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`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 .
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`mt
`I 1674
`Examiner Name
`Not Yet Assigned
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` Attorney Docket Number
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`I AVN-OOQDVCNB oligonucleotide diagrams, 5 pages (Exhibit Number 1053 filed in interferences 106008, 106007 on November 18,
`
`orcos, Paul A., "Gene switching: analyzing a broad range of mutations using steric block antisense Oligonucleotides,"
`ethods in Enzymology, Vol. 313:174-189 (1999)
`
`OULTON, H.M., "Compound and Method for Treating Myotonic Dystrophy," US. Application No. 12/493,140, 82
`ages, filed June 26, 2009
`
`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_
`
`UNTONI, Francesco et at, "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 at, "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)
`
`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.
`
`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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`EFS Web 2.1.17
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`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
`
`'altial European Search Report for Application No. 100042746, 6 pages, dated October 2, 2012
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`'altial European Search Report for Application No. 121629950, 6 pages, dated October 2, 2012
`
`'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)
`
`'CT Application as—filed for application No. PCT/NL03l00214, 71 pages, dated September 21, 2005 (Exhibit Number
`'042 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`'D-10 Desalting Columns, Pages 12, Exhibit Number 1141 filed in lnterferences 106,007 and 106,008 on February 16
`
`'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
`
`'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)
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`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
`
`'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
`
`'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).
`
`'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.
`
`'reliminary Amendment for Application No. 121976381, 4 pages, dated December 22, 2010 (Exhibit Number 2066
`"ed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`' reliminary Amendment for Application No. 12l198,007, 3 pages, dated November 7, 2008 (Exhibit Number 2067 filed
`n interferences 106008, 106013, 106007 on November 18, 2014)
`
`'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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`|AVN-009DVCN6
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`0000
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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. & |nterf.), 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)
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`

`

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`
`
`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
`
`'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)
`
`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)
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`|AVN-009DVCN6
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`00000'14:
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`4:-000000O(.003"-I
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`EFS Web 2.1.17
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`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
`06,007 and 106,008 on February 17, 2015.
`
`'NA Isolation Using RNA-BEE, Pages 1, Exhibit Number 1175 filed in Interferences 106,007 and 106,008 on
`ebruary 16, 2015.
`
`'OBERTS, Roland G. et al., "Exon Structure of the Human Dystrophin Gene," Genomics, Vol. 16:536—538 (1993)
`
`oest et al., "Application of In Vitro Myo—Differentiation of Non-Muscle Cells to Enhance Gene Expression and
`acilitate Analysis of Muscle Proteins," Neuromuscul. Disord., Vol. 6, No. 3, pp. 195-202 (May, 1996), Exhibit Number
`124 filed in interferences 106,007 and 106,008 on February 17, 2015.
`
`'OSSO, Mario G. et al., "An Arabidopsis thaliana T-DNA mutagenized population (GABI—Kat) for flanking sequence
`ag-based reverse genetics," Plant Molecular Biology, Vol. 53:247—259 (2003)
`
`

`

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`
`
`
`Application Number 15349778
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`.
`.
`( Not for submissmn 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
`
`.aito, T. et al., "First—in—Human Study of NS-0651'NCNP-01; the Morpholino Based Antisense Oligonucleotide for Exon
`3 Skipping in Duchenne Muscular Dystrophy," ASGCT meeting , May 13, 2015, Abstract [136] 1 page.
`
`.aito, T. et al., "First—in—Human Study of NS-0651'NCNP-01; the Morpholino Based Antisense Oligonucleotide for Exon
`3 Skipping in Duchenne Muscular Dystrophy," ASGCT meeting , May 13, 2015, pages 1—1 1.
`
` Attorney Docket Number
`ated January 22, 2016.
`
`
`
`I AVN-OOQDVCNB
`
`.arepta Therapeutics Press Release, dated January 12, 2015, Exhibit Number 1119 filed in interferences 106,007
`: nd 106,008 on February 17, 2015.
`
`arepta Therapeutics, Advisory Committee Briefing Materials: Available for Public Release, "Peripheral and Central
`ervous System Drugs Advisory Committee," Eteplirsen Briefing Document Addendum, NDA 206488, pages 1-9,
`
`If you wish to add additional non-patent literature document citation information please click the Add button Add
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`
`
`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
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`IN FORMATION DISCLOSU RE
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