HRP20210612T1 - Postupci i pripravci za specifičnu inhibiciju glikolat oksidaze (hao1) pomoću dvolančane rnk - Google Patents
Postupci i pripravci za specifičnu inhibiciju glikolat oksidaze (hao1) pomoću dvolančane rnk Download PDFInfo
- Publication number
- HRP20210612T1 HRP20210612T1 HRP20210612TT HRP20210612T HRP20210612T1 HR P20210612 T1 HRP20210612 T1 HR P20210612T1 HR P20210612T T HRP20210612T T HR P20210612TT HR P20210612 T HRP20210612 T HR P20210612T HR P20210612 T1 HRP20210612 T1 HR P20210612T1
- Authority
- HR
- Croatia
- Prior art keywords
- dsna
- chain
- nucleotides
- hao1
- nucleotide
- Prior art date
Links
Classifications
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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)
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- 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)
-
- 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]
Landscapes
- 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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Animal Behavior & Ethology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- Veterinary Medicine (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)
- Medicinal Preparation (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361921181P | 2013-12-27 | 2013-12-27 | |
| US201461937838P | 2014-02-10 | 2014-02-10 | |
| EP19182616.3A EP3581654B1 (en) | 2013-12-27 | 2014-12-26 | Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20210612T1 true HRP20210612T1 (hr) | 2021-08-20 |
Family
ID=53479713
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20210612TT HRP20210612T1 (hr) | 2013-12-27 | 2021-04-16 | Postupci i pripravci za specifičnu inhibiciju glikolat oksidaze (hao1) pomoću dvolančane rnk |
Country Status (21)
| Country | Link |
|---|---|
| US (7) | US9701966B2 (2) |
| EP (3) | EP3581654B1 (2) |
| JP (2) | JP6886818B2 (2) |
| AU (2) | AU2014369850B2 (2) |
| CA (1) | CA2935220A1 (2) |
| CY (2) | CY1124203T1 (2) |
| DK (2) | DK3087184T3 (2) |
| ES (2) | ES2749855T3 (2) |
| FI (1) | FIC20210025I1 (2) |
| FR (1) | FR21C1044I2 (2) |
| HR (1) | HRP20210612T1 (2) |
| HU (2) | HUE055470T2 (2) |
| LT (2) | LT3581654T (2) |
| LU (1) | LUC00218I2 (2) |
| NO (1) | NO2021038I1 (2) |
| PL (1) | PL3581654T3 (2) |
| PT (1) | PT3581654T (2) |
| RS (1) | RS61892B1 (2) |
| SI (1) | SI3581654T1 (2) |
| SM (1) | SMT202100325T1 (2) |
| WO (1) | WO2015100436A1 (2) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9228186B2 (en) | 2002-11-14 | 2016-01-05 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
| US9879266B2 (en) | 2002-11-14 | 2018-01-30 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
| EP3581654B1 (en) | 2013-12-27 | 2021-03-31 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna |
| TW202503057A (zh) * | 2014-10-10 | 2025-01-16 | 美商艾爾妮蘭製藥公司 | 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法 |
| EP3310918B1 (en) * | 2015-06-18 | 2020-08-05 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof |
| WO2018065303A1 (en) * | 2016-10-05 | 2018-04-12 | Syngenta Participations Ag | Methods of preserving the biological activity of ribonucleic acids |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| CA3069451A1 (en) | 2017-07-13 | 2019-01-17 | Alnylam Pharmaceuticals, Inc. | Methods for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression |
| MX2021001070A (es) * | 2018-07-31 | 2021-05-27 | Intellia Therapeutics Inc | Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1). |
| WO2020132659A1 (en) * | 2018-12-21 | 2020-06-25 | Precision Biosciences, Inc. | Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria |
| AU2020376792B2 (en) | 2019-11-01 | 2025-10-02 | Lilac Therapeutics, Inc. | Heterocyclic carboxylate compounds as glycolate oxidase inhibitors |
| US20230242918A1 (en) * | 2020-05-22 | 2023-08-03 | President And Fellows Of Harvard College | Interferon- inducing oligonucleotide duplexes and methods of use |
| JP7642809B2 (ja) | 2020-11-12 | 2025-03-10 | プレシジョン バイオサイエンシズ，インク． | ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ |
| EP4043018A1 (en) | 2021-02-10 | 2022-08-17 | Charité - Universitätsmedizin Berlin | Composition and method for reducing oxalate levels in patients receiving maintenance dialysis |
| IL308803A (en) * | 2021-06-04 | 2024-01-01 | Arbor Biotechnologies Inc | Gene editing systems comprising guide RNA targeting hydroxy acid 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 |
