PT3581654T - Métodos e composições para a inibição específica de glicolato oxidase (hao1) por rna de cadeia dupla - Google Patents

Métodos e composições para a inibição específica de glicolato oxidase (hao1) por rna de cadeia dupla

Info

Publication number
PT3581654T
PT3581654T PT191826163T PT19182616T PT3581654T PT 3581654 T PT3581654 T PT 3581654T PT 191826163 T PT191826163 T PT 191826163T PT 19182616 T PT19182616 T PT 19182616T PT 3581654 T PT3581654 T PT 3581654T
Authority
PT
Portugal
Prior art keywords
hao1
compositions
methods
double chain
specific inhibition
Prior art date
Application number
PT191826163T
Other languages
English (en)
Portuguese (pt)
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 PT3581654T publication Critical patent/PT3581654T/pt

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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 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)
  • 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)
PT191826163T 2013-12-27 2014-12-26 Métodos e composições para a inibição específica de glicolato oxidase (hao1) por rna de cadeia dupla PT3581654T (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361921181P 2013-12-27 2013-12-27
US201461937838P 2014-02-10 2014-02-10

Publications (1)

Publication Number Publication Date
PT3581654T true PT3581654T (pt) 2021-06-02

Family

ID=53479713

Family Applications (1)

Application Number Title Priority Date Filing Date
PT191826163T PT3581654T (pt) 2013-12-27 2014-12-26 Métodos e composições para a inibição específica de glicolato oxidase (hao1) por rna de cadeia dupla

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)

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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

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Also Published As

Publication number Publication date
US11873493B2 (en) 2024-01-16
DK3581654T3 (da) 2021-04-26
US20170306333A1 (en) 2017-10-26
CA2935220A1 (en) 2015-07-02
JP7109416B2 (ja) 2022-07-29
ES2875558T3 (es) 2021-11-10
EP3087184B1 (en) 2019-07-03
EP3581654A1 (en) 2019-12-18
CY2021022I1 (el) 2021-12-31
SMT202100325T1 (it) 2021-07-12
EP3892727A1 (en) 2021-10-13
JP2020036603A (ja) 2020-03-12
US20190211340A1 (en) 2019-07-11
HRP20210612T1 (hr) 2021-08-20
US9701966B2 (en) 2017-07-11
US20210230604A1 (en) 2021-07-29
HUE055470T2 (hu) 2021-11-29
FR21C1044I2 (fr) 2022-09-23
US20180291379A1 (en) 2018-10-11
EP3087184A4 (en) 2017-08-16
LTPA2021008I1 (2) 2021-09-27
LUC00218I2 (2) 2022-10-07
US10487330B2 (en) 2019-11-26
US10465195B2 (en) 2019-11-05
JP2017502668A (ja) 2017-01-26
LT3581654T (lt) 2021-06-25
US20190194665A1 (en) 2019-06-27
US9828606B2 (en) 2017-11-28
US10435692B2 (en) 2019-10-08
SI3581654T1 (sl) 2021-08-31
DK3087184T3 (da) 2019-07-29
FR21C1044I1 (2) 2021-11-19
ES2749855T3 (es) 2020-03-24
LTC3581654I2 (2) 2023-07-10
US20200002705A1 (en) 2020-01-02
EP3581654B1 (en) 2021-03-31
CY1124203T1 (el) 2021-12-31
FIC20210025I1 (fi) 2021-08-25
RS61892B1 (sr) 2021-06-30
US20150184160A1 (en) 2015-07-02
AU2014369850A1 (en) 2016-07-14
JP6886818B2 (ja) 2021-06-16
HUS2100041I1 (hu) 2021-10-28
AU2014369850B2 (en) 2021-04-08
PL3581654T3 (pl) 2021-09-13
EP3087184A1 (en) 2016-11-02
CY2021022I2 (el) 2021-12-31
AU2021201953A1 (en) 2021-04-29
WO2015100436A1 (en) 2015-07-02
NO2021038I1 (no) 2021-09-16
US11060093B2 (en) 2021-07-13

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