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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AU2012345638A1 (en
AU2012345638B2 (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)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • 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-DMAC10.html)
EP (1) EP2785840B8 (cg-RX-API-DMAC10.html)
JP (2) JP6496549B2 (cg-RX-API-DMAC10.html)
AU (1) AU2012345638C1 (cg-RX-API-DMAC10.html)
CA (1) CA2857664A1 (cg-RX-API-DMAC10.html)
ES (1) ES2727481T3 (cg-RX-API-DMAC10.html)
WO (1) WO2013082551A1 (cg-RX-API-DMAC10.html)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
ES2727481T3 (es) 2011-11-30 2019-10-16 Sarepta Therapeutics Inc Inclusión inducida de exón en atrofia muscular espinal
WO2013082548A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
WO2014113540A1 (en) 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A 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
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
WO2017040271A1 (en) * 2015-08-28 2017-03-09 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
US10357543B2 (en) 2015-11-16 2019-07-23 Ohio State Innovation Foundation Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein
US11060089B2 (en) 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
US10947533B2 (en) 2016-05-24 2021-03-16 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
TWI737736B (zh) * 2016-05-24 2021-09-01 美商薩羅塔治療公司 製備磷醯二胺嗎啉代寡聚物之方法
MA45158A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Composition pharmaceutique comprenant de l'éteplirsen
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MX384727B (es) 2016-05-24 2025-03-14 Sarepta Therapeutics Inc Procesos para preparar oligómeros
MX390906B (es) * 2016-05-24 2025-03-21 Sarepta Therapeutics Inc Procesos para preparar oligómeros de morfolino fosforodiamidato.
US11198867B2 (en) 2016-06-16 2021-12-14 Ionis Pharmaceuticals, Inc. Combinations for the modulation of SMN expression
CN109152775B (zh) * 2016-06-30 2022-04-26 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
CA3024153A1 (en) 2016-06-30 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
AU2017330276B2 (en) * 2016-09-20 2022-01-20 The Regents Of The University Of Colorado, A Body Corporate Synthesis of backbone modified morpholino oligonucleotides and chimeras using phosphoramidite chemistry
PT4122497T (pt) 2016-12-19 2024-06-12 Sarepta Therapeutics Inc Conjugados oligoméricos de salto de exão para a distrofia muscular
ES3051834T3 (en) 2016-12-19 2025-12-30 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
NZ755416A (en) 2016-12-19 2023-05-26 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
TWI812647B (zh) 2017-09-25 2023-08-21 美商薩羅塔治療公司 經由速流合成以製備磷醯二胺嗎啉代寡聚物之製程
US11555189B2 (en) * 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
EP3765030A4 (en) * 2018-03-16 2022-01-26 Sarepta Therapeutics, Inc. CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION
WO2019183570A1 (en) * 2018-03-22 2019-09-26 Board Of Regents, The University Of Texas System Soluble interleukin-7 receptor (sil7r) molulating therapy to treat autoimmune diseases and cancer
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
BR112021011018A2 (pt) 2018-12-13 2021-08-31 Sarepta Therapeutics, Inc. Conjugados de oligômero para exon skipping para distrofia muscular
WO2021025899A1 (en) 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer pharmaceutical compositions
AU2021225957A1 (en) * 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
US12378267B2 (en) 2021-06-17 2025-08-05 Entrada Therapeutics, Inc. Synthesis of FMOC-protected morpholino monomers and oligomers
US20250289851A1 (en) 2022-04-22 2025-09-18 Entrada Therapeutics, Inc. Cyclic peptides for delivering therapeutics
US20260207778A1 (en) * 2022-12-23 2026-07-23 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Treatment of GEMIN5-Mediated Disorders

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008036127A2 (en) * 2006-05-10 2008-03-27 Avi Biopharma, Inc. Oligonucleotide analogs having cationic intersubunit linkages

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン，ジエ−ムス ポリヌクレオチド測定試薬と方法
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
AU659482B2 (en) 1991-06-28 1995-05-18 Massachusetts Institute Of Technology Localized oligonucleotide therapy
WO1997022371A1 (en) 1995-12-18 1997-06-26 Collagen Corporation Crosslinked polymer compositions and methods for their use
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
UA67760C2 (uk) 1997-12-11 2004-07-15 Міннесота Майнінг Енд Мануфакчурінг Компані Імідазонафтиридин та тетрагідроімідазонафтиридин, фармацевтична композиція, спосіб індукування біосинтезу цитокінів та спосіб лікування вірусної інфекції, проміжні сполуки
FR2802206B1 (fr) * 1999-12-14 2005-04-22 Sod Conseils Rech Applic Derives de 4-aminopiperidine et leur utilisation en tant que medicament
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
PT2548560E (pt) 2005-06-23 2015-09-22 Isis Pharmaceuticals Inc Composições e métodos de modulação da excisão-união de smn2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
JP6047270B2 (ja) 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー．フェー． Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
MX2010004955A (es) 2007-11-15 2010-06-30 Avi Biopharma Inc Metodo de sintesis de oligomeros de morfolina.
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
KR20220084437A (ko) 2009-06-17 2022-06-21 바이오젠 엠에이 인코포레이티드 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
JP5585822B2 (ja) 2010-05-11 2014-09-10 東レ・ファインケミカル株式会社 光学活性ニペコチン酸誘導体の製造方法
KR101981705B1 (ko) 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
AU2011367230B2 (en) 2011-05-05 2017-08-10 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2013082548A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
ES2727481T3 (es) 2011-11-30 2019-10-16 Sarepta Therapeutics Inc Inclusión inducida de exón en atrofia muscular espinal

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008036127A2 (en) * 2006-05-10 2008-03-27 Avi Biopharma, Inc. Oligonucleotide analogs having cationic intersubunit linkages

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
A. H. M. BURGHES ET AL, "Antisense oligonucleotides and spinal muscular atrophy: skipping along", GENES & DEVELOPMENT, (20100801), vol. 24, no. 15, doi:10.1101/gad.1961710, ISSN 0890-9369, pages 1574 - 1579 *

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Publication number Publication date
AU2012345638A1 (en) 2014-07-24
ES2727481T3 (es) 2019-10-16
JP6496549B2 (ja) 2019-04-03
JP2015504650A (ja) 2015-02-16
US10577605B2 (en) 2020-03-03
US20180273954A1 (en) 2018-09-27
WO2013082551A1 (en) 2013-06-06
JP2019059769A (ja) 2019-04-18
EP2785840B1 (en) 2019-03-20
HK1202891A1 (en) 2015-10-09
CA2857664A1 (en) 2013-06-06
US20140329772A1 (en) 2014-11-06
EP2785840A1 (en) 2014-10-08
EP2785840B8 (en) 2019-05-22
US9944926B2 (en) 2018-04-17
AU2012345638B2 (en) 2018-07-12

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