JP2012533587A - 自然に存在する細胞内輸送経路を介して化合物を送達するための送達システム及びコンジュゲート - Google Patents
自然に存在する細胞内輸送経路を介して化合物を送達するための送達システム及びコンジュゲート Download PDFInfo
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- JP2012533587A JP2012533587A JP2012520958A JP2012520958A JP2012533587A JP 2012533587 A JP2012533587 A JP 2012533587A JP 2012520958 A JP2012520958 A JP 2012520958A JP 2012520958 A JP2012520958 A JP 2012520958A JP 2012533587 A JP2012533587 A JP 2012533587A
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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
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- 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
- A61K47/6415—Toxins or lectins, e.g. clostridial toxins or Pseudomonas exotoxins
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- 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
- A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
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- 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]
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- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- 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
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
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- 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
- C12N2310/351—Conjugate
- C12N2310/3517—Marker; Tag
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- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US22766909P | 2009-07-22 | 2009-07-22 | |
| US61/227,669 | 2009-07-22 | ||
| PCT/EP2010/004512 WO2011009624A1 (en) | 2009-07-22 | 2010-07-22 | Delivery system and conjugates for compound delivery via naturally occurring intracellular transport routes |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2012533587A true JP2012533587A (ja) | 2012-12-27 |
| JP2012533587A5 JP2012533587A5 (enExample) | 2013-09-12 |
Family
ID=42731966
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012520958A Pending JP2012533587A (ja) | 2009-07-22 | 2010-07-22 | 自然に存在する細胞内輸送経路を介して化合物を送達するための送達システム及びコンジュゲート |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20120258104A1 (enExample) |
| EP (1) | EP2456470A1 (enExample) |
| JP (1) | JP2012533587A (enExample) |
| CA (1) | CA2768598A1 (enExample) |
| WO (1) | WO2011009624A1 (enExample) |
Cited By (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2014505064A (ja) * | 2011-01-26 | 2014-02-27 | セニックス バイオサイエンス ゲーエムベーハー | 自然に存在する細胞内輸送経路を介して化合物を送達するための送達システム及びコンジュゲート |
| JP2017513459A (ja) * | 2014-03-11 | 2017-06-01 | モレキュラー テンプレーツ, インク.Molecular Templates, Inc. | 結合領域、志賀毒素aサブユニットのエフェクター領域、及びカルボキシ末端小胞体局在化シグナルモチーフを含むタンパク質 |
| JP2017534598A (ja) * | 2014-09-30 | 2017-11-24 | ザ メディカル カレッジ オブ ウィスコンシン インクThe Medical College Of Wisconsin, Inc. | 神経系疾患を治療する標的療法のための汎用的なプラットフォーム |
| JP2018529386A (ja) * | 2015-10-05 | 2018-10-11 | シンジェンタ パーティシペーションズ アーゲー | リボ核酸の生物活性を保存する方法 |
| US11389542B1 (en) | 2016-12-07 | 2022-07-19 | Molecular Templates, Inc. | Shiga toxin a subunit effector polypeptides, Shiga toxin effector scaffolds, and cell-targeting molecules for site-specific conjugation |
| US12037367B2 (en) | 2014-01-27 | 2024-07-16 | Molecular Templates, Inc. | MHC class I epitope delivering polypeptides |
Families Citing this family (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11246915B2 (en) | 2010-09-15 | 2022-02-15 | Applied Molecular Transport Inc. | Cholix toxin-derived fusion molecules for oral delivery of biologically active cargo |
| EP2616045B1 (en) | 2010-09-15 | 2017-10-25 | Randall J. Mrsny | Systems and methods of delivery of bioactive agents using bacterial toxin-derived transport sequences |
| LT2646470T (lt) | 2010-11-30 | 2017-06-12 | F. Hoffmann-La Roche Ag | Mažo giminingumo antitransferino receptorių antikūnai ir jų panaudojimas pernešti gydomąjį vienos grandinės antikūną (scfv) per hematoencefalinį barjerą |
| WO2012113413A1 (en) * | 2011-02-21 | 2012-08-30 | Curevac Gmbh | Vaccine composition comprising complexed immunostimulatory nucleic acids and antigens packaged with disulfide-linked polyethyleneglycol/peptide conjugates |
