Doc code: IDS
`
`PTOISBI'DBa (03-15)
`Approved for use through 071312016. OMB 0651-0031
`Doc description: Information Disclosure Statement (IDS) Filed
`U.S. Patent and Trademark Office; U.S. DEPARTMENT OF COMMERCE
`Under the Paperwork Reduction Act of 1995, no persons are required to respond to a collection of information unless it contains a valid OMB control number.
`
`
`IN FORMATION DISCLOSU RE
`
`STATEMENT BY APPLICANT
`
`( Not for submission under 37 CFR 1.99)
`
`
`
`Filing Date
`
`First Named Inventor
`
`Art Unit
`
`Examiner Name
`
`Application Number I—
`Not Yet Assigned Attorney Docket Number
`
`
`I AVN-OOQDVCNB
`
`
`
`U.S.PATENTS
`-—
`
`Examiner Cite
`'
`
`Kind
`Code1
`
`Issue Date
`
`Name of Patentee or Applicant
`of cited Document
`
`Pages,Columns,Lines where
`Relevant Passages or Relevant
`Figures Appear
`
`
`
`If you wish to add additional US. Patent citation information please click the Add button.
`Add
`U.S.PATENT APPLICATION PUBLICATIONS -
`
`
`Examiner
`.
`.
`*
`InItIal
`
`Cite No
`
`Publication
`Number
`
`Publication
`Kind
`Code1 Date
`
`Name of Patentee or Applicant
`of cited Document
`
`Pages,Columns,Lines where
`Relevant Passages or Relevant
`Figures Appear
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`If you wish to add additional U.S. Published Application citation information please click the Add button. Add
`FOREIGN PATENT DOCUMENTS
`-—
`
`
`
`Pages,Columns,Lines
`
`Name of Patentee or
`
`Examiner Cite
`where Relevant
`Kind
`Publication
`
`Applicant of cited
`
`Initial*
`No
`Code4 Date
`Passages or Relevant
`
`
`Document
`
`Figures Appear
`
`
`Foreign Document
`Number3
`
`5
`
`T
`
`
`If you wish to add additional Foreign Patent Document citation information please click the Add button
`NON-PATENT LITERATURE DOCUMENTS
`-—
`
`Include name of the author (in CAPITAL LETTERS), title of the article (when appropriate), title of the item
`T5
`(book, magazine, journal, serial, symposium, catalog, etc), date, pages(s), volume-issue number(s),
`publisher, city and/or country where published.
`
`
`Examiner Cite
`
`EFS Web 2.1.17
`
`

`

`
`
`
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`.
`.
`( Not for submissmn under 37 CFR 1.99)
`
`Application Number 15349778
`
`Filing Date
`2016-11-11
`First Named Inventor
`Peter SAZANI
`
`Art U .
`
`mt
`I 1674
`Examiner Name
`Not Yet Assigned
`
` Attorney Docket Number
`
`
`
`
`I AVN-009DVCN6
`
`aboratory Notebook Entry (Exon 51 Experiments): RT—PCR Analysis of 8036 Cells, Pages 2, Exhibit Number 1179
`"ed in Interferences 106,007 and 106,008 on February 16, 2015.
`
`aboratory Notebook Entry (Exon 51 Experiments): RT—PCR Analysis of KM155.C25 Cells, Pages 2, Exhibit Number
`178 filed in Interferences 106,007 and 106,008 on February 16, 2015.
`
`aboratory Notebook Entry (Exon 51 Experiments): Transfection of 8036 Cells, Pages 1, Exhibit Number 1172 filed in
`nterferences 106,007 and 106,008 on February 16, 2015.
`
`aboratory Notebook Entry (Exon 51 Experiments): Transfection of KM155.C25 Cells, Pages 1, Exhibit Number 1171
`"ed in Interferences 106,007 and 106,008 on February 16, 2015.
`
`aboratory Notebook Entry (Exon 53 Experiments): RT—PCR Analysis of KM155.C25 Cells, Pages 2, Exhibit Number
`180 filed in Interferences 106,007 and 106,008 on February 16, 2015.
`
`aboratory Notebook Entry (Exon 53 Experiments): RT—PCR Analysis of R1809 Cells, Pages 2, Exhibit Number 1181
`"ed in Interferences 106,007 and 106,008 on February 16, 2015.
`
`aboratory Notebook Entry (Exon 53 Experiments): Transfection of KM155.C25 Cells, Pages 1, Exhibit Number 1173
`"ed in Interferences 106,007 and 106,008 on February 16, 2015.
`
`aboratory Notebook Entry (Exon 53 Experiments): Transfection of R1809 Cells, Pages 1, Exhibit Number 1174 filed
`n Interferences 106,007 and 106,008 on February 16, 2015.
`
`aboratory Notebook Entry: General RNA recovery, 1 Page, Exhibit Number 1176 filed in lnterferences 106,007 and
`06,008 on February 16, 2015.
`
`aboratory Notebook Entry: Lab—on—a—Chip Analysis, Pages 3, Exhibit Number 1184 filed in lnterferences 106,007 and
`06,008 on February 16, 2015.
`
`arsen et al., "Antisense properties of peptide nucleic acid," Biochim. Et Biophys. Acta, Vol. 1489, pp. 159—166 (1999),
`xhibit Number 1190 filed in Interferences 106,007 and 106,008 on February 17, 2015.
`
`EFS Web 2.1.17
`
`

