ES2535654T3 - ADN triciclo-fosforotioato - Google Patents
ADN triciclo-fosforotioato Download PDFInfo
- Publication number
- ES2535654T3 ES2535654T3 ES11185129.1T ES11185129T ES2535654T3 ES 2535654 T3 ES2535654 T3 ES 2535654T3 ES 11185129 T ES11185129 T ES 11185129T ES 2535654 T3 ES2535654 T3 ES 2535654T3
- Authority
- ES
- Spain
- Prior art keywords
- dna
- nucleic acid
- acid molecule
- mrna
- exon
- 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
Links
Classifications
-
- 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
Landscapes
- 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)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Epidemiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Pain & Pain Management (AREA)
- Cardiology (AREA)
- Neurosurgery (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)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP11185129.1A EP2581448B1 (en) | 2011-10-13 | 2011-10-13 | Tricyclo-phosphorothioate DNA |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2535654T3 true ES2535654T3 (es) | 2015-05-13 |
Family
ID=44799824
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES11185129.1T Active ES2535654T3 (es) | 2011-10-13 | 2011-10-13 | ADN triciclo-fosforotioato |
| ES12770503.6T Active ES2651216T3 (es) | 2011-10-13 | 2012-10-12 | Triciclo-ADN fosforotioato |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES12770503.6T Active ES2651216T3 (es) | 2011-10-13 | 2012-10-12 | Triciclo-ADN fosforotioato |
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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| CN110218727A (zh) | 2013-03-14 | 2019-09-10 | 萨勒普塔医疗公司 | 用于治疗肌营养不良的外显子跳跃组合物 |
| US20140329762A1 (en) | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| US11236338B2 (en) | 2013-09-05 | 2022-02-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| EP3044315B1 (en) | 2013-09-11 | 2019-02-20 | Synthena AG | Nucleic acids and methods for the treatment of pompe disease |
| EP3174981B1 (en) | 2014-07-31 | 2020-03-25 | Association Institut de Myologie | Treatment of amyotrophic lateral sclerosis |
| 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 |
| MA50829A (fr) | 2015-06-01 | 2018-04-11 | Sarepta Therapeutics Inc | Exclusion d'exon induite pat technologie antisens dans le collagène de type vii |
| US20180237775A1 (en) | 2015-09-21 | 2018-08-23 | Association Institut De Myologie | Antisense oligonucleotides and uses thereof |
| EP3858993A1 (en) | 2015-10-09 | 2021-08-04 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| 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 |
| MX2018013157A (es) | 2016-04-29 | 2019-05-23 | Sarepta Therapeutics Inc | Analogos de oligonucleotidos dirigidos a lmna humana. |
| CA3024456A1 (en) | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| BR112018074346B1 (pt) | 2016-05-24 | 2023-01-03 | Sarepta Therapeutics, Inc. | Composto oligomérico e processo para preparar um composto oligomérico |
| MD3464306T2 (ro) | 2016-05-24 | 2024-08-31 | Sarepta Therapeutics Inc | Procedee de preparare a oligomerilor morfolino fosforodiamidați |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| US10947533B2 (en) | 2016-05-24 | 2021-03-16 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| JP2019525742A (ja) | 2016-06-30 | 2019-09-12 | 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 |
| EP3516061A1 (en) | 2016-09-23 | 2019-07-31 | Synthena AG | Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof |
| WO2018055577A1 (en) * | 2016-09-23 | 2018-03-29 | Synthena Ag | Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof |
