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`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`.
`.
`( Not for submrssron under 37 CFR 1.99)
`
`Application Number 15349778
`
`Filing Date
`2016-11-11
`First Named Inventor
`Peter SAZANI
`
`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-009DVCN6
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`uropean Search Report, EP15168694.6, dated July 23, 2015, pages 1-8.
`
`xcerpts from Prosecution History of Application No. 13l741,150: Notice of Allowance dated March 16, 2015; List of
`eferences cited by Applicant and Considered by Examiner; Notice of Allowance and Fees due dated September 18,
`'014; Amendment in Response to Non—Final Office Action dated July 11, 2014, (Academisch Ziekenhuis Leiden
`xhibit 1229, filed April 3, 2015 in Interference 106007 and 106008, pages 1—133).
`
`xcerpts from Prosecution History of Application No. 13/826,880: Notice of Allowance dated January 26, 2015 and
`‘ mendment in Response to Non-Final Office Action dates October 15, 2014, (Academisch Ziekenhuis Leiden Exhibit
`228, filed April 3, 2015 in Interference 106007 and 106008, pages 1—16).
`
`xcerpts from Yeo (Ed.), "Systems Biology of RNA Binding Proteins," Adv. Exp. Med. Biol., Chapter 9, 56 pages
`2014), (Academisch Ziekenhuis Leiden Exhibit 1232, filed April 3, 2015 in Interference 106007 and 106008, pages
`—56).
`
`xcerpts of SEC Form 8-K, dated November 23 2014, for BioMarin Pharmaceutical Inc., (University of Western
`‘ ustralia Exhibit 2129,
`filed April 3, 2015 in Interferences 106007, 106008, and 106013, pages 1—9).
`
`xon 51 Internal Sequence Schematic, Pages 1, Exhibit Number 1224 filed in Interferences 106,007 and 106,008 on
`ebruary 17, 2015.
`
`xon 53 Internal Sequence Schematic, Pages 1, Exhibit Number 1225 filed in Interferences 106,007 and 106,008 on
`ebruary 17, 2015.
`
`xtended European Search Report, EP 151903416, dated April 28, 2016, 9 pages.
`
`airclough et al., "Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches," Nature
`eviews, Vol. 14, pp. 373-378 (June, 2013), Exhibit Number 1112 filed in interferences 106,007 and 106,008 on
`ebruary 17, 2015.
`
`ALL, Abbie M. et al., "Induction of revertant fibres in the mdx mouse using antisense oligonucleotides," Genetic
`accines and Therapy, Vol. 4:3, doi:10.1186/1479—0556-4—3, 12 pages (2006)
`
`DA Briefing Document, "Peripheral and Central Nervous System," Drugs Advisory Committee Meeting, NDA 206488
`teplirsen, Food and Drug Administration, pages 1—73, January 22, 2016.
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`EFS Web 2.1.17
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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
`
`ederal Register, Vol. 58, No. 183, pp. 49432-49434, September 23, 1993 (6 pages); [Cited as: 58 FR 49432-01, 1993
`L 371451 (F_R_)], Exhibit Number 1221 filed in Interferences 106,007 and 106,008 on February 17, 2015.
`
`ederal Register, Vol. 69, No. 155, pp. 49960-50020 dated August 12, 2004 (62 pages), Exhibit Number 1220 filed in
`nterferences 106,007 and 106,008 on February 17, 2015.
