CY1124203T1 - Μεθοδοι και συνθεσεις για την ειδικη αναστολη της οξειδασης του γλυκολικου (ηαο1) απο το δικλωνο rna - Google Patents

Μεθοδοι και συνθεσεις για την ειδικη αναστολη της οξειδασης του γλυκολικου (ηαο1) απο το δικλωνο rna

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Publication number
CY1124203T1
CY1124203T1 CY20211100465T CY211100465T CY1124203T1 CY 1124203 T1 CY1124203 T1 CY 1124203T1 CY 20211100465 T CY20211100465 T CY 20211100465T CY 211100465 T CY211100465 T CY 211100465T CY 1124203 T1 CY1124203 T1 CY 1124203T1
Authority
CY
Cyprus
Prior art keywords
hao1
compositions
methods
double
stranded rna
Prior art date
Application number
CY20211100465T
Other languages
English (en)
Inventor
Bob D. Brown
Henryk T. Dudek
Original Assignee
Dicerna Pharmaceuticals, Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Dicerna Pharmaceuticals, Inc filed Critical Dicerna Pharmaceuticals, Inc
Publication of CY1124203T1 publication Critical patent/CY1124203T1/el

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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
    • 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)
    • 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 N.A.

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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)
  • 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)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)

Abstract

Η παρούσα εφεύρεση αναφέρεται σε ενώσεις, συνθέσεις και μεθόδους χρήσιμες για τη μείωση των επιπέδων RNA και πρωτεΐνης στόχου της οξειδάσης γλυκολικού (ΗΑO1) μέσω της χρήσης των dsRNA, π.χ. των παραγόντων του Dicer υποστρώματος siRNA (DsiRNA).
CY20211100465T 2013-12-27 2021-05-28 Μεθοδοι και συνθεσεις για την ειδικη αναστολη της οξειδασης του γλυκολικου (ηαο1) απο το δικλωνο rna CY1124203T1 (el)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361921181P 2013-12-27 2013-12-27
US201461937838P 2014-02-10 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
EP14874374.3A EP3087184B1 (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
CY1124203T1 true CY1124203T1 (el) 2021-12-31

Family

ID=53479713

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20211100465T CY1124203T1 (el) 2013-12-27 2021-05-28 Μεθοδοι και συνθεσεις για την ειδικη αναστολη της οξειδασης του γλυκολικου (ηαο1) απο το δικλωνο rna

Country Status (19)

Country Link
US (7) US9701966B2 (el)
EP (3) EP3581654B1 (el)
JP (2) JP6886818B2 (el)
AU (2) AU2014369850B2 (el)
CA (1) CA2935220A1 (el)
CY (1) CY1124203T1 (el)
DK (2) DK3087184T3 (el)
ES (2) ES2749855T3 (el)
FR (1) FR21C1044I2 (el)
HR (1) HRP20210612T1 (el)
HU (2) HUE055470T2 (el)
LT (2) LT3581654T (el)
LU (1) LUC00218I2 (el)
NO (1) NO2021038I1 (el)
PL (1) PL3581654T3 (el)
PT (1) PT3581654T (el)
RS (1) RS61892B1 (el)
SI (1) SI3581654T1 (el)
WO (1) WO2015100436A1 (el)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
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
US9701966B2 (en) * 2013-12-27 2017-07-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA
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
AU2017339022B2 (en) * 2016-10-05 2022-06-02 Devgen Nv Methods of preserving the biological activity of ribonucleic acids
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
JP7337044B2 (ja) 2017-07-13 2023-09-01 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｈａｏ１（ヒドロキシ酸オキシダーゼ１（グリコール酸オキシダーゼ））遺伝子発現の阻害方法
CN112867795A (zh) * 2018-07-31 2021-05-28 因特利亚治疗公司 用于执行羟基酸氧化酶1（hao1）基因编辑以治疗1型原发性高草酸尿症（ph1）的组合物和方法
EP3898661A1 (en) * 2018-12-21 2021-10-27 Precision BioSciences, Inc. Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria
JP7439248B2 (ja) 2019-11-01 2024-02-27 ライラック セラピューティクス， インク． グリコール酸オキシダーゼ阻害剤としてのヘテロ環式カルボキシレート化合物
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
CA3222159A1 (en) * 2021-06-04 2022-12-08 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

