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`Receiptdajze; 10/10/2013
`15/789j52 —GAU: 1574
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`Application Number
`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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`|AVN-009DVCN7
`
`ing et al., "A Dictionary of Genetics," Oxford University Press, 4th Ed. (1990), Exhibit Number 1189 filed in
`nterferences 106,007 and 106,008 on February 17, 2015.
`
`OENIG, M. et al., "The Complete Sequence of Dystrophin Predicts a Rod-Shaped Cytoskeleton Protein," Cell, Vol.
`. 3:219—228 (1988) (Exhibit Number 1010 filed in interferences 106008, 106007 on November 18, 2014)
`
`OENIG, M. et al., "The Molecular Basis for Duchenne versus Becker Muscular Dystrophy: Correlation of Severity with
`ype of Deletion," Am. J. Hum. Genet, Vol. 45:498—506 (1989) (Exhibit Number 1011 filed in interferences 106008,
`06007 on November 18, 2014)
`
`ohler M, et al., "Quality of life, physical disability and respiratory impairment in Duchenne muscular dystrophy," Am J
`espir Crit Care Med 2005;172:1032-6.
`
`OLE et al. "Exon skipping therapy for Duchenne muscular dystrophy," Advanced Drug Delivery Reviews, vol.
`.7:104—107 (2015).
`
`OSHKIN, Alexei A. et al., "LNA (Locked Nucleic Acids): Synthesis of the Adenine, Cytosine, Guanine, 5-
`ethylcytosine, Thymine and Uracil Bicyclonucleoside Monomers, Oligomerisation, and Unprecedented Nucleic Acid
`ecognition," Tetrahedron, Vol. 54:3607—3630 (1998) (Exhibit Number 2007 filed in interferences 106008, 106013,
`06007 on November 18, 2014)
`
`urreck J., "Antisense Technologies: Improvement Through Novel Chemical Modifications", European Journal of
`iochemistry, Vol.270(8):1628—1644 (2003)
`
`ab-on-a-Chip Data, Pages 28, Exhibit Number 1185 filed in Interferences 106,007 and 106,008 on February 16,
`
`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.
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`Receiptdajze; 10/10/2013
`15/789j52 —GAU: 1574
`
`Application Number
`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
`
`
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`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
`|AVN-009DVCN7
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`(A)N
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`4:.
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`—l—l"NlU'i
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`—l
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`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.
`
`|\J|\J—‘O
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`|\JN
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`etter from the FDA to Sarepta Therapeutics, Inc., Re: ACCELERATED APPROVAL for the use of Exondys 51
`eteplirsen), FDA Reference ID: 3987286, dated September 19, 2016, 11 pages.
`
`etter to the US Food and Drug Administration, (Dr. Billy Dunn, MD. Director Division of Neurology Products, Office
`f Drug Evaluation 1, Center for Drug Evaluation and Research), for The Peripheral and Central Nervous System
`dvisory Committee Meeting (AdComm) supporting approval of eteplirsen, dated February 24, 2016, 4 pages.
`
`etter to the US Food and Drug Administration, (Dr. Janet Woodcock, MD. Director, CDER), from The Congress of
`he United States regarding Duchenne muscular dystrophy, dated February 17, 2016, 7 pages.
`
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`15789862
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`IN FORMATION DISCLOSU RE
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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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`Not Yet Assigned
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`Attorney Docket Number |AVN-009DVCNT
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`|\J00
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`|\J4:.
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`|\JUt
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`00|\J|\J|\J|\JO(.0CO"-I0')
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`00 _.
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`0000
`
`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.
`
`EFSWeb2.1.17
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`Receipt. date; Ill/WELT
`15/7&9,_8_62 — GAU: 1574
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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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`Examiner Name
`Not Yet Assigned
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`Attorney Docket Number |AVN-009DVCNT
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`004:.
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`00UI
`
`000')
`
`4:-000000O(.003"-l
`
`4:. _.
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`4:.N
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`4:.00
`
`4:.4:.
`
`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. 325:114-126 (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 al., "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.
`
`
`
`CCARVILLE, M. Beth at 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 al., "Antisense oligonucleotide—induced exon skipping restores dystrophin expression in vitro in a
`. nine model of DMD," Gene Therapy, Vol. 13:1373-1381 (2006)
`
`EFSWeb2.1.17
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`ALL REFERENCES CONSIDERED EXCEPT WHERE LINED THROUGH.
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`Receipt. date; 111/10/21113
`15/739,1162 — GAU: 1574
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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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` Application Number
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`Examiner Name
`Not Yet Assigned
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`Attorney Docket Number |AVN-009DVCNT
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`CCLOREY, G. et aI., "Induced dystrophin exon skipping in human muscle explants," Neuromuscular Disorders, Vol.
`6:583—590 (2006)
`
`CCLOREY, Graham et aI., "Splicing intervention for Duchenne muscular dystrophy," Current Opinion in
`'harmacology, Vol. 5:529-534 (2005)
`
`'012;T1:304—13.
`
`cDonaId CM, et aI., "Profiles of Neuromuscular Diseases, Duchenne muscular dystrophy," Am J Phys Med Rehabil
`99574370692
`
`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
`
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`Receipt. date; 10/10/2013
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`15789862
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