HUE055470T2 - Eljárások és kompozíciók a glikolát-oxidáz (HAO1) kettõs szálú RNA általi specifikus gátlására - Google Patents

Eljárások és kompozíciók a glikolát-oxidáz (HAO1) kettõs szálú RNA általi specifikus gátlására

Info

Publication number
HUE055470T2
HUE055470T2 HUE19182616A HUE19182616A HUE055470T2 HU E055470 T2 HUE055470 T2 HU E055470T2 HU E19182616 A HUE19182616 A HU E19182616A HU E19182616 A HUE19182616 A HU E19182616A HU E055470 T2 HUE055470 T2 HU E055470T2
Authority
HU
Hungary
Prior art keywords
hao1
compositions
methods
double strand
strand rna
Prior art date
Application number
HUE19182616A
Other languages
English (en)
Hungarian (hu)
Inventor
Bob Brown
Henryk 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 HUE055470T2 publication Critical patent/HUE055470T2/hu

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Classifications

    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
HUE19182616A 2013-12-27 2014-12-26 Eljárások és kompozíciók a glikolát-oxidáz (HAO1) kettõs szálú RNA általi specifikus gátlására HUE055470T2 (hu)

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
HUE055470T2 true HUE055470T2 (hu) 2021-11-29

Family

ID=53479713

Family Applications (2)

Application Number Title Priority Date Filing Date
HUE19182616A HUE055470T2 (hu) 2013-12-27 2014-12-26 Eljárások és kompozíciók a glikolát-oxidáz (HAO1) kettõs szálú RNA általi specifikus gátlására
HUS2100041C HUS2100041I1 (hu) 2013-12-27 2021-09-28 Eljárások és kompozíciók a glikolát-oxidáz (HAO1) kettõs szálú RNA általi specifikus gátlására

Family Applications After (1)

Application Number Title Priority Date Filing Date
HUS2100041C HUS2100041I1 (hu) 2013-12-27 2021-09-28 Eljárások és kompozíciók a glikolát-oxidáz (HAO1) kettõs szálú RNA általi specifikus gátlására

Country Status (21)

Country Link
US (7) US9701966B2 (enExample)
EP (3) EP3892727A1 (enExample)
JP (2) JP6886818B2 (enExample)
AU (2) AU2014369850B2 (enExample)
CA (1) CA2935220A1 (enExample)
CY (2) CY1124203T1 (enExample)
DK (2) DK3087184T3 (enExample)
ES (2) ES2875558T3 (enExample)
FI (1) FIC20210025I1 (enExample)
FR (1) FR21C1044I2 (enExample)
HR (1) HRP20210612T1 (enExample)
HU (2) HUE055470T2 (enExample)
LT (2) LT3581654T (enExample)
LU (1) LUC00218I2 (enExample)
NO (1) NO2021038I1 (enExample)
PL (1) PL3581654T3 (enExample)
PT (1) PT3581654T (enExample)
RS (1) RS61892B1 (enExample)
SI (1) SI3581654T1 (enExample)
SM (1) SMT202100325T1 (enExample)
WO (1) WO2015100436A1 (enExample)

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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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RU2769831C2 (ru) * 2016-10-05 2022-04-06 Зингента Партисипейшнс Аг Способы сохранения биологической активности рибонуклеиновых кислот
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2019014491A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals, Inc. METHODS OF INHIBITING GENE EXPRESSION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE))
BR112021001546A2 (pt) * 2018-07-31 2021-05-04 Intellia Therapeutics, Inc. composições e métodos para a edição de gene do hidroxiácido oxidase 1 (hao1) para o tratamento da hiperoxalúria primária tipo 1 (ph1)
US20220090047A1 (en) * 2018-12-21 2022-03-24 Precision Biosciences, Inc. Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria
WO2021086874A1 (en) 2019-11-01 2021-05-06 GyanRx Sciences, Inc. Heterocyclic carboxylate compounds as glycolate oxidase inhibitors
JP2023527525A (ja) * 2020-05-22 2023-06-29 プレジデント・アンド・フェロウズ・オブ・ハーバード・カレッジ インターフェロン誘導オリゴヌクレオチド二重鎖および使用方法
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
EP4347833A2 (en) * 2021-06-04 2024-04-10 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof
EP4373934A1 (en) * 2021-07-19 2024-05-29 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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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
AU2014369850B2 (en) 2021-04-08
US20170306333A1 (en) 2017-10-26
NO2021038I1 (no) 2021-09-16
ES2875558T3 (es) 2021-11-10
LTPA2021008I1 (enExample) 2021-09-27
PT3581654T (pt) 2021-06-02
PL3581654T3 (pl) 2021-09-13
EP3581654B1 (en) 2021-03-31
US20210230604A1 (en) 2021-07-29
WO2015100436A1 (en) 2015-07-02
HUS2100041I1 (hu) 2021-10-28
FIC20210025I1 (fi) 2021-08-25
CY1124203T1 (el) 2021-12-31
US9828606B2 (en) 2017-11-28
US10465195B2 (en) 2019-11-05
LT3581654T (lt) 2021-06-25
SI3581654T1 (sl) 2021-08-31
LUC00218I2 (enExample) 2022-10-07
EP3087184A4 (en) 2017-08-16
US20200002705A1 (en) 2020-01-02
CA2935220A1 (en) 2015-07-02
DK3087184T3 (da) 2019-07-29
FR21C1044I1 (enExample) 2021-11-19
EP3087184A1 (en) 2016-11-02
US10487330B2 (en) 2019-11-26
DK3581654T3 (da) 2021-04-26
US11873493B2 (en) 2024-01-16
JP2020036603A (ja) 2020-03-12
EP3581654A1 (en) 2019-12-18
AU2014369850A1 (en) 2016-07-14
US9701966B2 (en) 2017-07-11
US20180291379A1 (en) 2018-10-11
EP3087184B1 (en) 2019-07-03
US20190194665A1 (en) 2019-06-27
JP7109416B2 (ja) 2022-07-29
US11060093B2 (en) 2021-07-13
EP3892727A1 (en) 2021-10-13
US10435692B2 (en) 2019-10-08
FR21C1044I2 (fr) 2022-09-23
SMT202100325T1 (it) 2021-07-12
JP6886818B2 (ja) 2021-06-16
ES2749855T3 (es) 2020-03-24
CY2021022I1 (el) 2021-12-31
CY2021022I2 (el) 2021-12-31
HRP20210612T1 (hr) 2021-08-20
US20190211340A1 (en) 2019-07-11
RS61892B1 (sr) 2021-06-30
JP2017502668A (ja) 2017-01-26
LTC3581654I2 (enExample) 2023-07-10
US20150184160A1 (en) 2015-07-02
AU2021201953A1 (en) 2021-04-29

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