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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
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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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`mt
`I N/A
`Examiner Name
`Not Yet Assigned
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` Attorney Docket Number
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`I AVN-009DVCN7
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`S Transmittal, application as—filed, and Preliminary Amendment for Application No. 131271,080, 115 pages, dated
`october 11, 2011 (Exhibit Number 2111 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`S Updated Filing Receipt for Application No. 131550210, 3 pages, dated December 11, 2012 (Exhibit Number 2044
`"ed in interferences 106008, 106013, 106007 on November 18, 2014)
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`SPTO "2014 Procedure for Subject Matter Eligibility Analysis of Claims Reciting or Involving. . .Natural Products" ("the
`arch Guidance“), 19 pages, (Exhibit Number 2118 filed in interferences 106,007 and 106,008 on February 17, 2015.
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`SPTO Written Description Training Materials, Revised March 25, 2008, Example 12, 6 pages, (Exhibit Number 1068
`"ed in interferences 106008, 106007 on December 23, 2014)
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`WA Clean Copy of Claims and Sequence, as filed in Interference No. 106,007 on August 1, 2014 (Paper 12), 8
`ages, (Exhibit Number 2126 filed in interferences 106,007 and 106,008 on February 17, 2015.
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`WA Clean Copy of Claims and Sequence, as filed in Interference No. 106,007 on August 7, 2014 (Paper 12), 8
`ages, (Exhibit Number 2127 filed in interferences 106,007 and 106,008 on February 17, 2015.
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`WA Motion 1 (For Judgment Under 35 § 112(a)) from Int. No. 106,007 (PN210), 40 Pages, Exhibit Number 1005 filed
`n Interference 106,013 on February 17, 2015.
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`WA Motion 1 (For Judgment Under 35 § 112(a)) from Int. No. 106,008 (Doc 213), Pages 38, Exhibit Number 1004
`"ed in Interference 106,013 on February 17, 2015.
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`WA submission of teleconference transcript , 28 pages, dated December 12, 2014 (Exhibit Number 2114 filed in
`nterferences 106008 and 106007 on December 12, 2014)
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`alorization Memorandum published by the Dutch Federation of University Medical Centers in March 2009, (University
`f Western Australia Exhibit 2140, filed April 3, 2015 in lnterferences 106007, 106008, and 106013, pages 1—33).
`
`AN DEUTEKOM et al., "Antisense-induced exon skipping restores dystrophin expression in DMD patient derived
`uscle cells," HUMAN MOLECULAR GENETICS Vol. 10, No. 15: 1547—1554 (2001) (Exhibit Number 1084 filed in
`nterferences 106008, 106007 on December 23,2014)
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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
`
`( 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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`AN DEUTEKOM et at, "Local Dystrophin Restoration with Antisense Oligonucleotide PR0051," N. Engl. J. Med., Vol.
`:57, No. 26, pp. 2677—2686 (December, 2007), Exhibit Number 1213 filed in Interferences 106,007 and 106,008 on
`ebruary 17, 2015.
`
`AN DEUTEKOM, Judith C. T. et al., "Advances in Duchenne Muscular Dystrophy Gene Therapy," Nature Reviews
`enetics, Vol. 4(10):774—783 (2003)
`
`an Ommen 2002 PCT (WO 02724906 AI), 43 pages,(Exhibit Number 1071 filed in interferences 106008, 106007 on
`I ecember 23, 2014)
`
`an Putten M, et al., "The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology. PLoS ONE
`'012;7:e31937, 13 pages
`
`an Vliet, Laura et al., "Assessment of the Feasibility of Exon 45—55 Multiexon Skipping for Duchenne Muscular
`Iystrophy", BMC Medical Genetics, Vol.9(1):105 (2008)
`
`ERMA, Sandeep et al., "Modified Oligonucleotides: Synthesis and Strategy for Users," Annu. Rev. Biochem, Vol.
