CA2720363A1 - Compositions and methods for delivering inhibitory oligonucleotides - Google Patents

Compositions and methods for delivering inhibitory oligonucleotides Download PDF

Info

Publication number
CA2720363A1
CA2720363A1 CA2720363A CA2720363A CA2720363A1 CA 2720363 A1 CA2720363 A1 CA 2720363A1 CA 2720363 A CA2720363 A CA 2720363A CA 2720363 A CA2720363 A CA 2720363A CA 2720363 A1 CA2720363 A1 CA 2720363A1
Authority
CA
Canada
Prior art keywords
nucleic acid
cell
complex
rna
targeting polypeptide
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.)
Abandoned
Application number
CA2720363A
Other languages
English (en)
French (fr)
Inventor
Bira Arya
Purevdorj Olkhanud
Juan Espinoza
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.)
US Department of Health and Human Services
Original Assignee
US 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 US Department of Health and Human Services filed Critical US Department of Health and Human Services
Publication of CA2720363A1 publication Critical patent/CA2720363A1/en
Abandoned legal-status Critical Current

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)
CA2720363A 2008-04-15 2009-04-15 Compositions and methods for delivering inhibitory oligonucleotides Abandoned CA2720363A1 (en)

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

Publications (1)

Publication Number Publication Date
CA2720363A1 true CA2720363A1 (en) 2009-10-22

Family

ID=41172206

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2720363A Abandoned CA2720363A1 (en) 2008-04-15 2009-04-15 Compositions and methods for delivering inhibitory oligonucleotides

Country Status (6)

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5667043B2 (ja) 2008-04-15 2015-02-12 アメリカ合衆国ＧＯＶＥＲＮＭＥＮＴ ＯＦ ＴＨＥ ＵＮＩＴＥＤ ＳＴＡＴＥＳ ＯＦ ＡＭＥＲＩＣＡ， ａｓ ｒｅｐｒｅｓｅｎｔｅｄ ｂｙ ＴＨＥ ＳＥＣＲＥＴＡＲＹ， ＤＥＰＡＲＴＭＥＮＴ ＯＦ ＨＥＡＬＴＨＡＮＤ ＨＵＭＡＮ ＳＥＲＶＩＣＥＳ 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
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
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
WO2018094195A1 (en) 2016-11-21 2018-05-24 Tribiotica Llc Methods for directed folding assembly or dimerization of proteins by templated assembly reactions
US11186839B2 (en) 2016-11-21 2021-11-30 Tribiotica Llc Methods for preventing titration of bimolecular templated assembly reactions by structurally-determined differential hybridizations
JP6661797B2 (ja) * 2016-11-22 2020-03-11 株式会社東芝 核酸凝縮ペプチド、核酸凝縮ペプチドセット、核酸送達キャリア、核酸送達方法、細胞作製方法、細胞検出方法及びキット
JP7506601B2 (ja) 2017-12-06 2024-06-26 アビディティー バイオサイエンシーズ，インク． 筋萎縮症と筋緊張性ジストロフィーを処置するための組成物および方法
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy

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.
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
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
CA2297070A1 (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
JP2003528024A (ja) 1997-12-16 2003-09-24 バレンティス・インコーポレーテッド 製剤化された核酸分子の無針注入
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
DE60040274D1 (de) 1999-03-10 2008-10-30 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
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
ES2327382T3 (es) 1999-07-20 2009-10-29 Morphosys Ag Metodos para presentar (poli)peptidos/proteinas en particulas de bacteriofagos a traves de enlaces disulfuro.
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
EP1635832A2 (en) 2003-06-06 2006-03-22 Merck & Co., Inc. 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
CA2624662A1 (en) 2005-09-30 2007-04-12 The Government Of The United States Of America, As Represented By The Se Cretary 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
EP2121929B1 (en) * 2007-01-26 2011-11-23 Industry-University Cooperation Foundation Hanyang University Targeted delivery of sirna
JP5667043B2 (ja) 2008-04-15 2015-02-12 アメリカ合衆国ＧＯＶＥＲＮＭＥＮＴ ＯＦ ＴＨＥ ＵＮＩＴＥＤ ＳＴＡＴＥＳ ＯＦ ＡＭＥＲＩＣＡ， ａｓ ｒｅｐｒｅｓｅｎｔｅｄ ｂｙ ＴＨＥ ＳＥＣＲＥＴＡＲＹ， ＤＥＰＡＲＴＭＥＮＴ ＯＦ ＨＥＡＬＴＨＡＮＤ ＨＵＭＡＮ ＳＥＲＶＩＣＥＳ 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

Also Published As

Publication number Publication date
WO2009129281A3 (en) 2010-12-16
JP2011520424A (ja) 2011-07-21
US9415116B2 (en) 2016-08-16
US20140377179A1 (en) 2014-12-25
EP2276511A2 (en) 2011-01-26
US20110150768A1 (en) 2011-06-23
AU2009236270B2 (en) 2014-06-26
JP5667043B2 (ja) 2015-02-12
WO2009129281A2 (en) 2009-10-22
JP2015096073A (ja) 2015-05-21
US10765694B2 (en) 2020-09-08
US20160310609A1 (en) 2016-10-27
AU2009236270A1 (en) 2009-10-22
US8703921B2 (en) 2014-04-22

Similar Documents

Publication Publication Date Title
US10765694B2 (en) Compositions and methods for delivering inhibitory oligonucleotides
US20250002558A1 (en) Constitutively active cytokine receptors for cell therapy
US10590417B2 (en) RNA ligand-displaying exosomes for specific delivery of therapeutics to cell by RNA nanotechnology
US20030055014A1 (en) Inhibition of angiogenesis by nucleic acids
US20100016409A1 (en) Rna Nanoparticles and Nanotubes
EP3269811B1 (en) Nucleic acid having adjuvant activity and use thereof
KR20140051182A (ko) 특정한 키토산-기재의 나노복합체를 사용하여 ｓｉｒｎａ를 효과적이고 안전하게 전달하기 위한 조성물 및 방법
WO2003097835A2 (en) Pharmaceutical compositions for the treatment of cancer
CN117343965B (zh) 核酸载体、核酸药及其制备方法和应用
AU2016336868B2 (en) CXCR6-transduced T cells for targeted tumor therapy
Mao et al. Targeting fibroblast activation protein with chimeric antigen receptor macrophages
Yang et al. Engineering M1 macrophages with targeting aptamers for enhanced adoptive immunotherapy by modifying the cell surface
CA3146298A1 (en) T cell receptors and methods of use thereof
CA2734389A1 (en) S0x9, prostaglandin d2 and retinoic acid for treating pigmentary conditions and melanoma
EP3974531A1 (en) Asymmetric sirna inhibiting expression of pd-1
AU2014221306A1 (en) Compositions and methods for delivering inhibitory oligonucleotides
RU2757502C1 (ru) Генетическая кассета, содержащая кодон-оптимизированные нуклеотидные последовательности генов TRAIL, PTEN и IFNβ-1, и фармацевтическая композиция для лечения онкологических заболеваний
US20230277628A1 (en) Methods for promoting anti-tumor immune response in a subject in need thereof using encapsulated interleukin 12
WO2025250826A1 (en) Persistent t-cell engagers targeting fshr and methods of use in cancer therapeutics
WO2025250823A1 (en) Dna-encoded multivalent antibodies targeting carbonic anhydrase 9 and methods of use in cancer therapeutics

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20140228

FZDE Discontinued

Effective date: 20190416