CA3062316A1 - Methods for treatment of alport syndrome - Google Patents
Methods for treatment of alport syndrome Download PDFInfo
- Publication number
- CA3062316A1 CA3062316A1 CA3062316A CA3062316A CA3062316A1 CA 3062316 A1 CA3062316 A1 CA 3062316A1 CA 3062316 A CA3062316 A CA 3062316A CA 3062316 A CA3062316 A CA 3062316A CA 3062316 A1 CA3062316 A1 CA 3062316A1
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- Prior art keywords
- subject
- modified oligonucleotide
- nucleosides
- alport syndrome
- certain embodiments
- Prior art date
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- 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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- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
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- A—HUMAN NECESSITIES
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- 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
- 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
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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
- A61K9/0021—Intradermal administration, e.g. through microneedle arrays, needleless injectors
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- 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
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- 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
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- C12N2310/11—Antisense
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- 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
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- C12N2310/10—Type of nucleic acid
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- 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
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
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- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- 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
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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- 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
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- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
Landscapes
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- Chemical & Material Sciences (AREA)
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- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
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- Zoology (AREA)
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- Microbiology (AREA)
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- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Urology & Nephrology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Inorganic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
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| US201762501699P | 2017-05-04 | 2017-05-04 | |
| US62/501,699 | 2017-05-04 | ||
| PCT/US2018/031094 WO2018204788A1 (en) | 2017-05-04 | 2018-05-04 | Methods for treatment of alport syndrome |
Publications (1)
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| CA3062316A1 true CA3062316A1 (en) | 2018-11-08 |
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| US (3) | US20210100828A1 (enExample) |
| EP (1) | EP3618838A1 (enExample) |
| JP (2) | JP2020518633A (enExample) |
| KR (1) | KR20190141770A (enExample) |
| CN (2) | CN116585344A (enExample) |
| AU (1) | AU2018261792A1 (enExample) |
| BR (1) | BR112019022824A2 (enExample) |
| CA (1) | CA3062316A1 (enExample) |
| CL (1) | CL2019003153A1 (enExample) |
| MA (1) | MA50749A (enExample) |
| MX (1) | MX2019013158A (enExample) |
| SG (1) | SG11201910099WA (enExample) |
| WO (1) | WO2018204788A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022076452A1 (en) * | 2020-10-05 | 2022-04-14 | Father Flanagan's Boys' Home Doing Business As Boys Town National Research Hospital | Combination therapy for alport renal disease |
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| CN120731923B (zh) * | 2025-09-05 | 2025-11-04 | 内蒙古医科大学附属医院(内蒙古自治区心血管研究所) | 一种Alport综合征小鼠NMD逃逸模型的构建方法及其应用 |
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| US7524827B2 (en) * | 2004-06-01 | 2009-04-28 | Pronai Therapeutics, Inc. | Methods and compositions for the inhibition of gene expression |
| GB2465902B (en) * | 2008-08-25 | 2010-12-08 | Excaliard Pharmaceuticals Inc | Antisense oligonucleotides directed against connective tissue growth factor and uses thereof |
| DK2825648T3 (en) * | 2012-03-15 | 2018-10-15 | Curna Inc | TREATMENT OF BRAIN-DERIVATIVE NEUROTROPHIC FACTOR (BDNF) -related DISEASES BY INHIBITATION OF NATURAL ANTISENCE TRANSCRIPTION TO BDNF |
| PE20142404A1 (es) * | 2012-04-25 | 2015-02-02 | Regulus Therapeutics Inc | Compuestos de microarn y metodos de modulacion de la actividad de mir-21 |
| UA116639C2 (uk) * | 2012-10-09 | 2018-04-25 | Рег'Юлес Терап'Ютікс Інк. | Способи лікування синдрому альпорта |
| GB201507926D0 (en) * | 2015-05-08 | 2015-06-24 | Proqr Therapeutics N V | Improved treatments using oligonucleotides |
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- 2018-05-04 SG SG11201910099W patent/SG11201910099WA/en unknown
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- 2018-05-04 KR KR1020197035568A patent/KR20190141770A/ko not_active Ceased
- 2018-05-04 CN CN202310591916.8A patent/CN116585344A/zh active Pending
- 2018-05-04 MA MA050749A patent/MA50749A/fr unknown
- 2018-05-04 MX MX2019013158A patent/MX2019013158A/es unknown
- 2018-05-04 CN CN201880037914.1A patent/CN110740737A/zh active Pending
- 2018-05-04 BR BR112019022824A patent/BR112019022824A2/pt not_active IP Right Cessation
- 2018-05-04 AU AU2018261792A patent/AU2018261792A1/en not_active Abandoned
- 2018-05-04 US US16/607,971 patent/US20210100828A1/en not_active Abandoned
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- 2018-05-04 EP EP18726668.9A patent/EP3618838A1/en not_active Withdrawn
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2019
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022076452A1 (en) * | 2020-10-05 | 2022-04-14 | Father Flanagan's Boys' Home Doing Business As Boys Town National Research Hospital | Combination therapy for alport renal disease |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023075111A (ja) | 2023-05-30 |
| WO2018204788A1 (en) | 2018-11-08 |
| CL2019003153A1 (es) | 2020-03-27 |
| CN110740737A (zh) | 2020-01-31 |
| RU2019139261A (ru) | 2021-06-04 |
| KR20190141770A (ko) | 2019-12-24 |
| SG11201910099WA (en) | 2019-11-28 |
| MA50749A (fr) | 2020-03-11 |
| BR112019022824A2 (pt) | 2020-06-09 |
| RU2019139261A3 (enExample) | 2021-09-06 |
| AU2018261792A1 (en) | 2020-01-02 |
| US20210100828A1 (en) | 2021-04-08 |
| EP3618838A1 (en) | 2020-03-11 |
| CN116585344A (zh) | 2023-08-15 |
| US20220133769A1 (en) | 2022-05-05 |
| MX2019013158A (es) | 2020-02-07 |
| US20240115598A1 (en) | 2024-04-11 |
| JP2020518633A (ja) | 2020-06-25 |
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