DK2417257T3 - Tricyclo-dna-antisense-oligonukleotider, sammensætninger, og fremgangsmåder til behandlingen af sygdom - Google Patents
Tricyclo-dna-antisense-oligonukleotider, sammensætninger, og fremgangsmåder til behandlingen af sygdom Download PDFInfo
- Publication number
- DK2417257T3 DK2417257T3 DK10713338.1T DK10713338T DK2417257T3 DK 2417257 T3 DK2417257 T3 DK 2417257T3 DK 10713338 T DK10713338 T DK 10713338T DK 2417257 T3 DK2417257 T3 DK 2417257T3
- Authority
- DK
- Denmark
- Prior art keywords
- procedures
- disease
- compositions
- treatment
- antisense oligonucleotides
- Prior art date
Links
- 108020000948 Antisense Oligonucleotides Proteins 0.000 title 1
- 239000000074 antisense oligonucleotide Substances 0.000 title 1
- 238000012230 antisense oligonucleotides Methods 0.000 title 1
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
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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
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
-
- 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
- A61P21/04—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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N2310/32—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)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurosurgery (AREA)
- Rheumatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21238409P | 2009-04-10 | 2009-04-10 | |
| PCT/EP2010/054735 WO2010115993A1 (en) | 2009-04-10 | 2010-04-09 | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2417257T3 true DK2417257T3 (da) | 2016-06-06 |
Family
ID=42293782
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK10713338.1T DK2417257T3 (da) | 2009-04-10 | 2010-04-09 | Tricyclo-dna-antisense-oligonukleotider, sammensætninger, og fremgangsmåder til behandlingen af sygdom |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20120149756A1 (da) |
| EP (1) | EP2417257B1 (da) |
| JP (3) | JP2012523225A (da) |
| CN (2) | CN109576268A (da) |
| CA (1) | CA2758189C (da) |
| DK (1) | DK2417257T3 (da) |
| ES (1) | ES2573981T3 (da) |
| HU (1) | HUE028036T2 (da) |
| IL (1) | IL215682A (da) |
| PL (1) | PL2417257T3 (da) |
| PT (1) | PT2417257E (da) |
| WO (1) | WO2010115993A1 (da) |
Families Citing this family (96)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| DK1766010T3 (da) | 2004-06-28 | 2011-06-06 | Univ Western Australia | Antisense-oligonukleotider til induktion af exon-skipping og fremgangsmåder til anvendelse deraf |
| CA2596506C (en) | 2005-02-09 | 2021-04-06 | Avi Biopharma, Inc. | Antisense composition and method for treating muscle atrophy |
| ES2639852T3 (es) | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| IL313162A (en) | 2008-10-24 | 2024-07-01 | Sarepta Therapeutics Inc | Antisense compounds for inducing axon termination and preparations containing them for the treatment of muscular dystrophy |
| WO2010119368A2 (en) * | 2009-04-13 | 2010-10-21 | Sulur Subramaniam Vanangamudi | A medicinal cream made using miconazole nitrate and chitosan, and a process to make the same |
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| KR20230137491A (ko) | 2009-11-12 | 2023-10-04 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
| JP5981428B2 (ja) * | 2010-07-19 | 2016-08-31 | アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整 |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
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| WO2012144906A1 (en) * | 2011-04-22 | 2012-10-26 | Prosensa Technologies B.V. | New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1) |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| ES2867054T3 (es) * | 2011-10-11 | 2021-10-20 | Inst Nat Sante Rech Med | Tratamiento de salto de exones para epidermólisis ampollosa distrófica |
| PL2581448T3 (pl) * | 2011-10-13 | 2015-08-31 | Association Inst De Myologie | Tricyklo-tiofosforanowy DNA |
| CA2855241A1 (en) * | 2011-11-11 | 2013-05-16 | Santaris Pharma A/S | Compounds for the modulation of smn2 splicing |
| CN118581086A (zh) | 2012-01-27 | 2024-09-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| EA201492116A1 (ru) | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| CA2873794A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating smn gene family expression |
| KR20230116945A (ko) | 2013-03-14 | 2023-08-04 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 엑손 스키핑 조성물 |
| KR20150133768A (ko) | 2013-03-15 | 2015-11-30 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 개선된 조성물 |
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2010
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- 2010-04-09 US US13/263,949 patent/US20120149756A1/en not_active Abandoned
- 2010-04-09 ES ES10713338.1T patent/ES2573981T3/es active Active
- 2010-04-09 PT PT107133381T patent/PT2417257E/pt unknown
- 2010-04-09 CN CN201811113867.2A patent/CN109576268A/zh active Pending
- 2010-04-09 EP EP10713338.1A patent/EP2417257B1/en active Active
- 2010-04-09 JP JP2012504034A patent/JP2012523225A/ja active Pending
- 2010-04-09 CA CA2758189A patent/CA2758189C/en active Active
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- 2010-04-09 PL PL10713338.1T patent/PL2417257T3/pl unknown
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| JP6675851B2 (ja) | 2020-04-08 |
| CA2758189A1 (en) | 2010-10-14 |
| JP2019055990A (ja) | 2019-04-11 |
| US10465191B2 (en) | 2019-11-05 |
| US20170260524A1 (en) | 2017-09-14 |
| EP2417257B1 (en) | 2016-03-09 |
| EP2417257A1 (en) | 2012-02-15 |
| CN102625840A (zh) | 2012-08-01 |
| PL2417257T3 (pl) | 2016-11-30 |
| PT2417257E (pt) | 2016-06-03 |
| CA2758189C (en) | 2020-12-29 |
| JP2012523225A (ja) | 2012-10-04 |
| CN109576268A (zh) | 2019-04-05 |
| IL215682A0 (en) | 2012-01-31 |
| IL215682A (en) | 2017-06-29 |
| US20120149756A1 (en) | 2012-06-14 |
| WO2010115993A1 (en) | 2010-10-14 |
| HUE028036T2 (en) | 2016-11-28 |
| ES2573981T3 (es) | 2016-06-13 |
| JP2016027823A (ja) | 2016-02-25 |
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