FR21C1044I2 - Procédé et composition pour l'inhibition spécifique de la glycolate oxydase (hao1) par un arn bicaténaire - Google Patents

Procédé et composition pour l'inhibition spécifique de la glycolate oxydase (hao1) par un arn bicaténaire

Info

Publication number
FR21C1044I2
FR21C1044I2 FR21C1044C FR21C1044C FR21C1044I2 FR 21C1044 I2 FR21C1044 I2 FR 21C1044I2 FR 21C1044 C FR21C1044 C FR 21C1044C FR 21C1044 C FR21C1044 C FR 21C1044C FR 21C1044 I2 FR21C1044 I2 FR 21C1044I2
Authority
FR
France
Prior art keywords
hao1
double
composition
stranded rna
specific inhibition
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.)
Active
Application number
FR21C1044C
Other languages
English (en)
French (fr)
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Dicerna Pharmaceuticals Inc
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 FR21C1044I1 publication Critical patent/FR21C1044I1/fr
Application granted granted Critical
Publication of FR21C1044I2 publication Critical patent/FR21C1044I2/fr
Active legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/02Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0006Oxidoreductases (1.) acting on CH-OH groups as donors (1.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y101/00Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/03Oxidoreductases 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)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.

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)
  • 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)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (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)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Medicinal Preparation (AREA)
FR21C1044C 2013-12-27 2021-09-20 Procédé et composition pour l'inhibition spécifique de la glycolate oxydase (hao1) par un arn bicaténaire Active FR21C1044I2 (fr)

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 (2)

Publication Number Publication Date
FR21C1044I1 FR21C1044I1 (es) 2021-11-19
FR21C1044I2 true FR21C1044I2 (fr) 2022-09-23

Family

ID=53479713

Family Applications (1)

Application Number Title Priority Date Filing Date
FR21C1044C Active FR21C1044I2 (fr) 2013-12-27 2021-09-20 Procédé et composition pour l'inhibition spécifique de la glycolate oxydase (hao1) par un arn bicaténaire

Country Status (19)

Country Link
US (7) US9701966B2 (es)
EP (3) EP3087184B1 (es)
JP (2) JP6886818B2 (es)
AU (2) AU2014369850B2 (es)
CA (1) CA2935220A1 (es)
CY (2) CY1124203T1 (es)
DK (2) DK3581654T3 (es)
ES (2) ES2875558T3 (es)
FR (1) FR21C1044I2 (es)
HR (1) HRP20210612T1 (es)
HU (2) HUE055470T2 (es)
LT (2) LT3581654T (es)
LU (1) LUC00218I2 (es)
NO (1) NO2021038I1 (es)
PL (1) PL3581654T3 (es)
PT (1) PT3581654T (es)
RS (1) RS61892B1 (es)
SI (1) SI3581654T1 (es)
WO (1) WO2015100436A1 (es)

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US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
RS61892B1 (sr) 2013-12-27 2021-06-30 Dicerna Pharmaceuticals Inc Postupci i kompozicije za specifičnu inhibiciju glikolat oksidaze (hao1) pomoću dvolančane rnk
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
CA3036620A1 (en) * 2016-10-05 2018-04-12 Syngenta Participations Ag 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
WO2019014491A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals, Inc. METHODS OF INHIBITING GENE EXPRESSION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE))
CA3113190A1 (en) * 2018-07-31 2020-02-06 Intellia Therapeutics, Inc. Compositions and methods for hydroxyacid oxidase 1 (hao1) gene editing for treating primary hyperoxaluria type 1 (ph1)
EP3898661A1 (en) * 2018-12-21 2021-10-27 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
WO2021237067A1 (en) * 2020-05-22 2021-11-25 President And Fellows Of Harvard College Interferon- inducing oligonucleotide duplexes and methods of use
CA3172481A1 (en) 2020-11-12 2022-05-19 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

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Also Published As

Publication number Publication date
LTC3581654I2 (es) 2023-07-10
US9828606B2 (en) 2017-11-28
PT3581654T (pt) 2021-06-02
US20180291379A1 (en) 2018-10-11
US10435692B2 (en) 2019-10-08
CA2935220A1 (en) 2015-07-02
PL3581654T3 (pl) 2021-09-13
US20190194665A1 (en) 2019-06-27
US9701966B2 (en) 2017-07-11
US20170306333A1 (en) 2017-10-26
US20190211340A1 (en) 2019-07-11
LT3581654T (lt) 2021-06-25
JP7109416B2 (ja) 2022-07-29
AU2014369850A1 (en) 2016-07-14
US10487330B2 (en) 2019-11-26
SI3581654T1 (sl) 2021-08-31
US20200002705A1 (en) 2020-01-02
DK3087184T3 (da) 2019-07-29
EP3892727A1 (en) 2021-10-13
EP3087184A4 (en) 2017-08-16
CY1124203T1 (el) 2021-12-31
CY2021022I1 (el) 2021-12-31
EP3581654B1 (en) 2021-03-31
DK3581654T3 (da) 2021-04-26
NO2021038I1 (no) 2021-09-16
US20210230604A1 (en) 2021-07-29
LTPA2021008I1 (es) 2021-09-27
US20150184160A1 (en) 2015-07-02
JP2017502668A (ja) 2017-01-26
HUS2100041I1 (hu) 2021-10-28
WO2015100436A1 (en) 2015-07-02
EP3087184B1 (en) 2019-07-03
JP2020036603A (ja) 2020-03-12
HUE055470T2 (hu) 2021-11-29
AU2014369850B2 (en) 2021-04-08
ES2749855T3 (es) 2020-03-24
LUC00218I2 (es) 2022-10-07
US11060093B2 (en) 2021-07-13
HRP20210612T1 (hr) 2021-08-20
US11873493B2 (en) 2024-01-16
JP6886818B2 (ja) 2021-06-16
EP3087184A1 (en) 2016-11-02
US10465195B2 (en) 2019-11-05
AU2021201953A1 (en) 2021-04-29
RS61892B1 (sr) 2021-06-30
FR21C1044I1 (es) 2021-11-19
ES2875558T3 (es) 2021-11-10
CY2021022I2 (el) 2021-12-31
EP3581654A1 (en) 2019-12-18

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