DE60042984D1 - Arzneitmittel zur Behandlung von Duchenne-Muskeldystrophie - Google Patents
Arzneitmittel zur Behandlung von Duchenne-MuskeldystrophieInfo
- Publication number
- DE60042984D1 DE60042984D1 DE60042984T DE60042984T DE60042984D1 DE 60042984 D1 DE60042984 D1 DE 60042984D1 DE 60042984 T DE60042984 T DE 60042984T DE 60042984 T DE60042984 T DE 60042984T DE 60042984 D1 DE60042984 D1 DE 60042984D1
- Authority
- DE
- Germany
- Prior art keywords
- drugs
- treatment
- muscular dystrophy
- duchenne muscular
- duchenne
- 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.)
- Expired - Lifetime
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
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/06—Anabolic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Animal Behavior & Ethology (AREA)
- Physical Education & Sports Medicine (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Endocrinology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP2000125448 | 2000-04-26 |
Publications (1)
Publication Number | Publication Date |
---|---|
DE60042984D1 true DE60042984D1 (de) | 2009-10-29 |
Family
ID=18635405
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DE60039038T Expired - Lifetime DE60039038D1 (de) | 2000-04-26 | 2000-09-27 | "Splicing enhancer sequences" im Dystrophie Gen |
DE60042984T Expired - Lifetime DE60042984D1 (de) | 2000-04-26 | 2000-09-27 | Arzneitmittel zur Behandlung von Duchenne-Muskeldystrophie |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DE60039038T Expired - Lifetime DE60039038D1 (de) | 2000-04-26 | 2000-09-27 | "Splicing enhancer sequences" im Dystrophie Gen |
Country Status (6)
Country | Link |
---|---|
US (1) | US6653467B1 (de) |
EP (2) | EP1544297B1 (de) |
JP (2) | JP4590033B2 (de) |
AU (1) | AU780517B2 (de) |
CA (1) | CA2319149A1 (de) |
DE (2) | DE60039038D1 (de) |
Families Citing this family (49)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6653467B1 (en) * | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
AU5752601A (en) | 2000-05-04 | 2001-11-12 | Avi Biopharma Inc | Splice-region antisense composition and method |
US6727355B2 (en) * | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
EP1191097A1 (de) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induktion von "exon-skipping" in eukaryotischen Zellen |
ES2561851T3 (es) * | 2002-11-25 | 2016-03-01 | Masafumi Matsuo | Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm |
WO2004083432A1 (en) | 2003-03-21 | 2004-09-30 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
ES2361325T3 (es) * | 2004-06-28 | 2011-06-16 | The University Of Western Australia | Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos. |
FR2874384B1 (fr) * | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
JP2008538500A (ja) * | 2005-04-22 | 2008-10-30 | アカデミス ツィーケンホイス ライデン | SRタンパク質の結合に対する干渉とRNA二次構造に対する干渉による、mRNA前駆体におけるエクソン認識の調節 |
US20070017138A1 (en) * | 2005-04-25 | 2007-01-25 | Young Kevin D | Weapon grip assist |
WO2007123391A1 (en) * | 2006-04-20 | 2007-11-01 | Academisch Ziekenhuis Leiden | Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene. |
EP1857548A1 (de) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Mittel und Verfahren zur Induktion von Exon-skipping |
US8097596B2 (en) * | 2006-06-30 | 2012-01-17 | Lakewood-Amedex, Inc. | Compositions and methods for the treatment of muscle wasting |
PL2049664T3 (pl) | 2006-08-11 | 2012-02-29 | Biomarin Tech Bv | Jednoniciowe oligonukleotydy komplementarne do powtarzalnych elementów do leczenia chorób genetycznych związanych z niestabilnością powtórzeń DNA |
CN101790385A (zh) * | 2007-07-12 | 2010-07-28 | 普罗森那技术公司 | 用于使化合物靶向多种选定器官、组织或肿瘤细胞的分子 |
DK2167136T3 (en) * | 2007-07-12 | 2016-07-25 | Biomarin Tech Bv | Molecules for targeting compounds at different selected organs or tissues |
AU2008317566B2 (en) | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
NZ587178A (en) * | 2008-02-08 | 2011-11-25 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
EP2119783A1 (de) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel |
