AR069019A1 - Arn bicatenario modificado con lipido que tiene un potente efecto de interferencia por arn, composicion farmaceutica que lo comprende y uso del mismo - Google Patents

Arn bicatenario modificado con lipido que tiene un potente efecto de interferencia por arn, composicion farmaceutica que lo comprende y uso del mismo

Info

Publication number
AR069019A1
AR069019A1 ARP080104637A ARP080104637A AR069019A1 AR 069019 A1 AR069019 A1 AR 069019A1 AR P080104637 A ARP080104637 A AR P080104637A AR P080104637 A ARP080104637 A AR P080104637A AR 069019 A1 AR069019 A1 AR 069019A1
Authority
AR
Argentina
Prior art keywords
rna
bicatenary
arn
understands
interference
Prior art date
Application number
ARP080104637A
Other languages
English (en)
Inventor
Kubo Takanori
Toyobuku Hidekazu
Ohba Hideki
Hayashi Hirotake
Original Assignee
Aist
Otsuka Pharma Co Ltd
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 Aist, Otsuka Pharma Co Ltd filed Critical Aist
Publication of AR069019A1 publication Critical patent/AR069019A1/es

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system

Landscapes

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

Abstract

Un objeto de la presente solicitud es brindar un ARN bicatenario que tiene una alta resistencia a la nucleasa y una alta eficacia de captación celular, y que es capaz de producir un excelente efecto de interferencia por ARN. La presente brinda un ARN bicatenario modificado con lípido que comprende una cadena sentido que tiene una secuencia de nucleótidos complementaria de una secuencia blanco, y una cadena antisentido que tiene una secuencia de nucleótidos complementaria de la cadena sentido, el ARN bicatenario es capaz de inhibir la expresión del gen blanco, y la cadena sentido tiene un lípido unido a al menos uno del primer al sexto nucleótido del extremo 5' directamente o por medio de un enlace.
ARP080104637A 2007-10-24 2008-10-24 Arn bicatenario modificado con lipido que tiene un potente efecto de interferencia por arn, composicion farmaceutica que lo comprende y uso del mismo AR069019A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP2007276985 2007-10-24

Publications (1)

Publication Number Publication Date
AR069019A1 true AR069019A1 (es) 2009-12-23

Family

ID=40524987

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP080104637A AR069019A1 (es) 2007-10-24 2008-10-24 Arn bicatenario modificado con lipido que tiene un potente efecto de interferencia por arn, composicion farmaceutica que lo comprende y uso del mismo

Country Status (19)

Country Link
US (1) US8981074B2 (es)
EP (1) EP2217284A2 (es)
JP (2) JP5887648B2 (es)
KR (1) KR20100106314A (es)
CN (2) CN101835497A (es)
AR (1) AR069019A1 (es)
AU (1) AU2008314860B2 (es)
BR (1) BRPI0818846A2 (es)
CA (1) CA2703496A1 (es)
CO (1) CO6270240A2 (es)
IL (1) IL204992A (es)
MX (1) MX2010004452A (es)
NZ (1) NZ584509A (es)
RU (1) RU2489167C2 (es)
SG (1) SG185295A1 (es)
TW (1) TW200927177A (es)
UA (1) UA104131C2 (es)
WO (1) WO2009054551A2 (es)
ZA (1) ZA201002427B (es)

