CA2995996A1 - Methods for treatment of polycystic kidney disease - Google Patents
Methods for treatment of polycystic kidney disease Download PDFInfo
- Publication number
- CA2995996A1 CA2995996A1 CA2995996A CA2995996A CA2995996A1 CA 2995996 A1 CA2995996 A1 CA 2995996A1 CA 2995996 A CA2995996 A CA 2995996A CA 2995996 A CA2995996 A CA 2995996A CA 2995996 A1 CA2995996 A1 CA 2995996A1
- Authority
- CA
- Canada
- Prior art keywords
- subject
- mir
- certain embodiments
- modified oligonucleotide
- kidney
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
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
-
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/12—Antihypertensives
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
-
- 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
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/322—2'-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
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Urology & Nephrology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562210031P | 2015-08-26 | 2015-08-26 | |
| US62/210,031 | 2015-08-26 | ||
| PCT/US2016/048603 WO2017035319A1 (en) | 2015-08-26 | 2016-08-25 | Methods for treatment of polycystic kidney disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA2995996A1 true CA2995996A1 (en) | 2017-03-02 |
Family
ID=56855838
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2995996A Pending CA2995996A1 (en) | 2015-08-26 | 2016-08-25 | Methods for treatment of polycystic kidney disease |
Country Status (14)
| Country | Link |
|---|---|
| US (3) | US10633657B2 (https=) |
| EP (2) | EP4268891A3 (https=) |
| JP (1) | JP6929269B2 (https=) |
| KR (2) | KR20250049446A (https=) |
| CN (2) | CN108135922A (https=) |
| AU (1) | AU2016312590B2 (https=) |
| CA (1) | CA2995996A1 (https=) |
| DK (1) | DK3340993T5 (https=) |
| ES (1) | ES2954151T3 (https=) |
| IL (1) | IL257596B (https=) |
| MA (1) | MA44836A (https=) |
| MX (1) | MX2018002354A (https=) |
| RU (1) | RU2742300C2 (https=) |
| WO (1) | WO2017035319A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018106568A1 (en) * | 2016-12-05 | 2018-06-14 | Regulus Therapeutics Inc. | Methods for treatment of polycystic kidney disease |
| IL322679A (en) * | 2016-12-05 | 2025-10-01 | Regulus Therapeutics Inc | Modified oligonucleotides for the treatment of polycystic kidney disease |
| EP3841220A1 (en) | 2018-08-23 | 2021-06-30 | Roche Innovation Center Copenhagen A/S | Microrna-134 biomarker |
| CA3234547A1 (en) | 2021-10-08 | 2023-04-13 | Regulus Therapeutics Inc. | Methods and compositions for treatment of polycystic kidney disease |
| WO2024026354A2 (en) * | 2022-07-27 | 2024-02-01 | The General Hospital Corporation | Methods and compositions for use in treating autosomal recessive polycystic kidney disease (arpkd) |
| IL324441A (en) * | 2023-06-16 | 2026-01-01 | Pyc Therapeutics Ltd | Compounds and methods for treating kidney diseases |
| WO2025110217A1 (ja) * | 2023-11-22 | 2025-05-30 | 国立大学法人東海国立大学機構 | 嚢胞性腎疾患の予防及び／又は治療剤 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7683036B2 (en) * | 2003-07-31 | 2010-03-23 | Regulus Therapeutics Inc. | Oligomeric compounds and compositions for use in modulation of small non-coding RNAs |
| NZ549535A (en) | 2004-03-17 | 2010-11-26 | Novartis Ag | Use of aliskiren for treating renal and other disorders |
| JPWO2007126150A1 (ja) * | 2006-04-27 | 2009-09-17 | 国立大学法人名古屋大学 | 癌の新規治療用組成物 |
| US20120283411A9 (en) * | 2006-06-29 | 2012-11-08 | Ironwood Pharmaceuticals, Inc. | Methods and compositions for the treatment of gastrointestinal disorders |
| EP3192788A1 (en) | 2006-10-03 | 2017-07-19 | Arbutus Biopharma Corporation | Lipid containing formulations |
| US20090105174A1 (en) * | 2007-04-20 | 2009-04-23 | Amgen Inc. | Nucleic acids hybridizable to micro rna and precursors thereof |
| WO2009143403A1 (en) * | 2008-05-23 | 2009-11-26 | Targacept, Inc. | Combination therapy for the management hypertension |
| WO2014082644A1 (en) * | 2012-11-30 | 2014-06-05 | WULFF, Peter, Samuel | Circular rna for inhibition of microrna |