`

`
`
`
`15349778
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`
`( Not for submission under 37 CFR 1.99)
`
`
`Filing Date
`2016-11-11
`First Named Inventor
`Peter SAZANI
`
`Art Unit
`|1574
`
` Application Number
`
`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
`
`(A)N
`
`4:.
`
`—l—l
`
`ist of Publications for Matthew J. A. Wood, M.D., D. PHIL, 11 pages, (Exhibit Number 2124 filed in interferences
`06,007 and 106,008 on February 17, 2015.
`
`IU, Hong—Xiang et al., "Identification of functional exonic splicing enhancer motifs recognized by individual SR
`roteins," Genes & Development, Vol. 12:1998—2012 (1998)
`
`u et al, "Massive Idiosyncratic Exon Skipping Corrects the Nonsense Mutation in Dystrophic Mouse Muscle and
`'roduces Functional Revertant Fibers by Clonal Expansion," THE JOURNAL OF CELL BIOLOGY, Vol. 148(5):
`.85—995, March 6, 2000 ("Lu et al.") (Exhibit Number 1082 filed in interferences 106008, 106007 on December 23,
`
`U, Qi Long et al., "Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic
`ouse," Nature Medicine, Vol. 9(8):1009—1014 (2003)
`
`U, Qi-Iong et al., "What Can We Learn From Clinical Trials of Exon Skipping for DMD?" Molecular Therapy — Nucleic
`‘ cids, Vol. 3:e152, doi:10.1038lmtna.2014.6, 4 pages (2014)
`
`yophilisation of Oligonucleotides, Pages 2, Exhibit Number 1133 filed in lnterferences 106,007 and 106,008 on
`ebruary 17, 2015.
`
`ANN, Christopher J. et al., "Antisense—induced exon skipping and synthesis of dystrophin in the mdx mouse," PNAS,
`ol. 98(1):42—47 (2001)
`
`ANN, Christopher J. et al., "Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of
`uscular dystrophy," The Journal of Gene Medicine, Vol. 4:644—654 (2002)
`
`ANNINO, Raphael J. et aI., "Liposome Mediated Gene Transfer," BioTechniques, Vol. 6(7):682-690 (1988)
`
`anual of Patent Examining Procedure 2308.02 (6th ed., rev. 3, July 1997), (University of Western Australia Exhibit
`' 143, filed April 3, 2015 in lnterferences 106007, 106008, and 106013, pages 1—2).
`
`anzur A, et al.,. "Glucocorticoid corticosteroids for Duchenne muscular dystrophy," Cochrane Database Syst Rev.
`'004;(2):CDOO3725.
`
`
`
`
`|AVN-009DVCN6
`
`—l—l"NIU'i
`
`—l
`
`|\J|\J—‘O
`
`|\JN
`
`EFS Web 2.1.17
`
`

