CO6270240A2 - Arn bicatenario con lipido modificado que tiene un potente efecto de interferencia del arn - Google Patents

Arn bicatenario con lipido modificado que tiene un potente efecto de interferencia del arn

Info

Publication number
CO6270240A2
CO6270240A2 CO10048045A CO10048045A CO6270240A2 CO 6270240 A2 CO6270240 A2 CO 6270240A2 CO 10048045 A CO10048045 A CO 10048045A CO 10048045 A CO10048045 A CO 10048045A CO 6270240 A2 CO6270240 A2 CO 6270240A2
Authority
CO
Colombia
Prior art keywords
double
coding chain
stranded rna
rna
lipid
Prior art date
Application number
CO10048045A
Other languages
English (en)
Inventor
Takanori Kubo
Hideki Ohba
Hidekazu Toyobuku
Hirotake Hayashi
Original Assignee
Nat Inst Of Advanced Ind Scien
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 Nat Inst Of Advanced Ind Scien, Otsuka Pharma Co Ltd filed Critical Nat Inst Of Advanced Ind Scien
Publication of CO6270240A2 publication Critical patent/CO6270240A2/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)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Oncology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Communicable Diseases (AREA)
  • Hematology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Endocrinology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (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 invencion es suministrar un ARN novedoso bicatenario que tiene alta resistencia a la nucleasa y alta eficiencia de tomas celular, y que es capaz de producir un excelente efecto en la interferencia del ARN. La presente invencion suministra un ARN bicatenario con lipido modificado que comprende una cadena codificante que tiene una secuencia de nucleotido complementaria a la secuencia objetivo, y una cadena contrasentido que tiene una secuencia de nucleotido complementaria a la cadena codificante, el ARN bicatenario es capaz de inhibir la expresion del gen objetivo, la cadena codificante tiene un lipido ligado a al menos uno de los primeros seis nucleotidos desde el lado del extremo 5' directamente o por via de un ligador. 1.- Un ARN bicatenario modificado con lipido que comprende una cadena codificante que tiene una secuencia de nucleotidos complementaria a la secuencia blanco en un gen objetivo, y una cadena anticodificante que tiene una secuencia de nucleotido complementaria a la cadena codificante, y el ARN bicatenario es capaz de inhibir la expresion del gen objetivo, y la cadena codificante que tiene un lipido ligado a por lo menos uno del primero al sexto nucleotido del extremo 5' directamente o por via de un ligador. 2.- Un ARN bicatenario modificado con lipido de acuerdo con la reivindicacion 1 que tiene un extremo truncado sobre el lado del extremo 5' de la cadena codificante, y tiene un extremo truncado o tiene un extremo en suspension sobre el lado del extremo 3' de la cadena codificante.
CO10048045A 2007-10-24 2010-04-23 Arn bicatenario con lipido modificado que tiene un potente efecto de interferencia del arn CO6270240A2 (es)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
CO6270240A2 true CO6270240A2 (es) 2011-04-20

Family

ID=40524987

Family Applications (1)

Application Number Title Priority Date Filing Date
CO10048045A CO6270240A2 (es) 2007-10-24 2010-04-23 Arn bicatenario con lipido modificado que tiene un potente efecto de interferencia del arn

Country Status (19)

Country Link
US (1) US8981074B2 (es)
EP (1) EP2217284A2 (es)
JP (2) JP5887648B2 (es)
KR (1) KR20100106314A (es)
CN (2) CN104726458A (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)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2679867A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting vegf family gene expression and uses thereof
ES2605618T3 (es) 2008-03-31 2017-03-15 National Institute Of Advanced Industrial Science And Technology ARN de doble cadena, modificado con lípidos con elevado efecto de interferencia por ARN
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
EP2857513A4 (en) * 2012-05-26 2016-05-25 Bonac Corp SINGLE STRANDED NUCLEIC ACID MOLECULE FOR CONTROLLING THE EXPRESSION OF A GENE HAVING AN ADMINISTRATION 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
EP3088524A4 (en) 2013-12-26 2017-08-09 Tokyo Medical University Artificial mimic mirna for controlling gene expression, and use of same
US10934542B2 (en) 2013-12-27 2021-03-02 Bonac Corporation Artificial match-type miRNA for controlling gene expression and use therefor
AU2015368293B2 (en) 2014-12-27 2021-07-22 Bonac Corporation Naturally occuring miRNA for controlling gene expression, and use of same
JP6602847B2 (ja) 2015-03-27 2019-11-06 株式会社ボナック デリバリー機能と遺伝子発現制御能を有する一本鎖核酸分子
LT3277814T (lt) 2015-04-03 2020-10-26 University Of Massachusetts Oligonukleotidų junginiai, skirti nukreipimui į hantingtino mrnr
EP3334499A4 (en) * 2015-08-14 2019-04-17 University of Massachusetts BIOACTIVE CONJUGATES FOR THE RELEASE OF OLIGONUCLEOTIDES
AU2017210726B2 (en) 2016-01-31 2023-08-03 University Of Massachusetts Branched oligonucleotides
CA3033368A1 (en) * 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
AU2017364806A1 (en) 2016-11-28 2019-06-13 Napajen Pharma, Inc. Chemically-modified siRNA
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
JP7208911B2 (ja) * 2017-10-11 2023-01-19 日東電工株式会社 核酸分子発現の調節
KR20210018267A (ko) 2018-05-07 2021-02-17 알닐람 파마슈티칼스 인코포레이티드 간외 전달
WO2019217997A1 (en) * 2018-05-14 2019-11-21 Murdoch University Methods for treating vegf-related conditions
SG11202101288TA (en) 2018-08-10 2021-03-30 Univ Massachusetts Modified oligonucleotides targeting snps
CA3174095A1 (en) 2021-06-23 2022-12-29 Vignesh Narayan HARIHARAN Optimized anti-flt1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
WO2023245060A2 (en) 2022-06-15 2023-12-21 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
EP1667522B1 (en) * 2003-09-09 2018-01-17 Geron Corporation Modified oligonucleotides for telomerase inhibition
US8084599B2 (en) * 2004-03-15 2011-12-27 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
EP1750776A2 (en) 2004-04-30 2007-02-14 Alnylam Pharmaceuticals Inc. Oligonucleotides comprising a c5-modified pyrimidine
US20070105803A1 (en) * 2005-08-18 2007-05-10 Muthiah Manoharan Methods and compositions for treating neurological disease

