HK1257658A1 - Sirna及其在抑制nrarp基因表達的方法和組合物中的用途 - Google Patents

Sirna及其在抑制nrarp基因表達的方法和組合物中的用途

Info

Publication number
HK1257658A1
HK1257658A1 HK19100018.7A HK19100018A HK1257658A1 HK 1257658 A1 HK1257658 A1 HK 1257658A1 HK 19100018 A HK19100018 A HK 19100018A HK 1257658 A1 HK1257658 A1 HK 1257658A1
Authority
HK
Hong Kong
Prior art keywords
sirna
inhibiting
expression
compositions
methods
Prior art date
Application number
HK19100018.7A
Other languages
English (en)
Inventor
Ana Isabel Jimenez
Covadonga Pañeda
Tamara Martinez
Original Assignee
Sylentis Sau
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 Sylentis Sau filed Critical Sylentis Sau
Publication of HK1257658A1 publication Critical patent/HK1257658A1/zh

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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 N.A.
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3235—Chemical structure of the sugar modified ring structure having the O of the ribose replaced by another atom
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
HK19100018.7A 2015-09-08 2019-01-02 Sirna及其在抑制nrarp基因表達的方法和組合物中的用途 HK1257658A1 (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP15382440 2015-09-08
PCT/EP2016/071122 WO2017042238A1 (en) 2015-09-08 2016-09-07 Sirna and their use in methods and compositions for inhibiting the expression of the nrarp gene

Publications (1)

Publication Number Publication Date
HK1257658A1 true HK1257658A1 (zh) 2019-10-25

Family

ID=54199152

Family Applications (1)

Application Number Title Priority Date Filing Date
HK19100018.7A HK1257658A1 (zh) 2015-09-08 2019-01-02 Sirna及其在抑制nrarp基因表達的方法和組合物中的用途

Country Status (16)

Country Link
US (1) US10752896B2 (zh)
EP (1) EP3347470A1 (zh)
JP (3) JP6946297B2 (zh)
KR (2) KR20180039760A (zh)
CN (2) CN114632090B (zh)
AU (1) AU2016319072B2 (zh)
CA (1) CA2997648A1 (zh)
CO (1) CO2018003596A2 (zh)
HK (1) HK1257658A1 (zh)
IL (2) IL295206B2 (zh)
MA (1) MA44908A (zh)
MX (2) MX2018002857A (zh)
MY (1) MY185638A (zh)
RU (1) RU2738971C2 (zh)
SG (1) SG10202002084UA (zh)
WO (1) WO2017042238A1 (zh)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017104270A1 (ja) 2015-12-15 2017-06-22 日立オートモティブシステムズ株式会社 車両用制御装置

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* Cited by examiner, † Cited by third party
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US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
JP4095895B2 (ja) 2000-12-01 2008-06-04 マックス−プランク−ゲゼルシャフト ツール フォーデルング デル ヴィッセンシャフテン エー．ヴェー． Ｒｎａ干渉を媒介する短鎖ｒｎａ分子
US20060040262A1 (en) * 2002-12-27 2006-02-23 Morris David W Novel compositions and methods in cancer
WO2003068961A2 (en) * 2002-02-13 2003-08-21 Axordia Limited Method to modify differentiation of pluripotential stem cells
EP2258847B2 (en) 2002-08-05 2020-07-01 Silence Therapeutics GmbH Futher novel forms of interfering RNA molecules
EP2314691A3 (en) * 2002-11-14 2012-01-18 Dharmacon, Inc. Fuctional and hyperfunctional siRNA
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
GB0521716D0 (en) 2005-10-25 2005-11-30 Genomica Sau Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のｓｉＲＮＡおよびその使用方法
WO2008104978A2 (en) 2007-02-28 2008-09-04 Quark Pharmaceuticals, Inc. Novel sirna structures
BRPI0817605A2 (pt) 2007-10-03 2017-05-09 Quark Pharmaceuticals Inc novas estruturas de sirna
NZ591391A (en) 2008-10-22 2012-10-26 Quark Pharmaceuticals Inc Non-invasive methods for treating eye disorders using a topical oligonucleotide composition
US20110293625A1 (en) * 2008-11-21 2011-12-01 Krishna Addepalli Murali Inhibition of vegf-a secretion, angiogenesis and/or neoangiogenesis by sina mediated knockdown of vegf-c and rhoa
KR20120059553A (ko) * 2009-08-14 2012-06-08 제넨테크, 인크. Ｖｅｇｆ 길항제에 대한 환자 반응을 모니터링하기 위한 생물학적 마커
US9260470B2 (en) 2009-11-04 2016-02-16 Sungkyunkwan University Foundation For Corporate Collaboration SiRNA structure for minimizing off-target effects caused by antisense strands, and use thereof
EP2390327A1 (en) * 2010-05-27 2011-11-30 Sylentis S.A. siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
WO2012140234A1 (en) * 2011-04-13 2012-10-18 Vib Vzw Modulation of mirna in diseases with aberrant angiogenesis
US20130123330A1 (en) 2011-07-15 2013-05-16 Patrick Y. Lu Dual Targeted siRNA Therapeutics for Treatment of Diabetic Retinopathy and Other Ocular Neovascularization Diseases
KR101535341B1 (ko) * 2012-07-02 2015-07-13 한화케미칼 주식회사 Dll4에 특이적으로 결합하는 신규한 단일클론항체 및 이의 용도
KR20140085790A (ko) * 2012-12-27 2014-07-08 한화케미칼 주식회사 Ｎｏｔｃｈ 신호전달을 효과적으로 저해하는 ｄｌｌ４에 특이적인 신규한 인간 단일클론항체
EP2865757A1 (en) * 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the PDK1 gene.
EP2865758A1 (en) * 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the ORAI1 gene
EP2865756A1 (en) * 2013-10-22 2015-04-29 Sylentis, S.A.U. siRNA and their use in methods and compositions for inhibiting the expression of the FLAP gene.
CN105624159B (zh) * 2016-02-22 2019-02-05 中国人民解放军第二军医大学 一种针对人EDIL3基因的siRNA及其应用

Also Published As

Publication number Publication date
MX2018002857A (es) 2018-09-12
JP6946297B2 (ja) 2021-10-06
EP3347470A1 (en) 2018-07-18
RU2738971C2 (ru) 2020-12-21
AU2016319072A1 (en) 2018-03-15
AU2016319072B2 (en) 2022-03-17
CN108026530B (zh) 2022-03-04
CN114632090A (zh) 2022-06-17
US10752896B2 (en) 2020-08-25
MA44908A (fr) 2018-07-18
IL257524A (en) 2018-04-30
JP2022000035A (ja) 2022-01-04
SG10202002084UA (en) 2020-05-28
CO2018003596A2 (es) 2018-08-31
MY185638A (en) 2021-05-27
KR20240000615A (ko) 2024-01-02
IL295206B2 (en) 2024-02-01
CN114632090B (zh) 2023-09-29
IL257524B1 (en) 2023-09-01
JP2023144029A (ja) 2023-10-06
IL257524B2 (en) 2024-01-01
MX2022000554A (es) 2022-02-10
US20180340177A1 (en) 2018-11-29
CA2997648A1 (en) 2017-03-16
RU2018112453A (ru) 2019-10-10
IL295206B1 (en) 2023-10-01
IL295206A (en) 2022-10-01
WO2017042238A1 (en) 2017-03-16
JP2018526031A (ja) 2018-09-13
RU2018112453A3 (zh) 2020-05-18
CN108026530A (zh) 2018-05-11
KR20180039760A (ko) 2018-04-18
JP7394815B2 (ja) 2023-12-08

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