`
`ile Excerpt from AZL US. Patent Application 11/233,495: Amendment Afler Non—Final Office Action, as—filed
`ovember 1, 2010 (Exhibit Number 1085 filed in interferences 106008, 106007 on December 23, 2014)
`
`ile Excerpt from AZL US. Patent Application 11/233,495: Claims examined in Non-Final Office Action, dated
`December 1, 2008 (Exhibit Number 1079 filed in interferences 106008, 106007 on December 23, 2014)
`
`ile Excerpt from AZL US. Patent Application 11/233,495: Final Office Action dated August 31, 2010 (Exhibit Number
`086 filed in interferences 106008, 106007 on December 23, 2014)
`
`ile Excerpt from U.S. Patent Application 11/233,495: Non-Final Office Action dated December 1, 2008 and Final
`office Action dated June 25, 2009 (Exhibit Number 1078 filed in interferences 106008, 106007 on December 23, 2014
`
`ile Excerpt from U.S. Patent Application No. 12/198,007: AZL's Preliminary Amendment and Response, as—filed
`ovember 7, 2008 (Exhibit Number 1075 filed in interferences 106008, 106007 on December 23, 2014)
`
`ile Excerpt from U.S. Patent Application No. 12/976,381: AZL's First Preliminary Amendment, as—filed December 22,
`'010 (Exhibit Number 1076 filed in interferences 106008, 106007 on December 23,2014)
`
`ile Excerpts from Prosecution History of US. Patent Application No. 13/270,992 ("UWA's US. Patent 8,486,907),
`'ages 122, Exhibit Number 1006 filed in Interference 106,013 on February 17, 2015.
`
`ile Excerpts from US. Patent Application No. 11/233,495: Response to Non- Final Office Action, as filed July 26,
`0'11 (14 pages), Exhibit Number 1222 filed in Interferences 106,007 and 106,008 on February 17,2015.
`
`ile Excerpts from US. Patent Application No. 13/270,992 ("UWA's US. Patent 8,486,907): NFOA, dated 7/30/2012;
`‘ pplicant—Initiated Interview Summary, dated 11/8/2012; Amendment, as filed January 30, 2013; NOA, dated 4/4/2013,
`xhibit Number 1118 (122 pages) filed in interferences 106,007 and 106,008 on February 17, 2015.
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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
`
`Ar, Unit
`|1574
`
` Application Number
`
`Examiner Name
`Not Yet Assigned
`Attorney Docket Number
`
`lanagan, W. Michael, et al., "A cytosine analog that confers enhanced potency to antisense oligonucleotides," Proc.
`at'l Acad. Sci. USA, Vol. 96, pp. 3513-3518 (March, 1999), Exhibit Number 1211 filed in lnterferences 106,007 and
`06,008 on February 17, 2015.
`
`LANIGAN, Kevin M. et al., "Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects
`ith Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial," Neuromuscular Disorders, Vol.
`4:16—24 (2014) (Exhibit Number 2038 filed in interferences 106008, 106013, 106007 on November 18, 2014)
`
`Ianigan, Kevin M., et al. (2003) "Rapid Direct Sequence Analysis of the Dystrophin Gene," Am. J. Hum. Genet.
`2:931—939, dated February 17, 2015 (Exhibit Number 2120 filed in interferences 106,007 and 106,008 on February
`7, 2015.
`
`letcher S., et al, "Morpholino oligomer—mediated exon skipping averts the onset of dystrophic pathology in the mdx
`ouse. Mol Ther 2007;15:1587—1592.
`
`LETCH ER, Sue et al., "Dystrophin lsoform Induction In Vivo by Antisense—mediated Alternative Splicing," Molecular
`herapy, Vol. 18(6):1218—1223 (2010)
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`EFS Web 2.1.17
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`LETCHER, Sue et al., "Targeted Exon Skipping to Address 'Leaky' Mutations in the Dystrophin Gene," Molecular
`herapy—Nucleic Acids, Vol. 1, e48, doi:10.1038lmtna.2012.40, 11 pages (2012)
`
`LETCH ER, Susan et al., "Dystrophin expression in the mdx mouse after localised and systemic administration of a
`orpholino antisense oligonucleotide," J. Gene Med, Vol. 8:207—216 (2006)
`
`LETCHER, Susan et al., "Gene therapy and molecular approaches to the treatment of hereditary muscular
`isorders," Curr. Opin. Neurol., Vol. 13:553—560 (2000)
`
`OSTER, Helen et al., "Genetic Therapeutic Approaches for Duchenne Muscular Dystrophy," Human Gene Therapy,
`ol. 23:676—687 (2012)
`
`ourth Declaration of Erik Sontheimer, Ph.D. (Pursuant to Bd.R. 41 .155(b)(2) and SO 155.1.3 and 155.1.4), dated
`arch 9, 2015, (University of Western Australia Exhibit 2138, filed April 3, 2015 in lnterferences 106007, 106008, and
`06013, pages 1-4).
