ZA201502087B - Methods for treatment of alport syndrome - Google Patents
Methods for treatment of alport syndromeInfo
- Publication number
- ZA201502087B ZA201502087B ZA2015/02087A ZA201502087A ZA201502087B ZA 201502087 B ZA201502087 B ZA 201502087B ZA 2015/02087 A ZA2015/02087 A ZA 2015/02087A ZA 201502087 A ZA201502087 A ZA 201502087A ZA 201502087 B ZA201502087 B ZA 201502087B
- Authority
- ZA
- South Africa
- Prior art keywords
- treatment
- methods
- alport syndrome
- alport
- syndrome
- Prior art date
Links
Classifications
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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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- 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
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
- A61K31/4015—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil having oxo groups directly attached to the heterocyclic ring, e.g. piracetam, ethosuximide
-
- 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
- A61K31/59—Compounds containing 9, 10- seco- cyclopenta[a]hydrophenanthrene ring systems
- A61K31/593—9,10-Secocholestane derivatives, e.g. cholecalciferol, i.e. vitamin D3
-
- 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
-
- 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
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/315—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
- C12N2310/321—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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Urology & Nephrology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Investigating Or Analysing Biological Materials (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261711514P | 2012-10-09 | 2012-10-09 | |
| US201361779137P | 2013-03-13 | 2013-03-13 | |
| PCT/US2013/063884 WO2014058881A1 (en) | 2012-10-09 | 2013-10-08 | Methods for treatment of alport syndrome |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ZA201502087B true ZA201502087B (en) | 2016-06-29 |
Family
ID=49382663
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ZA2015/02087A ZA201502087B (en) | 2012-10-09 | 2015-03-26 | Methods for treatment of alport syndrome |
Country Status (34)
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2005333165B2 (en) | 2004-11-12 | 2012-07-19 | Asuragen, Inc. | Methods and compositions involving miRNA and miRNA inhibitor molecules |
| HRP20170557T2 (hr) | 2011-04-25 | 2020-02-21 | Regulus Therapeutics Inc. | Mikrorna spojevi i postupci za modulaciju aktivnosti mir-21 |
| 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 |
| EP3060664B1 (en) | 2013-10-25 | 2021-07-07 | Sanofi | Microrna compounds and methods for modulating mir-21 activity |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| EP3171889A4 (en) * | 2014-07-25 | 2018-03-14 | Goldfinch Biopharma, Inc. | Collagen iv replacement |
| US10744178B2 (en) | 2014-08-21 | 2020-08-18 | Stealth Biotherapeutics Corp | Methods and compositions for the prevention and treatment of disease |
| KR102620328B1 (ko) | 2014-10-03 | 2024-01-02 | 콜드스프링하버러보러토리 | 핵 유전자 산출량의 표적화 증강 |
| KR20220105174A (ko) | 2015-10-09 | 2022-07-26 | 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 |
| CN109312343B (zh) | 2015-12-14 | 2022-09-27 | 冷泉港实验室 | 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体 |
| CN110198718B (zh) | 2016-11-08 | 2023-01-10 | 里亚塔医药控股有限责任公司 | 使用甲基巴多索隆或其类似物治疗阿尔波特综合征的方法 |
| JP2019535839A (ja) | 2016-11-29 | 2019-12-12 | ピュアテック ヘルス エルエルシー | 治療剤の送達のためのエクソソーム |
| WO2018123925A1 (ja) | 2016-12-28 | 2018-07-05 | 第一三共株式会社 | アルポート症候群治療薬 |
| WO2018204788A1 (en) * | 2017-05-04 | 2018-11-08 | Regulus Therapeutics Inc. | Methods for treatment of alport syndrome |
| WO2018226900A2 (en) | 2017-06-06 | 2018-12-13 | Zymergen Inc. | A htp genomic engineering platform for improving fungal strains |
| EP3485013B1 (en) | 2017-06-06 | 2021-03-24 | Zymergen, Inc. | A htp genomic engineering platform for improving escherichia coli |
| SMT202400071T1 (it) | 2017-08-25 | 2024-03-13 | Stoke Therapeutics Inc | Oligomeri antisenso per il trattamento di condizioni e malattie |
| DK3700570T3 (da) | 2017-10-23 | 2025-03-31 | Stoke Therapeutics Inc | Antisense-oligomerer til behandling af ikke-sense medieret rna henfald baserede tilstande og sygdomme |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| US11066663B2 (en) | 2018-10-31 | 2021-07-20 | Zymergen Inc. | Multiplexed deterministic assembly of DNA libraries |
| JP2022524043A (ja) | 2019-03-08 | 2022-04-27 | ザイマージェン インコーポレイテッド | 微生物の反復ゲノム編集 |
| US11053515B2 (en) | 2019-03-08 | 2021-07-06 | Zymergen Inc. | Pooled genome editing in microbes |
| EP4121088A4 (en) | 2020-03-20 | 2024-07-03 | Keros Therapeutics, Inc. | Methods of using activin receptor type iib variants |
| CA3173647A1 (en) | 2020-05-11 | 2021-11-18 | Isabel AZNAREZ | Opa1 antisense oligomers for treatment of conditions and diseases |
| AU2023267259A1 (en) * | 2022-05-09 | 2024-11-14 | Calliditas Therapeutics Suisse Sa | Nox inhibitors for use in the treatment of alport syndrome |
