AU2014369850B2 - Methods and compositions for the specific inhibition of Glycolate Oxidase (HAO1) by double-stranded RNA - Google Patents
Methods and compositions for the specific inhibition of Glycolate Oxidase (HAO1) by double-stranded RNA Download PDFInfo
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- AU2014369850B2 AU2014369850B2 AU2014369850A AU2014369850A AU2014369850B2 AU 2014369850 B2 AU2014369850 B2 AU 2014369850B2 AU 2014369850 A AU2014369850 A AU 2014369850A AU 2014369850 A AU2014369850 A AU 2014369850A AU 2014369850 B2 AU2014369850 B2 AU 2014369850B2
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- Australia
- Prior art keywords
- strand
- rna
- nucleotides
- methyl rna
- methyl
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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
- C12N15/1137—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 enzymes
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- 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
- A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0006—Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
- C12Y101/03—Oxidoreductases acting on the CH-OH group of donors (1.1) with a oxygen as acceptor (1.1.3)
- C12Y101/03015—(S)-2-Hydroxy-acid oxidase (1.1.3.15)
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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
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Urology & Nephrology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2021201953A AU2021201953A1 (en) | 2013-12-27 | 2021-03-29 | Methods And Compositions For The Specific Inhibition Of Glycolate Oxidase (HAO1) By Double-Stranded RNA |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361921181P | 2013-12-27 | 2013-12-27 | |
| US61/921,181 | 2013-12-27 | ||
| US201461937838P | 2014-02-10 | 2014-02-10 | |
| US61/937,838 | 2014-02-10 | ||
| PCT/US2014/072410 WO2015100436A1 (en) | 2013-12-27 | 2014-12-26 | Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2021201953A Division AU2021201953A1 (en) | 2013-12-27 | 2021-03-29 | Methods And Compositions For The Specific Inhibition Of Glycolate Oxidase (HAO1) By Double-Stranded RNA |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2014369850A1 AU2014369850A1 (en) | 2016-07-14 |
| AU2014369850B2 true AU2014369850B2 (en) | 2021-04-08 |
Family
ID=53479713
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2014369850A Active AU2014369850B2 (en) | 2013-12-27 | 2014-12-26 | Methods and compositions for the specific inhibition of Glycolate Oxidase (HAO1) by double-stranded RNA |
| AU2021201953A Abandoned AU2021201953A1 (en) | 2013-12-27 | 2021-03-29 | Methods And Compositions For The Specific Inhibition Of Glycolate Oxidase (HAO1) By Double-Stranded RNA |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2021201953A Abandoned AU2021201953A1 (en) | 2013-12-27 | 2021-03-29 | Methods And Compositions For The Specific Inhibition Of Glycolate Oxidase (HAO1) By Double-Stranded RNA |
Country Status (21)
| Country | Link |
|---|---|
| US (7) | US9701966B2 (OSRAM) |
| EP (3) | EP3087184B1 (OSRAM) |
| JP (2) | JP6886818B2 (OSRAM) |
| AU (2) | AU2014369850B2 (OSRAM) |
| CA (1) | CA2935220A1 (OSRAM) |
| CY (2) | CY1124203T1 (OSRAM) |
| DK (2) | DK3087184T3 (OSRAM) |
| ES (2) | ES2749855T3 (OSRAM) |
| FI (1) | FIC20210025I1 (OSRAM) |
| FR (1) | FR21C1044I2 (OSRAM) |
| HR (1) | HRP20210612T1 (OSRAM) |
| HU (2) | HUE055470T2 (OSRAM) |
| LT (2) | LT3581654T (OSRAM) |
| LU (1) | LUC00218I2 (OSRAM) |
| NO (1) | NO2021038I1 (OSRAM) |
| PL (1) | PL3581654T3 (OSRAM) |
| PT (1) | PT3581654T (OSRAM) |
| RS (1) | RS61892B1 (OSRAM) |
| SI (1) | SI3581654T1 (OSRAM) |
| SM (1) | SMT202100325T1 (OSRAM) |
| WO (1) | WO2015100436A1 (OSRAM) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9879266B2 (en) * | 2002-11-14 | 2018-01-30 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
| US9228186B2 (en) | 2002-11-14 | 2016-01-05 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
