JP6827148B2 - 状態および疾患の処置のためのアンチセンスオリゴマー - Google Patents
状態および疾患の処置のためのアンチセンスオリゴマー Download PDFInfo
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- JP6827148B2 JP6827148B2 JP2020511436A JP2020511436A JP6827148B2 JP 6827148 B2 JP6827148 B2 JP 6827148B2 JP 2020511436 A JP2020511436 A JP 2020511436A JP 2020511436 A JP2020511436 A JP 2020511436A JP 6827148 B2 JP6827148 B2 JP 6827148B2
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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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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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
- 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
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
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- A—HUMAN NECESSITIES
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- 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
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- 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
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
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- 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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/06—Antimigraine agents
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- 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
- A61P25/08—Antiepileptics; Anticonvulsants
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- 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
- A61P25/08—Antiepileptics; Anticonvulsants
- A61P25/12—Antiepileptics; Anticonvulsants for grand-mal
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- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- 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
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- 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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- 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
- C12N15/1138—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 against receptors or cell surface proteins
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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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- C12N2310/11—Antisense
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- C12N2310/32—Chemical structure of the sugar
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- C12N2320/33—Alteration of splicing
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- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
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- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
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- General Health & Medical Sciences (AREA)
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- Organic Chemistry (AREA)
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- Molecular Biology (AREA)
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- Animal Behavior & Ethology (AREA)
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- Pharmacology & Pharmacy (AREA)
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- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
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- Biophysics (AREA)
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- Plant Pathology (AREA)
- Pain & Pain Management (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
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Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020136962A JP7420679B2 (ja) | 2017-08-25 | 2020-08-14 | 状態および疾患の処置のためのアンチセンスオリゴマー |
| JP2024002090A JP2024045199A (ja) | 2017-08-25 | 2024-01-10 | 状態および疾患の処置のためのアンチセンスオリゴマー |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762550462P | 2017-08-25 | 2017-08-25 | |
| US62/550,462 | 2017-08-25 | ||
| US201762575901P | 2017-10-23 | 2017-10-23 | |
| US62/575,901 | 2017-10-23 | ||
| US201862667356P | 2018-05-04 | 2018-05-04 | |
| US62/667,356 | 2018-05-04 | ||
| US201862671745P | 2018-05-15 | 2018-05-15 | |
| US62/671,745 | 2018-05-15 | ||
| PCT/US2018/048031 WO2019040923A1 (en) | 2017-08-25 | 2018-08-24 | ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASE CONDITIONS AND OTHER DISEASES |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020136962A Division JP7420679B2 (ja) | 2017-08-25 | 2020-08-14 | 状態および疾患の処置のためのアンチセンスオリゴマー |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020534802A JP2020534802A (ja) | 2020-12-03 |
| JP6827148B2 true JP6827148B2 (ja) | 2021-02-10 |
Family
ID=65439262
