CA2776651C - Tricyclo-phosphorothioate dna - Google Patents

Tricyclo-phosphorothioate dna Download PDF

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Publication number
CA2776651C
CA2776651C CA2776651A CA2776651A CA2776651C CA 2776651 C CA2776651 C CA 2776651C CA 2776651 A CA2776651 A CA 2776651A CA 2776651 A CA2776651 A CA 2776651A CA 2776651 C CA2776651 C CA 2776651C
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CA
Canada
Prior art keywords
nucleic acid
acid molecule
dna
tricyclo
treatment
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
CA2776651A
Other languages
English (en)
French (fr)
Other versions
CA2776651A1 (en
Inventor
Christian Leumann
Luis Garcia
Thomas Voit
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Universitaet Bern
Centre National de la Recherche Scientifique CNRS
Universite Pierre et Marie Curie
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Original Assignee
Universitaet Bern
Centre National de la Recherche Scientifique CNRS
Universite Pierre et Marie Curie
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Universitaet Bern, Centre National de la Recherche Scientifique CNRS, Universite Pierre et Marie Curie, Institut National de la Sante et de la Recherche Medicale INSERM, Association Institut de Myologie filed Critical Universitaet Bern
Priority to PCT/EP2012/070349 priority Critical patent/WO2013053928A1/en
Priority to BR112014009066A priority patent/BR112014009066B8/pt
Priority to AU2012322903A priority patent/AU2012322903B2/en
Priority to US14/351,733 priority patent/US9738891B2/en
Priority to JP2014535113A priority patent/JP6181653B2/ja
Priority to CN201280057688.6A priority patent/CN104245935B/zh
Priority to CA2851970A priority patent/CA2851970A1/en
Priority to ES12770503.6T priority patent/ES2651216T3/es
Priority to EP12770503.6A priority patent/EP2766479B1/en
Publication of CA2776651A1 publication Critical patent/CA2776651A1/en
Priority to IL231983A priority patent/IL231983B/en
Priority to IN3463DEN2014 priority patent/IN2014DN03463A/en
Application granted granted Critical
Publication of CA2776651C publication Critical patent/CA2776651C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Pain & Pain Management (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
CA2776651A 2011-10-13 2012-04-27 Tricyclo-phosphorothioate dna Active CA2776651C (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
CA2851970A CA2851970A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna
AU2012322903A AU2012322903B2 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna
US14/351,733 US9738891B2 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate DNA
JP2014535113A JP6181653B2 (ja) 2011-10-13 2012-10-12 トリシクロ−ホスホロチオエートｄｎａ
CN201280057688.6A CN104245935B (zh) 2011-10-13 2012-10-12 三环硫代磷酸酯dna
ES12770503.6T ES2651216T3 (es) 2011-10-13 2012-10-12 Triciclo-ADN fosforotioato
PCT/EP2012/070349 WO2013053928A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna
EP12770503.6A EP2766479B1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna
BR112014009066A BR112014009066B8 (pt) 2011-10-13 2012-10-12 molécula de ácido nucleico, método para a sintetização de uma molécula de dna de triciclo-fosforotioato e composição farmacêutica
IL231983A IL231983B (en) 2011-10-13 2014-04-07 Dna containing tricyclo-phosphorothioate
IN3463DEN2014 IN2014DN03463A (enExample) 2011-10-13 2014-04-30

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201161546942P 2011-10-13 2011-10-13
US61/546.942 2011-10-13
EP11185129.1 2011-10-13
EP11185129.1A EP2581448B1 (en) 2011-10-13 2011-10-13 Tricyclo-phosphorothioate DNA

Publications (2)

Publication Number Publication Date
CA2776651A1 CA2776651A1 (en) 2013-04-13
CA2776651C true CA2776651C (en) 2021-06-08

Family

ID=44799824

Family Applications (2)

Application Number Title Priority Date Filing Date
CA2776651A Active CA2776651C (en) 2011-10-13 2012-04-27 Tricyclo-phosphorothioate dna
CA2851970A Abandoned CA2851970A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2851970A Abandoned CA2851970A1 (en) 2011-10-13 2012-10-12 Tricyclo-phosphorothioate dna

Country Status (14)

