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`IN FORMATION DISCLOSU RE
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`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.
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`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
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`( Not for submrssron 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-009DVCNT
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`'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.
`
`'0550, 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)
`
`.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.
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`.arepta Briefing Information for the April 25, 2016 Meeting of the Peripheral and Central Nervous System Drugs
`dvisory Committee, Eteplirsen Briefing Document, NDA 206488, 186 pages.
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`.arepta Presentation at Peripheral and Central Nervous System Drugs Advisory Committee, April 25, 2016, 133
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`.arepta Press Release, Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the
`reatment of Duchenne Muscular Dystrophy, April 25, 2016, 2 pages
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`.arepta Therapeutics Press Release, dated January 12, 2015, Exhibit Number 1119 filed in interferences 106,007
`: nd 106,008 on February 17, 2015.
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`arepta Therapeutics, Advisory Committee Briefing Materials: Available for Public Release, "Peripheral and Central
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`ervous System Drugs Advisory Committee," Eteplirsen Briefing Document Addendum, NDA 206488, pages 1-9,
`ated January 22, 2016.
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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)
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`
`Filing Date
`2017-10-20
`First Named Inventor
`Peter SAZANI
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`Art Unit
`|NIA
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`|AVN-009DVCN7 equence of Exon 46 of Dyslrophin Gene, 1 page
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` Application Number
`‘ menable to Skipping Exon 51," September 19, 2016, 2 pages.
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`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
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`arepta Therapeutics, Advisory Committee Briefing Materials: Available for Public Release, "Peripheral and Central
`ervous System Drugs Advisory Committee," Eteplirsen Briefing Document, NDA 206488, pages 1-166, dated
`anuary 22, 2016.
`
`arepta Therapeutics, Inc. News Release, "Sarepta Therapeutics Announces FDA Accelerated Approval of EXONDY
`.1T" (eteplirsen) injection, an Exon Skipping Therapy to Treat Duchenne Muscular Dystrophy (DMD) Patients
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`
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`arepta, "AVI BioPharma Initiates Dosing in Phase 2 Study of Eteplirsen in Duchenne Muscular Dystrophy Patients,"
`.ress release, 4 pages, dated August 15, 2011 (Exhibit Number 2082 filed in interferences 106008, 106013, 106007
`n November 18, 2014)
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`arepta, "Sarepta Therapeutics Announces Eteplirsen Demonstrates Continued Stability on Walking Test through 120
`eeks in Phase lib Study in Duchenne Muscular Dystrophy," press release, 3 pages, dated January 15, 2014 (Exhibit
`umber 2034 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`arepta, "Sarepta Therapeutics Reports Long—Term Outcomes through 144 Weeks from Phase llb Study of Eteplirsen
`n Duchenne Muscular Dystrophy," press release, http:/Iinvestorrelations.sareptacomlphoenix.zhtml?c=64231&
`=ir0l—newsArticle&id=1946426, 4 pages, dated July 10, 2014
`
`.cully, Michele et al., "Review of Phase II and Phase III Clinical Trials for Duchenne Muscular Dystrophy", Expert
`opinion on Orphan Drugs, Vol.1 (1 ):33—46 (2013)
`
`.econd Preliminary Amendment filed in US Application No. 13l550,210, 5 pages, dated January 3, 2013 (Exhibit
`umber 2062 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`econd Written Opinion for Application No. PCT/AU2010/001520, 7 pages, dated October 13, 2011
`
`.emi Quantitative Lab-on—Chip Analysis of Second PCR Product, Pages 1, Exhibit Number 1183 filed in lnlerferences
`06,007 and 106,008 on February 16, 2015.
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`.equence Listing — Serial No. 137550210, as filed July 16, 2012 (9 pages), Exhibit Number 1205 filed in lnterferences
`06,007 and 106,008 on February 17, 2015.
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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
`
`equence of Exon 51 of Dystrophin Gene, 1 page
`
`habanpoor et al., "Bi—specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a
`ouse model of Duchenne muscular dystrophy," Nucleic Acids Res., pp. 1—11 (December, 2014), Exhibit Number
`114 filed in interferences 106,007 and 106,008 on February 17, 2015.
`
`.. HAPIRO, Marvin B. el al., "RNA splice junctions of different classes of eukaryotes: sequence statistics and functional
`mplications in gene expression," Nucleic Acids Research, Vol. 15(17):?1 55-7174 (1987)
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`.HERRA'I'I', Tim G. et al., "Exon Skipping and Translation in Patients with Frameshift Deletions in the Dystrophin
`ene," Am. J. Hum. Genet, Vol. 53:1007-1015 (1993)
`
`HIGA, Nobuyuki et al., "Disruption of the Splicing Enhancer Sequence within Exon 27 of the Dystrophin Gene by a
`onsense Mutation Induced Partial Skipping of the Exon and Is Responsible for Becker Muscular Dystrophy," J. Clin.
`nvest., Vol. 100(9):2204—2210 (1997)
`
`.. HIMIZU, Miho et al., "OIigo(2'-O—methyl)ribonucleotides Effective probes for duplex DNA," FEBS Letters, Vol. 302
`2):155-158 (1992) (Exhibit Number 1035 filed in interferences 106008, 106007 on November 18, 2014)
`
`iemens Healthcare Diagnostics, Inc. v. Enzo Life Sciences, Inc., 2013 WL 4411227, *11 [Parallel cite: U.S.D.C., D.