`

`
`
`
`15349778
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`
`( Not for submission under 37 CFR 1.99)
`
`
`Filing Date
`2016-11-11
`First Named Inventor
`Peter SAZANI
`
`Art Unit
`|1574
`
` Application Number
`
`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
`
`ARSHALL, N.B. et al., "Arginine—rich cell-penetrating peptides facilitate delivery of antisense oligomers into murine
`eukocytes and alter pre—mRNA splicing," Journal of Immunological Methods, Vol. 325:1 14-126 (2007)
`
`athews et al., "Expanded Sequence Dependence of Thermodynamic Parameters Improves Prediction of RNA
`econdary Structure," J. Mol. Biol. 288:911-940 (1999), (University of Western Australia Exhibit 2131, filed April 3,
`' 015 in Interferences 106007, 106008, and 106013, pages 1-31).
`
`athews et al., "Expanded Sequence Dependence of Thermodynamic Parameters Improves Prediction of RNA
`. econdary Structure," J. Mol. Biol., Vol. 288, pp. 911-940 (1999), Exhibit Number 1212 filed in Interferences 106,007
`: nd 106,008 on February 17, 2015.
`
`ATSUO, Masafumi et aI., "Exon Skipping during Splicing of Dystrophin mRNA Precursor due to an Intraexon
`Deletion in the Dystrophin Gene of Duchenne Muscular Dystrophy Kobe," J. Clin. Invest, Vol. 87:2127—2131 (1991)
`
`ATSUO, Masafumi et al., "Treatment of Duchenne Muscular Dystrophy with Oligonucleotides against an Exonic
`. plicing Enhancer Sequence," Basic Appl. Myol., Vol. 13(6):281—285 (2003)
`
`ATSUO, Masafumi, "Duchenne and Becker Muscular Dystrophy: From Gene Diagnosis to Molecular Therapy,"
`UBMB Life, Vol. 53:147-152 (2002)
`
`ATSUO, Masafumi, "Duchenne/Becker muscular dystrophy: from molecular diagnosis to gene therapy," Brain &
`Development, Vol. 18:167—172 (1996)
`
`A'I'I'EUCCI, Mark, "Structural modifications toward improved antisense Oligonucleotides," Perspectives in Drug
`iscovery and Design, Vol. 4:1-16 (1996)
`
`azzone E, et aI. "Functional changes in Duchenne muscular dystrophy: a 12—month longitudinal cohort study,"
`eurology 201 1;77(3):250—6.
`
`
`
`
`|AVN-009DVCN6
`
`|\J|\J4k00
`
`|\J0'1
`
`00|\J|\J|\J|\JO(.003"-l0')
`
`0000N_n
`
`0000
`
`EFS Web 2.1.17
`
`CCARVILLE, M. Beth et al., "Rhabdomyosarcoma in Pediatric Patients: The Good, the Bad, and the Unusual," AJR,
`0|. 176:1563—1569 (2001) (Exhibit Number 1034 filed in interferences 106008, 106007 on November 18, 2014)
`
`CCLOREY, G. et aI., "Antisense oligonucleotide—induced exon skipping restores dystrophin expression in vitro in a
`. nine model of DMD," Gene Therapy, Vol. 13:1373-1381 (2006)
`
`

`

`
`
`
`15349778
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`
`( Not for submission under 37 CFR 1.99)
`
`
`Filing Date
`2016-11-11
`First Named Inventor
`Peter SAZANI
`
`Art Unit
`|1574
`
` Application Number
`
`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
`
`CCLOREY, G. et al., "Induced dystrophin exon skipping in human muscle explants," Neuromuscular Disorders, Vol.
`6:583—590 (2006)
`
`CCLOREY, Graham et al., "Splicing intervention for Duchenne muscular dystrophy," Current Opinion in
`'harmacology, Vol. 5:529-534 (2005)
`
`
`
`
`|AVN-009DVCN6
`
`00000'14:
`
`000')
`
`4:-000000O(.003"-l
`4:-4:-4:-00N—‘
`
`4:.4:.
`
`EFS Web 2.1.17
`
`cDonaId CM, et al., "Profiles of Neuromuscular Diseases, Duchenne muscular dystrophy," Am J Phys Med Rehabil
`995;?4370-392
`
`cDonaId CM, et al., "The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy," Muscle
`erve 2010;41:500—10.
`
`cDonaId CM, et al., "The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations,"
`uscle Nerve 2010;42: 966-74.
`
`endell JR et al., "Evidence—based path to newborn screening for Duchenne muscular Dystrophy," Ann Neurol
`'012;T1:304—13.
`
`endell JR, et al., "Dystrophin immunity revealed by gene therapy in Duchenne muscular dystrophy," N Engl J Med
`'010;363:1429-37.
`
`endell JR, et al., “Randomized, double—blind six-month trial of prednisone in Duchenne's muscular dystrophy," N Eng
`Med 1989;320:1592-97.
`
`ENDELL, Jerry R. et al., "Eteplirsen for the Treatment of Duchenne Muscular Dystrophy," Ann. Neurol., Vol.
`4:637—647 (2013) (Exhibit Number 2058 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`ENDELL, Jerry R. et al., "Eteplirsen in Duchenne Muscular Dystrophy (DMD): 144 Week Update on Six—Minute Walk
`est (6MWT) and Safety," slideshow, presented at the 19th lntemational Congress of the WOI1d Muscle Society, 1?
`ages (2014) (Exhibit Number 2059 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`ONACO, Anthony P. et al., "An Explanation for the Phenotypic Differences between Patients Bearing Partial
`Deletions of the DMD Locus," Genomics, Vol. 2:90—95 (1988)
`
`

