NZ732507A - Improved compositions for treating muscular dystrophy - Google Patents
Improved compositions for treating muscular dystrophyInfo
- Publication number
- NZ732507A NZ732507A NZ732507A NZ73250714A NZ732507A NZ 732507 A NZ732507 A NZ 732507A NZ 732507 A NZ732507 A NZ 732507A NZ 73250714 A NZ73250714 A NZ 73250714A NZ 732507 A NZ732507 A NZ 732507A
- Authority
- NZ
- New Zealand
- Prior art keywords
- muscular dystrophy
- improved compositions
- treating muscular
- eteplirsen
- pharmaceutically acceptable
- Prior art date
Links
Classifications
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive 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
-
- 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
-
- 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
-
- 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
- C12N2310/33—Chemical structure of the base
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Pain & Pain Management (AREA)
- Dermatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361793463P | 2013-03-15 | 2013-03-15 | |
| NZ631289A NZ631289A (en) | 2013-03-15 | 2014-03-14 | Improved compositions for treating muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| NZ732507A true NZ732507A (en) | 2018-08-31 |
Family
ID=50678303
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ732507A NZ732507A (en) | 2013-03-15 | 2014-03-14 | Improved compositions for treating muscular dystrophy |
| NZ631289A NZ631289A (en) | 2013-03-15 | 2014-03-14 | Improved compositions for treating muscular dystrophy |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| NZ631289A NZ631289A (en) | 2013-03-15 | 2014-03-14 | Improved compositions for treating muscular dystrophy |
Country Status (14)
| Country | Link |
|---|---|
| US (8) | US9506058B2 (cg-RX-API-DMAC7.html) |
| EP (2) | EP2968991A2 (cg-RX-API-DMAC7.html) |
| JP (2) | JP2016516066A (cg-RX-API-DMAC7.html) |
| KR (2) | KR20200139271A (cg-RX-API-DMAC7.html) |
| CN (2) | CN105307723A (cg-RX-API-DMAC7.html) |
| AU (3) | AU2014233456B2 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112015022998A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2906812A1 (cg-RX-API-DMAC7.html) |
| EA (1) | EA201591792A1 (cg-RX-API-DMAC7.html) |
| HK (1) | HK1220154A1 (cg-RX-API-DMAC7.html) |
| IL (3) | IL241558B (cg-RX-API-DMAC7.html) |
| MX (2) | MX373959B (cg-RX-API-DMAC7.html) |
| NZ (2) | NZ732507A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2014144978A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (86)
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| EP2206781B1 (en) | 2004-06-28 | 2015-12-02 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| CA2596506C (en) | 2005-02-09 | 2021-04-06 | Avi Biopharma, Inc. | Antisense composition and method for treating muscle atrophy |
| CA2740328A1 (en) | 2008-10-24 | 2010-04-29 | Avi Biopharma, Inc. | Multiple exon skipping compositions for dmd |
| LT2499249T (lt) | 2009-11-12 | 2018-12-27 | The University Of Western Australia | Priešprasmės molekulės ir patologijų gydymo būdai |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
| BR112015023001B8 (pt) | 2013-03-14 | 2022-08-09 | Sarepta Therapeutics Inc | Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd) |
| AU2014233456B2 (en) | 2013-03-15 | 2019-02-21 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| WO2016037165A1 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating exon skipping anti-viral transfer vector immune responses |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| CN107921092B (zh) | 2015-05-19 | 2022-04-08 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
| EP3351633B1 (en) | 2015-09-15 | 2020-06-24 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid |
| CN108738310A (zh) * | 2015-09-30 | 2018-11-02 | 萨勒普塔医疗公司 | 用于治疗肌营养不良的方法 |
| NZ741397A (en) | 2015-10-09 | 2025-07-25 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods 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 |
| US11970710B2 (en) | 2015-10-13 | 2024-04-30 | Duke University | Genome engineering with Type I CRISPR systems in eukaryotic cells |
| EP3384055B1 (en) * | 2015-11-30 | 2025-07-16 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| JP2018538302A (ja) * | 2015-12-15 | 2018-12-27 | サレプタ セラピューティクス， インコーポレイテッド | ペプチドオリゴヌクレオチド複合体 |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
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| SG10202101836TA (en) | 2016-05-24 | 2021-03-30 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| SG11201809494VA (en) * | 2016-05-24 | 2018-12-28 | Sarepta Therapeutics Inc | Pharmaceutical composition comprising eteplirsen |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| HRP20241428T1 (hr) | 2016-05-24 | 2025-01-03 | Sarepta Therapeutics, Inc. | Postupci za pripremu oligomera |
| BR112018074270B1 (pt) | 2016-05-24 | 2021-02-02 | Sarepta Therapeutics, Inc | processo para preparar compostos oligoméricos bem como os ditos compostos |
| MA45362A (fr) | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
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| US12214056B2 (en) | 2016-07-19 | 2025-02-04 | Duke University | Therapeutic applications of CPF1-based genome editing |
| CN106581043A (zh) * | 2016-10-24 | 2017-04-26 | 佳木斯大学 | 一种适用于杜氏肌营养不良症的药物制剂及其制备方法 |
| MX2019006157A (es) * | 2016-11-28 | 2019-11-21 | Univ Texas | Prevención de la distrofia muscular mediante la edición de genes mediada por crispr/cpf1. |
| JP7160807B2 (ja) | 2016-12-19 | 2022-10-25 | 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コンジュゲート |
| FI4122497T3 (fi) * | 2016-12-19 | 2024-06-04 | Sarepta Therapeutics Inc | Eksonin ohittavia oligomeerikonjugaatteja lihasdystrofiaan |
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| US11622960B2 (en) | 2017-06-19 | 2023-04-11 | University Of Maryland, Baltimore | Microtubule polymerization inhibitor prodrugs and methods of using the same |
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