| CN117041337A (zh) * | 2023-08-14 | 2023-11-10 | 中国电信股份有限公司技术创新中心 | 网络流量的处理方法、系统以及risc装置 |
| EP4725949A1 (en) * | 2024-10-08 | 2026-04-15 | BioSpring Gesellschaft für Biotechnologie mbH | Improved solid-phase phosphoramidite-synthesis of long oligonucleotide molecules |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| ES2061416T3 (es) | 1990-10-12 | 1997-03-01 | Max Planck Gesellschaft | Ribozimas modificadas. |
| 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 |
| ATE227342T1 (de) | 1993-09-02 | 2002-11-15 | Ribozyme Pharm 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 |
| US6218108B1 (en) | 1997-05-16 | 2001-04-17 | Research Corporation Technologies, Inc. | Nucleoside analogs with polycyclic aromatic groups attached, methods of synthesis and uses therefor |
| US6248878B1 (en) | 1996-12-24 | 2001-06-19 | Ribozyme Pharmaceuticals, Inc. | Nucleoside analogs |
| US6111086A (en) | 1998-02-27 | 2000-08-29 | Scaringe; Stephen A. | Orthoester protecting groups |
| US7678897B2 (en) * | 2002-02-20 | 2010-03-16 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of platelet-derived endothelial cell growth factor (ECGF1) gene expression using short interfering nucleic acid (siNA) |
| WO2006006948A2 (en) | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
| US9879266B2 (en) * | 2002-11-14 | 2018-01-30 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
| AU2003295600A1 (en) | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| CA2559955C (en) | 2004-03-15 | 2016-02-16 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded rna |
| 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 |
| WO2005116204A1 (ja) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
| US20080213891A1 (en) | 2004-07-21 | 2008-09-04 | Alnylam Pharmaceuticals, Inc. | RNAi Agents Comprising Universal Nucleobases |
| WO2007030167A1 (en) | 2005-09-02 | 2007-03-15 | Nastech Pharmaceutical Company Inc. | Modification of double-stranded ribonucleic acid molecules |
| US8178503B2 (en) * | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| 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 |
| US20100249214A1 (en) | 2009-02-11 | 2010-09-30 | Dicerna Pharmaceuticals | Multiplex dicer substrate rna interference molecules having joining sequences |
| KR101728655B1 (ko) | 2008-12-18 | 2017-04-19 | 다이서나 파마수이티컬, 인크. | 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법 |
| 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 |
| EP3730618A1 (en) | 2011-11-18 | 2020-10-28 | Alnylam Pharmaceuticals, Inc. | Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases |
| US9133461B2 (en) * | 2012-04-10 | 2015-09-15 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the ALAS1 gene |
| EP3581654B1 (en) * | 2013-12-27 | 2021-03-31 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna |
| TW202503057A (zh) * | 2014-10-10 | 2025-01-16 | 美商艾爾妮蘭製藥公司 | 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法 |
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2014
- 2014-12-26 EP EP19182616.3A patent/EP3581654B1/en active Active
- 2014-12-26 RS RS20210652A patent/RS61892B1/sr unknown
- 2014-12-26 DK DK14874374.3T patent/DK3087184T3/da active
- 2014-12-26 CA CA2935220A patent/CA2935220A1/en active Pending
- 2014-12-26 US US14/583,200 patent/US9701966B2/en active Active
- 2014-12-26 PL PL19182616T patent/PL3581654T3/pl unknown
- 2014-12-26 EP EP21163279.9A patent/EP3892727A1/en not_active Withdrawn
- 2014-12-26 HU HUE19182616A patent/HUE055470T2/hu unknown
- 2014-12-26 SM SM20210325T patent/SMT202100325T1/it unknown
- 2014-12-26 LT LTEP19182616.3T patent/LT3581654T/lt unknown
- 2014-12-26 EP EP14874374.3A patent/EP3087184B1/en active Active
- 2014-12-26 AU AU2014369850A patent/AU2014369850B2/en active Active
- 2014-12-26 ES ES14874374T patent/ES2749855T3/es active Active
- 2014-12-26 PT PT191826163T patent/PT3581654T/pt unknown
- 2014-12-26 ES ES19182616T patent/ES2875558T3/es active Active
- 2014-12-26 DK DK19182616.3T patent/DK3581654T3/da active
- 2014-12-26 JP JP2016543143A patent/JP6886818B2/ja active Active
- 2014-12-26 WO PCT/US2014/072410 patent/WO2015100436A1/en not_active Ceased
- 2014-12-26 SI SI201431812T patent/SI3581654T1/sl unknown
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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
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