| WO2024238950A1 (en) * | 2023-05-18 | 2024-11-21 | Keros Therapeutics, Inc. | Methods of using activin receptor type ii variants |
| 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 |
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| KR100574597B1 (ko) * | 1998-05-22 | 2006-04-28 | 보이스 타운 내셔널 리서치 호스피탈 | α1β1 인테그린 수용체 저해제 및 ＴＧＦ-β1 저해제의신장 질환의 치료용 용도 |
| US20060008817A1 (en) | 2000-12-08 | 2006-01-12 | Invitrogen Corporation | Methods and compositions for generating recombinant nucleic acid molecules |
| EP2428568B1 (en) | 2001-09-28 | 2018-04-25 | Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. | Microrna molecules |
| US7585501B2 (en) * | 2002-06-14 | 2009-09-08 | Stowers Institute For Medical Research | Compositions and methods for treating kidney disease |
| US20050124591A1 (en) | 2003-07-29 | 2005-06-09 | Jin Tian | Use of vitamin Ds to treat kidney disease |
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| ITMI20032528A1 (it) * | 2003-12-19 | 2005-06-20 | Francesco Santangelo | Uso di cistina o di cisteina per la prevenzione e il |
| US7585550B2 (en) | 2004-02-02 | 2009-09-08 | College Of William And Mary | Process for modifying polymeric surfaces using deep UV irradiation |
| EP2290071B1 (en) | 2004-05-28 | 2014-12-31 | Asuragen, Inc. | Methods and compositions involving microRNA |
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| AU2005333165B2 (en) | 2004-11-12 | 2012-07-19 | Asuragen, Inc. | Methods and compositions involving miRNA and miRNA inhibitor molecules |
| WO2006069584A2 (en) | 2004-12-29 | 2006-07-06 | Exiqon A/S | NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF microRNAs AND THEIR TARGET mRNAs |
| US6995235B1 (en) | 2005-05-02 | 2006-02-07 | Univation Technologies, Llc | Methods of producing polyolefins and films therefrom |
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| EA201100813A1 (ru) | 2006-04-03 | 2012-06-29 | Сантарис Фарма А/С | Фармацевтическая композиция |
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| US8361980B2 (en) | 2008-03-07 | 2013-01-29 | Santaris Pharma A/S | Pharmaceutical compositions for treatment of microRNA related diseases |
| EP2401405A4 (en) | 2009-02-26 | 2013-10-16 | Univ Ohio State Res Found | MICRO-RNAS FOR NON-SMOKERS AND RELATED MATERIALS AND METHODS |
| EA022757B1 (ru) | 2009-06-08 | 2016-02-29 | Мираджен Терапьютикс | МОТИВЫ ХИМИЧЕСКИХ МОДИФИКАЦИЙ ДЛЯ ИНГИБИТОРОВ И МИМЕТИКОВ мкРНК |
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| HRP20170557T2 (hr) | 2011-04-25 | 2020-02-21 | Regulus Therapeutics Inc. | Mikrorna spojevi i postupci za modulaciju aktivnosti mir-21 |
| WO2013013165A2 (en) | 2011-07-21 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Use of lna compounds for therapeutic inhibition of mir-21 in systemic lupus erythematosus |
| PE20142404A1 (es) | 2012-04-25 | 2015-02-02 | Regulus Therapeutics Inc | Compuestos de microarn y metodos de modulacion de la actividad de mir-21 |
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| WO2014048441A1 (en) | 2012-09-26 | 2014-04-03 | Mirrx Therapeutics | Oligomers with improved off-target profile |
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2013
- 2013-08-10 UA UAA201504453A patent/UA116639C2/uk unknown
- 2013-10-08 ES ES19188663T patent/ES2960488T3/es active Active
- 2013-10-08 EP EP19188663.9A patent/EP3620522B1/en active Active
- 2013-10-08 EP EP13777458.4A patent/EP2906698B1/en active Active
- 2013-10-08 HU HUE13777458A patent/HUE045858T2/hu unknown
- 2013-10-08 DK DK13777458T patent/DK2906698T3/da active
- 2013-10-08 NZ NZ630596A patent/NZ630596A/en not_active IP Right Cessation
- 2013-10-08 RS RS20191380A patent/RS60014B1/sr unknown
- 2013-10-08 UY UY35069A patent/UY35069A/es not_active Application Discontinuation
- 2013-10-08 ES ES13777458T patent/ES2753174T3/es active Active
- 2013-10-08 KR KR1020207028577A patent/KR102349184B1/ko active Active
- 2013-10-08 PT PT137774584T patent/PT2906698T/pt unknown
- 2013-10-08 JP JP2015535891A patent/JP6272880B2/ja active Active
- 2013-10-08 AR ARP130103643A patent/AR092940A1/es not_active Application Discontinuation
- 2013-10-08 LT LT13777458T patent/LT2906698T/lt unknown
- 2013-10-08 KR KR1020157009045A patent/KR102165189B1/ko active Active
- 2013-10-08 SI SI201331597T patent/SI2906698T1/sl unknown
- 2013-10-08 MX MX2015004460A patent/MX356340B/es active IP Right Grant
- 2013-10-08 AU AU2013329427A patent/AU2013329427B2/en not_active Ceased
- 2013-10-08 PL PL13777458T patent/PL2906698T3/pl unknown
- 2013-10-08 KR KR1020227000173A patent/KR20220005634A/ko not_active Ceased
- 2013-10-08 SG SG11201502748RA patent/SG11201502748RA/en unknown
- 2013-10-08 MY MYPI2015000876A patent/MY183921A/en unknown
- 2013-10-08 US US14/048,827 patent/US9012423B2/en active Active
- 2013-10-08 BR BR112015007709-9A patent/BR112015007709B1/pt not_active IP Right Cessation
- 2013-10-08 TW TW102136388A patent/TWI641388B/zh active
- 2013-10-08 KR KR1020237031594A patent/KR20230136686A/ko not_active Withdrawn
- 2013-10-08 EA EA201590711A patent/EA201590711A1/ru unknown
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