`
`RAGALL, Clayton T. et al., "Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin
`plice switching," BMC Medical Genetics, Vol. 12:141, 8 pages (2011) (Exhibit Number 2019 filed in interferences
`06008, 106013, 106007 on November 18, 2014)
`
`

`

`
`
`
`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
`
`RALEY, Robert et al., "New generation liposomes: the engineering of an efficient vehicle for intracellular delivery of
`ucleic acids," Trends Biochem, Vol. 6:77—80 (1981)
`
`RAZIER, Kendall S. et aI., "Species-specific Inflammatory Responses as a Primary Component for the Development
`f Glomerular Lesions in Mice and Monkeys Following Chronic Administration of a Second—generation Antisense
`oligonucleotide," Toxicologica Pathology, 13 pages (2013)
`
`RIEDMAN N, Theodore, "Progress Toward Human Gene Therapy," Science, Vol. 244(4910):1275—1281 (1989)
`
`EBSKI, Bianca L. et al., "Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse
`uscle,“ Human Molecular Genetics, Vol. 12(15):1801-1811 (2003)
`
`eneric Method for Average Mass Determination Using LC-UV—MS in the Negative Mode, Pages 15, Exhibit Number
`145 filed in Interferences 106,007 and 106,008 on February 16, 2015.
`
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`4:-4:-4:-00N—‘
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`EFS Web 2.1.17
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`eneric UPLC Purity Method for Oligonucleotides (19— to 25—mers), Pages 18, Exhibit Number 1156 filed in
`nterferences 106,007 and 106,008 on February 16, 2015.
`
`ENNARO, Alfonso R., (ed.), Remington's Pharmaceutical Sciences, 18th Edition, Mack Publishing, 00., Easton PA,
`'020 pages (1990)
`
`ILES, Richard V. et aI., "Antisense Morpholino Oligonucleotide Analog Induces Missplicing of C—myc mRNA,"
`‘ ntisense & Nucleic Acid Drug Development, Vol. 9:213-220 (1999)
`
`IaxoSmithKline Press Release, Issued in London, UK, dated June 27,2013 (5 pages), Exhibit Number 1202 filed in
`nterferences 106,007 and 106,008 on February 17, 2015.
`
`IaxoSmithKline, "GSK and Prosensa announce start of Phase III study of investigational Duchenne Muscular
`Iystrophy medication," press release, 6 pages, dated January 19, 2011 (Exhibit Number 2060 filed in interferences
`06008, 106013, 106007 on November 18, 2014)
`
`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).
`
`

`

`
`
`
`
`Application Number 15349778
`
`IN FORMATION DISCLOSU RE
`STATEMENT BY APPLICANT
`.
`.
`( Not for submlssmn under 37 CFR 1.99)
`
`
`Filing Date
`2016-11-11
`First Named Inventor
`Peter SAZANI
`
`Art U .
`M
`I 1674
`
`Examiner Name
`Not Yet Assigned
`
`OEMANS, Nathalie M. et al., "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 al., "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)
`
` Attorney Docket Number
`ice by Morpholino-oligomer—mediated Exon—skipping," Molecular Therapy, Vol. 18(1):198—205 (2010)
`
`
`
`I AVN-009DVCN6
`
`ordon, Peter M. et al. ,"Kinetic Characterization of the Second Step of Group II Intron Splicing: Role of Metal Ions
`=nd the Cleavage Site 2'O—H 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
`
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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
`STATEMENT BY APPLICANT
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