| US9045750B2 (en) * | 2011-03-18 | 2015-06-02 | Yuelong Ma | Humanized lewis-Y specific antibody-based delivery of dicer substrate siRNA (D-siRNA) against STAT3 |
| HK1200484A1 (zh) | 2011-09-14 | 2015-08-07 | Translate Bio Ma, Inc. | 多聚寡核苷酸化合物 |
| WO2013103146A1 (ja) * | 2012-01-07 | 2013-07-11 | 株式会社ボナック | アミノ酸骨格を有する一本鎖核酸分子 |
| US9475840B2 (en) | 2012-02-27 | 2016-10-25 | Universitat De Barcelona | Protease-resistant compounds useful as shuttles through the blood-brain barrier and shuttle-cargo constructs |
| CA2873779A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics Inc. | Compositions and methods for modulating mecp2 expression |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| US10174323B2 (en) | 2012-05-16 | 2019-01-08 | The General Hospital Corporation | Compositions and methods for modulating ATP2A2 expression |
| AU2013262700A1 (en) | 2012-05-16 | 2015-01-22 | Rana Therapeutics, Inc. | Compositions and methods for modulating hemoglobin gene family expression |
| US10058623B2 (en) | 2012-05-16 | 2018-08-28 | Translate Bio Ma, Inc. | Compositions and methods for modulating UTRN expression |
| EP2850186B1 (en) | 2012-05-16 | 2018-12-19 | Translate Bio MA, Inc. | Compositions and methods for modulating smn gene family expression |
| EP2857513A4 (en) | 2012-05-26 | 2016-05-25 | Bonac Corp | Single-stranded nucleic acid molecule controlling the expression of a gene transporter |
| WO2014043544A1 (en) | 2012-09-14 | 2014-03-20 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| CA2902432A1 (en) | 2013-03-12 | 2014-10-09 | Molecular Templates, Inc. | Cytotoxic proteins comprising cell-targeting binding regions and shiga toxin a subunit regions for selective killing of specific cell types |
| DK3035969T3 (da) | 2013-08-19 | 2022-10-24 | S M Discovery Holdings Inc | Peptidsammensætning til nanofremføring målrettet to receptorer |
| CA2915728A1 (en) | 2013-08-21 | 2015-02-26 | Thomas Kramps | Respiratory syncytial virus (rsv) vaccine |
| EP3052632A4 (en) | 2013-10-04 | 2017-03-29 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| JP6486836B2 (ja) | 2013-12-26 | 2019-03-20 | 学校法人東京医科大学 | 遺伝子発現制御のための人工ミミックmiRNAおよびその用途 |
| RU2697094C2 (ru) | 2013-12-27 | 2019-08-12 | Бонак Корпорейшн | ИСКУССТВЕННАЯ мкРНК С СООТВЕТСТВИЕМ ДЛЯ КОНТРОЛЯ ЭКСПРЕССИИ ГЕНОВ И ЕЕ ПРИМЕНЕНИЕ |
| US11142584B2 (en) | 2014-03-11 | 2021-10-12 | Molecular Templates, Inc. | CD20-binding proteins comprising Shiga toxin A subunit effector regions for inducing cellular internalization and methods using same |
| US10273484B2 (en) | 2014-03-24 | 2019-04-30 | Riboxx Gmbh | Double-stranded RNA conjugates and their use |
| SI3139965T1 (sl) | 2014-05-07 | 2022-01-31 | Applied Molecular Transport Inc. | Fuzijske molekule, izpeljane iz toksina cholix, za peroralno dostavljanje biološko aktivnega tovora |
| JP6735237B2 (ja) | 2014-06-11 | 2020-08-05 | モレキュラー テンプレーツ, インク.Molecular Templates, Inc. | プロテアーゼ切断耐性、志賀毒素aサブユニットエフェクターポリペプチド及びそれを含む細胞標的化分子 |
| WO2016073488A1 (en) * | 2014-11-04 | 2016-05-12 | The Regents Of The University Of California | Targeted delivery platform for delivery of therapeutics |
| AU2015368293B2 (en) | 2014-12-27 | 2021-07-22 | Bonac Corporation | Naturally occuring miRNA for controlling gene expression, and use of same |
| JP6444486B2 (ja) | 2015-02-05 | 2018-12-26 | モレキュラー テンプレーツ, インク.Molecular Templates, Inc. | 志賀毒素aサブユニットエフェクター領域を含む多価cd20結合分子及びそれらの強化組成物 |
| HK1244031A1 (en) | 2015-03-27 | 2018-07-27 | Bonac Corporation | Single-chain nucleic acid molecule having delivery function and gene expression control ability |
| EP3303373B1 (en) | 2015-05-30 | 2020-04-08 | Molecular Templates, Inc. | De-immunized, shiga toxin a subunit scaffolds and cell-targeting molecules comprising the same |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| CN110536695B (zh) | 2017-01-25 | 2024-05-10 | 分子模板公司 | 包括去免疫化的志贺毒素a亚基效应子和cd8+ t细胞表位的细胞靶向分子 |
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| WO2019204272A1 (en) | 2018-04-17 | 2019-10-24 | Molecular Templates, Inc. | Her2-targeting molecules comprising de-immunized, shiga toxin a subunit scaffolds |