| ES2749855T3 (es) | 2013-12-27 | 2020-03-24 | Dicerna Pharmaceuticals Inc | Procedimientos y composiciones para la inhibición específica de glicolato oxidasa (hao1) por ARN bicatenario |
| JOP20200115A1 (ar) * | 2014-10-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) |
| WO2016205323A1 (en) * | 2015-06-18 | 2016-12-22 | Alnylam Pharmaceuticals, Inc. | Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof |
| RU2769831C2 (ru) * | 2016-10-05 | 2022-04-06 | Зингента Партисипейшнс Аг | Способы сохранения биологической активности рибонуклеиновых кислот |
| CN110177544A (zh) | 2016-11-29 | 2019-08-27 | 普尔泰克健康有限公司 | 用于递送治疗剂的外泌体 |
| AU2018301829B2 (en) | 2017-07-13 | 2024-08-01 | Alnylam Pharmaceuticals, Inc. | Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase) gene expression |
| JP2021531804A (ja) * | 2018-07-31 | 2021-11-25 | インテリア セラピューティクス，インコーポレイテッド | 原発性高シュウ酸尿症１型（ｐｈ１）を治療するためのヒドロキシ酸オキシダーゼ１（ｈａｏ１）遺伝子編集のための組成物および方法 |
| WO2020132659A1 (en) * | 2018-12-21 | 2020-06-25 | Precision Biosciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| KR20220097442A (ko) | 2019-11-01 | 2022-07-07 | 기안알엑스 사이언스 인코포레이티드 | 글리콜레이트 산화효소 억제제로서의 헤테로사이클릭 카르복실레이트 화합물 |
| US20230242918A1 (en) * | 2020-05-22 | 2023-08-03 | President And Fellows Of Harvard College | Interferon- inducing oligonucleotide duplexes and methods of use |
| EP4244342A1 (en) | 2020-11-12 | 2023-09-20 | Precision BioSciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| EP4043018A1 (en) | 2021-02-10 | 2022-08-17 | Charité - Universitätsmedizin Berlin | Composition and method for reducing oxalate levels in patients receiving maintenance dialysis |
| PH12023553297A1 (en) | 2021-06-04 | 2024-03-18 | Arbor Biotechnologies Inc | Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof |
| WO2023003805A1 (en) * | 2021-07-19 | 2023-01-26 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder |
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| US513207A (en) | 1894-01-23 | Rail-brake | ||
| US5432272A (en) | 1990-10-09 | 1995-07-11 | Benner; Steven A. | Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases |
| PL169576B1 (pl) | 1990-10-12 | 1996-08-30 | Max Planck Gesellschaft | Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| US6469158B1 (en) | 1992-05-14 | 2002-10-22 | Ribozyme Pharmaceuticals, Incorporated | Synthesis, deprotection, analysis and purification of RNA and ribozymes |
| US5977343A (en) | 1992-05-14 | 1999-11-02 | Ribozyme Pharmaceuticals, Inc. | Synthesis, deprotection, analysis and purification of RNA and ribozymes |
| US5804683A (en) | 1992-05-14 | 1998-09-08 | Ribozyme Pharmaceuticals, Inc. | Deprotection of RNA with alkylamine |
| DE69431669T2 (de) | 1993-09-02 | 2003-10-23 | Ribozyme Pharmaceuticals, Inc. | Enzymatische nukleiksaüre die nicht-nukleotide enthaltet |
| US5889136A (en) | 1995-06-09 | 1999-03-30 | The Regents Of The University Of Colorado | Orthoester protecting groups in RNA synthesis |
| US5998203A (en) | 1996-04-16 | 1999-12-07 | Ribozyme Pharmaceuticals, Inc. | Enzymatic nucleic acids containing 5'-and/or 3'-cap structures |
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| US20070265220A1 (en) | 2004-03-15 | 2007-11-15 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
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| EP1752536A4 (en) | 2004-05-11 | 2008-04-16 | Alphagen Co Ltd | POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME |
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| JP4644619B2 (ja) | 2006-03-27 | 2011-03-02 | 富士通株式会社 | 基地局装置、端末および帯域制御方法 |
| DE102007021853A1 (de) | 2007-05-10 | 2008-11-13 | Norma Germany Gmbh | Steckkupplung und Stutzen für eine Steckkupplung |
| US20110288147A1 (en) | 2008-09-22 | 2011-11-24 | Bob Dale Brown | Compositions and methods for the specific inhibition of gene expression by DSRNA containing a tetraloop |
| CN101752443B (zh) | 2008-12-08 | 2012-06-20 | 鸿富锦精密工业（深圳）有限公司 | 光伏电池 |
