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`| 5789862
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`IN FORMATION DISCLOSU RE
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`STATEMENT BY APPLICANT
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`( Not for submission under 37 CFR 1.99)
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`U.S.PATENTS
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`Include name of the author (in CAPITAL LETTERS), title Of the article (when appropriate), title of the item
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`(book, magazine, journal, serial, symposium, catalog, etc), date, pageS(S), volume-issue number(s),
`publisher, city and/or country where published.
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`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
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`( Not for submissmn under 37 CFR 1.99)
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`Application Number 15789862
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`Filing Date
`2017-10-20
`First Named Inventor
`Peter SAZANI
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`Art U .
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`mt
`I N/A
`Examiner Name
`Not Yet Assigned
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` Attorney Docket Number
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`I AVN-009DVCN7
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`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.
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`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, 17
`ages (2014) (Exhibit Number 2059 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`ENDELL, Jerry R. et al., "Gene therapy for muscular dystrophy: Lessons learned and path fowvard," Neuroscience
`etters, Vol. 527:90-99 (2012)
`
`erlini L, et al., "Early conicosteroid treatment in 4 Duchenne muscular dystrophy patients: 14—year follow—up," Muscle
`erve 2012;45:796—802.
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`fold illustrations for Exon 51 and Exon 53 with varying amounts of intron sequence, (University of Western Australia
`xhibit 2132, filed April 3, 2015 in lnterferences 106007, 106008, and 106013, pages 1-2).
`
`ITRPANT, Chalermchai et al., "Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping,"
`olecular Therapy, Vol. 17(8):1418—1426 (2009)
`
`ONACO, Anthony P. et al., "An Explanation for the Phenolypic Differences between Patients Bearing Partial
`Deletions of the DMD Locus," Genomics, Vol. 2:90—95 (1988)
`
`orcos, Paul A., "Gene switching: analyzing a broad range of mutations using steric block antisense oligonucleotides,"
`ethods in Enzymology, Vol. 313:174—189 (1999)
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`OULTON, H.M., "Compound and Method for Treating Myotonic Dystrophy," US. Application No. 12/493,140, 82
`ages, filed June 26, 2009
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`EFS Web 2.1.17
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`15789862
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`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
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`( Not for submission under 37 CFR 1.99)
`
`
`Filing Date
`2017-10-20
`First Named Inventor
`Peter SAZANI
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`Art Unit
`|NIA
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` Application Number
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`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
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`(A)N
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`4:.
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`OULTON, Hong M. et al., "Morpholinos and their peptide conjugates: Therapeutic promise and challenge for
`I uchenne muscular dystrophy," Biochimica et Biophysica Acta, Vol. 1798:2296—2303 (2010)
`
`untoni F, et al., "Dystrophin and mutations: one gene, several proteins, multiple phenotypes," Lancet Neurol.
`'003;2:731—40.
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`UNTONI, Francesco et al., "128th ENMC lntemational Workshop on 'Preclinical optimization and Phase IIII Clinical
`rials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy' 22—24 October 2004, Naarden, The
`etherlands," Neuromuscular Disorders, Vol. 15:450—457 (2005) (Exhibit Number 2025 filed in interferences 106008,
`06013, 106007 on November 18, 2014)
`
`UNTONI, Francesco et al., "149th ENMC lntemational Workshop and 1st TREAT-N MD Workshop on: 'Planning
`'hase II” Clinical trials using Systemically Delivered Antisense Oligonucleotides in Duchenne Muscular Dystrophy,
`euromuscular Disorders, Vol. 18:268—275 (2008)
`
`ELSON, David L. et al., "Nucleotides and Nucleic Acids," Lehninger Principles of Biochemistry, 3rd Edition, Chapter
`0, pages 325-328 and glossary page 6—1 1, Worth Publishers, New York (2000)
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`|AVN-009DVCN7 'altial European Search Report for Application No. 121629950, 6 pages, dated October 2,2012
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`guyen TM, et. A|., "Use of Epitope libraries to identify exon-specific monoclonal antibodies for characterization of
`ltered dystrophins in muscular dystrophy," Am J Hum Genet 1993;52:1057—66.
`
`oberbauer, "Renal uptake of an 18—mer phosphorothioate oligonucleotide," Kidney lnt'l, Vol. 48, pp. 1226—1232 (1995),
`xhibit Number 1191 filed in Interferences 106,007 and 106,008 on February 17, 2015.