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020511436A Active JP6827148B2 (ja) | 2017-08-25 | 2018-08-24 | 状態および疾患の処置のためのアンチセンスオリゴマー |
| JP2020136962A Active JP7420679B2 (ja) | 2017-08-25 | 2020-08-14 | 状態および疾患の処置のためのアンチセンスオリゴマー |
| JP2024002090A Pending JP2024045199A (ja) | 2017-08-25 | 2024-01-10 | 状態および疾患の処置のためのアンチセンスオリゴマー |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020136962A Active JP7420679B2 (ja) | 2017-08-25 | 2020-08-14 | 状態および疾患の処置のためのアンチセンスオリゴマー |
| JP2024002090A Pending JP2024045199A (ja) | 2017-08-25 | 2024-01-10 | 状態および疾患の処置のためのアンチセンスオリゴマー |
Country Status (23)
| Country | Link |
|---|---|
| US (4) | US10683503B2 (enExample) |
| EP (2) | EP3673080B1 (enExample) |
| JP (3) | JP6827148B2 (enExample) |
| KR (3) | KR20240035631A (enExample) |
| CN (2) | CN111278991B (enExample) |
| AU (3) | AU2018322319B2 (enExample) |
| CA (1) | CA3073515A1 (enExample) |
| DK (1) | DK3673080T3 (enExample) |
| ES (1) | ES2965696T3 (enExample) |
| FI (1) | FI3673080T3 (enExample) |
| GB (3) | GB2582457B (enExample) |
| HR (1) | HRP20231637T1 (enExample) |
| HU (1) | HUE064635T2 (enExample) |
| IL (2) | IL321831A (enExample) |
| LT (1) | LT3673080T (enExample) |
| MX (2) | MX380890B (enExample) |
| PL (1) | PL3673080T3 (enExample) |
| PT (1) | PT3673080T (enExample) |
| RS (1) | RS65031B1 (enExample) |
| SG (2) | SG11202001590RA (enExample) |
| SI (1) | SI3673080T1 (enExample) |
| SM (1) | SMT202400071T1 (enExample) |
| WO (1) | WO2019040923A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022522205A (ja) * | 2019-02-27 | 2022-04-14 | ストーク セラピューティクス,インク. | 病態及び疾患の治療用アンチセンスオリゴマー |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201408623D0 (en) | 2014-05-15 | 2014-07-02 | Santaris Pharma As | Oligomers and oligomer conjugates |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3201339A4 (en) | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| KR102422625B1 (ko) | 2015-10-09 | 2022-07-20 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| SG11202001590RA (en) | 2017-08-25 | 2020-03-30 | Stoke Therapeutics Inc | Antisense oligomers for treatment of conditions and diseases |
| US11713463B2 (en) | 2018-01-17 | 2023-08-01 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for increasing expression of SCN2A |
| EP3788169A4 (en) | 2018-05-04 | 2022-08-10 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| EA202190581A1 (ru) | 2018-08-20 | 2021-07-13 | Рогкон, Инк. | Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a |
| US10851376B2 (en) * | 2018-12-28 | 2020-12-01 | The Florida International University Board Of Trustees | Long noncoding RNAs in pulmonary airway inflammation |
| US20220090087A1 (en) * | 2019-01-23 | 2022-03-24 | The Florey Institute Of Neuroscience And Mental Health | Antisense oligonucleotides targeting scn2a retained introns |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| BR112022002905A2 (pt) * | 2019-08-19 | 2022-07-12 | Stoke Therapeutics Inc | Composições e métodos para modular splicing e expressão de proteína |
| JP7560544B2 (ja) * | 2019-09-19 | 2024-10-02 | アルネー サイエンシズ、エルエルシー | 遺伝子スプライシングを調節するために有用な化合物および方法 |
| CA3159162A1 (en) * | 2019-12-06 | 2021-06-10 | Isabel AZNAREZ | Antisense oligomers for treatment of conditions and diseases |
| JP2023515974A (ja) * | 2020-02-28 | 2023-04-17 | アイオーニス ファーマシューティカルズ, インコーポレーテッド | Scn1a発現を調節するための化合物及び方法 |
| MX2022014151A (es) | 2020-05-11 | 2022-11-30 | Stoke Therapeutics Inc | Oligomeros antisentido de opa1 para tratamiento de afecciones y enfermedades. |
| CA3204779A1 (en) * | 2021-01-26 | 2022-08-04 | Brage Storstein Andresen | Splice switching oligonucleotides targeting pseudoexons |
| BR112023022439A2 (pt) | 2021-04-26 | 2023-12-26 | Celanese Eva Performance Polymers Llc | Dispositivo implantável para liberação sustentada de um composto de fármaco macromolecular |
| MX2024002553A (es) * | 2021-08-27 | 2024-03-13 | Ionis Pharmaceuticals Inc | Compuestos y metodos para modular la expresion de scn1a. |
| CA3232214A1 (en) * | 2021-09-21 | 2023-03-30 | Jeffery COLLER | Mrna regulon therapy for the treatment of haploinsufficiency disorders |
| US20250101419A1 (en) * | 2022-01-26 | 2025-03-27 | Syddansk Universitet | Allele specific splice switching oligonucleotides targeting pseudoexons |
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Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
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| JP2022522205A (ja) * | 2019-02-27 | 2022-04-14 | ストーク セラピューティクス,インク. | 病態及び疾患の治療用アンチセンスオリゴマー |
| JP7660515B2 (ja) | 2019-02-27 | 2025-04-11 | ストーク セラピューティクス,インク. | 病態及び疾患の治療用アンチセンスオリゴマー |
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