Country Link
US (1) US9738891B2 (enExample)
EP (2) EP2581448B1 (enExample)
JP (1) JP6181653B2 (enExample)
CN (1) CN104245935B (enExample)
AU (1) AU2012322903B2 (enExample)
BR (1) BR112014009066B8 (enExample)
CA (2) CA2776651C (enExample)
DK (1) DK2581448T3 (enExample)
ES (2) ES2535654T3 (enExample)
IL (1) IL231983B (enExample)
IN (1) IN2014DN03463A (enExample)
PL (1) PL2581448T3 (enExample)
PT (1) PT2581448E (enExample)
WO (1) WO2013053928A1 (enExample)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20110352T1 (hr) 2004-06-28 2011-06-30 The University Of Western Australia Antisens olinukleotidi za induciranje zaobilaženja egzona i postupci njihove upotrebe
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
WO2010048586A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
KR102113306B1 (ko) 2009-11-12 2020-05-21 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
NZ731587A (en) 2013-03-14 2021-07-30 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
CN113633787A (zh) 2013-03-15 2021-11-12 萨勒普塔医疗公司 改进的用于治疗肌营养不良的组合物
CN111235149B (zh) 2013-09-05 2024-04-16 萨罗塔治疗公司(美国) 酸性α-葡糖苷酶中反义诱导的外显子2纳入
ES2739850T3 (es) 2013-09-11 2020-02-04 Synthena Ag Acidos nucleicos y procedimientos para el tratamiento de la enfermedad de Pompe
BR112017001725A2 (pt) 2014-07-31 2018-02-14 Association Inst De Myologie tratamento de esclerose lateral amiotrófica
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
HK1253335A1 (zh) 2015-06-01 2019-06-14 Sarepta Therapeutics, Inc. 在vii型胶原蛋白中反义诱导外显子的排除
EP3353301A1 (en) 2015-09-21 2018-08-01 Association Institut de Myologie Antisense oligonucleotides and uses thereof
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
SG11201809468XA (en) 2016-04-29 2018-11-29 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
SG10202101836TA (en) 2016-05-24 2021-03-30 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
LT3464306T (lt) 2016-05-24 2024-05-10 Sarepta Therapeutics, Inc. Fosforodiamidato morfolino oligomerų gamybos būdas
WO2017205496A1 (en) 2016-05-24 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing oligomers
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
TW201811807A (zh) 2016-06-30 2018-04-01 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
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
EP3516061A1 (en) 2016-09-23 2019-07-31 Synthena AG Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
HRP20221316T1 (hr) 2016-12-19 2022-12-23 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
ES2928486T3 (es) 2016-12-19 2022-11-18 Sarepta Therapeutics Inc Conjugados de oligómeros con omisión de exón para la distrofia muscular
PL3554553T3 (pl) 2016-12-19 2022-11-07 Sarepta Therapeutics, Inc. Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej
CN110536694A (zh) 2017-04-20 2019-12-03 Atyr 医药公司 用于治疗肺部炎症的组合物和方法
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
EP4036101A1 (en) 2017-04-20 2022-08-03 Synthena AG Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
JP2020536058A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
EP3687547A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
JP2020536057A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
EP3697910A4 (en) 2017-10-18 2021-07-14 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMER COMPOUNDS
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3806868A4 (en) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
TW202020153A (zh) 2018-07-27 2020-06-01 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
AU2019397461A1 (en) 2018-12-13 2021-07-29 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2020214763A1 (en) 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
CA3155381A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
KR20220148230A (ko) 2020-02-28 2022-11-04 아이오니스 파마수티컬즈, 인코포레이티드 Smn2를 조절하기 위한 화합물 및 방법
IL296702A (en) 2020-04-09 2022-11-01 Association Inst De Myologie Antisense sequences for the treatment of amyotrophic lateral sclerosis
EP3978608A1 (en) 2020-10-05 2022-04-06 SQY Therapeutics Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
EP4392558A1 (en) 2021-09-30 2024-07-03 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
US20240318179A1 (en) 2021-10-22 2024-09-26 Murdoch University Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
AR132529A1 (es) 2023-04-27 2025-07-16 Sarepta Therapeutics Inc Oligómeros antisentido para el tratamiento de la enfermedad renal crónica
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
JP2003528024A (ja) 1997-12-16 2003-09-24 バレンティス・インコーポレーテッド 製剤化された核酸分子の無針注入
DE60040274D1 (de) 1999-03-10 2008-10-30 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
FI20051017A7 (fi) 2003-04-13 2005-11-28 Enzon Pharmaceutivals Inc Polymeeriset oligonukleotidiaihiolääkkeet
WO2007073149A1 (en) * 2005-12-22 2007-06-28 Keygene N.V. Alternative nucleotides for improved targeted nucleotide exchange
MX2009002859A (es) * 2006-09-15 2009-03-30 Enzon Pharmaceuticals Inc Enlazadores biodegradables a base de ester impedido para suministro de oligonucleotidos.
US20100016215A1 (en) * 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
EP3443976A1 (en) * 2007-06-29 2019-02-20 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
US20110305770A1 (en) * 2008-11-17 2011-12-15 Enzon Pharmaceuticals, Inc. Releasable polymeric lipids for nucleic acids delivery system
DK2417257T3 (da) * 2009-04-10 2016-06-06 Ass Inst De Myologie Tricyclo-dna-antisense-oligonukleotider, sammensætninger, og fremgangsmåder til behandlingen af sygdom
US20140022812A1 (en) 2011-02-25 2014-01-23 Lumitex, Inc. Display front lighting device

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Publication number Publication date
AU2012322903B2 (en) 2017-09-14
EP2766479B1 (en) 2017-09-13
CA2851970A1 (en) 2013-04-18
US9738891B2 (en) 2017-08-22
CN104245935B (zh) 2018-05-08
EP2581448B1 (en) 2015-01-28
AU2012322903A1 (en) 2014-05-29
PL2581448T3 (pl) 2015-08-31
DK2581448T3 (en) 2015-04-27
WO2013053928A1 (en) 2013-04-18
ES2651216T3 (es) 2018-01-25
PT2581448E (pt) 2015-05-21
CN104245935A (zh) 2014-12-24
EP2766479A1 (en) 2014-08-20
BR112014009066A2 (pt) 2017-04-18
BR112014009066A8 (pt) 2018-01-09
BR112014009066B1 (pt) 2020-08-25
IL231983B (en) 2018-04-30
HK1186205A1 (en) 2014-03-07
US20140296323A1 (en) 2014-10-02
EP2581448A1 (en) 2013-04-17
IL231983A0 (en) 2014-05-28
ES2535654T3 (es) 2015-05-13
IN2014DN03463A (enExample) 2015-06-05
JP6181653B2 (ja) 2017-08-16
JP2015501144A (ja) 2015-01-15
CA2776651A1 (en) 2013-04-13
BR112014009066B8 (pt) 2021-02-23

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