PT3351633T - Ácido nucleico antissentido - Google Patents
Ácido nucleico antissentidoInfo
- Publication number
- PT3351633T PT3351633T PT168465789T PT16846578T PT3351633T PT 3351633 T PT3351633 T PT 3351633T PT 168465789 T PT168465789 T PT 168465789T PT 16846578 T PT16846578 T PT 16846578T PT 3351633 T PT3351633 T PT 3351633T
- Authority
- PT
- Portugal
- Prior art keywords
- nucleic acid
- antisense nucleic
- antisense
- acid
- nucleic
- Prior art date
Links
- 230000000692 anti-sense effect Effects 0.000 title 1
- 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
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
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
-
- C—CHEMISTRY; METALLURGY
- 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
-
- 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
- C12N2310/313—Phosphorodithioates
-
- 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
-
- 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
- C12N2310/321—2'-O-R Modification
-
- 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
- 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)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (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)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2015182145 | 2015-09-15 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT3351633T true PT3351633T (pt) | 2020-07-29 |
Family
ID=58289325
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT168465789T PT3351633T (pt) | 2015-09-15 | 2016-09-15 | Ácido nucleico antissentido |
Country Status (29)
| Country | Link |
|---|---|
| US (4) | US10144931B2 (enExample) |
| EP (2) | EP3778895A1 (enExample) |
| JP (4) | JP6384845B2 (enExample) |
| KR (3) | KR102473431B1 (enExample) |
| CN (3) | CN113913426B (enExample) |
| AU (1) | AU2016324800B2 (enExample) |
| CA (1) | CA2996280C (enExample) |
| CO (1) | CO2018002557A2 (enExample) |
| CY (1) | CY1123119T1 (enExample) |
| DK (1) | DK3351633T3 (enExample) |
| ES (1) | ES2808049T3 (enExample) |
| HR (1) | HRP20201125T1 (enExample) |
| HU (1) | HUE050061T2 (enExample) |
| IL (1) | IL258065B (enExample) |
| LT (1) | LT3351633T (enExample) |
| MX (1) | MX391304B (enExample) |
| MY (1) | MY185390A (enExample) |
| PH (1) | PH12018500568B1 (enExample) |
| PL (1) | PL3351633T3 (enExample) |
| PT (1) | PT3351633T (enExample) |
| RS (1) | RS60493B1 (enExample) |
| RU (1) | RU2724554C2 (enExample) |
| SG (1) | SG11201802138TA (enExample) |
| SI (1) | SI3351633T1 (enExample) |
| SM (1) | SMT202000379T1 (enExample) |
| TW (1) | TWI725990B (enExample) |
| UA (1) | UA123359C2 (enExample) |
| WO (1) | WO2017047707A1 (enExample) |
| ZA (1) | ZA201801682B (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RS60493B1 (sr) * | 2015-09-15 | 2020-08-31 | Nippon Shinyaku Co Ltd | Antismisaona nukleinska kiselina |
| US20190127733A1 (en) | 2015-10-09 | 2019-05-02 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| WO2020028864A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| 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 |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| EP3959319A4 (en) | 2019-04-25 | 2023-06-07 | Avidity Biosciences, Inc. | NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING |
| KR20240004609A (ko) | 2021-04-30 | 2024-01-11 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근이영양증에 대한 치료 방법 |
| MX2023014417A (es) * | 2021-06-23 | 2023-12-15 | Nippon Shinyaku Co Ltd | Combinacion de oligomeros antisentido. |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CA3226366A1 (en) | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations 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 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| JPWO2023127918A1 (enExample) | 2021-12-27 | 2023-07-06 | ||
| EP4486386A1 (en) | 2022-03-03 | 2025-01-08 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| US20250195667A1 (en) | 2022-03-17 | 2025-06-19 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
| US12071621B2 (en) | 2022-04-05 | 2024-08-27 | Avidity Biosciences, Inc. | Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping |
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 |
| AU655164B2 (en) | 1989-12-20 | 1994-12-08 | Antivirals Inc. | 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 |
| 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 |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| ES2509140T3 (es) | 2002-11-25 | 2014-10-17 | 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 |
| HUE028632T2 (en) | 2004-06-28 | 2016-12-28 | Univ Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| 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 |
