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MA44908A - Molécules d'arnsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène nrarp - Google Patents

Molécules d'arnsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène nrarp

Info

Publication number
MA44908A
MA44908A MA044908A MA44908A MA44908A MA 44908 A MA44908 A MA 44908A MA 044908 A MA044908 A MA 044908A MA 44908 A MA44908 A MA 44908A MA 44908 A MA44908 A MA 44908A
Authority
MA
Morocco
Prior art keywords
molecules
arnsi
inhibit
compositions
processes
Prior art date
Application number
MA044908A
Other languages
English (en)
Inventor
Ana Isabel Jimenez
Tamara Martinez
Covadonga Pañeda
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.)
Publication date
Publication of MA44908A publication Critical patent/MA44908A/fr

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • 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]
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3235Chemical structure of the sugar modified ring structure having the O of the ribose replaced by another atom
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

L'invention concerne des molécules d'arn si et leur utilisation dans des procédés et des compositions pharmaceutiques destinés à inhiber l'expression du gène nrarp. L'invention concerne également l'utilisation desdites molécules d'arn si dans le traitement et / ou la prévention d'une maladie ou d'un trouble lié à la néovascularisation caractérisé par une expression et / ou une activité accrue du gène nrarp, ledit trouble de la vue étant choisi dans le groupe comprenant dégénérescence maculaire apparentée (dmla), rétinopathie ischémique, œdème maculaire diabétique (dme), rétinopathie diabétique proliférante (pdr), ischémie rétinienne diabétique (dri), œdème rétinien diabétique (dre) et rétinopathie de prématurité (rop) et leurs combinaisons.
MA044908A 2015-09-08 Molécules d'arnsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène nrarp MA44908A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP15382440 2015-09-08

Publications (1)

Publication Number Publication Date
MA44908A true MA44908A (fr) 2018-07-18

Family

ID=54199152

Family Applications (1)

Application Number Title Priority Date Filing Date
MA044908A MA44908A (fr) 2015-09-08 Molécules d'arnsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène nrarp

Country Status (23)

Country Link
US (1) US10752896B2 (fr)
EP (2) EP4599889A2 (fr)
JP (3) JP6946297B2 (fr)
KR (2) KR102741279B1 (fr)
CN (2) CN108026530B (fr)
AU (1) AU2016319072B2 (fr)
CA (1) CA2997648A1 (fr)
CO (1) CO2018003596A2 (fr)
DK (1) DK3347470T3 (fr)
ES (1) ES3042567T3 (fr)
FI (1) FI3347470T3 (fr)
HR (1) HRP20251155T1 (fr)
IL (2) IL295206B2 (fr)
LT (1) LT3347470T (fr)
MA (1) MA44908A (fr)
MX (2) MX2018002857A (fr)
MY (1) MY185638A (fr)
PL (1) PL3347470T3 (fr)
PT (1) PT3347470T (fr)
RS (1) RS67288B1 (fr)
RU (1) RU2738971C2 (fr)
SG (1) SG10202002084UA (fr)
WO (1) WO2017042238A1 (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017104270A1 (fr) 2015-12-15 2017-06-22 日立オートモティブシステムズ株式会社 Dispositif de commande de véhicule
EP4560020A1 (fr) 2023-11-22 2025-05-28 Sylentis S.A.U. Arnsi et compositions pour le traitement prophylactique et thérapeutique d'états rétiniens oculaires
EP4560019A1 (fr) 2023-11-22 2025-05-28 Sylentis S.A.U. Arnsi et compositions pour le traitement prophylactique et thérapeutique d'états rétiniens oculaires
CN120137968B (zh) * 2025-02-13 2025-12-09 上海市第一人民医院 Rna、药物组合物、缀合物及应用

Family Cites Families (25)