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US20100047909A1 (en) * 2007-03-02 2010-02-25 Mdrna, Inc. Nucleic acid compounds for inhibiting vegf family gene expression and uses thereof
KR20100131509A (ko) 2008-03-31 2010-12-15 내셔날 인스티튜트 오브 어드밴스드 인더스트리얼 사이언스 앤드 테크놀로지 Ｒｎａ 간섭 효과가 높은 2본쇄 지질 수식 ｒｎａ
KR101224828B1 (ko) * 2009-05-14 2013-01-22 (주)바이오니아 ｓｉＲＮＡ 접합체 및 그 제조방법
GB0910723D0 (en) 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
US9663784B2 (en) 2012-05-26 2017-05-30 Bonac Corporation Single-stranded nucleic acid molecule for regulating expression of gene having delivering function
RU2523119C2 (ru) * 2012-09-28 2014-07-20 Общество с ограниченной ответственностью "ВИТАЛАНГ" Способ количественной оценки эффективности олеиновой кислоты как переносчика рнк через биологические мембраны
CN105899663B (zh) 2013-12-26 2019-07-26 学校法人东京医科大学 用于基因表达控制的人工模拟miRNA及其用途
WO2015099187A1 (ja) 2013-12-27 2015-07-02 株式会社ボナック 遺伝子発現制御のための人工マッチ型ｍｉＲＮＡおよびその用途
KR20170098914A (ko) 2014-12-27 2017-08-30 가부시키가이샤 보낙 유전자 발현 제어를 위한 천연형 miRNA 및 그의 용도
EP3276003B1 (en) 2015-03-27 2022-07-20 Bonac Corporation Single-chain nucleic acid molecule having delivery function and gene expression control ability
PL3277814T3 (pl) 2015-04-03 2020-11-30 University Of Massachusetts Związki oligonukleotydowe ukierunkowane na mrna huntingtyny
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP2019503394A (ja) 2016-01-31 2019-02-07 ユニバーシティ・オブ・マサチューセッツＵｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍａｓｓａｃｈｕｓｅｔｔｓ 分岐オリゴヌクレオチド
US11753638B2 (en) 2016-08-12 2023-09-12 University Of Massachusetts Conjugated oligonucleotides
KR20190090836A (ko) 2016-11-28 2019-08-02 나파젠 파마, 인크. 화학 수식 siRNA
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
WO2019074071A1 (ja) * 2017-10-11 2019-04-18 日東電工株式会社 核酸分子発現の調節
EP3790970A1 (en) 2018-05-07 2021-03-17 Alnylam Pharmaceuticals Inc. Extrahepatic delivery
WO2019217997A1 (en) * 2018-05-14 2019-11-21 Murdoch University Methods for treating vegf-related conditions
CA3109133A1 (en) 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps
EP4359539A1 (en) 2021-06-23 2024-05-01 University Of Massachusetts Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
US11912997B2 (en) 2022-06-15 2024-02-27 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of Superoxide Dismutase 1 (SOD1), compositions thereof, and methods of use

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
DE10302421A1 (de) 2003-01-21 2004-07-29 Ribopharma Ag Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit
CN101293908B (zh) * 2003-09-09 2015-05-06 杰龙公司 用于端粒酶抑制的改性寡核苷酸
US20050277610A1 (en) * 2004-03-15 2005-12-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
CA2562151C (en) * 2004-04-30 2016-09-06 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a c5-modified pyrimidine
US20070161591A1 (en) * 2005-08-18 2007-07-12 University Of Massachusetts Methods and compositions for treating neurological disease

Also Published As

Publication number Publication date
WO2009054551A2 (en) 2009-04-30
CO6270240A2 (es) 2011-04-20
CN104726458A (zh) 2015-06-24
TW200927177A (en) 2009-07-01
US20100298411A1 (en) 2010-11-25
ZA201002427B (en) 2011-06-29
JP2016028605A (ja) 2016-03-03
UA104131C2 (uk) 2014-01-10
JP5887648B2 (ja) 2016-03-16
AU2008314860B2 (en) 2014-01-30
IL204992A0 (en) 2010-11-30
IL204992A (en) 2015-11-30
RU2010120715A (ru) 2011-11-27
CN101835497A (zh) 2010-09-15
WO2009054551A3 (en) 2010-01-28
KR20100106314A (ko) 2010-10-01
CA2703496A1 (en) 2009-04-30
RU2489167C2 (ru) 2013-08-10
MX2010004452A (es) 2010-08-04
US8981074B2 (en) 2015-03-17
NZ584509A (en) 2012-08-31
SG185295A1 (en) 2012-11-29
EP2217284A2 (en) 2010-08-18
AU2008314860A1 (en) 2009-04-30
BRPI0818846A2 (pt) 2015-04-14
JP2011500002A (ja) 2011-01-06

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