MA42695A - Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale - Google Patents
Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinaleInfo
- Publication number
- MA42695A MA42695A MA042695A MA42695A MA42695A MA 42695 A MA42695 A MA 42695A MA 042695 A MA042695 A MA 042695A MA 42695 A MA42695 A MA 42695A MA 42695 A MA42695 A MA 42695A
- Authority
- MA
- Morocco
- Prior art keywords
- exons
- inclusion
- muscle atrophy
- antisense oligomers
- modified antisense
- Prior art date
Links
- 108700024394 Exon Proteins 0.000 title 1
- 206010028289 Muscle atrophy Diseases 0.000 title 1
- 230000000692 anti-sense effect Effects 0.000 title 1
- 230000020763 muscle atrophy Effects 0.000 title 1
- 201000000585 muscular atrophy Diseases 0.000 title 1
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
- A61K31/712—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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
-
- 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
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
-
- 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
- C12N2310/3233—Morpholino-type ring
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562211678P | 2015-08-28 | 2015-08-28 | |
| US201662379696P | 2016-08-25 | 2016-08-25 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MA42695A true MA42695A (fr) | 2018-07-04 |
Family
ID=56883867
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA042695A MA42695A (fr) | 2015-08-28 | 2016-08-26 | Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US10905709B2 (enExample) |
| EP (1) | EP3341480A1 (enExample) |
| JP (2) | JP6987041B2 (enExample) |
| AU (1) | AU2016317667A1 (enExample) |
| CA (1) | CA2996164A1 (enExample) |
| HK (1) | HK1257498A1 (enExample) |
| MA (1) | MA42695A (enExample) |
| WO (1) | WO2017040271A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| WO2019079637A2 (en) * | 2017-10-18 | 2019-04-25 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGOMERIC COMPOUNDS |
| US12215382B2 (en) | 2019-03-01 | 2025-02-04 | The General Hospital Corporation | Liver protective MARC variants and uses thereof |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| TW202517787A (zh) | 2020-02-28 | 2025-05-01 | 美商Ionis製藥公司 | 用於調節smn2之化合物及方法 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2802206B1 (fr) | 1999-12-14 | 2005-04-22 | Sod Conseils Rech Applic | Derives de 4-aminopiperidine et leur utilisation en tant que medicament |
| JP2003533986A (ja) | 2000-05-04 | 2003-11-18 | エイブイアイ バイオファーマ， インコーポレイテッド | スプライス領域アンチセンス組成物および方法 |
| SI2548560T1 (sl) * | 2005-06-23 | 2015-12-31 | Isis Pharmaceuticals, Inc. | Sestavki in postopki za moduliranje izrezovanja smn2 |
| US8067571B2 (en) | 2005-07-13 | 2011-11-29 | Avi Biopharma, Inc. | Antibacterial antisense oligonucleotide and method |
| PL2735568T3 (pl) * | 2006-05-10 | 2018-02-28 | Sarepta Therapeutics, Inc. | Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami |
| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| US20120149757A1 (en) | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
| LT3305302T (lt) * | 2009-06-17 | 2018-12-10 | Biogen Ma Inc. | Junginiai ir būdai smn2 splaisingo moduliavimui subjekte |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| JP5585822B2 (ja) | 2010-05-11 | 2014-09-10 | 東レ・ファインケミカル株式会社 | 光学活性ニペコチン酸誘導体の製造方法 |
| KR102095478B1 (ko) * | 2010-05-28 | 2020-04-01 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| EP2582397A4 (en) * | 2010-06-15 | 2014-10-29 | Isis Pharmaceuticals Inc | COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS |
| US9161948B2 (en) | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| CN103619356B (zh) * | 2011-05-05 | 2017-09-12 | 萨勒普塔医疗公司 | 肽寡核苷酸缀合物 |
| CA2842742A1 (en) * | 2011-06-23 | 2012-12-27 | Cold Spring Harbor Laboratory | Phenocopy model of disease |
| ES2727481T3 (es) * | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| JP6317675B2 (ja) | 2011-11-30 | 2018-04-25 | サレプタ セラピューティクス， インコーポレイテッド | 延長リピート病を処置するためのオリゴヌクレオチド |
| WO2013086207A1 (en) * | 2011-12-06 | 2013-06-13 | The Ohio State University | Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease |
| BR112014023347A2 (pt) | 2012-03-20 | 2017-07-18 | Sarepta Therapeutics Inc | conjugados de ácido borônico de análogos oligonucleotídeos |
| HK1208700A1 (en) * | 2012-05-16 | 2016-03-11 | Rana Therapeutics Inc. | Compositions and methods for modulating smn gene family expression |
| US20160002624A1 (en) | 2012-05-17 | 2016-01-07 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide compositions |
| WO2014110291A1 (en) | 2013-01-09 | 2014-07-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| EP2946013A1 (en) * | 2013-01-16 | 2015-11-25 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| EP2983676B1 (en) * | 2013-04-12 | 2019-03-20 | The Curators of the University of Missouri | Smn2 element 1 antisense compositions and methods and uses thereof |
| CA2955698A1 (en) * | 2013-07-29 | 2015-02-05 | Universitat Zu Koln | Neurocalcin delta inhibitors and therapeutic and non-therapeutic uses thereof |
| JP2016530887A (ja) * | 2013-09-13 | 2016-10-06 | ザ ユニバーシティ オブ ウェスタン オーストラリア | Ｓｍｎ関連病理を処置するためのアンチセンスオリゴマーおよび方法 |
| US9845469B2 (en) * | 2014-02-10 | 2017-12-19 | Ohio State Innovation Foundation | Antisense oligonucleotides for treatment of spinal muscular atrophy |
| US10905709B2 (en) | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
-
2016
- 2016-08-26 US US15/754,782 patent/US10905709B2/en active Active
- 2016-08-26 WO PCT/US2016/048965 patent/WO2017040271A1/en not_active Ceased
- 2016-08-26 MA MA042695A patent/MA42695A/fr unknown
- 2016-08-26 HK HK18116288.7A patent/HK1257498A1/zh unknown
- 2016-08-26 AU AU2016317667A patent/AU2016317667A1/en not_active Abandoned
- 2016-08-26 CA CA2996164A patent/CA2996164A1/en not_active Abandoned
- 2016-08-26 JP JP2018510718A patent/JP6987041B2/ja active Active
- 2016-08-26 EP EP16762935.1A patent/EP3341480A1/en not_active Withdrawn
-
2020
- 2020-11-23 US US16/949,980 patent/US12121532B2/en active Active
- 2020-12-25 JP JP2020216610A patent/JP2021048877A/ja not_active Withdrawn
-
2024
- 2024-08-23 US US18/813,696 patent/US20250241940A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA2996164A1 (en) | 2017-03-09 |
| EP3341480A1 (en) | 2018-07-04 |
| US12121532B2 (en) | 2024-10-22 |
| AU2016317667A1 (en) | 2018-03-22 |
| JP2018525015A (ja) | 2018-09-06 |
| JP2021048877A (ja) | 2021-04-01 |
| US20210169918A1 (en) | 2021-06-10 |
| US20190015440A1 (en) | 2019-01-17 |
| US20250241940A1 (en) | 2025-07-31 |
| JP6987041B2 (ja) | 2021-12-22 |
| HK1257498A1 (zh) | 2019-10-25 |
| US10905709B2 (en) | 2021-02-02 |
| WO2017040271A1 (en) | 2017-03-09 |
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