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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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`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
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`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
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`AMMOND, Suzan M. et al., "Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel
`' EN—modified Oligonucleotides," Molecular Therapy — Nucleic Acids, Vol. 3:1, 11 pages (2014) (Exhibit Number 2011
`"ed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`ammond, Suzan M., et al., "Genetic therapies for RNA mis—splicing diseases," Cell, Vol.27, No. 5, pp. 196—205 (May,
`'011), Exhibit Number 1113 filed in interferences 106,007 and 106,008 on February 17, 2015.
`
`ammond,Suzan M., et al., "PRO—051, an antisense oligonucleotide for the potential treatment of Duchenne muscular
`ystrophy," Curr. Opinion MoI. Therap., Vol. 12, No.4, pp. 478—486 (2010), Exhibit Number 1121 filed in interferences
`06,007 and 106,008 on February 13,2015.
`
`ARDING, PL et al., "The Influence of Antisense Oligonucleotide Length on Dystrophin Exon Skipping," Molecular
`herapy, Vol. 15(1):157—166 (2007) (Exhibit Number 1030 filed in interferences 106008, 106007 on November 18,
`
`AREL—BELLAN, Annick et al., "Specific Inhibition of c—myc Protein Biosynthesis Using an Antisense Synthetic Deoxy—
`oligonucleotide in Human T Lymphocytes," The Journal of Immunology, Vol. 140(7):2431-2435 (1988)
`
`avenga, M.J.E., et al., "Exploiting the Natural Diversity in Adenovirus Tropism for Therapy and Prevention of
`Iisease," J. Virol., Vol. 76, No. 9, pp. 4612—4620 (May, 2002), Exhibit Number 1123 filed in interferences 106,007 and
`06,008 on February 13, 2015.
`
`EASMAN, Janet, "Morpholino Oligos: Making Sense of Antisense?" Developmental Biology, Vol. 243:209—214 (2002)
`
`EEMSKERK, Hans A. et al., "In vivo comparison of 2'—O—methyl phosphorothioate and morpholino antisense
`ligonucleotides for Duchenne muscular dystrophy exon skipping," The Journal of Gene Medicine, Vol. 11:257-266
`2009) (Exhibit Number 2020 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`EID, Christian A. et al., "Real Time Quantitative PCR," Genome Research, Vol. 6:986—994 (1996) (Exhibit Number
`061 filed in interferences 106008, 106007 on November 18,2014)
`
`ERSCHLAG, Daniel et al., "Contributions of 2' Hydroxyl Groups of the RNA Substrate to Binding and Catalysis by the
`etrahymena Ribozyme: An Energetic Picture of an Active Site Composed of RNA," Biochemistry, Vol. 32:8299—8311
`1993) (Exhibit Number 1031 filed in interferences 106008, 106007 on November 18, 2014)
`
`offman EP, et al., "Characterization of dystrophin in muscle—biopsy specimens from patients with Duchenne's or
`:ecker‘s muscular dystrophy" N Engl J Med 1988;318:1363-68.
`
`EFS Web 2.1.17
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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
`
`offman EP, et al., "Restoring dystrophin expression in Duchenne muscular dystrophy muscle: Progress in exon
`kipping and stop codon read through," Am J Path 2011;179:12—22.
`
`UDZIAK, Robert M. et al., "Antiproliferative Effects of Steric Blocking Phosphorodiamidate Morpholino Antisense
`gents Directed against c—myc," Antisense & Nucleic Acid Drug Development, Vol. 10:163-176 (2000) (Exhibit Numbe
`032 filed in interferences 106008, 106007 on November 18, 2014)
`
`UDZIAK, Robert M. et al., "Resistance of Morpholino Phosphorodiamidate Oligomers to Enzymatic Degradation,"
`‘ ntisense & Nucleic Acid Drug Development, Vol. 6:267-272 (1996)
`
`USSEY, Nicole D. et al., "Analysis of five Duchenne muscular dystrophy exons and gender determination using
`nventional duplex polymerase chain reaction on single cells," Molecular Human Reproduction, Vol. 5(11):1089—1094
`1999)
`
`nterim Guidance on Patent Subject Matter Eligibility ("the December Guidance," 16 pages,(Exhibit Number 2119 filed
`n interferences 106,007 and 106,008 on February 17, 2015.
`
`ntemational Patent Application No. PCT/AU2000l00693 ("Wraight"), published as WO 00/78341 on December 28,
`'000, 201 pages, (Exhibit Number 2125 filed in interferences 106,007 and 106,008 on February 17, 2015.
`
`ntemational Preliminary Report on Patentability and Written Opinion for Application No. PCT/US2009I061960, 8
`ages, dated April 26, 2011
`
`ntemational Preliminary Report on Patentability for Application No. PCT/AU2005/000943, 8 pages, dated December
`
`ntemational Preliminary Report on Patentability, PCT/U32013l077216, dated June 23, 2015, pages 1—7.
`
`ntemational Preliminary Report on Patentability, PCT/U32014l029610, dated July 1, 2015, pages 1-122.
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`|AVN-009DVCN6 ntemational Preliminary Report on Patentability, PCT/U32014l029689, dated September 15, 2015, pages 1-10.
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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
`
`ntemational Preliminary Report on Patentability, PCT/U32014l029766, dated September 15, 2015, pages 1-10.
`
`ntemational Search Report and Written Opinion of the lntemational Searching Authority issued in International Patent
`‘ pplication No. PCT/US2013I077216, 5 pages, dated March 27, 2014
`
`ntemational Search Report and Written Opinion of the lntemational Searching Authority issued in International Patent
`‘ pplication No. PCT/US2014I029610, 6 pages, dated September 18, 2014
`
`ntemational Search Report and Written Opinion of the lntemational Searching Authority issued in International Patent
`‘ pplication No. PCT/US2014I029689, 8 pages, dated October 21, 2014
`
`ntemational Search Report and Written Opinion of the lntemational Searching Authority issued in International Patent
`‘ pplication No. PCT/US2014I029766, 8 pages, dated October 21, 2014
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`|AVN-009DVCN6
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`EFS Web 2.1.17
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`ntemational Search Report for Application No. PCT/AU2005/000943, 5 pages, dated October 20, 2005
`
`ntemational Search Report for Application No. PCT/USO1I14410, 5 pages, dated March 6, 2002
`
`ntemational Search Report for Application No. PCT/U32009l061960, 9 pages, dated April 6, 2010
`
`nvitation to pay fees and Partial lntemational Search Report issued by the International Search Authority in
`ntemational Patent Application No. PCT/USZO14I029689, 8 pages, dated July 29, 2014
`
`SIS Pharmaceuticals website, 2 pages, http:/lwwwisispharm.comlPipelinelTherapeutic—Arealether.htm (2014)
`Exhibit Number 2021 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`VERSEN, Patrick L. et al., "Efficacy of Antisense Morpholino Oligomer Targeted to c—myc in Prostate Cancer
`' enograft Murine Model and a Phase I Safety Study in Humans," Clinical Cancer Research, Vol. 92510-2519 (2003)
`
`

