/304,519

Compositions and Methods for Treating Spinal Muscular Atrophy

Non-Final OA §102§DP
Filed
Apr 21, 2023
Examiner
POLIAKOVA-GEORGAN, EKATERINA
Art Unit
1637
Tech Center
1600 — Biotechnology & Organic Chemistry
Assignee
Genzyme Corporation
OA Round
1 (Non-Final)
65%
Grant Probability
Favorable
1-2
OA Rounds
2y 8m
To Grant
81%
With Interview

Examiner Intelligence

Grants 65% — above average
65%
Career Allow Rate
434 granted / 668 resolved
+5.0% vs TC avg
Strong +16% interview lift
Without
With
+16.2%
Interview Lift
resolved cases with interview
Typical timeline
2y 8m
Avg Prosecution
55 currently pending
Career history
723
Total Applications
across all art units

Statute-Specific Performance

§101
5.4%
-34.6% vs TC avg
§103
28.6%
-11.4% vs TC avg
§102
22.8%
-17.2% vs TC avg
§112
24.2%
-15.8% vs TC avg
Black line = Tech Center average estimate • Based on career data from 668 resolved cases

Office Action

§102 §DP
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 . Claim Objections Claims 13-20 are objected to because of the following informalities: claims 13-20 are not listed in the list of claims. For the purpose of examination it will be considered that claims 13-20 are cancelled, but appropriate correction is required. 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 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. Claim(s) 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 is/are rejected under 35 U.S.C. 102(a)(1) as being anticipated by Passini et al (WO 2010/129021, November 2010, cited from IDS). The claimed invention embraces a method for treating or ameliorating a symptom of spinal muscular atrophy in a primate, or for delivering a heterologous transgene encoding a primate SMN in a motor neuron in a primate with spinal muscular atrophy, comprising administering to the spinal cord and/or cisterna magna of the primate at least 1x1012 genome copies of a recombinant adeno-associated virus (rAAV) viral particle comprising a vector encoding a primate SMN, wherein the AAV viral particle comprises an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAVrh8, AAV10, AAVrh10, AAV11, or AAV12 serotype capsid. The symptoms may include one or more of muscle wasting, inability to achieve motor milestones, inability to sit, inability to walk, paralysis, respiratory dysfunction, bulbar dysfunction, motor neuron cell loss and neuromuscular junction pathology. The claimed invention specifies that at least 10-30% of the motor neurons in the lumbar, thoracic and cervical regions of the spinal cord are transduced and/or wherein at least 30% of SMN wild type levels are generated throughout the spinal cord. The rAAV may be administered via direct injection into the spinal cord, via intrathecal injection, or via intracisternal injection, or administered to one or more of a lumbar subarachnoid space, thoracic subarachnoid space and a cervical subarachnoid space of the spinal cord or the cisterna magna. At least 3.5 x 1011, 3.5 x 1012, 5x1012 or 5x1013 genome copies per kg body weight of rAAV may be administered to the primate, or at least 2.5 x 1012 or 1.25 x 1013 genome copies may be administered to the primate. The rAAV viral particle may comprise AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAVrh8, AAV10, AAVrh10, AAV11, or AAV12 ITRs. The vector may be a self- complimenting vector. The vector may encode a SMN-1 transgene that is operably linked to a promoter, which may be capable of expressing the SMN-1 transgene in neurons of the spinal cord. The promoter may a human β-glucuronidase promoter or a cytomegalovirus enhancer linked to a chicken β-actin promoter. The vector may comprise a polynucleotide encoding the amino acid sequence of SEQ ID NO:1. The primate may be human, which may be a pediatric subject or a young adult. The rAAV viral particle may be in a pharmaceutical composition. Passini teach a self-complementary adeno-associated virus (scAAV) vector comprising a polynucleotide that encodes a survival motor neuron (SMN) protein as depicted in figure 9B (see lines 29-32 on page 3, lines 1-8 on page 4), which corresponds to SEQ ID NO: 1 of the instant specification and claims, for the purpose of treatment of spinal muscular atrophy in humans (see lines 22-27 on page 16). One of the symptoms of spinal muscular dystrophy is inability to walk (see bridging paragraph between pages 2 and 3). Passini teach delivering to the spinal cord and intrathecal injections (see lines 25-31 on page 4), more specifically into subarachnoid space (see lines 22-30 on page 42). Passini does not teach administering to the cisterna magna, but intrathecal injection constitutes delivery to the cisterna magna since it is well known in the art that they share the same fluid-filled contiguous compartment. Passini teach the use of adenoviral vectors of AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, or AAV9 serotypes (see lines 21-23 on page 31), vectors comprising AAV ITRs (see lines 8-25 on page 26), self-complementary AAV vectors (see lines 20-25 on page 3), and injection of up to 1015 genome particles (see lines 25-30 on page 44), which meets the claimed requirement for administering at least 1 x 1012 genome copies as recited in independent claims 1, 2 and 4, or at least 2.5 x 1012 or 1.25 x 1013 genome copies in claim 12. Passini teach the use of a cytomegalovirus enhancer and chicken β-actin promoter in the disclosed scAAV vector (see lines 15-20 on page 29). The outcomes as described in instant claim 5 are expected to happen in the absence of evidence to the contrary upon practicing the described treatment. Further Passini teach pharmaceutical compositions comprising AAV vectors (see lines 10-12 on page 4). The invention is thus anticipated. 