| KR102810425B1 (ko) | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR20240006023A (ko) | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| PT3554552T (pt) | 2016-12-19 | 2022-10-03 | Sarepta Therapeutics Inc | Conjugados de oligómero de skipping de exão para distrofia muscular |
| EP4036101A1 (en) | 2017-04-20 | 2022-08-03 | Synthena AG | Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof |
| CA3060514A1 (en) | 2017-04-20 | 2018-10-25 | Atyr Pharma, Inc. | Compositions and methods for treating lung inflammation |
| WO2018193428A1 (en) | 2017-04-20 | 2018-10-25 | 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 |
| EP3687547A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| WO2019067981A1 (en) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| WO2019067979A1 (en) | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| US11555189B2 (en) | 2017-10-18 | 2023-01-17 | 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 |
| 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 |
| IL318474A (en) | 2018-12-13 | 2025-03-01 | Sarepta Therapeutics Inc | Axon-skipping oligomer complexes for muscular dystrophy |
| WO2020214763A1 (en) | 2019-04-18 | 2020-10-22 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| WO2021089736A1 (en) | 2019-11-06 | 2021-05-14 | Association Institut De Myologie | Combined therapy for muscular diseases |
| CN120505310A (zh) | 2020-02-28 | 2025-08-19 | Ionis制药公司 | 用于调节smn2的化合物和方法 |
| BR112022020412A2 (pt) | 2020-04-09 | 2023-05-02 | Association Inst De Myologie | Sequências antissenso para tratar esclerose lateral amiotrófica |
| EP3978608A1 (en) | 2020-10-05 | 2022-04-06 | SQY Therapeutics | Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51 |
| WO2023055774A1 (en) | 2021-09-30 | 2023-04-06 | Sarepta Therapeutics, Inc. | Antisense oligonucleotides having one or more abasic units |
| EP4419680A1 (en) | 2021-10-22 | 2024-08-28 | Sarepta Therapeutics, Inc. | Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases |
| JP2025533454A (ja) | 2022-09-21 | 2025-10-07 | サレプタ セラピューティクス， インコーポレイテッド | Ｄｍｄアンチセンスオリゴヌクレオチド介在性エクソンスキッピング効率 |
| TW202442246A (zh) | 2023-04-27 | 2024-11-01 | 美商薩羅塔治療公司 | 用於治療慢性腎病之反義寡聚物 |
| WO2025085810A2 (en) | 2023-10-18 | 2025-04-24 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of centronuclear myopathies |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| 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 |
| CA2315256A1 (en) | 1997-12-16 | 1999-06-24 | Valentis, Inc. | Needle-free injection of formulated nucleic acid molecules |
| DE60040274D1 (de) | 1999-03-10 | 2008-10-30 | Phogen Ltd | Verabreichung von nukleinsäuren und proteinen an zellen |
| NZ542687A (en) * | 2003-04-13 | 2010-02-26 | Enzon Pharmaceuticals Inc | Polymeric oligonucleotide prodrugs |
| 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. |
| WO2009005793A2 (en) * | 2007-06-29 | 2009-01-08 | Avi Biopharma, Inc. | Tissue specific peptide conjugates and methods |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| CA2742838A1 (en) * | 2008-11-17 | 2010-05-20 | Enzon Pharmaceuticals, Inc. | Releasable polymeric lipids for nucleic acids delivery systems |
| CA2758189C (en) * | 2009-04-10 | 2020-12-29 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
| JP2014511547A (ja) | 2011-02-25 | 2014-05-15 | ルミテックス， インコーポレイテッド | ディスプレイ前方照明装置 |
-
2011
- 2011-10-13 PL PL11185129T patent/PL2581448T3/pl unknown
- 2011-10-13 PT PT111851291T patent/PT2581448E/pt unknown
- 2011-10-13 DK DK11185129T patent/DK2581448T3/en active
- 2011-10-13 ES ES11185129.1T patent/ES2535654T3/es active Active
- 2011-10-13 EP EP11185129.1A patent/EP2581448B1/en active Active
-
2012
- 2012-04-27 CA CA2776651A patent/CA2776651C/en active Active
- 2012-10-12 BR BR112014009066A patent/BR112014009066B8/pt active IP Right Grant
- 2012-10-12 CN CN201280057688.6A patent/CN104245935B/zh active Active
