LT3581654T - Būdai ir kompozicijos, skirti glikolato oksidazės (hao1) specifinei inhibicijai dvigrande rnr - Google Patents

Būdai ir kompozicijos, skirti glikolato oksidazės (hao1) specifinei inhibicijai dvigrande rnr

Info

Publication number
LT3581654T
LT3581654T LTEP19182616.3T LT19182616T LT3581654T LT 3581654 T LT3581654 T LT 3581654T LT 19182616 T LT19182616 T LT 19182616T LT 3581654 T LT3581654 T LT 3581654T
Authority
LT
Lithuania
Prior art keywords
hao1
compositions
methods
specific inhibition
glycolate oxidase
Prior art date
Application number
LTEP19182616.3T
Other languages
English (en)
Lithuanian (lt)
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 LT3581654T publication Critical patent/LT3581654T/lt

Links

Classifications

    • 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 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)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (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)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
LTEP19182616.3T 2013-12-27 2014-12-26 Būdai ir kompozicijos, skirti glikolato oksidazės (hao1) specifinei inhibicijai dvigrande rnr LT3581654T (lt)

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
LT3581654T true LT3581654T (lt) 2021-06-25

Family

ID=53479713

Family Applications (2)

Application Number Title Priority Date Filing Date
LTEP19182616.3T LT3581654T (lt) 2013-12-27 2014-12-26 Būdai ir kompozicijos, skirti glikolato oksidazės (hao1) specifinei inhibicijai dvigrande rnr
LTPA2021008C LTC3581654I2 (enExample) 2013-12-27 2021-09-01

Family Applications After (1)

Application Number Title Priority Date Filing Date
LTPA2021008C LTC3581654I2 (enExample) 2013-12-27 2021-09-01

Country Status (21)

Country Link
US (7) US9701966B2 (enExample)
EP (3) EP3087184B1 (enExample)
JP (2) JP6886818B2 (enExample)
AU (2) AU2014369850B2 (enExample)
CA (1) CA2935220A1 (enExample)
CY (2) CY1124203T1 (enExample)
DK (2) DK3581654T3 (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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US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
SI3581654T1 (sl) * 2013-12-27 2021-08-31 Dicerna Pharmaceuticals, Inc. Postopki in sestavki za specifično inhibicijo glikolat-oksidaze (HA01) z dvoverižno RNA
TWI864340B (zh) * 2014-10-10 2024-12-01 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
WO2016205323A1 (en) * 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
EP3522719A1 (en) * 2016-10-05 2019-08-14 Syngenta Participations AG Methods of preserving the biological activity of ribonucleic acids
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
AU2018301829B2 (en) 2017-07-13 2024-08-01 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).
EP3898661A1 (en) * 2018-12-21 2021-10-27 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
WO2021237067A1 (en) * 2020-05-22 2021-11-25 President And Fellows Of Harvard College Interferon- inducing oligonucleotide duplexes and methods of use
KR20250099412A (ko) 2020-11-12 2025-07-01 프리시젼 바이오사이언시스 인코포레이티드 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제
EP4043018A1 (en) 2021-02-10 2022-08-17 Charité - Universitätsmedizin Berlin Composition and method for reducing oxalate levels in patients receiving maintenance dialysis
KR20240034746A (ko) * 2021-06-04 2024-03-14 아버 바이오테크놀로지스, 인크. 하이드록시산 산화효소 1(hao1)을 표적으로 하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도
JP2024528659A (ja) * 2021-07-19 2024-07-30 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 非原発性高シュウ酸尿症疾患または障害を有するまたは発症するリスクがある対象を治療するための方法および組成物
TW202430637A (zh) 2022-11-16 2024-08-01 美商戴瑟納製藥股份有限公司 Stat3靶向性寡核苷酸及其用途
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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SI3581654T1 (sl) * 2013-12-27 2021-08-31 Dicerna Pharmaceuticals, Inc. Postopki in sestavki za specifično inhibicijo glikolat-oksidaze (HA01) z dvoverižno RNA
TWI864340B (zh) * 2014-10-10 2024-12-01 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

Also Published As

Publication number Publication date
FR21C1044I2 (fr) 2022-09-23
EP3581654B1 (en) 2021-03-31
EP3087184A1 (en) 2016-11-02
JP2020036603A (ja) 2020-03-12
SMT202100325T1 (it) 2021-07-12
FIC20210025I1 (fi) 2021-08-25
CY2021022I2 (el) 2021-12-31
EP3581654A1 (en) 2019-12-18
AU2021201953A1 (en) 2021-04-29
RS61892B1 (sr) 2021-06-30
EP3087184A4 (en) 2017-08-16
HUS2100041I1 (hu) 2021-10-28
US10435692B2 (en) 2019-10-08
JP7109416B2 (ja) 2022-07-29
US20150184160A1 (en) 2015-07-02
US20180291379A1 (en) 2018-10-11
US9701966B2 (en) 2017-07-11
JP6886818B2 (ja) 2021-06-16
CA2935220A1 (en) 2015-07-02
HUE055470T2 (hu) 2021-11-29
DK3087184T3 (da) 2019-07-29
FR21C1044I1 (enExample) 2021-11-19
PL3581654T3 (pl) 2021-09-13
US20200002705A1 (en) 2020-01-02
ES2875558T3 (es) 2021-11-10
ES2749855T3 (es) 2020-03-24
LTPA2021008I1 (enExample) 2021-09-27
EP3087184B1 (en) 2019-07-03
EP3892727A1 (en) 2021-10-13
AU2014369850A1 (en) 2016-07-14
US20170306333A1 (en) 2017-10-26
US10487330B2 (en) 2019-11-26
NO2021038I1 (no) 2021-09-16
US20190211340A1 (en) 2019-07-11
LTC3581654I2 (enExample) 2023-07-10
HRP20210612T1 (hr) 2021-08-20
CY2021022I1 (el) 2021-12-31
LUC00218I2 (enExample) 2022-10-07
US10465195B2 (en) 2019-11-05
US11873493B2 (en) 2024-01-16
US20210230604A1 (en) 2021-07-29
JP2017502668A (ja) 2017-01-26
US11060093B2 (en) 2021-07-13
DK3581654T3 (da) 2021-04-26
AU2014369850B2 (en) 2021-04-08
WO2015100436A1 (en) 2015-07-02
PT3581654T (pt) 2021-06-02
US9828606B2 (en) 2017-11-28
US20190194665A1 (en) 2019-06-27
CY1124203T1 (el) 2021-12-31
SI3581654T1 (sl) 2021-08-31

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