AU2012345638C1 - Induced exon inclusion in spinal muscle atrophy - Google Patents

Induced exon inclusion in spinal muscle atrophy Download PDF

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Publication number
AU2012345638C1
AU2012345638C1 AU2012345638A AU2012345638A AU2012345638C1 AU 2012345638 C1 AU2012345638 C1 AU 2012345638C1 AU 2012345638 A AU2012345638 A AU 2012345638A AU 2012345638 A AU2012345638 A AU 2012345638A AU 2012345638 C1 AU2012345638 C1 AU 2012345638C1
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AU2012345638B2 (en
AU2012345638A1 (en
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Brian James Leppert
Peter Linsley
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Sarepta Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2012345638A 2011-11-30 2012-11-30 Induced exon inclusion in spinal muscle atrophy Ceased AU2012345638C1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161565499P 2011-11-30 2011-11-30
US61/565,499 2011-11-30
PCT/US2012/067475 WO2013082551A1 (en) 2011-11-30 2012-11-30 Induced exon inclusion in spinal muscle atrophy

Publications (3)

Publication Number Publication Date
AU2012345638A1 AU2012345638A1 (en) 2014-07-24
AU2012345638B2 AU2012345638B2 (en) 2018-07-12
AU2012345638C1 true AU2012345638C1 (en) 2018-10-18

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AU2012345638A Ceased AU2012345638C1 (en) 2011-11-30 2012-11-30 Induced exon inclusion in spinal muscle atrophy

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US (2) US9944926B2 (cg-RX-API-DMAC7.html)
EP (1) EP2785840B8 (cg-RX-API-DMAC7.html)
JP (2) JP6496549B2 (cg-RX-API-DMAC7.html)
AU (1) AU2012345638C1 (cg-RX-API-DMAC7.html)
CA (1) CA2857664A1 (cg-RX-API-DMAC7.html)
ES (1) ES2727481T3 (cg-RX-API-DMAC7.html)
WO (1) WO2013082551A1 (cg-RX-API-DMAC7.html)

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US9944926B2 (en) 2011-11-30 2018-04-17 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
US9856474B2 (en) 2013-01-16 2018-01-02 Iowa State University Research Foundation, Inc. Deep intronic target for splicing correction on spinal muscular atrophy gene
AU2014317961B2 (en) * 2013-09-05 2020-07-30 Murdoch University Antisense-induced exon2 inclusion in acid alpha-glucosidase
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
JP6987041B2 (ja) * 2015-08-28 2021-12-22 サレプタ セラピューティクス，インコーポレイテッド 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー
EP3377073A4 (en) * 2015-11-16 2019-06-26 Ohio State Innovation Foundation METHOD AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES WITH THE SURVIVAL MOTOR NEURON (SMN) PROTEIN
JP7033547B2 (ja) 2016-04-18 2022-03-10 サレプタ セラピューティクス， インコーポレイテッド 酸性アルファ－グルコシダーゼ遺伝子に関連する疾患を処置するためのアンチセンスオリゴマーおよびそれを用いる方法
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HRP20241428T1 (hr) * 2016-05-24 2025-01-03 Sarepta Therapeutics, Inc. Postupci za pripremu oligomera
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
SG10202101836TA (en) * 2016-05-24 2021-03-30 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
AU2017270598B2 (en) * 2016-05-24 2022-12-01 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
BR112018074270B1 (pt) 2016-05-24 2021-02-02 Sarepta Therapeutics, Inc processo para preparar compostos oligoméricos bem como os ditos compostos
EP3471779A4 (en) 2016-06-16 2020-07-08 Ionis Pharmaceuticals, Inc. COMBINATIONS FOR MODULATING SMN EXPRESSION
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CN116804031A (zh) * 2016-09-20 2023-09-26 科罗拉多州立大学董事会法人团体 使用亚磷酰胺化学法合成主链修饰的吗啉代寡核苷酸和嵌合体
JP7160807B2 (ja) 2016-12-19 2022-10-25 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート
FI4122497T3 (fi) 2016-12-19 2024-06-04 Sarepta Therapeutics Inc Eksonin ohittavia oligomeerikonjugaatteja lihasdystrofiaan
LT3554553T (lt) 2016-12-19 2022-08-25 Sarepta Therapeutics, Inc. Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos
US12370261B2 (en) 2018-03-16 2025-07-29 Sarepta Therapeutics, Inc. Chimeric peptides for antisense delivery
JP7576835B2 (ja) * 2018-03-22 2024-11-01 ボード・オブ・リージェンツ・オブ・ザ・ユニバーシティ・オブ・テキサス・システム 自己免疫疾患および癌を治療するための可溶性インターロイキン７受容体（ｓＩＬ７Ｒ）調節療法
EP4219717A3 (en) 2018-06-13 2023-12-20 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
TW202449155A (zh) 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
WO2020123574A1 (en) 2018-12-13 2020-06-18 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2021025899A1 (en) 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer pharmaceutical compositions
BR112022016238A2 (pt) * 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular smn2
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Publication number Publication date
JP2015504650A (ja) 2015-02-16
US10577605B2 (en) 2020-03-03
US20140329772A1 (en) 2014-11-06
AU2012345638B2 (en) 2018-07-12
JP2019059769A (ja) 2019-04-18
US20180273954A1 (en) 2018-09-27
JP6496549B2 (ja) 2019-04-03
US9944926B2 (en) 2018-04-17
EP2785840A1 (en) 2014-10-08
HK1202891A1 (en) 2015-10-09
EP2785840B8 (en) 2019-05-22
CA2857664A1 (en) 2013-06-06
AU2012345638A1 (en) 2014-07-24
EP2785840B1 (en) 2019-03-20
ES2727481T3 (es) 2019-10-16
WO2013082551A1 (en) 2013-06-06

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