`.7z99—134 (1998) (Exhibit Number 1040 filed in interferences 106008, 106007 on November 18, 2014)
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`ikase Corp. v. Am. Nat'l. Can 00., No. 93—7651, 1996 WL 377054 (N.D. |||. July 1, 1996), 3 pages (Exhibit Number
`' 152 filed in interference 106013 on October 29, 2015)
`
`OIT, Thomas et al., "Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND
`I): an exploratory, randomised, placebo—controlled phase 2 study," Lancet Neuro|., Vol. 13:987-996 (2014) (Exhibit
`umber 2037 filed in interferences 106008, 106013, 106007 on November 18, 2014)
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`O
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`OLLOCH, Vladimir et al., "Inhibition of Pre—mRNA Splicing by Antisense RNA in Vitro: Effect of RNA Containing
`equences Complementary to Exons," Biochemical and Biophysical Research Communications, Vol. 179
`3):1593—1599 (1991)
`
`ahlestedt et al., "Potent and nontoxic antisense oligonucleotides containing locked nucleic acids," PNAS, Vol. 97,
`o. 10, pp. 5633—5638 (May, 2000), Exhibit Number 1201 filed in Interferences 106,007 and 106,008 on February 17,
`
`ang et al., "In Vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for
`I uchenne muscular dystrophy," J. Gene Medicine, Vol. 12, pp. 354-364 (March, 2010), Exhibit Number 1115 filed in
`nterferences 106,007 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
`
`Ar, Unit
`|NIA
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` Application Number
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`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
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`ANG, Chen-Yen et al., "pH—sensitive immunoliposomes mediate target—ceII-specific delivery and controlled
`xpression of a foreign gene in mouse," Proc. Natl. Acad. Sci. USA, Vol. 847851-7855 (1987)
`
`ATAKABE, Akiya et al., "The role of exon sequences in splice site selection," Genes & Development, Vol. 7:407-418
`1993)
`
`atanabe et al., "Plasma Protein Binding of an Antisense Oligonucleotide Targeting Human ICAM—1 (ISIS 2302),"
`oligonucleotides, Vol. 16, pp. 169— 180 (2006), Exhibit Number 1197 filed in Interferences 106,007 and 106,008 on
`ebruary 17, 2015.
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`H0 Drug Information, International Nonproprietary Names for Pharmaceutical Substances (INN), Proposed INN: List
`15, "CASIMERSEN," vol. 30(2): 3 pages (2016)
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`H0 Drug Information, International Nonproprietary Names for Pharmaceutical Substances (INN), Proposed INN: List
`15, "Golodirsen," vol. 30(2): 3 pages (2016)
`
`IJNAENDTS, L.C.D. et al., "Prognostic importance of DNA flow cytometric variables in rhabdomyosarcomas," J. Clin.
`'athol., Vol. 46:948—952 (1993) (Exhibit Number 1041 filed in interferences 106008, 106007 on November 18, 2014)
`
`ilton et al. (2007) "Antisense Oligonucleotide—induced Exon Skipping Across the Human Dystrophin Gene
`ranscript," Molecular Therapy 15(7):1288—1296, 10 pages, (Exhibit Number 2121 filed in interferences 106,007 and
`06,008 on February 17, 2015
`
`ILTON, Stephen D. et al., "Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: where are
`e now?" Neuromuscular Disorders, Vol. 15:399-402 (2005)
`
`ILTON, Stephen D. et al., "Specific removal of the nonsense mutation from the mdx dystrophin mRNA using
`: ntisense oligonucleotides," Neuromuscular Disorders, Vol. 9:330—338 (1999)
`
`0 200224906 A1 of AZL, (University of Western Australia Exhibit 2134, filed April 3, 2015 in Interferences 106007,
`06008, and 106013, pages 1-43.)
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`0 2004/083432 (the published AZL PCT Application, "Van Ommen"), Pages 71, Exhibit Number 1003 filed in
`nterference 106,013 on February 17, 2015.
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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
`
`0 2013/112053 A1, (University of Western Australia Exhibit 2130,
`06008, and 106013, pages 1—177).
