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

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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.)
Filing date
Publication date
Application filed by Sylentis Sau filed Critical Sylentis Sau
Publication of MA44908A publication Critical patent/MA44908A/fr

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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 nucleic acids [NA]
    • 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
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    • 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
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    • 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
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    • 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
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    • 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

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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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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 2016-09-07 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 2016-09-07 Molécules d'arnsi et leur utilisation dans des procédés et des compositions pour inhiber l'expression du gène nrarp

Country Status (24)

Country Link
US (1) US10752896B2 (fr)
EP (2) EP3347470B1 (fr)
JP (3) JP6946297B2 (fr)
KR (2) KR102708889B1 (fr)
CN (2) CN108026530B (fr)
AU (1) AU2016319072B2 (fr)
CO (1) CO2018003596A2 (fr)
DK (1) DK3347470T3 (fr)
ES (1) ES3042567T3 (fr)
FI (1) FI3347470T3 (fr)
HR (1) HRP20251155T1 (fr)
HU (1) HUE073212T2 (fr)
IL (2) IL257524B2 (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)
SI (1) SI3347470T1 (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
US10862292B2 (en) 2015-12-15 2020-12-08 Hitachi Automotive Systems, Ltd. Vehicle control device
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

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US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
CA2429814C (fr) 2000-12-01 2014-02-18 Thomas Tuschl Petites molecules d'arn mediant l'interference arn
US20060040262A1 (en) * 2002-12-27 2006-02-23 Morris David W Novel compositions and methods in cancer
EP1474512A2 (fr) * 2002-02-13 2004-11-10 Axordia Limited Cellules souches pluripotentielles
DE60310944T3 (de) 2002-08-05 2017-08-03 Silence Therapeutics Gmbh Weitere neue formen von interferierende rns moleküle
AU2003295600A1 (en) * 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
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
WO2005116204A1 (fr) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucléotide provoquant l'interférence rna et procédé de regulation d'expression génétique avec l’usage de ce dernier
GB0521716D0 (en) * 2005-10-25 2005-11-30 Genomica Sau Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases
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US20100292301A1 (en) 2007-02-28 2010-11-18 Elena Feinstein Novel sirna structures
RU2487716C2 (ru) 2007-10-03 2013-07-20 Кварк Фармасьютикалс, Инк. Новые структуры малых интерферирующих рнк (sirna)
CA2962219C (fr) 2008-10-22 2020-08-25 Quark Pharmaceuticals, Inc. Procedes de traitement de troubles oculaires
WO2010058426A2 (fr) * 2008-11-21 2010-05-27 Reliance Life Sciences Pvt. Ltd. Inhibition de la sécrétion de vegf-a, l’angiogenèse et/ou la néo-angiogenèse par inactivation véhiculée par ansi de vegf-c et rhoa
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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
KR20180039760A (ko) 2018-04-18
MX2018002857A (es) 2018-09-12
DK3347470T3 (da) 2025-09-29
US10752896B2 (en) 2020-08-25
KR20240000615A (ko) 2024-01-02
SI3347470T1 (sl) 2025-12-31
CN108026530A (zh) 2018-05-11
IL295206B1 (en) 2023-10-01
IL257524B1 (en) 2023-09-01
US20180340177A1 (en) 2018-11-29
HUE073212T2 (hu) 2026-01-28
HK1257658A1 (en) 2019-10-25
RS67288B1 (sr) 2025-11-28
CA2997648A1 (fr) 2017-03-16
WO2017042238A1 (fr) 2017-03-16
RU2018112453A3 (fr) 2020-05-18
FI3347470T3 (fi) 2025-10-08
CN114632090A (zh) 2022-06-17
KR102708889B1 (ko) 2024-09-25
AU2016319072A1 (en) 2018-03-15
ES3042567T3 (en) 2025-11-21
IL295206B2 (en) 2024-02-01
JP2022000035A (ja) 2022-01-04
JP7394815B2 (ja) 2023-12-08
JP2018526031A (ja) 2018-09-13
CO2018003596A2 (es) 2018-08-31
IL257524B2 (en) 2024-01-01
PT3347470T (pt) 2025-09-25
IL257524A (en) 2018-04-30
IL295206A (en) 2022-10-01
EP3347470A1 (fr) 2018-07-18
RU2018112453A (ru) 2019-10-10
EP4599889A3 (fr) 2026-04-29
MY185638A (en) 2021-05-27
SG10202002084UA (en) 2020-05-28
JP6946297B2 (ja) 2021-10-06
PL3347470T3 (pl) 2025-11-24
MX2022000554A (es) 2022-02-10
CN114632090B (zh) 2023-09-29
EP3347470B1 (fr) 2025-07-09
JP2023144029A (ja) 2023-10-06
AU2016319072B2 (en) 2022-03-17
CN108026530B (zh) 2022-03-04
LT3347470T (lt) 2025-12-10
KR102741279B1 (ko) 2024-12-11
RU2738971C2 (ru) 2020-12-21
HRP20251155T1 (hr) 2025-12-05
EP4599889A2 (fr) 2025-08-13

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