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`oligonucleotide Cleavage and Deprotection Laboratory Notebook Entry, Pages 1, Exhibit Number 1138 filed in
`nterferences 106,007 and 106,008 on February 16, 2015.
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`O
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`oligonucleotide diagrams, 5 pages (Exhibit Number 1053 filed in interferences 106008, 106007 on November 18,
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`'altial European Search Report for Application No. 100042746, 6 pages, dated October 2, 2012
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`15789862
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`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`
`( Not for submission under 37 CFR 1.99)
`
`
`Filing Date
`2017-10-20
`First Named Inventor
`Peter SAZANI
`
`Art Unit
`|NIA
`
` Application Number
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`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
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`'atentee's Response to European Patent Application No. 050767706, dated July 28, 2006, 4 pages
`
`'atrick 0. Brown and Tidear D. Shalon v. Stephen P.A. Fodor, Dennis W. Solas and William J. Dower: Interference
`erits Panel, Interference No. 104,358, 24 pages, dated August 9, 1999 (Exhibit Number 2113 filed in interferences
`06008, 106013, 106007 on November 18, 2014)
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`'CT Application as—filed for application No. PCT/NL03700214, 71 pages, dated September 21, 2005 (Exhibit Number
`'042 filed in interferences 106008, 106013, 106007 on November 18, 2014)
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`'D-10 Desalting Columns, Pages 12, Exhibit Number 1141 filed in lnterferences 106,007 and 106,008 on February 16
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`'opplewell, et al., Design of Phosphorodiamidate Morpholino Oligomers (PMOs) For the Induction of Exon Skipping of
`he Human DMD Gene, DSGT Poster, 2008, 1 page.
`
`'OPPLEWELL, Linda et al., "Design of phosphorodiamidate morpholino oligmers (PMOs) for the induction of exon
`kipping of the human DMD gene," Human Gene Therapy 19(10): ESGCT 2008 Poster Presentations, Page 1174,
`'oster N0. P203
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`'OPPLEWELL, Linda J. et al., "Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the
`uman DMD gene: Implications for future clinical trials," Neuromuscular Disorders, Vol. 20(2):102—1 10 (2010) 9 pages
`Exhibit Number 2031 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`'OPPLEWELL, Linda J. et al., "Design of Antisense Oligonucleotides for Exon Skipping of the Human Dystrophin
`ene," Human Gene Therapy 19(4): BSGT 2008 Poster Presentation, Page 407, Poster No. P—35
`
`'OPPLEWELL, Linda J. et al., "Design of Phosphorodiamidate Morpholino Oligomers (PMOs) for the Induction of
`xon Skipping of the Human DMD Gene," Molecular Therapy, Vol. 17(3):554—561 (2009)
`
`'OPPLEWELL, Linda J. et al., "Targeted Skipping of Exon 53 of the Human DMD Gene Recommendation of the
`ighly Efficient Antisense Oligonucleotide for Clinical Trial," Human Gene Therapy 20(4): BSGT 2009 Poster
`' resentations, Page 399, Poster N0. P10
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`'oster Abstract Listing for The Tenth Annual Meeting of the RNA Society, held at the Banff Centre for Conferences, in
`anff, Alberta, Canada, from May 24-29, 2005, (University of Western Australia Exhibit 2137, filed April 3, 2015 in
`nterferences 106007, 106008, and 106013, pages 1—11).
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`15789862
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`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`
`( Not for submission under 37 CFR 1.99)
`
`
`Filing Date
`2017-10-20
`First Named Inventor
`Peter SAZANI
`
`Art Unit
`|NIA
`
` Application Number
`
`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
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`'ramono, "Induction of Exon Skipping of the Dystrophin Transcript in Lymphoblastoid Cells by Transfecting an
`. ntisense Oligodeoxynucleotide Complementary to an Exon Recognition Sequence," Biochem. and Biophy. Res.
`omm., Vol. 226, pp. 445-449 (1996), Exhibit Number 1192 filed in Interferences 106,007 and 106,008 on February
`7, 2015.