| US20210139892A1 (en) * | 2018-07-05 | 2021-05-13 | The Regents Of The University Of California | Compositions and methods for delivery of rna to a cell |
| IL282986B2 (en) | 2018-11-07 | 2024-01-01 | Applied Molecular Transport Inc | Colics-derivative carriers for oral administration of heterologous cargo |
| PL3650037T3 (pl) | 2018-11-07 | 2022-06-06 | Applied Molecular Transport Inc. | Konstrukty dostarczające do transcytozy i powiązane z nimi sposoby |
| CA3150859A1 (en) | 2019-08-16 | 2021-02-25 | Applied Molecular Transport Inc. | Compositions, formulations, and interleukin production and purification |
| EP3868882A1 (en) * | 2020-02-21 | 2021-08-25 | European Molecular Biology Laboratory | Archaeal pyrrolysyl trna synthetases for orthogonal use |
| MX2022011499A (es) | 2020-03-19 | 2022-10-07 | Avidity Biosciences Inc | Composiciones y metodos para tratar la distrofia muscular facioescapulohumeral. |
| US20230158061A1 (en) * | 2020-04-23 | 2023-05-25 | Cornell University | Nanotherapy Targeting RHAMM-Positive Tumors |
| CA3231330A1 (en) | 2021-09-16 | 2023-03-23 | Avidity Biosciences, Inc. | Compositions and methods of treating facioscapulohumeral muscular dystrophy |
| EP4422679A4 (en) * | 2021-10-26 | 2025-11-12 | Navicure Biopharmaceuticals Ltd | Immunogenic fusion proteins against coronavirus |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1997013529A1 (en) * | 1995-10-13 | 1997-04-17 | The Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services | Immunotoxin containing a disulfide-stabilized antibody fragment |
| WO1998020135A2 (en) * | 1996-11-06 | 1998-05-14 | The Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services | Protease-activatable pseudomonas exotoxin a-like proproteins |
| US20040071731A1 (en) * | 2000-12-21 | 2004-04-15 | Fitzgerald David J. | Chimeric protein comprising non-toxic pseudomonas exotoxin a and type iv pilin sequences |
| WO2008157263A2 (en) * | 2007-06-15 | 2008-12-24 | Arkansas State University | Methods of delivery of molecules to cells using a ricin subunit and compositions relating to same |
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| US4935233A (en) | 1985-12-02 | 1990-06-19 | G. D. Searle And Company | Covalently linked polypeptide cell modulators |
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| US4957773A (en) | 1989-02-13 | 1990-09-18 | Syracuse University | Deposition of boron-containing films from decaborane |
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| AU2002360353A1 (en) | 2001-12-11 | 2003-06-23 | University Of Iowa Research Foundation | Receptor-targeted adenoviral vectors |
| JP4597976B2 (ja) | 2003-04-17 | 2010-12-15 | アルナイラム ファーマシューティカルズ インコーポレイテッド | 修飾iRNA剤 |
| US8017762B2 (en) | 2003-04-17 | 2011-09-13 | Alnylam Pharmaceuticals, Inc. | Modified iRNA agents |
| CA2529752A1 (en) | 2003-06-20 | 2005-09-15 | The Regents Of The University Of California | Polypeptide transduction and fusogenic peptides |
| EP2205740A2 (en) | 2007-10-02 | 2010-07-14 | Rxi Pharmaceuticals Corp. | Tripartite rnai constructs |
-
2010
- 2010-07-22 WO PCT/EP2010/004512 patent/WO2011009624A1/en not_active Ceased
- 2010-07-22 US US13/386,347 patent/US20120258104A1/en not_active Abandoned
- 2010-07-22 CA CA2768598A patent/CA2768598A1/en not_active Abandoned
- 2010-07-22 JP JP2012520958A patent/JP2012533587A/ja active Pending
- 2010-07-22 EP EP10737781A patent/EP2456470A1/en not_active Withdrawn
Patent Citations (5)
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| WO1998020135A2 (en) * | 1996-11-06 | 1998-05-14 | The Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services | Protease-activatable pseudomonas exotoxin a-like proproteins |
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| US20040071731A1 (en) * | 2000-12-21 | 2004-04-15 | Fitzgerald David J. | Chimeric protein comprising non-toxic pseudomonas exotoxin a and type iv pilin sequences |
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| EP2456470A1 (en) | 2012-05-30 |
| CA2768598A1 (en) | 2011-01-27 |
| US20120258104A1 (en) | 2012-10-11 |
| WO2011009624A1 (en) | 2011-01-27 |
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