JP5667043B2 - 阻害性オリゴヌクレオチドを送達するための組成物および方法 - Google Patents

阻害性オリゴヌクレオチドを送達するための組成物および方法 Download PDF

Info

Publication number
JP5667043B2
JP5667043B2 JP2011505154A JP2011505154A JP5667043B2 JP 5667043 B2 JP5667043 B2 JP 5667043B2 JP 2011505154 A JP2011505154 A JP 2011505154A JP 2011505154 A JP2011505154 A JP 2011505154A JP 5667043 B2 JP5667043 B2 JP 5667043B2
Authority
JP
Japan
Prior art keywords
seq
complex
polypeptide
nucleic acid
rna
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Fee Related
Application number
JP2011505154A
Other languages
English (en)
Japanese (ja)
Other versions
JP2011520424A (ja
JP2011520424A5 (enExample
Inventor
アリャ,ビラ
オークハヌド,ピュレヴドルジ
エスピノザ,ホアン
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Government Of United States, REPRESENTED BY SECRETARY Department Of Health And Human Services AS
United States Department of Health and Human Services
Original Assignee
Government Of United States, REPRESENTED BY SECRETARY Department Of Health And Human Services AS
United States Department of Health and Human Services
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 Government Of United States, REPRESENTED BY SECRETARY Department Of Health And Human Services AS, United States Department of Health and Human Services filed Critical Government Of United States, REPRESENTED BY SECRETARY Department Of Health And Human Services AS
Publication of JP2011520424A publication Critical patent/JP2011520424A/ja
Publication of JP2011520424A5 publication Critical patent/JP2011520424A5/ja
Application granted granted Critical
Publication of JP5667043B2 publication Critical patent/JP5667043B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/642—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent the peptide or protein in the drug conjugate being a cytokine, e.g. IL2, chemokine, growth factors or interferons being the inactive part of the conjugate
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • 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
    • A61P31/12—Antivirals
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/521—Chemokines
    • C07K14/523—Beta-chemokines, e.g. RANTES, I-309/TCA-3, MIP-1alpha, MIP-1beta/ACT-2/LD78/SCIF, MCP-1/MCAF, MCP-2, MCP-3, LDCF-1, LDCF-2
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Oncology (AREA)
  • Toxicology (AREA)
  • Reproductive Health (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Communicable Diseases (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2011505154A 2008-04-15 2009-04-15 阻害性オリゴヌクレオチドを送達するための組成物および方法 Expired - Fee Related JP5667043B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4508808P 2008-04-15 2008-04-15
US61/045,088 2008-04-15
PCT/US2009/040607 WO2009129281A2 (en) 2008-04-15 2009-04-15 Compositions and methods for delivering inhibitory oligonucleotides

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2014250963A Division JP2015096073A (ja) 2008-04-15 2014-12-11 阻害性オリゴヌクレオチドを送達するための組成物および方法

Publications (3)

Publication Number Publication Date
JP2011520424A JP2011520424A (ja) 2011-07-21
JP2011520424A5 JP2011520424A5 (enExample) 2012-06-07
JP5667043B2 true JP5667043B2 (ja) 2015-02-12

Family

ID=41172206

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2011505154A Expired - Fee Related JP5667043B2 (ja) 2008-04-15 2009-04-15 阻害性オリゴヌクレオチドを送達するための組成物および方法
JP2014250963A Pending JP2015096073A (ja) 2008-04-15 2014-12-11 阻害性オリゴヌクレオチドを送達するための組成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2014250963A Pending JP2015096073A (ja) 2008-04-15 2014-12-11 阻害性オリゴヌクレオチドを送達するための組成物および方法

Country Status (6)