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`'reliminary Amendment for Application No. 127976381, 4 pages, dated December 22, 2010 (Exhibit Number 2066
`"ed in interferences 106008, 106013, 106007 on November 18, 2014)
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`' reliminary Amendment for Application No. 127198007, 3 pages, dated November 7, 2008 (Exhibit Number 2067 filed
`n interferences 106008, 106013, 106007 on November 18, 2014)
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`'rescribing Information for EXONDYS 51 (eteplirsen) Injection, dated 09/2016, 10 pages
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`'rogram Schedule for The Tenth Annual Meeting of the RNA Society, held at the Banff Centre for Conferences, in
`anff, Alberta, Canada, from May 24-29, 2005, (University of Western Australia Exhibit 2136, filed April 3, 2015 in
`nterferences 106007, 106008, and 106013, pages 1-4).
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`EFS Web 2.1.17
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`'roliferation and Differentiation of Myoblast Cultures, Pages 2, Exhibit Number 1169 filed in Interferences 106,007
`: nd 106,008 on February 16, 2015.
`
`'rosensa Press Release, dated October 10, 2014 (2 pages), Exhibit Number 1203 filed in Interferences 106,007 and
`06,008 on February 17, 2015.
`
`'rosensa, "GSK and Prosensa Announce Primary Endpoint Not Met in Phase I” Study of Drisapersen in Patients With
`I uchenne Muscular Dystrophy," press release, 4 pages, dated September 20, 2013 (Exhibit Number 2039 filed in
`nterferences 106008, 106013, 106007 on November 18, 2014)
`
`az et al. v. Davis et al., Board of Patent Appeals and Inteferences, Patent and Trademark Office, Int. No. 105,712,
`ech. Ctr. 1600, September 29, 2011 (24 pages) (2011 WL 4568986 (Bd.Pat.App. & Interf.), Exhibit Number 1209 filed
`n Interferences 106,007 and 106,008 on February 17, 2015.
`
`'EESE, Colin B. et al., "Reaction Between 1-ArenesulphonyI-3-Nitro-1,2,4-Triazo|es and Nucleoside Base Residues.
`lucidation of the Nature of Side-Reactions During Oligonucleotide Synthesis," Tetrahedron Letters, Vol. 21 :2265—226
`1980)
`
`'EESE, Colin B. et al., "The Protection of Thymine and Guanine Residues in Oligodeoxyribonucleotide Synthesis," J.
`hem. Soc. Perkin Trans. 1, pages 1263—1271 (1984)
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`

`

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`
`
`Application Number 15789862
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`.
`.
`( Not for submissmn under 37 CFR 1.99)
`
`
`Filing Date
`2017-10-20
`First Named Inventor
`Peter SAZANI
`
`Art U .
`mt
`I N/A
`
`Examiner Name
`Not Yet Assigned
`
` Attorney Docket Number
`
`
`06,007 and 106,008 on February 17, 2015.
`
`I AVN-OOQDVCNT
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`eexamination Certificate — Application No. 90/011,320, issued March 27, 2012, 2 pages, (Exhibit Number 1072 filed i
`nterferences 106008, 106007 on December 23, 2014)
`
`eply to EPO Communication dated June 26, 2014 in European Application Serial No. 13160338, (University of
`estern Australia Exhibit 2145, filed April 3, 2015 in Interferences 106007, 106008, and 106013, pages 1—4).
`
`eply to EPO Communication dated October 21, 2014 in European Application Serial No. 12198517, (University of
`estern Australia Exhibit 2148, filed April 3,2015 in Interferences 106007, 106008, and 106013, pages 1—7).
`
`eply to EPO Communication dated October 23, 2014 in European Application Serial No. 12198485, (University of
`estern Australia Exhibit 2147, filed April 3,2015 in Interferences 106007, 106008, and 106013, pages 1—8).
`
`esponse to Office Action and Amendments to the Claims for Application No. 131550210, 10 pages, dated May 12,
`'014 (Exhibit Number 2064 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`'hodes et al., "BioMarin Bulks Up," BioCentury, pp. 6—8 (December, 2014), Exhibit Number 1193 filed in Interferences
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`Application Number 15789862
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`Filing Date
`2017-10-20
`First Named Inventor
`Peter SAZANI
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`Art U .
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`mt
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`Examiner Name
`Not Yet Assigned
`I AVN-009DVCN7
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`IN FORMATION DISCLOSU RE
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