- 2012-10-12 CA CA2851970A patent/CA2851970A1/en not_active Abandoned
- 2012-10-12 US US14/351,733 patent/US9738891B2/en active Active
- 2012-10-12 EP EP12770503.6A patent/EP2766479B1/en active Active
- 2012-10-12 AU AU2012322903A patent/AU2012322903B2/en active Active
- 2012-10-12 WO PCT/EP2012/070349 patent/WO2013053928A1/en not_active Ceased
- 2012-10-12 ES ES12770503.6T patent/ES2651216T3/es active Active
- 2012-10-12 JP JP2014535113A patent/JP6181653B2/ja active Active
-
2014
- 2014-04-07 IL IL231983A patent/IL231983B/en active IP Right Grant
- 2014-04-30 IN IN3463DEN2014 patent/IN2014DN03463A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA2776651C (en) | 2021-06-08 |
| BR112014009066B8 (pt) | 2021-02-23 |
| CN104245935A (zh) | 2014-12-24 |
| PT2581448E (pt) | 2015-05-21 |
| ES2651216T3 (es) | 2018-01-25 |
| EP2581448B1 (en) | 2015-01-28 |
| EP2581448A1 (en) | 2013-04-17 |
| BR112014009066B1 (pt) | 2020-08-25 |
| BR112014009066A2 (pt) | 2017-04-18 |
| CA2851970A1 (en) | 2013-04-18 |
| JP6181653B2 (ja) | 2017-08-16 |
| IN2014DN03463A (enExample) | 2015-06-05 |
| WO2013053928A1 (en) | 2013-04-18 |
| JP2015501144A (ja) | 2015-01-15 |
| PL2581448T3 (pl) | 2015-08-31 |
| DK2581448T3 (en) | 2015-04-27 |
| CA2776651A1 (en) | 2013-04-13 |
| IL231983A0 (en) | 2014-05-28 |
| AU2012322903A1 (en) | 2014-05-29 |
| IL231983B (en) | 2018-04-30 |
| HK1186205A1 (en) | 2014-03-07 |
| CN104245935B (zh) | 2018-05-08 |
| EP2766479A1 (en) | 2014-08-20 |
| US9738891B2 (en) | 2017-08-22 |
| BR112014009066A8 (pt) | 2018-01-09 |
| US20140296323A1 (en) | 2014-10-02 |
| AU2012322903B2 (en) | 2017-09-14 |
| EP2766479B1 (en) | 2017-09-13 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| ES2535654T3 (es) | ADN triciclo-fosforotioato | |
| ES2551154T3 (es) | Procedimiento para la eficiente omisión del exón (44) en distrofia muscular de Duchenne y medios asociados | |
| ES2573981T3 (es) | Oligonucleótidos antisentido de triciclo-ADN, composiciones, y métodos para el tratamiento de enfermedades | |
| DK2850186T3 (en) | COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION | |
| ES2700277T3 (es) | Agentes de doble cadena para el suministro de oligonucleótidos terapéuticos | |
| ES2699827T3 (es) | Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto | |
| ES2566553T3 (es) | ARNnp U7 modificados para el tratamiento de las enfermedades neuromusculares | |
| ES2707232T3 (es) | Acido nucleico bicatenario quimérico | |
| ES2935658T3 (es) | Compuestos y métodos para la modulación de SMN2 | |
| US12178855B2 (en) | Compositions and methods for facilitating delivery of synthetic nucleic acids to cells | |
| CN104540946A (zh) | 用于调节utrn表达的组合物和方法 | |
| JP2019527549A (ja) | 転写プロセシングの調節のための化合物及び方法 | |
| ES2918975T3 (es) | Tratamiento de la distrofia facioescapulohumeral | |
| WO2019183005A1 (en) | 2'f-ana-let7 mediated utrophin upregulation for dmd therapy | |
| CN107466323A (zh) | 酰基‑氨基‑lna和/或烃基‑氨基‑lna寡核苷酸 | |
| JP7536187B2 (ja) | 核内受容体サブファミリー１グループｈメンバー３（ｎｒ１ｈ３）発現を阻害するための組成物および方法 | |
| HK1186205B (en) | Tricyclo-phosphorothioate dna | |
| Leumann et al. | des brevets (11) GG 0 000 000 L0 (12) LLLLLGGG GGGGGGGG LLLLL GGGGGGGG | |
| EP4426839A1 (en) | Lipid conjugation for targeting astrocytes of the central nervous system | |
| CN121793974A (zh) | 用于治疗肌少症的组合物和方法 | |
| WO2024233864A2 (en) | Galnac-conjugated rnai oligonucleotides | |
| WO2023081822A1 (en) | Lipid conjugation for targeting oligodendrocytes of the central nervous system | |
| BR122020025437B1 (pt) | Uso de um conjugado de oligômero antissenso para tratar distrofia muscular de duchenne | |
| BR112019012647B1 (pt) | Conjugados de oligômero antissenso que induzem o salto de éxon 51 no gene da distrofia humana |