PL3133160T3 (pl) | 2008-10-24 | 2019-06-28 | Sarepta Therapeutics, Inc. | Kompozycje do pomijania eksonów w DMD |
US20120046342A1 (en) | 2009-04-24 | 2012-02-23 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
KR102239374B1 (ko) | 2009-11-12 | 2021-04-14 | 더 유니버시티 오브 웨스턴 오스트레일리아 | 안티센스 분자 및 이를 이용한 질환 치료방법 |
CA2785451C (en) | 2009-12-24 | 2019-01-22 | Prosensa Technologies B.V. | Molecule for treating an inflammatory disorder |
KR20150082685A (ko) * | 2010-08-20 | 2015-07-15 | 레플리코르 인코포레이티드 | 올리고뉴클레오티드 킬레이트 복합체 |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
EP3260540A1 (de) | 2010-11-12 | 2017-12-27 | The General Hospital Corporation | Polycombassoziierte nichtcodierende rnas |
EP2672977A1 (de) | 2011-02-08 | 2013-12-18 | The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System | Antisense-oligonukleotide |
US20130085139A1 (en) * | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
JP2015509922A (ja) | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
EP2900686B1 (de) | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Gegen oligodendrozyten gerichtete aav-vektoren |
JP6449231B2 (ja) | 2013-03-14 | 2019-01-09 | サレプタ セラピューティクス, インコーポレイテッド | 筋ジストロフィを処置するためのエキソンスキッピング組成物 |
KR20200139271A (ko) | 2013-03-15 | 2020-12-11 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 개선된 조성물 |
WO2016070060A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
CN107532173A (zh) | 2014-11-21 | 2018-01-02 | 北卡罗来纳-查佩尔山大学 | 靶向中枢神经系统的aav载体 |
US10308705B2 (en) | 2015-02-06 | 2019-06-04 | The University Of North Carolina At Chapel Hill | Optimized human clotting factor VIII gene expression cassettes and their use |
US10900036B2 (en) | 2015-03-17 | 2021-01-26 | The General Hospital Corporation | RNA interactome of polycomb repressive complex 1 (PRC1) |
MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
CN109311919A (zh) | 2016-06-30 | 2019-02-05 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体 |
RU2022107926A (ru) * | 2016-07-05 | 2022-04-27 | Биомарин Текнолоджис Б.В. | Олигонуклеотиды, производящие переключение или модулирование сплайсинга пре-рнк и содержащие бициклические каркасные фрагменты, с улучшенными характеристиками для лечения генетических заболеваний |
PT3554553T (pt) | 2016-12-19 | 2022-08-04 | Sarepta Therapeutics Inc | Conjugados oligoméricos de salto de exão para a distrofia muscular |
IL297528A (en) | 2016-12-19 | 2022-12-01 | Sarepta Therapeutics Inc | Exon-skipping oligomer conjugates for muscular dystrophy |
EP4406601A2 (de) | 2016-12-19 | 2024-07-31 | Sarepta Therapeutics, Inc. | Exon-skipping-oligomerkonjugate für muskeldystrophie |
EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
CA3098266A1 (en) * | 2018-05-11 | 2019-11-14 | Alpha Anomeric Sas | Oligonucleotides conjugates comprising 7'-5'-alpha-anomeric-bicyclic sugar nucleosides |
US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
Family Cites Families (16)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5151520A (en) | 1985-11-20 | 1992-09-29 | The Mead Corporation | Cationic dye-triarylmonoalkylorate anion complexes |
US5239060A (en) * | 1986-07-25 | 1993-08-24 | The Children's Medical Center Corporation | Muscular dystrophy protein, dystrophin |
US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
EP0698092B1 (de) | 1993-05-11 | 2007-07-25 | The University Of North Carolina At Chapel Hill | Antisense Oligonukleotide die anomales Splicing verhindern und deren Verwendung |
JPH11140930A (ja) | 1997-11-13 | 1999-05-25 | Matsushita Electric Works Ltd | シャワー装置 |
EP1071753A2 (de) * | 1998-04-20 | 2001-01-31 | Ribozyme Pharmaceuticals, Inc. | Nukeinsäure moleküle mit einer neuen chemischen zusammensetzung die fähig sind die expression von gene zu modulieren |
US6417169B1 (en) * | 1998-04-23 | 2002-07-09 | Genesense Technologies Inc. | Insulin-like growth factor II antisense oligonucleotide sequences and methods of using same to inhibit cell growth |
EP1078053B1 (de) * | 1998-05-14 | 2005-09-28 | Coley Pharmaceutical GmbH | Verfahren zur regulierung der hematopoiese mit hilfe von cpg oligonukleotide |