`

`
`
`
`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
`
`ARVER, Peter at al., "A Chemical View of Oligonucleotides for Exon Skipping and Related Drug Applications,"
`ucleic Acid Therapeutics, Vol. 24(1):37-47 (2014) (Exhibit Number 2061 filed in interferences 106008, 106013,
`06007 on November 18, 2014)
`
`ASON, Tracey L.H. et al., "Toxicology of antisense therapeutics," Toxicology and Applied Pharmacology, Vol.
`'01 :66—83 (2004) (Exhibit Number 2027 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`EARAWIRIYAPAISARN, Natee et al., "Long-term improvement in mdx cardiomyopathy afler therapy with peptide—
`njugated morpholino oligomers," Cardiovascular Research, Vol. 85:444-453 (2010)
`
`EARAWIRIYAPAISARN, Natee et al., "Sustained Dystrophin Expression Induced by Peptide—conjugated Morpholino
`oligomers in the Muscles of mdx Mice," Mol. Ther_, Vol. 16(9):1624—1629 (2008)
`
`ob Posting by Sarepta for "Scientist II, Muscle Biology" (2 pages), (Academisch Ziekenhuis Leiden Exhibit 1233, filed
`‘ pril 3, 2015 in Interference 106007 and 106008).
`
`
`
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`|AVN-009DVCN6
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`00000'14:
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`000')
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`4:-000000O(.003"-I
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`4:-4:-N_n
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`4:.00
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`4:.4:.
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`EFS Web 2.1.17
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`ONES, Simon S. et al., "The Protection of Uracil and Guanine Residues in Oligonucleotide Synthesis," Tetrahedron
`etters, Vol. 22(47):4755—4758 (1981)
`
`A RLEN, Yann et al., "Statistical significance of quantitative PCR," BMC Bioinforrnatics, 8:131, 16 pages (2007)
`Exhibit Number 1033 filed in interferences 106008, 106007 on November 18, 2014)
`
`‘ RRAS, James G. et al., "Deletion of Individual Exons and Induction of Soluble Murine Interleukin—5 Receptor—alpha
`hain Expression through Antisense Oligonucleotide-Mediated Redirection of Pre-mRNA splicing," Molecular
`'harmacology, Vol. 58:380-387 (2000)
`
`YE, Ed, "Results of the Eteplirsen Phase 2b and Phase 2b Extension Study in Duchenne Muscular Dystrophy," 8th
`‘ nnual Meeting of the Oligonucleotide Therapeutics Society, Session 9: Advances in Oligonucleotide Clinical
`Development II, Page 48 (2012)
`
`INALI, Maria et al., "Local restoration of dystrophin expression with the morpholino oligomer AVI—4658 in Duchenne
`uscular dystrophy: a single-blind, placebo—controlled, dose—escalation, proof-of—concept study," Lancet Neuro|., Vol.
`: :918—928 (2009)
`
`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.
`
`

`

`
`
`
`
`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
`
` Attorney Docket Number
`
`
`ab-on-a-Chip Data, Pages 28, Exhibit Number 1185 filed in lnterferences 106,007 and 106,008 on February 16,
`
`I AVN-009DVCN6
`
`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 aI., "Quality of life, physical disability and respiratory impairment in Duchenne muscular dystrophy," Am J
`espir Crit Care Med 2005;172:1032-6.
`
`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)
`
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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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