| US8513207B2 (en) | 2008-12-18 | 2013-08-20 | Dicerna Pharmaceuticals, Inc. | Extended dicer substrate agents and methods for the specific inhibition of gene expression |
| WO2010093788A2 (en) | 2009-02-11 | 2010-08-19 | Dicerna Pharmaceuticals, Inc. | Multiplex dicer substrate rna interference molecules having joining sequences |
| EP2756845B1 (en) | 2009-04-03 | 2017-03-15 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA |
| US8632982B2 (en) * | 2009-05-21 | 2014-01-21 | Institute For Systems Biology | Biomarkers for liver injury |
| PE20142362A1 (es) | 2011-11-18 | 2015-01-30 | Alnylam Pharmaceuticals Inc | Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr) |
| US9133461B2 (en) * | 2012-04-10 | 2015-09-15 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the ALAS1 gene |
| ES2749855T3 (es) | 2013-12-27 | 2020-03-24 | Dicerna Pharmaceuticals Inc | Procedimientos y composiciones para la inhibición específica de glicolato oxidasa (hao1) por ARN bicatenario |
| JOP20200115A1 (ar) * | 2014-10-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) |
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2014
- 2014-12-26 ES ES14874374T patent/ES2749855T3/es active Active
- 2014-12-26 ES ES19182616T patent/ES2875558T3/es active Active
- 2014-12-26 EP EP14874374.3A patent/EP3087184B1/en active Active
- 2014-12-26 EP EP19182616.3A patent/EP3581654B1/en active Active
- 2014-12-26 PL PL19182616T patent/PL3581654T3/pl unknown
- 2014-12-26 LT LTEP19182616.3T patent/LT3581654T/lt unknown
- 2014-12-26 JP JP2016543143A patent/JP6886818B2/ja active Active
- 2014-12-26 RS RS20210652A patent/RS61892B1/sr unknown
- 2014-12-26 SI SI201431812T patent/SI3581654T1/sl unknown
- 2014-12-26 US US14/583,200 patent/US9701966B2/en active Active
- 2014-12-26 WO PCT/US2014/072410 patent/WO2015100436A1/en not_active Ceased
- 2014-12-26 SM SM20210325T patent/SMT202100325T1/it unknown
- 2014-12-26 DK DK14874374.3T patent/DK3087184T3/da active
- 2014-12-26 DK DK19182616.3T patent/DK3581654T3/da active
- 2014-12-26 PT PT191826163T patent/PT3581654T/pt unknown
- 2014-12-26 AU AU2014369850A patent/AU2014369850B2/en active Active
- 2014-12-26 EP EP21163279.9A patent/EP3892727A1/en not_active Withdrawn
- 2014-12-26 HU HUE19182616A patent/HUE055470T2/hu unknown
- 2014-12-26 CA CA2935220A patent/CA2935220A1/en active Pending
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2017
- 2017-06-07 US US15/616,254 patent/US9828606B2/en active Active
- 2017-10-25 US US15/793,441 patent/US10435692B2/en active Active
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2019
- 2019-03-08 US US16/297,354 patent/US10487330B2/en active Active
- 2019-03-08 US US16/297,316 patent/US10465195B2/en active Active
- 2019-08-20 US US16/545,149 patent/US11060093B2/en active Active
- 2019-11-08 JP JP2019202937A patent/JP7109416B2/ja active Active
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2021
- 2021-01-25 US US17/157,803 patent/US11873493B2/en active Active
- 2021-03-29 AU AU2021201953A patent/AU2021201953A1/en not_active Abandoned
- 2021-04-16 HR HRP20210612TT patent/HRP20210612T1/hr unknown
- 2021-05-28 CY CY20211100465T patent/CY1124203T1/el unknown
- 2021-08-18 LU LU00218C patent/LUC00218I2/fr unknown
- 2021-08-25 FI FIC20210025C patent/FIC20210025I1/fi unknown
- 2021-08-26 CY CY2021022C patent/CY2021022I2/el unknown
- 2021-09-01 LT LTPA2021008C patent/LTC3581654I2/lt unknown
- 2021-09-16 NO NO2021038C patent/NO2021038I1/no unknown
- 2021-09-20 FR FR21C1044C patent/FR21C1044I2/fr active Active
- 2021-09-28 HU HUS2100041C patent/HUS2100041I1/hu unknown
Non-Patent Citations (1)
| Title |
|---|
| Cochat et al,, N. E. J. M., vol. 369, No. 7, pp. 649-658 (Aug. 2013) (Year: 2013). * |
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| FGA | Letters patent sealed or granted (standard patent) | ||
| PC | Assignment registered |
Owner name: NOVO NORDISK A/S Free format text: FORMER OWNER(S): DICERNA PHARMACEUTICALS, INC. |