| WO2008096690A1 (ja) | 2007-02-05 | 2008-08-14 | Nippon Shinyaku Co., Ltd. | ポリエチレングリコール誘導体 |
| US20100016215A1 (en) * | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| NZ584793A (en) * | 2007-10-26 | 2012-05-25 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| RU2606627C2 (ru) | 2007-11-15 | 2017-01-10 | Серепта Терапьютикс,Инк. | Способ синтеза морфолиновых олигомеров |
| 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 |
| 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 |
| SI2607484T1 (sl) * | 2008-10-27 | 2016-11-30 | Biomarin Technologies B.V. | Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije |
| EP2421971B1 (en) * | 2009-04-24 | 2016-07-06 | BioMarin Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
| 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 |
| NZ626359A (en) | 2009-11-12 | 2016-02-26 | Univ Western Australia | Antisense molecules and methods for treating pathologies |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| BR112013020273A2 (pt) * | 2011-02-08 | 2016-10-18 | Charlotte Mecklenburg Hospital | oligonucleotídeos antissenso |
| CA3132111A1 (en) | 2011-12-28 | 2013-07-04 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acids for skipping of exon 55 of human dystrophin gene |
| EP4043039A1 (en) * | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| EP3608407A1 (en) | 2012-07-03 | 2020-02-12 | BioMarin Technologies B.V. | Oligonucleotide for the treatment of muscular dystrophy patients |
| EP3662912B1 (en) | 2013-03-15 | 2025-12-03 | Sarepta Therapeutics, Inc. | Improved dosages of eteplirsen for treating duchenne muscular dystrophy |
| RS58573B1 (sr) | 2014-03-12 | 2019-05-31 | Nippon Shinyaku Co Ltd | Antisensna nukleinska kiselina |
| 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 |
| PT3159409T (pt) | 2014-06-17 | 2020-01-21 | Nippon Shinyaku Co Ltd | Ácido nucleico anti-sentido para utilização no tratamento da distrofia muscular de duchenne |
| RS60493B1 (sr) * | 2015-09-15 | 2020-08-31 | Nippon Shinyaku Co Ltd | Antismisaona nukleinska kiselina |
-
2016
- 2016-09-15 RS RS20200811A patent/RS60493B1/sr unknown
- 2016-09-15 SG SG11201802138TA patent/SG11201802138TA/en unknown
- 2016-09-15 KR KR1020197010069A patent/KR102473431B1/ko active Active
- 2016-09-15 RU RU2018113276A patent/RU2724554C2/ru active
- 2016-09-15 US US15/759,267 patent/US10144931B2/en active Active
- 2016-09-15 KR KR1020227012681A patent/KR20220053048A/ko not_active Ceased
- 2016-09-15 PT PT168465789T patent/PT3351633T/pt unknown
- 2016-09-15 SM SM20200379T patent/SMT202000379T1/it unknown
- 2016-09-15 EP EP20181465.4A patent/EP3778895A1/en active Pending
- 2016-09-15 HR HRP20201125TT patent/HRP20201125T1/hr unknown
- 2016-09-15 MX MX2018002955A patent/MX391304B/es unknown
- 2016-09-15 SI SI201630848T patent/SI3351633T1/sl unknown
- 2016-09-15 PH PH1/2018/500568A patent/PH12018500568B1/en unknown
- 2016-09-15 AU AU2016324800A patent/AU2016324800B2/en active Active
- 2016-09-15 CN CN202111073029.9A patent/CN113913426B/zh active Active
- 2016-09-15 PL PL16846578T patent/PL3351633T3/pl unknown
- 2016-09-15 EP EP16846578.9A patent/EP3351633B1/en active Active
- 2016-09-15 JP JP2017539975A patent/JP6384845B2/ja active Active
- 2016-09-15 MY MYPI2018000333A patent/MY185390A/en unknown
- 2016-09-15 HU HUE16846578A patent/HUE050061T2/hu unknown
- 2016-09-15 WO PCT/JP2016/077305 patent/WO2017047707A1/ja not_active Ceased
- 2016-09-15 UA UAA201802093A patent/UA123359C2/uk unknown
- 2016-09-15 ES ES16846578T patent/ES2808049T3/es active Active
- 2016-09-15 CA CA2996280A patent/CA2996280C/en active Active
- 2016-09-15 CN CN202111073060.2A patent/CN113930426A/zh active Pending
- 2016-09-15 CN CN201680053726.9A patent/CN108026531B/zh active Active
- 2016-09-15 LT LTEP16846578.9T patent/LT3351633T/lt unknown
- 2016-09-15 KR KR1020187001380A patent/KR101968880B1/ko active Active
- 2016-09-15 DK DK16846578.9T patent/DK3351633T3/da active
- 2016-09-19 TW TW105130156A patent/TWI725990B/zh active
-
2018
- 2018-03-09 CO CONC2018/0002557A patent/CO2018002557A2/es unknown
- 2018-03-12 ZA ZA2018/01682A patent/ZA201801682B/en unknown
- 2018-03-13 IL IL258065A patent/IL258065B/en unknown
- 2018-07-31 JP JP2018144099A patent/JP6977998B2/ja active Active
- 2018-10-16 US US16/161,946 patent/US10851373B2/en active Active
-
2020
- 2020-07-10 CY CY20201100640T patent/CY1123119T1/el unknown
- 2020-11-17 US US16/950,449 patent/US11981894B2/en active Active
-
2021
- 2021-11-02 JP JP2021179353A patent/JP2022033738A/ja not_active Withdrawn
-
2023
- 2023-12-26 JP JP2023218764A patent/JP2024038104A/ja active Pending
-
2024
- 2024-05-13 US US18/662,046 patent/US20240368597A1/en not_active Abandoned
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