* 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
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
US20060040262A1 (en) * 2002-12-27 2006-02-23 Morris David W Novel compositions and methods in cancer
AU2003214363A1 (en) * 2002-02-13 2003-09-04 Axordia Limited Method to modify differentiation of pluripotential stem cells
PT1527176E (pt) 2002-08-05 2007-04-30 Atugen Ag Novas formas de muléculas de arn de interferência
DK2284266T3 (da) * 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
WO2005062937A2 (fr) 2003-12-22 2005-07-14 University Of Massachusetts Procedes et compositions d'amelioration de l'efficacite de la specificite d'un siarn a extremite emoussee simple et double
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
US20100292301A1 (en) 2007-02-28 2010-11-18 Elena Feinstein Novel sirna structures
US8614309B2 (en) 2007-10-03 2013-12-24 Quark Pharmaceuticals, Inc. Double-stranded RNA directed to CASP2 and methods of use thereof
KR101672563B1 (ko) 2008-10-22 2016-11-03 쿠아크 파마수티칼스 인코퍼레이티드 안질환을 치료하는 방법
CN102272307A (zh) * 2008-11-21 2011-12-07 利莱恩斯生命科学有限公司 siNA介导的VEGF-C和RhoA的敲低对VEGF-A分泌、血管生成和/或新血管生成的抑制
JP2013501526A (ja) * 2009-08-14 2013-01-17 ジェネンテック, インコーポレイテッド Vegfアンタゴニストに対する患者の応答をモニターするための生物学的マーカー
WO2011056005A2 (fr) 2009-11-04 2011-05-12 성균관대학교산학협력단 Nouvelle structure d'arnsi pour réduire au minimum les effets hors cible causés par des brins antisens, et utilisation de celle-ci
EP2390327A1 (fr) * 2010-05-27 2011-11-30 Sylentis S.A. ARNsi et leur utilisation dans des procédés et des compositions pour le traitement et/ou la prévention de maladies oculaires
WO2012140234A1 (fr) * 2011-04-13 2012-10-18 Vib Vzw Modulation du miarn dans les maladies caractérisées par une angiogenèse aberrante
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 (fr) * 2013-10-22 2015-04-29 Sylentis, S.A.U. ARNsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène PDK1
EP2865756A1 (fr) * 2013-10-22 2015-04-29 Sylentis, S.A.U. ARNsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène FLAP
EP2865758A1 (fr) * 2013-10-22 2015-04-29 Sylentis, S.A.U. ARNsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène ORAI1
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
PT3347470T (pt) 2025-09-25
US20180340177A1 (en) 2018-11-29
CO2018003596A2 (es) 2018-08-31
FI3347470T3 (fi) 2025-10-08
HK1257658A1 (en) 2019-10-25
IL257524A (en) 2018-04-30
KR102708889B1 (ko) 2024-09-25
ES3042567T3 (en) 2025-11-21
CN108026530B (zh) 2022-03-04
EP4599889A2 (fr) 2025-08-13
MY185638A (en) 2021-05-27
CN114632090A (zh) 2022-06-17
RU2738971C2 (ru) 2020-12-21
RU2018112453A (ru) 2019-10-10
KR102741279B1 (ko) 2024-12-11
JP6946297B2 (ja) 2021-10-06
CN108026530A (zh) 2018-05-11
JP2018526031A (ja) 2018-09-13
IL257524B1 (en) 2023-09-01
IL295206B1 (en) 2023-10-01
WO2017042238A1 (fr) 2017-03-16
MX2018002857A (es) 2018-09-12
RS67288B1 (sr) 2025-11-28
JP2023144029A (ja) 2023-10-06
DK3347470T3 (da) 2025-09-29
KR20240000615A (ko) 2024-01-02
US10752896B2 (en) 2020-08-25
MX2022000554A (es) 2022-02-10
AU2016319072B2 (en) 2022-03-17
AU2016319072A1 (en) 2018-03-15
SG10202002084UA (en) 2020-05-28
EP3347470A1 (fr) 2018-07-18
KR20180039760A (ko) 2018-04-18
CA2997648A1 (fr) 2017-03-16
IL295206B2 (en) 2024-02-01
IL295206A (en) 2022-10-01
HRP20251155T1 (hr) 2025-12-05
PL3347470T3 (pl) 2025-11-24
IL257524B2 (en) 2024-01-01
LT3347470T (lt) 2025-12-10
JP2022000035A (ja) 2022-01-04
CN114632090B (zh) 2023-09-29
EP3347470B1 (fr) 2025-07-09
RU2018112453A3 (fr) 2020-05-18
JP7394815B2 (ja) 2023-12-08

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