`
`filed April 3, 2015 in Interferences 106007,
`
`OLFF, Jon A. et al., "Direct Gene Transfer into Mouse Muscle in Vivo," Science, Vol. 247:1465-1468 (1990)
`
`ONG, Marisa L. et al., "Real-time PCR for mRNA quantitation," BioTechniques, Vol. 39:75—85 (2005) (Exhibit
`umber 1066 filed in interferences 106008, 106007 on November 18, 2014)
`
`ood, "Toward an Oligonucleotide Therapy for Duchenne Muscular Dystrophy: A Complex Development Challenge,"
`. cience Translational Medicine, Vol. 2, No. 25, pp. 1—6 (March, 2010), Exhibit Number 1116 filed in interferences
`06,007 and 106,008 on February 17, 2015,D0c 335.
`
`ritten Opinion for Application No. PCT/AU2010/001520, 6 pages, dated January 21, 2011
`
`U, B. et al., "Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by
`ystemically delivered morpholino," Gene Therapy, Vol. 17:132-140 (2010)
`
`U, Bo et al., "Effective rescue of dystrophin improves cardiac function in dystrophin—deficient mice by a modified
`orpholino oligomer," PNAS, Vol. 105(39):14814-14819 (2008)
`
`U, Bo et al., "Targeted Skipping of Human Dystrophin Exons in Transgenic Mouse Model Systemically for Antisense
`I rug Development," PLoS One, Vol. 6(5):e19906, 11 pages (2011)
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`4:-000000O(.003"-l
`4:-4:-4:-00N—‘
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`EFS Web 2.1.17
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`U, George Y. et al., "Receptor-mediated Gene Delivery and Expression in Vivo," The Journal of Biological
`hemistry, Vol. 263(29):14621-14624 (1988)
`
`U, George Y. et al., "Receptor-mediated in Vitro Gene Transformation by a Soluble DNA Carrier System," The
`ournal of Biological Chemistry, Vol. 262(10):4429—4432 (1987)
`
`yatt et al. "Site—specific cross—linking of mammalian U5 snRNP to the 5' splice site before the first step of pre-mRNA
`plicing," Genes & Development, Vol. 6, pp. 2542-2553 (1992), Exhibit Number 1198 filed in Interferences 106,007
`: nd 106,008 on February 17, 2015.
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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
`
`in et al., "A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in
`ystrophin—deficient mdx mice," Human Mol. Gen., Vol. 18, No. 22, pp. 4405-4414(2009), Exhibit Number 1200 filed in
`nterferences 106,007 and 106,008 on February 17, 2015.
`
`in et al., "Cell Penetrating peptide—conjugated antisense oligonucleotides restore systemic muscle and cardiac
`ystrophin expression and function," Human Mol. Gen., Vol. 17, No. 24, pp. 3909-3918 (2008), Exhibit Number 1199
`"ed in Interferences 106,007 and 106,008 on February 17, 2015.
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`I AVN-009DVCN7
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`in et al., "Functional Rescue of Dystrophin—deficient mdx Mice by a ChimericPeptide-PMO," Mol. Therapy, Vol. 18,
`o. 10, pp. 1822—1829 (October, 2010), Exhibit Number 1117 filed in interferences 106,007 and 106,008 on February
`7, 2015.
`
`okota et al., "Efficacy of Systematic Morpholino Exon—Skipping in Duchenne Dystrophy Dogs," American Neurological
`‘ ssoc., Vol. 65, No. 6, pp. 667—676 (June, 2009), Exhibit Number 1214 filed in Interferences 106,007 and 106,008 on
`ebruary 17, 2015.
`
`'oltek Corp. v. US, 95 Fed. CI. 681 (2011), 23 pages, (Academisch Ziekenhuis Leiden Exhibit 1236, filed May 5,
`'015 in Interference 106007 and 106008).
`
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`Application Number 15789862
`
`Filing Date
`2017-10-20
`First Named Inventor
`Peter SAZANI
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`Art U .
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`mt
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`Examiner Name
`Not Yet Assigned
`I AVN-009DVCN7
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`IN FORMATION DISCLOSU RE
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