HRP20201125T1 - Antisens nukleinska kiselina - Google Patents
Antisens nukleinska kiselina Download PDFInfo
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- HRP20201125T1 HRP20201125T1 HRP20201125TT HRP20201125T HRP20201125T1 HR P20201125 T1 HRP20201125 T1 HR P20201125T1 HR P20201125T T HRP20201125T T HR P20201125TT HR P20201125 T HRP20201125 T HR P20201125T HR P20201125 T1 HRP20201125 T1 HR P20201125T1
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- Prior art keywords
- nucleotide sequence
- pharmaceutically acceptable
- acceptable salt
- hydrate
- antisense oligomer
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- 230000000692 anti-sense effect Effects 0.000 title claims 15
- 150000007523 nucleic acids Chemical class 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 title 1
- 239000002773 nucleotide Substances 0.000 claims 19
- 125000003729 nucleotide group Chemical group 0.000 claims 19
- 150000003839 salts Chemical class 0.000 claims 14
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims 5
- 230000000295 complement effect Effects 0.000 claims 5
- 108091034117 Oligonucleotide Proteins 0.000 claims 4
- 201000006938 muscular dystrophy Diseases 0.000 claims 4
- 229910019142 PO4 Inorganic materials 0.000 claims 2
- 239000000825 pharmaceutical preparation Substances 0.000 claims 2
- 239000010452 phosphate Substances 0.000 claims 2
- HMFHBZSHGGEWLO-SOOFDHNKSA-N D-ribofuranose Chemical compound OC[C@H]1OC(O)[C@H](O)[C@@H]1O HMFHBZSHGGEWLO-SOOFDHNKSA-N 0.000 claims 1
- 108010069091 Dystrophin Proteins 0.000 claims 1
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims 1
- 239000004480 active ingredient Substances 0.000 claims 1
- 125000000217 alkyl group Chemical group 0.000 claims 1
- 125000002947 alkylene group Chemical group 0.000 claims 1
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-N phosphoramidic acid Chemical compound NP(O)(O)=O PTMHPRAIXMAOOB-UHFFFAOYSA-N 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
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- 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
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- 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
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- 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
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- 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
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/313—Phosphorodithioates
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- 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
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Biomedical Technology (AREA)
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- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
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- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
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- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Claims (15)
1. Antisens oligomer 14 do 32 baza u dužini koji sadrži spojena dva jedinična oligomera odabrana iz skupine koja sadrži (a) do (e) prikazane dolje, ili njihovu farmaceutski prihvatljivu sol ili hidrat, pri čemu dva jedinična oligomera nisu međusobno neprekidni ili ne preklapaju se jedan s drugim:
(a) jedinični oligomer koji se sastoji od nukleotidne sekvence komplementarne nukleotidnoj sekvenci koja se sastoji od međusobnih 7 do 16 baza odabranih iz nukleotidne sekvence smještene na pozicijama -5 do 15 od 5'-terminalnog kraja egzona 45 u ljudskom genu distrofina;
(b) jedinični oligomer koji se sastoji od nukleotidne sekvence komplementarne nukleotidnoj sekvenci koja se sastoji od međusobnih 7 do 16 baza odabranih iz nukleotidne sekvence smještene na pozicijama 48 do 70 od 5'-terminalnog kraja egzona 45 u ljudskom genu distrofina;
(c) jedinični oligomer koji se sastoji od nukleotidne sekvence komplementarne nukleotidnoj sekvenci koja se sastoji od međusobnih 7 do 16 baza odabranih iz nukleotidne sekvence smještene na pozicijama 128 do 150 od 5'-terminalnog kraja egzona 45 u ljudskom genu distrofina;
(d) jedinični oligomer koji se sastoji od nukleotidne sekvence komplementarne nukleotidnoj sekvenci koja se sastoji od međusobnih 7 do 16 baza odabranih iz nukleotidne sekvence smještene na pozicijama 15 do 40 od 5'-terminalnog kraja egzona 45 u ljudskom genu distrofina; i
(e) jedinični oligomer koji se sastoji od nukleotidne sekvence komplementarne nukleotidnoj sekvenci koja se sastoji od međusobnih 7 do 16 baza odabranih iz nukleotidne sekvence smještene na pozicijama 110 do 125 od 5'-terminalnog kraja egzona 45 u ljudskom genu distrofina.
2. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat prema zahtjevu 1, naznačen time što jedan od dva jedinična oligomera je (a).
3. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat prema zahtjevu 1 ili 2, koji se sastoji od bilo koje nukleotidne sekvence odabrane iz skupine koja sadrži SEQ ID NO: 7 do 12, 14 do 33, 40 do 52, 57, 64, 65 i 79 do 86.
4. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat prema bilo kojem od zahtjeva 1 do 3, koji se sastoji od bilo koje skupine odabrane iz skupine koja sadrži SEQ ID NO: 8, 10, 25, 30, 33, 79 i 80.
5. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat prema bilo kojem od zahtjeva 1 do 4, naznačen time što je oligonukleotid.
6. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat prema zahtjevu 5, naznačen time što je barem jedan nukleotid koji čini oligonukleotid modificiran na šećernom dijelu i / ili na dijelu fosfatne veze.
7. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat prema zahtjevu 5 ili 6, naznačen time što je šećerni dio od najmanje jednog nukleotida koji čini oligonukleotid riboza u kojoj je -OH skupina na položaju 2' supstituirana s bilo kojom skupinom odabranom iz skupine koju čine OR, R, R'OR, SH, SR, NH2, NHR, NR2, N3, CN, F, Cl, Br i I (gdje R predstavlja alkil ili aril, a R' predstavlja alkilen).
8. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat prema zahtjevu 6 ili 7, naznačen time što je dio fosfatne veze od barem jednog nukleotida koji čini oligonukleotid bilo koji odabran iz skupine koja se sastoji od fosforotioatne veze, fosforoditioatne veze, alkilfosfonatne veze, veze fosforoamidata i boranofosfatne veze.
9. Antisens oligomer prema bilo kojem od zahtjeva 1 do 4, naznačen time što je morfolino oligomer, ili njegova farmaceutski prihvatljiva sol ili hidrat.
10. Antisens oligomer prema zahtjevu 4 ili 9, naznačen time što je fosfordiamidat morfolino oligomer, ili njegova farmaceutski prihvatljiva sol ili hidrat.
11. Antisens oligomer prema bilo kojem od zahtjeva 9 do 10, čiji je 5'-terminalni kraj bilo koja od skupina prikazanih kemijskim formulama (1) do (3), prikazanim dolje, ili njegova farmaceutski prihvatljiva sol ili hidrat.
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12. Farmaceutski pripravak za liječenje mišićne distrofije, koji sadrži antisens oligomer ili njegovu farmaceutski prihvatljivu sol ili hidrat prema bilo kojem od zahtjeva 1 do 11 kao aktivni sastojak.
13. Farmaceutski pripravak prema zahtjevu 12, koji nadalje sadrži farmaceutski prihvatljiv nosač.
14. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat prema bilo kojem od zahtjeva 1 do 11 za uporabu u liječenju mišićne distrofije kod bolesnika s mišićnom distrofijom, naznačen time što je pacijent izborno ljudski pacijent.
15. Antisens oligomer ili njegova farmaceutski prihvatljiva sol ili hidrat za uporabu u prema zahtjevu 14, naznačen time što kod liječenja, pacijent s mišićnom distrofijom ima mutaciju koju treba ciljati egzonom 45 preskakanjem gena distrofina, pri čemu je pacijent izborno ljudski pacijent.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2015182145 | 2015-09-15 | ||
| PCT/JP2016/077305 WO2017047707A1 (ja) | 2015-09-15 | 2016-09-15 | アンチセンス核酸 |
| EP16846578.9A EP3351633B1 (en) | 2015-09-15 | 2016-09-15 | Antisense nucleic acid |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20201125T1 true HRP20201125T1 (hr) | 2020-10-30 |
Family
ID=58289325
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20201125TT HRP20201125T1 (hr) | 2015-09-15 | 2020-07-17 | Antisens nukleinska kiselina |
Country Status (28)
| Country | Link |
|---|---|
| US (3) | US10144931B2 (hr) |
| EP (2) | EP3778895A1 (hr) |
| JP (4) | JP6384845B2 (hr) |
| KR (3) | KR20220053048A (hr) |
| CN (3) | CN113913426B (hr) |
| AU (1) | AU2016324800B2 (hr) |
| CA (1) | CA2996280C (hr) |
| CO (1) | CO2018002557A2 (hr) |
| CY (1) | CY1123119T1 (hr) |
| DK (1) | DK3351633T3 (hr) |
| ES (1) | ES2808049T3 (hr) |
| HR (1) | HRP20201125T1 (hr) |
| HU (1) | HUE050061T2 (hr) |
| IL (1) | IL258065B (hr) |
| LT (1) | LT3351633T (hr) |
| MX (1) | MX2018002955A (hr) |
| MY (1) | MY185390A (hr) |
| PH (1) | PH12018500568A1 (hr) |
| PL (1) | PL3351633T3 (hr) |
| PT (1) | PT3351633T (hr) |
| RS (1) | RS60493B1 (hr) |
| RU (1) | RU2724554C2 (hr) |
| SG (1) | SG11201802138TA (hr) |
| SI (1) | SI3351633T1 (hr) |
| TW (1) | TWI725990B (hr) |
| UA (1) | UA123359C2 (hr) |
| WO (1) | WO2017047707A1 (hr) |
| ZA (1) | ZA201801682B (hr) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| UA123359C2 (uk) * | 2015-09-15 | 2021-03-24 | Ніппон Шин'Яку Ко., Лтд. | Антисенсова нуклеїнова кислота |