`ass, Civil No. 10-40124—FDS], Decided Aug. 14, 2013 (12 pages); [Cited as: 2013 WL 4411227], Exhibit Number
`210 filed in Interferences 106,007 and 106,008 on February 17, 2015.
`
`.IERAKOWSKA, Halina et al., "Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense
`ligonucleotides," Proc. Natl. Acad. Sci. USA, Vol. 93:12840—12844 (1996)
`
`ontheimer et al., "Metal ion catalysis during group II intron self-splicing: parallels with the spliceosome," Genes &
`Ievelopment, Vol. 13, pp. 1729-1741 (1999), Exhibit Number 1195 filed in lnterferences 106,007 and 106,008 on
`ebruary 17, 2015.
`
`.ontheimer et al., "Three Novel Functional Variants of Human U5 Small Nuclear RNA," Vol. 12, No. 2, pp. 734—746
`Feb., 1992), Exhibit Number 1194 filed in Interferences 106,007 and 106,008 on February 17, 2015.
`
`.ONTHEIMER, Erik J. et al., "Metal ion catalysis during splicing of premessenger RNA," Nature, Vol. 388:801-805
`1997) (Exhibit Number 1036 filed in interferences 106008, 106007 on November 18, 2014)
`
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`
`15789862
`
`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
`
`ONTHEIMER, Erik J. et al., "The U5 and U6 Small Nuclear RNAs as Active Site Components of the Spliceosome,"
`. cience, Vol. 262: 1 989—1 997 (1993) (Exhibit Number 1058 filed in interferences 106008, 106007 on November 18,
`
`.tandard Operating Procedure FPLC Desalting, Pages 6, Exhibit Number 1144 filed in Interferences 106,007 and
`06,008 on February 16, 2015.
`
`.tanton, Robert et al., "Chemical Modification Study of Antisense Gapmers", Nucleic Acid Therapeuliw, Vol. 22(5):
`544—359 (2012)
`
`.tatement On A Nonproprietary Name Adopted By the USAN Council, ETEPLIRSEN, Chemical Structure, 2010,
`
`TEIN, CA, "Delivery of antisense oligonucleotides to cells: a consideration of some of the barriers," Monographic
`upplement sen'es: Oligos & Peptides - Chimica Oggi - Chemistry Today, Vol. 32(2):4—7 (2014) (Exhibit Number 2022
`"ed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`.TEIN, Cy A. et al., "Therapeutic Oligonucleotides: The Road Not Taken," Clin. Cancer Res, Vol. 17(20):6369-6372
`2011) (Exhibit Number 2026 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`.TEIN, David et al., "A Specificity Comparison of Four Antisense Types: Morpholino, 2'-O—Methyl RNA, DNA, and
`'hosphorothioate DNA," Antisense & Nucleic Acid Drug Development, Vol. 7:151-157 (1997)
`
`trober JB, "Therapeutics in Duchenne muscular dystrophy," NeuroRX 2006; 3225—34.
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`EFS Web 2.1.17
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`.ummary of Professional Experience (Dr. Erik J. Sontheimer), Pages 4, Exhibit Number 1223 filed in Interferences
`06,007 and 106,008 on February 17, 2015.
`
`.UMMERTON, James et al., "Morpholino and Phosphorothioate Antisense Oligomers Compared in Cell-Free and In-
`ell Systems," Antisense & Nucleic Acid Drug Development, Vol. 7:63-70 (1997)
`
`.. UMMERTON, James et al., "Morpholino Antisense Oligomers: Design, Preparation, and Propelties," Antisense &
`ucleic Acid Drug Development, Vol. 7:187—195 (1997)
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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
`
`
`nterferences 106,007 and 106,008 on February 17, 2015.
`
`I AVN-009DVCN7
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`. UMMERTON, James, "Morpholino antisense oligomers: the case for an Rnase H-independent structural type,"
`. iochimica et Biophysica Acta, Vol. 1489:141—158 (1999) (Exhibit Number 1038 filed in interferences 106008, 106013,
`06007 on November 18, 2014)
`
`upplementary European Search Report for Application No. 108293671, 8 pages, dated May 22, 2013
`
`uter et al., "Double-target antisense U7 snRNAs promote efficient skipping of an aberrant exon in three human Beta—
`halassemic mutations," 8:13 HUMAN MOLECULAR GENETICS 2415-2423 (1999) (Exhibit Number 1083 filed in
`nterferences 106008, 106007 on December 23,2014)
`
`HOEN, Peter A.C. et al., "Generation and Characterization of Transgenic Mice with the Full—length Human DMD
`ene," The Journal of Biological Chemistry, Vol. 283(9):5899—5907 (2008) Exhibit Number 2030 filed in interferences
`06008, 106013, 106007 on November 18, 2014)
`
`able 1: Primer and Product Details for Exon 51 and 53 Reports on AONs of 20 to 50 Nucleotides dd 07 JAN 2015,
`'ages 1, Exhibit Number 1177 filed in lnterferences 106,007 and 106,008 on February 16,2015.
`
`akeshima et al., "Oligonucleotides against a splicing enhancer sequence led to dystrophin production in muscle cells
`rom a Duchenne muscular dystrophy patient," Brain & Dev., Vol. 23, pp. 788—790 (2001 ), Exhibit Number 1196 filed in
`
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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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`Not Yet Assigned
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`IN FORMATION DISCLOSU RE
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