Family Cites Families (35)

* Cited by examiner, † Cited by third party
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
CA2093664C (en) 1990-10-12 2003-07-29 Fritz Eckstein Modified ribozymes
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
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
EP0748382B1 (en) 1993-09-02 2002-11-06 Ribozyme Pharmaceuticals, Inc. Non-nucleotide containing enzymatic nucleic acid
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)
US9879266B2 (en) * 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
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
EP2514758B2 (en) 2004-03-15 2021-06-23 City of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
AU2005248147A1 (en) 2004-05-11 2005-12-08 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
US20080213891A1 (en) 2004-07-21 2008-09-04 Alnylam Pharmaceuticals, Inc. RNAi Agents Comprising Universal Nucleobases
US20090018097A1 (en) 2005-09-02 2009-01-15 Mdrna, 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
CN102325534B (zh) 2008-12-18 2016-02-17 戴瑟纳制药公司 延长的dicer酶底物和特异性抑制基因表达的方法
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
PL3301177T3 (pl) 2011-11-18 2020-08-24 Alnylam Pharmaceuticals, Inc. ŚRODKI RNAi, KOMPOZYCJE I SPOSOBY ICH ZASTOSOWANIA DO LECZENIA CHORÓB ZWIĄZANYCH Z TRANSTYRETYNĄ (TTR)
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9701966B2 (en) 2013-12-27 2017-07-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA
JOP20200115A1 (ar) * 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))

Also Published As

Publication number Publication date
FR21C1044I1 (el) 2021-11-19
EP3581654B1 (en) 2021-03-31
EP3087184A4 (en) 2017-08-16
JP2017502668A (ja) 2017-01-26
LTC3581654I2 (el) 2023-07-10
US10487330B2 (en) 2019-11-26
AU2021201953A1 (en) 2021-04-29
FR21C1044I2 (fr) 2022-09-23
EP3087184B1 (en) 2019-07-03
US9701966B2 (en) 2017-07-11
WO2015100436A1 (en) 2015-07-02
JP2020036603A (ja) 2020-03-12
RS61892B1 (sr) 2021-06-30
DK3087184T3 (da) 2019-07-29
AU2014369850A1 (en) 2016-07-14
US11873493B2 (en) 2024-01-16
US10465195B2 (en) 2019-11-05
JP7109416B2 (ja) 2022-07-29
CA2935220A1 (en) 2015-07-02
ES2875558T3 (es) 2021-11-10
US20170306333A1 (en) 2017-10-26
JP6886818B2 (ja) 2021-06-16
US9828606B2 (en) 2017-11-28
US10435692B2 (en) 2019-10-08
ES2749855T3 (es) 2020-03-24
LTPA2021008I1 (el) 2021-09-27
SI3581654T1 (sl) 2021-08-31
HUS2100041I1 (hu) 2021-10-28
US20180291379A1 (en) 2018-10-11
PL3581654T3 (pl) 2021-09-13
EP3087184A1 (en) 2016-11-02
HUE055470T2 (hu) 2021-11-29
US20190194665A1 (en) 2019-06-27
PT3581654T (pt) 2021-06-02
LT3581654T (lt) 2021-06-25
EP3581654A1 (en) 2019-12-18
EP3892727A1 (en) 2021-10-13
NO2021038I1 (no) 2021-09-16
US20190211340A1 (en) 2019-07-11
US20200002705A1 (en) 2020-01-02
AU2014369850B2 (en) 2021-04-08
LUC00218I2 (el) 2022-10-07
US11060093B2 (en) 2021-07-13
US20210230604A1 (en) 2021-07-29
DK3581654T3 (da) 2021-04-26
HRP20210612T1 (hr) 2021-08-20
US20150184160A1 (en) 2015-07-02

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