| US10660890B2 (en) * | 2013-10-24 | 2020-05-26 | National Institutes Of Health (Nih), U.S. Dept. Of Health And Human Services (Dhhs), U.S. Government Nih Division Of Extramural Inventions And Technology Resources (Deitr) | Treatment of polycystic diseases with an HDAC6 inhibitor |
| EP3105327A4 (en) * | 2014-02-12 | 2017-10-18 | Thomas Jefferson University | Compositions and methods of using microrna inhibitors |
| WO2018106568A1 (en) | 2016-12-05 | 2018-06-14 | Regulus Therapeutics Inc. | Methods for treatment of polycystic kidney disease |
| IL322679A (en) | 2016-12-05 | 2025-10-01 | Regulus Therapeutics Inc | Modified oligonucleotides for the treatment of polycystic kidney disease |
-
2016
- 2016-08-25 DK DK16760627.6T patent/DK3340993T5/da active
- 2016-08-25 KR KR1020257010343A patent/KR20250049446A/ko active Pending
- 2016-08-25 AU AU2016312590A patent/AU2016312590B2/en active Active
- 2016-08-25 RU RU2018108206A patent/RU2742300C2/ru active
- 2016-08-25 MA MA044836A patent/MA44836A/fr unknown
- 2016-08-25 EP EP23179064.3A patent/EP4268891A3/en not_active Withdrawn
- 2016-08-25 JP JP2018510089A patent/JP6929269B2/ja active Active
- 2016-08-25 WO PCT/US2016/048603 patent/WO2017035319A1/en not_active Ceased
- 2016-08-25 KR KR1020187007997A patent/KR20180080181A/ko not_active Ceased
- 2016-08-25 IL IL257596A patent/IL257596B/en unknown
- 2016-08-25 CN CN201680049290.6A patent/CN108135922A/zh active Pending
- 2016-08-25 EP EP16760627.6A patent/EP3340993B1/en active Active
- 2016-08-25 CA CA2995996A patent/CA2995996A1/en active Pending
- 2016-08-25 MX MX2018002354A patent/MX2018002354A/es unknown
- 2016-08-25 CN CN202111177217.6A patent/CN114404440A/zh active Pending
- 2016-08-25 US US15/753,865 patent/US10633657B2/en active Active
- 2016-08-25 ES ES16760627T patent/ES2954151T3/es active Active
-
2020
- 2020-03-19 US US16/823,904 patent/US11168325B2/en active Active
-
2021
- 2021-10-05 US US17/494,234 patent/US20220025372A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP3340993B1 (en) | 2023-06-14 |
| AU2016312590B2 (en) | 2021-12-02 |
| NZ740040A (en) | 2024-07-26 |
| US20200231971A1 (en) | 2020-07-23 |
| HK1256883A1 (en) | 2019-10-04 |
| JP2018528945A (ja) | 2018-10-04 |
| RU2018108206A3 (https=) | 2020-01-14 |
| RU2018108206A (ru) | 2019-09-27 |
| WO2017035319A8 (en) | 2021-03-11 |
| US10633657B2 (en) | 2020-04-28 |
| ES2954151T3 (es) | 2023-11-20 |
| DK3340993T5 (da) | 2024-10-07 |
| WO2017035319A1 (en) | 2017-03-02 |
| DK3340993T3 (da) | 2023-08-21 |
| EP3340993A1 (en) | 2018-07-04 |
| CN108135922A (zh) | 2018-06-08 |
| AU2016312590A1 (en) | 2018-03-15 |
| RU2742300C2 (ru) | 2021-02-04 |
| MX2018002354A (es) | 2018-12-10 |
| EP4268891A2 (en) | 2023-11-01 |
| EP4268891A3 (en) | 2024-01-24 |
| KR20250049446A (ko) | 2025-04-11 |
| IL257596B (en) | 2022-08-01 |
| US11168325B2 (en) | 2021-11-09 |
| KR20180080181A (ko) | 2018-07-11 |
| JP6929269B2 (ja) | 2021-09-01 |
| CN114404440A (zh) | 2022-04-29 |
| US20190153442A1 (en) | 2019-05-23 |
| IL257596A (en) | 2018-04-30 |
| US20220025372A1 (en) | 2022-01-27 |
| MA44836A (fr) | 2018-07-04 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20210817 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 8TH ANNIV.) - STANDARD Year of fee payment: 8 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20240719 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT Effective date: 20240719 Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20240719 |
|
| D15 | Examination report completed |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT Effective date: 20241224 |
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| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION Effective date: 20250423 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20250424 |
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| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT Effective date: 20250721 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED Effective date: 20250721 |