Country Link
US (3) US8703921B2 (enExample)
EP (1) EP2276511A2 (enExample)
JP (2) JP5667043B2 (enExample)
AU (1) AU2009236270B2 (enExample)
CA (1) CA2720363A1 (enExample)
WO (1) WO2009129281A2 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8703921B2 (en) 2008-04-15 2014-04-22 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Compositions and methods for delivering inhibitory oligonucleotides
US10485879B2 (en) 2008-04-15 2019-11-26 Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services, National Institutes Of Health Plasma cell cytokine vehicle containing fusion proteins for targeted introduction of siRNA into cells and tissues
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
WO2018094070A1 (en) 2016-11-21 2018-05-24 Tribiotica Llc Methods for preventing titration of bimolecular templated assembly reactions by structurally-determined differential hybridizations
WO2018094195A1 (en) * 2016-11-21 2018-05-24 Tribiotica Llc Methods for directed folding assembly or dimerization of proteins by templated assembly reactions
WO2018096457A1 (en) * 2016-11-22 2018-05-31 Kabushiki Kaisha Toshiba Nucleic acid condensing peptide, nucleic acid condensing peptide set, nucleic acid delivery carrier, nucleic acid delivery method, cell production method, cell detection method and kit
CN118638787A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5108921A (en) 1989-04-03 1992-04-28 Purdue Research Foundation Method for enhanced transmembrane transport of exogenous molecules
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US6172208B1 (en) 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
DE614989T1 (de) 1993-02-17 1995-09-28 Morphosys Proteinoptimierung Verfahren für in vivo Selektion von Ligandenbindende Proteine.
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
JP3659261B2 (ja) 1994-10-20 2005-06-15 モルフォシス・アクチェンゲゼルシャフト 組換体タンパク質の多機能性複合体への標的化ヘテロ結合
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
WO1999006587A2 (en) 1997-08-01 1999-02-11 Morphosys Ag Novel method and phage for the identification of nucleic acid sequences encoding members of a multimeric (poly)peptide complex
GB9722131D0 (en) 1997-10-20 1997-12-17 Medical Res Council Method
EP1038016A2 (en) 1997-12-16 2000-09-27 Valentis Inc. Needle-free injection of formulated nucleic acid molecules
AR020078A1 (es) 1998-05-26 2002-04-10 Syngenta Participations Ag Metodo para alterar la expresion de un gen objetivo en una celula de planta
WO2000053722A2 (en) 1999-03-10 2000-09-14 Phogen Limited Delivery of nucleic acids and proteins to cells
ATE482275T1 (de) 1999-07-02 2010-10-15 Morphosys Ag Erzeugung spezifischer bindungspartner die an von genomischen dns-fragmenten oder ests kodierten (poly)peptiden binden
CA2347973C (en) 1999-07-20 2010-06-22 Morphosys Ag Novel methods for displaying (poly)peptides/proteins on bacteriophage particles via disulfide bonds
US20070042983A1 (en) 2001-05-18 2007-02-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
KR20040008526A (ko) 2002-07-18 2004-01-31 주식회사 하이닉스반도체 플래시 메모리 셀의 소거 방법
AU2003256857A1 (en) 2002-08-08 2004-02-25 Dharmacon, Inc. Short interfering rnas having a hairpin structure containing a non-nucleotide loop
US20070099884A1 (en) 2003-06-06 2007-05-03 Erondu Ngozi E Combination therapy for the treatment of diabetes
WO2006023491A2 (en) * 2004-08-16 2006-03-02 The Cbr Institute For Biomedical Research, Inc. Method of delivering rna interference and uses thereof
EP1937311A2 (en) * 2005-09-30 2008-07-02 The Government of the United States of America, as represented by the Secretary of the Department of Health and Human Services Methods and compositions for modulating immune tolerance
US20070231392A1 (en) 2006-01-23 2007-10-04 Ernst Wagner CHEMICALLY MODIFIED POLYCATION POLYMER FOR siRNA DELIVERY
US20130129752A1 (en) * 2006-04-25 2013-05-23 Immune Disease Institute, Inc. Targeted delivery to leukocytes using protein carriers
ATE534737T1 (de) 2007-01-26 2011-12-15 Iucf Hyu Gezielte abgabe von sirna
US8703921B2 (en) 2008-04-15 2014-04-22 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Compositions and methods for delivering inhibitory oligonucleotides

Also Published As

Publication number Publication date
AU2009236270B2 (en) 2014-06-26
US10765694B2 (en) 2020-09-08
AU2009236270A1 (en) 2009-10-22
US8703921B2 (en) 2014-04-22
WO2009129281A2 (en) 2009-10-22
WO2009129281A3 (en) 2010-12-16
US20110150768A1 (en) 2011-06-23
CA2720363A1 (en) 2009-10-22
US9415116B2 (en) 2016-08-16
JP2011520424A (ja) 2011-07-21
EP2276511A2 (en) 2011-01-26
US20140377179A1 (en) 2014-12-25
US20160310609A1 (en) 2016-10-27
JP2015096073A (ja) 2015-05-21

Similar Documents

Publication Publication Date Title
US10765694B2 (en) Compositions and methods for delivering inhibitory oligonucleotides
US10590417B2 (en) RNA ligand-displaying exosomes for specific delivery of therapeutics to cell by RNA nanotechnology
US9809821B2 (en) Compositions and methods for efficacious and safe delivery of siRNA using specific chitosan-based nanocomplexes
CN103003427B (zh) 具有佐剂活性的新型核酸及其应用
JP2023531509A (ja) Il-2コンジュゲートおよび抗egfr抗体を用いる免疫腫瘍学併用療法
CN117343965B (zh) 核酸载体、核酸药及其制备方法和应用
EP3600379B1 (en) Prevention and treatment of non-melanoma skin cancer (nmsc)
KR20180104692A (ko) Angpt2 및 pdgfb를 표적화하는 rna 복합체를 사용하는 혈관신생 관련 질환의 치료
US20250270250A1 (en) Spherical nucleic acids with tailored and active protein coronae
WO2008039254A2 (en) Rna nanoparticles and nanotubes
Mao et al. Targeting fibroblast activation protein with chimeric antigen receptor macrophages
EP3265132A1 (en) Nanoparticle conjugates and uses thereof
Yang et al. Engineering M1 macrophages with targeting aptamers for enhanced adoptive immunotherapy by modifying the cell surface
AU2014221306A1 (en) Compositions and methods for delivering inhibitory oligonucleotides
CN101333237B (zh) 一种具有治疗乳腺癌作用的寡核苷酸
US11364305B2 (en) Nanoparticle conjugates and uses thereof
WO2025110115A1 (ja) Cxcl12 rna配列に基づく新規二本鎖rna及びその利用
KR20250025781A (ko) Rna를 함유하는 항종양제 및 그의 용도
JP2020070252A (ja) 腫瘍免疫賦活剤

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20120413

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20120413

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20140107

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20140407

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20140414

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20140507

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20141111

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20141211

R150 Certificate of patent or registration of utility model

Ref document number: 5667043

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

LAPS Cancellation because of no payment of annual fees