WO1999061056A2 (en) * | 1998-05-22 | 1999-12-02 | Loeb Health Research Institute At The Ottawa Hospital | Methods and products for inducing mucosal immunity |
JP2000125448A (ja) | 1998-10-14 | 2000-04-28 | Yazaki Corp | 電気接続箱 |
US5948680A (en) | 1998-12-17 | 1999-09-07 | Isis Pharmaceuticals Inc. | Antisense inhibition of Elk-1 expression |
JP2000325085A (ja) | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
JP3585769B2 (ja) | 1999-06-02 | 2004-11-04 | 三菱電機株式会社 | 零相変流器 |
US6653467B1 (en) * | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
JP4836366B2 (ja) * | 2000-08-25 | 2011-12-14 | 雅文 松尾 | デュシェンヌ型筋ジストロフィー治療剤 |
US6727355B2 (en) * | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
-
2000
- 2000-09-22 US US09/667,327 patent/US6653467B1/en not_active Expired - Lifetime
- 2000-09-27 DE DE60039038T patent/DE60039038D1/de not_active Expired - Lifetime
- 2000-09-27 DE DE60042984T patent/DE60042984D1/de not_active Expired - Lifetime
- 2000-09-27 EP EP05001018A patent/EP1544297B1/de not_active Expired - Lifetime
- 2000-09-27 CA CA002319149A patent/CA2319149A1/en not_active Abandoned
- 2000-09-27 EP EP00121053A patent/EP1160318B1/de not_active Expired - Lifetime
- 2000-09-28 AU AU61354/00A patent/AU780517B2/en not_active Ceased
- 2000-11-16 JP JP2000348957A patent/JP4590033B2/ja not_active Expired - Fee Related
-
2010
- 2010-08-14 JP JP2010181478A patent/JP2010268815A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
JP2002010790A (ja) | 2002-01-15 |
AU6135400A (en) | 2001-11-01 |
EP1160318A2 (de) | 2001-12-05 |
CA2319149A1 (en) | 2001-10-26 |
JP4590033B2 (ja) | 2010-12-01 |
AU780517B2 (en) | 2005-03-24 |
DE60039038D1 (de) | 2008-07-10 |
EP1544297A2 (de) | 2005-06-22 |
EP1544297B1 (de) | 2009-09-16 |
EP1544297A3 (de) | 2005-07-06 |
EP1160318A3 (de) | 2004-06-23 |
JP2010268815A (ja) | 2010-12-02 |
US6653467B1 (en) | 2003-11-25 |
EP1160318B1 (de) | 2008-05-28 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
DE60042984D1 (de) | Arzneitmittel zur Behandlung von Duchenne-Muskeldystrophie | |
DE60118067D1 (de) | Pharmazeutische Zusammensetzung mit Dystrophin Exon 45 zur Behandlung von Duchenne-Muskeldystrophie | |
ATE293610T1 (de) | 2-amino-benzoxazinonderivate zur behandlung von fettleibigkeit | |
DE69929996D1 (de) | Pharmazeutisches mittel zur behandlung von diabetes | |
ATE275145T1 (de) | Chinazolinderivate zur behandlung von tumoren | |
DE60043305D1 (de) | Pharmazeutische zubereitungen zur behandlung von insulinresitenz | |
DE60124302D1 (de) | Thiazolderivate zur behandlung von ppar-lierte krankheiten | |
DE60142473D1 (de) | Intrakorporale medikamente zur photodynamischen behandlung von erkrankungen | |
ATE293447T1 (de) | 2-oxy-benzoxazinonderivate zur behandlung von fettleibigkeit | |
DE50206657D1 (de) | Pharmazeutische zusammensetzung zur behandlung von multiple sklerose | |
DE60234760D1 (de) | Phenylindole zur behandlung von hiv | |
DE60232251D1 (de) | Pharmazeutische zusammensetzungen zur behandlung von asthma | |
DE60132600D1 (de) | Medikamente zur chemotherapeutischen behandlung von erkrankungen | |
DE60103137D1 (de) | Impstoff zur behandlung von atherosclerosis | |
DE69934877D1 (de) | Biochanin-angereicherter extrakt zur behandlung von estrogenabhängigen erkrankungen | |
DE60020820D1 (de) | Thiadiazolyl-harnstoffe oder -thioharnstoffe zur antiviralen behandlung | |
DE10082139T1 (de) | Zusammensetzung zur Behandlung von Reibungspaaren | |
DE60136477D1 (de) | Retinoide zur behandlung von emphysem | |
DE60208186D1 (de) | Chinazolin- und chinazolinähnliche verbindungen zur behandlung von integrinvermittelten erkrankungen | |
ATE346614T1 (de) | Vaginale zusammensetzung enthaltend lidocaine zur behandlung von gebärmutterrhythmusstörung | |
DE60030554D1 (de) | Verwendungen von et743 zur behandlung von krebs | |
ATE295174T1 (de) | Arzneimittel zur behandlung von bluthochdruck | |
DE50013843D1 (de) | Arzneimittel zur behandlung von darmerkrankungen | |
ATE298569T1 (de) | Virus behandlung | |
DE60238765D1 (de) | Carbamatverbindungen zur behandlung von schmerz |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
8364 | No opposition during term of opposition |