| AU2016334232B2 (en) | 2015-10-09 | 2022-05-26 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020028864A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| MX2023012559A (es) | 2021-04-30 | 2023-12-15 | Sarepta Therapeutics Inc | Métodos de tratamiento para la distrofia muscular. |
| CA3224782A1 (en) * | 2021-06-23 | 2022-12-29 | Nippon Shinyaku Co., Ltd. | Combination of antisense oligomers |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| CN118488959A (zh) | 2021-12-27 | 2024-08-13 | 日本新药株式会社 | 低聚核酸化合物的制造方法 |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| WO2023178230A1 (en) | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US2567664A (en) | 1948-04-22 | 1951-09-11 | Willis G Ewell | Chicken culling device |
| WO1991009033A1 (en) | 1989-12-20 | 1991-06-27 | Anti-Gene Development Group | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| JP2924179B2 (ja) | 1993-02-19 | 1999-07-26 | 日本新薬株式会社 | グリセロール誘導体，デバイス及び医薬組成物 |
| 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 |
| US6727355B2 (en) * | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition 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 |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| CA2942791C (en) | 2002-11-25 | 2019-08-20 | Masafumi Matsuo | Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors |
| CA2524255C (en) | 2003-03-21 | 2014-02-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
| ATE498685T1 (de) | 2004-06-28 | 2011-03-15 | Univ Western Australia | Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon |
| JPWO2006038608A1 (ja) | 2004-10-05 | 2008-05-15 | 日本新薬株式会社 | オリゴ二本鎖ｒｎａ及び医薬組成物 |
| US20090069260A1 (en) | 2005-05-30 | 2009-03-12 | Nippon Shinyaku Co., Ltd | Method for producing a nucleic-acid-containing complex preparation |
| EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| US8466255B2 (en) | 2007-02-05 | 2013-06-18 | Nippon Shinyaku Co., Ltd. | Polyethylene glycol derivative |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| CN105641700B (zh) * | 2007-10-26 | 2021-01-01 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| WO2009064471A1 (en) | 2007-11-15 | 2009-05-22 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| 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 |
| US8084601B2 (en) * | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| CN102203253B (zh) | 2008-10-24 | 2016-04-06 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| PT2607484E (pt) | 2008-10-27 | 2016-03-09 | Academisch Ziekenhuis Leiden | Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne |
| JP2012524540A (ja) * | 2009-04-24 | 2012-10-18 | プロセンサ テクノロジーズ ビー．ブイ． | Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド |
| ITTO20090487A1 (it) * | 2009-06-26 | 2010-12-27 | Univ Ferrara | Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd) |
| US20120270930A1 (en) * | 2009-10-29 | 2012-10-25 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Methods and compositions for dysferlin exon-skipping |
| CN105838714B (zh) | 2009-11-12 | 2020-07-17 | 西澳大利亚大学 | 反义分子和治疗疾病的方法 |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| EP2672977A1 (en) * | 2011-02-08 | 2013-12-18 | The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System | Antisense oligonucleotides |
| CN117721110A (zh) * | 2011-12-28 | 2024-03-19 | 日本新药株式会社 | 反义核酸 |
| CN112251436A (zh) * | 2012-01-27 | 2021-01-22 | 比奥马林技术公司 | 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 |
| AU2013285698A1 (en) * | 2012-07-03 | 2015-02-19 | Biomarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| CA2906812A1 (en) | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| CN106459955B (zh) | 2014-03-12 | 2019-12-20 | 日本新药株式会社 | 反义核酸 |
| JP2015182145A (ja) | 2014-03-20 | 2015-10-22 | キヤノン株式会社 | ロボットシステムの制御方法、およびロボットシステム |
| ES2765463T3 (es) | 2014-06-17 | 2020-06-09 | Nippon Shinyaku Co Ltd | Acido nucleico antisentido para usar en el tratamiento de la distrofia muscular de Duchenne |
| UA123359C2 (uk) * | 2015-09-15 | 2021-03-24 | Ніппон Шин'Яку Ко., Лтд. | Антисенсова нуклеїнова кислота |
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