`

`
`
`
`
`Application Number 15349778
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`.
`.
`( Not for submrssron under 37 CFR 1.99)
`
`
`Filing Date
`2016-11-11
`First Named Inventor
`Peter SAZANI
`
`Art U .
`mt
`I 1674
`
`Examiner Name
`Not Yet Assigned
`
`ENDELL, Jerry R. et al., "Gene therapy for muscular dystrophy: Lessons learned and path fowvard," Neuroscience
`etters, Vol. 527290-99 (2012)
`
`erlini L, et al., "Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14—year follow—up," Muscle
`erve 2012;45:796—802.
`
` Attorney Docket Number
`xhibit 2132, filed April 3, 2015 in lnterferences 106007, 106008, and 106013, pages 1-2).
`
`
`
`I AVN-OOQDVCNB
`
`ITRPANT, Chalermchai et al., "Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping,"
`olecular Therapy, Vol. 17(8):1418—1426 (2009)
`
`fold illustrations for Exon 51 and Exon 53 with varying amounts of intron sequence, (University of Western Australia
`
`If you wish to add additional non-patent literature document citation information please click the Add button Add
`EXAMINER SIGNATURE
`
`*EXAMINER: Initial if reference considered, whether or not citation is in conformance with MPEP 609. Draw line through a
`citation if not in conformance and not considered.
`Include copy of this form with next communication to applicant.
`
`1 See Kind Codes of USPTO Patent Documents at www.USPTO.GOV or MPEP 901.04. 2 Enter office that issued the document, by the two-letter code (WIPO
`Standard ST.3). 3 For Japanese patent documents, the indication of the year of the reign of the Emperor must precede the serial number of the patent document.
`4 Kind of document by the appropriate symbols as indicated on the document under WIPO Standard ST.16 if possible. 5 Applicant is to place a check mark here if
`English language translation is attached.
`
`EFS Web 2.1.17
`
`

`

`
`
`Application Number 15349778
`
`Filing Date
`2016-11-11
`First Named Inventor
`Peter SAZANI
`
`Art U .
`
`mt
`I 1674
`Examiner Name
`Not Yet Assigned
`I AVN-009DVCN6
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`.
`.
`( Not for submissmn under 37 CFR 1.99)
`
`
`
` Attorney Docket Number
`
`
`
`
`
`Please see 37 CFR 1.97 and 1.98 to make the appropriate selection(s):
`
`CERTIFICATION STATEMENT
`
`That each item of information contained in the information disclosure statement was first cited in any communication
`from a foreign patent office in a counterpart foreign application not more than three months prior to the filing of the
`information disclosure statement. See 37 CFR 1.97(e)(1).
`
`OR
`
`That no item of information contained in the information disclosure statement was cited in a communication from a
`
`foreign patent office in a counterpart foreign application, and, to the knowledge of the person signing the certification
`after making reasonable inquiry, no item of information contained in the information disclosure statement was known to
`any individual designated in 37 CFR 1.56(c) more than three months prior to the filing of the information disclosure
`statement. See 37 CFR 1.97(e)(2).
`
`El
`
`See attached certification statement.
`
`The fee set forth in 37 CFR 1.17 (p) has been submitted herewith.
`
`A certification statement is not submitted herewith.
`
`SIGNATURE
`
`A signature of the applicant or representative is required in accordance with CFR 1.33, 10.18. Please see CFR 1.4(d) for the
`form of the signature.
`
`This collection of information is required by 37 CFR 1.97 and 1.98. The information is required to obtain or retain a benefit by the
`public which is to file (and by the USPTO to process) an application. Confidentiality is governed by 35 U.S.C. 122 and 37 CFR
`1.14. This collection is estimated to take 1 hour to complete, including gathering, preparing and submitting the completed
`application form to the USPTO. Time will vary depending upon the individual case. Any comments on the amount of time you
`require to complete this form andlor suggestions for reducing this burden, should be sent to the Chief Information Officer, U.S.
`Patent and Trademark Office, U.S. Department of Commerce, PO. Box 1450, Alexandria, VA 22313-1450. DO NOT SEND
`FEES OR COMPLETED FORMS TO THIS ADDRESS. SEND TO: Commissioner for Patents, P.O. Box 1450, Alexandria,
`VA 22313-1450.
`
`EFS Web 2.1.17
`
`
` NamelPrint
`Signature
`2016—12—21
`
`
`
`