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 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-19 of U.S. Patent No. 10,821,154. Although the claims at issue are not identical, they are not patentably distinct from each other because claims from ‘154 recite methods for treating spinal muscular atrophy in a primate with spinal muscular atrophy, comprising administering via intracisternal injection to the cisterna magna of the primate at least 1.25×1013 genome copies of a recombinant adeno-associated virus (rAAV) viral particle comprising a vector encoding a primate SMN and administering via direct injection into the spinal cord at of the primate at least 1.25×1013 genome copies of a recombinant adeno-associated virus (rAAV) viral particle comprising a vector encoding a primate SMN; wherein the rAAV viral particle comprises an AAV9 serotype capsid, anticipating instant claims. Claims 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-7 of U.S. Patent No. 9,415,119 in view of Passini, above. Claims from ‘119 recite methods of administering a therapeutically effective amount of a pharmaceutical composition comprising a recombinant adeno-associated virus (rAAV) virion to the central nervous system of the subject; wherein the rAAV virion comprises a self-complementary adeno-associated virus (scAAV) vector comprising a polynucleotide encoding a survival motor neuron (SMN) protein, wherein the rAAV virion comprises an AAV9 capsid. Teachings of Passini are discussed above. It would have been obvious to further improve method from ‘119 by applying teachings of Passini, arriving at instant invention. Claims 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-13 of U.S. Patent No. 10,369,193 in view of Passini, above. Claims from ‘193 recite methods of treatment of spinal muscular atrophy by administering AAV8 vector comprising AAV2 ITR and comprising a polynucleotide encoding a survival motor neuron (SMN) protein. Teachings of Passini are discussed above. It would have been obvious to further improve method from ‘193 by applying teachings of Passini, arriving at instant invention. Claims 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-20 of U.S. Patent No. 11,911,440 in view of Passini, above. Claims from ‘440 recite methods of treatment of spinal muscular atrophy by administering AAV8 vector comprising AAV2 ITR and comprising a polynucleotide encoding a survival motor neuron (SMN) protein. Teachings of Passini are discussed above. It would have been obvious to further improve method from ‘440 by applying teachings of Passini, arriving at instant invention. Claims 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-33 of U.S. Patent No. 11,975,043 in view of Passini, above. Claims from ‘043 recite methods of treatment of spinal muscular atrophy by administering AAV8 vector comprising AAV2 ITR and comprising a polynucleotide encoding a survival motor neuron (SMN) protein. Teachings of Passini are discussed above. It would have been obvious to further improve method from ‘043 by applying teachings of Passini, arriving at instant invention. Claims 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-21 of U.S. Patent No. 12,350,311 in view of Passini, above. Claims from ‘311 recite methods of treatment of spinal muscular atrophy by administering AAV8 vector comprising AAV2 ITR and comprising a polynucleotide encoding a survival motor neuron (SMN) protein. Teachings of Passini are discussed above. It would have been obvious to further improve method from ‘311 by applying teachings of Passini, arriving at instant invention. Claims 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 are provisionally rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-19 of copending Application No. 19/228,044 in view of Passini, above. Claims from ‘044 recite methods of treatment of spinal muscular atrophy by administering AAV vector comprising a polynucleotide encoding a survival motor neuron (SMN) protein. Teachings of Passini are discussed above. It would have been obvious to further improve method from ‘044 by applying teachings of Passini, arriving at instant invention. This is a provisional nonstatutory double patenting rejection. Claims 1-5, 7, 10, 12, 21, 27, 30-31, 33, 36, 39-40, 47 are rejected on the ground of nonstatutory double patenting as being unpatentable over claims 1-9 of U.S. Patent No. 9,034,836 in view of Passini, above. Claims from ‘836 recite methods of delivery of AAV vector comprising a polynucleotide encoding a survival motor neuron (SMN) protein. Teachings of Passini are discussed above. It would have been obvious to further improve method from ‘836 by applying teachings of Passini, arriving at instant invention. Conclusion Any inquiry concerning this communication or earlier communications from the examiner should be directed to EKATERINA POLIAKOVA whose telephone number is (571)270-5257. The examiner can normally be reached Mon-Fri 8-5. Examiner interviews are available via telephone, in-person, and video conferencing using a USPTO supplied web-based collaboration tool. To schedule an interview, applicant is encouraged to use the USPTO Automated Interview Request (AIR) at http://www.uspto.gov/interviewpractice. If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Jennifer Dunston can be reached at (571)272-2916. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300. Information regarding the status of published or unpublished applications may be obtained from Patent Center. Unpublished application information in Patent Center is available to registered users. To file and manage patent submissions in Patent Center, visit: https://patentcenter.uspto.gov. Visit https://www.uspto.gov/patents/apply/patent-center for more information about Patent Center and https://www.uspto.gov/patents/docx for information about filing in DOCX format. For additional questions, contact the Electronic Business Center (EBC) at 866-217-9197 (toll-free). If you would like assistance from a USPTO Customer Service Representative, call 800-786-9199 (IN USA OR CANADA) or 571-272-1000. /EKATERINA POLIAKOVA-GEORGANTAS/Primary Examiner, Art Unit 1637
Read full office action

Prosecution Timeline

Apr 21, 2023
Application Filed
Nov 09, 2025
Non-Final Rejection — §102, §DP (current)

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Study what changed to get past this examiner. Based on 5 most recent grants.

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Prosecution Projections

1-2
Expected OA Rounds
65%
Grant Probability
81%
With Interview (+16.2%)
2y 8m
Median Time to Grant
Low
PTA Risk
Based on 668 resolved cases by this examiner. Grant probability derived from career allow rate.

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