Also Published As

Publication number Publication date
MX2010004452A (es) 2010-08-04
WO2009054551A3 (en) 2010-01-28
NZ584509A (en) 2012-08-31
TW200927177A (en) 2009-07-01
IL204992A (en) 2015-11-30
JP2011500002A (ja) 2011-01-06
ZA201002427B (en) 2011-06-29
AR069019A1 (es) 2009-12-23
AU2008314860A1 (en) 2009-04-30
JP2016028605A (ja) 2016-03-03
US8981074B2 (en) 2015-03-17
RU2489167C2 (ru) 2013-08-10
EP2217284A2 (en) 2010-08-18
UA104131C2 (uk) 2014-01-10
RU2010120715A (ru) 2011-11-27
IL204992A0 (en) 2010-11-30
SG185295A1 (en) 2012-11-29
US20100298411A1 (en) 2010-11-25
CN101835497A (zh) 2010-09-15
CA2703496A1 (en) 2009-04-30
CN104726458A (zh) 2015-06-24
KR20100106314A (ko) 2010-10-01
JP5887648B2 (ja) 2016-03-16
AU2008314860B2 (en) 2014-01-30
WO2009054551A2 (en) 2009-04-30
BRPI0818846A2 (pt) 2015-04-14

Similar Documents

Publication Publication Date Title
CO6270240A2 (es) Arn bicatenario con lipido modificado que tiene un potente efecto de interferencia del arn
CO2017007335A2 (es) Supresión del gen de la huntingtina inducida por la arni
ES2618783T3 (es) Construcción de ADN inmunomodulador no codificante
NZ631512A (en) Compositions and methods for modulating apolipoprotein (a) expression
EA201001045A1 (ru) Модифицированный вирус гриппа
HRP20070012A2 (en) Immunostimulatory oligonucleotide multimers
ES2383716T3 (es) Composiciones y métodos que utilizan ARN de interferencia de un gen del tipo OPR3 para el control de nemátodos
AR069821A1 (es) Composiciones y metodos de uso de la interferencia de arn para el control de nematodos
AR100946A1 (es) Composiciones y métodos para regular la expresión génica por medio de la interferencia de arn
WO2010134939A3 (en) Mammalian genes involved in infection
CU20200054A7 (es) Composiciones de edb que se dirigen a il-12
IN2014MN01838A (es)
WO2010039778A3 (en) Mammalian genes involved in infection
WO2019147308A3 (en) Rna nanostructures and methods of making and using rna nanostructures
AR059005A1 (es) Virus quimericos del dengue
AR112230A1 (es) Fósforo y una composición
AR098819A1 (es) Método y composiciones para el control de insectos plaga en plantas por medio del silenciamiento de genes de la familia de la quitina sintasa y de la vitelogenina así como alternativamente por la expresión del gen de una toxina cry
PH12019502063A1 (en) Nucleic acid derivative having immunostimulatory activity
GT200500106A (es) Derivados de benceno halogenados sustituidos con trimeros macrociclicos
ES2647576T3 (es) Prueba genética para la predisposición genética a la hemofilia A en bichones habaneros
JP2015523858A5 (es)
ES2530716T3 (es) Especies de pestivirus
WO2013177595A3 (en) Recombinant influenza viruses and constructs and uses thereof
WO2010144908A3 (en) Mammalian genes involved in tularemia and other infections
GT200300176A (es) Acidos nucleicos inmunoestimuladores

Legal Events

Date Code Title Description
FC Application refused