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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
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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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`I N/A
`Examiner Name
`Not Yet Assigned
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`I AVN-OOQDVCNT
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`IaxoSmithKline, "Prosensa regains rights to drisapersen from GSK and retains rights to all other programmes for the
`reatment of Duchenne muscular dystrophy (DMD), press release, 4 pages, dated January 13, 2014 (Exhibit 2040 in
`nterferences 106007, 106008, and 106013 on November 18, 2014).
`
`OEMANS, Nathalie M. et aI., "Systemic Administration of PR0051 in Duchenne's Muscular Dystrophy," The New
`ngland Journal of Medicine, Vol. 364:1513-1522 (2011) (Exhibit Number 2036 filed in interferences 106008, 106013,
`06007 on November 18, 2014)
`
`ORDON, Peter M. et aI., "Metal ion catalysis during the axon-ligation step of nuclear pre-mRNA splicing: Extending
`he parallels between the spliceosome and group II introns," RNA, Vol. 6:199-205 (2000) (Exhibit Number 1055 filed in
`nterferences 106008, 106007 on November 18,2014)
`
`ordon, Peter M., et aI., "Kinetic Characterization of the Second Step of Group II Intron Splicing: Role of Metal Ions
`-nd the Cleavage Site 2'—OH in Catalysis," Biochemistry, Vol. 39, pp. 12939-12952 (2000), Exhibit Number 1188 filed
`n Interferences 106,007 and 106,008 on February 17, 2015.
`
`OYENVALLE, Aurelie et al., "Prevention of Dystrophic Pathology in Severely Affected DystrophinIUtrophin—deficient
`ice by Morpholino-oligomer—mediated Exon—skipping," Molecular Therapy, Vol. 18(1):198—205 (2010)
`
`AMMOND, Suzan M. et aI., "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)
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`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 aI., "PRO—051, an antisense oligonucleotide for the potential treatment of Duchenne muscular
`ystrophy," Curr. Opinion Mol. Therap., Vol. 12, No.4, pp. 478—486 (2010), Exhibit Number 1121 filed in interferences
`06,007 and 106,008 on February 13,2015.
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`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.
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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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`—l—l
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`EASMAN, Janet, "Morpholino Oligos: Making Sense of Antisense?" Developmental Biology, Vol. 243:209—214 (2002)
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`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.
`
`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)
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`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/AU2000I00693 ("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.
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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-OOQDVCNT
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`ntemational Preliminary Report on Patentability and Written Opinion for Application No. PCT/US2009/061960, 8
`ages, dated April 26, 2011
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`ntemational Preliminary Report on Patentability for Application No. PCT/AU2005/000943, 8 pages, dated December
`
`ntemational Preliminary Report on Patentability, PCT/U32013IO77216, dated June 23, 2015, pages 1—7.
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`ntemational Preliminary Report on Patentability, PCT/U320147029610, dated July 1, 2015, pages 1-122.
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`ntemational Preliminary Report on Patentability, PCT/U320147029689, dated September 15, 2015, pages 1-10.
`
`ntemational Preliminary Report on Patentability, PCT/U320147029766, 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/US20137077216, 5 pages, dated March 27, 2014
`
`ntemational Search Report and Written Opinion of the lntemational Searching Authority issued in International Patent
`‘ pplication No. PCT/US20147029610, 6 pages, dated September 18, 2014
`
`ntemational Search Report and Written Opinion of the lntemational Searching Authority issued in International Patent
`‘ pplication No. PCT/US20147029689, 8 pages, dated October 21, 2014
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`ntemational Search Report and Written Opinion of the lntemational Searching Authority issued in International Patent
`‘ pplication No. PCT/US20147029766, 8 pages, dated October 21, 2014
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`ntemational Search Report and Written Opinion, PCT/US2016/054534, dated January 17, 2017, 13 pages.
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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
`
`ntemational Search Report for Application No. PCT/AU2005I000943, 5 pages, dated October 20, 2005
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`ntemational Search Report for Application No. PCT/USO1I14410, 5 pages, dated March 6, 2002
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`4:-4:-4:-00N—‘
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`EFS Web 2.1.17
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`ntemational Search Report for Application No. PCT/US2009I061960, 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:/Iwwwisispharm.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)
`
`ARVER, Peter et 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)
`
`ett Foundation Presentation by McSherry, C. "Patient and Caregiver-Reported Outcomes of Patients in Clinical Trials
`f Eteplirsen for Treatment of Duchenne" at Peripheral and Central Nervous System Drugs Advisory Committee, April
`'5, 2016, 17 pages.
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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
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` Attorney Docket Number
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`
`: :918—928 (2009)
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`I AVN-009DVCN7
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`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).
`
`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 aI., "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 Neurot, Vol.
`
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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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