NO2021038I1 - lumasiran - Google Patents

lumasiran

Info

Publication number
NO2021038I1
NO2021038I1 NO2021038C NO2021038C NO2021038I1 NO 2021038 I1 NO2021038 I1 NO 2021038I1 NO 2021038 C NO2021038 C NO 2021038C NO 2021038 C NO2021038 C NO 2021038C NO 2021038 I1 NO2021038 I1 NO 2021038I1
Authority
NO
Norway
Prior art keywords
lumasiran
Prior art date
Application number
NO2021038C
Other languages
English (en)
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 NO2021038I1 publication Critical patent/NO2021038I1/no

Links

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 N.A.
NO2021038C 2013-12-27 2021-09-16 lumasiran NO2021038I1 (no)

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
NO2021038I1 true NO2021038I1 (no) 2021-09-16

Family

ID=53479713

Family Applications (1)

Application Number Title Priority Date Filing Date
NO2021038C NO2021038I1 (no) 2013-12-27 2021-09-16 lumasiran

Country Status (19)

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

Families Citing this family (16)

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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
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
EP3087184B1 (en) * 2013-12-27 2019-07-03 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))
EP3310918B1 (en) * 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
RU2769831C2 (ru) * 2016-10-05 2022-04-06 Зингента Партисипейшнс Аг Способы сохранения биологической активности рибонуклеиновых кислот
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
AU2018301829A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals, Inc. Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase) gene expression
AU2019313348A1 (en) * 2018-07-31 2021-03-04 Intellia Therapeutics, Inc. Compositions and methods for hydroxyacid oxidase 1 (HAO1) gene editing for treating primary hyperoxaluria type 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
AU2020376792A1 (en) 2019-11-01 2022-05-26 Lilac Therapeutics, Inc. Heterocyclic carboxylate compounds as glycolate oxidase inhibitors
EP4153752A1 (en) * 2020-05-22 2023-03-29 President And Fellows Of Harvard College Interferon- inducing oligonucleotide duplexes and methods of use
KR20230118841A (ko) 2020-11-12 2023-08-14 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
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
TW202333748A (zh) * 2021-07-19 2023-09-01 美商艾拉倫製藥股份有限公司 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物

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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)
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KR20110110776A (ko) 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
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
KR102263352B1 (ko) 2011-11-18 2021-06-11 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) 관련 질병을 치료하기 위한 RNAi 제제, 조성 및 그의 사용방법
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP3087184B1 (en) 2013-12-27 2019-07-03 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
US20200002705A1 (en) 2020-01-02
ES2749855T3 (es) 2020-03-24
US20150184160A1 (en) 2015-07-02
US20170306333A1 (en) 2017-10-26
US20190211340A1 (en) 2019-07-11
US10487330B2 (en) 2019-11-26
EP3087184A4 (en) 2017-08-16
RS61892B1 (sr) 2021-06-30
FR21C1044I2 (fr) 2022-09-23
DK3087184T3 (da) 2019-07-29
AU2014369850B2 (en) 2021-04-08
CY1124203T1 (el) 2021-12-31
US11060093B2 (en) 2021-07-13
US9701966B2 (en) 2017-07-11
LUC00218I2 (no) 2022-10-07
HUE055470T2 (hu) 2021-11-29
PT3581654T (pt) 2021-06-02
EP3087184B1 (en) 2019-07-03
LT3581654T (lt) 2021-06-25
HRP20210612T1 (hr) 2021-08-20
EP3581654A1 (en) 2019-12-18
US11873493B2 (en) 2024-01-16
FR21C1044I1 (no) 2021-11-19
ES2875558T3 (es) 2021-11-10
US10435692B2 (en) 2019-10-08
PL3581654T3 (pl) 2021-09-13
DK3581654T3 (da) 2021-04-26
CA2935220A1 (en) 2015-07-02
JP2020036603A (ja) 2020-03-12
US20210230604A1 (en) 2021-07-29
US10465195B2 (en) 2019-11-05
US20190194665A1 (en) 2019-06-27
JP6886818B2 (ja) 2021-06-16
LTPA2021008I1 (no) 2021-09-27
EP3892727A1 (en) 2021-10-13
US9828606B2 (en) 2017-11-28
EP3581654B1 (en) 2021-03-31
HUS2100041I1 (hu) 2021-10-28
EP3087184A1 (en) 2016-11-02
JP2017502668A (ja) 2017-01-26
JP7109416B2 (ja) 2022-07-29
LTC3581654I2 (no) 2023-07-10
AU2014369850A1 (en) 2016-07-14
WO2015100436A1 (en) 2015-07-02
US20180291379A1 (en) 2018-10-11
AU2021201953A1 (en) 2021-04-29
SI3581654T1 (sl) 2021-08-31

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