PL1850880T3 - Środki terapeutyczne na bazie aptamerów przydatne w leczeniu zaburzeń układu dopełniacza - Google Patents

Środki terapeutyczne na bazie aptamerów przydatne w leczeniu zaburzeń układu dopełniacza

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Publication number
PL1850880T3
PL1850880T3 PL06735059T PL06735059T PL1850880T3 PL 1850880 T3 PL1850880 T3 PL 1850880T3 PL 06735059 T PL06735059 T PL 06735059T PL 06735059 T PL06735059 T PL 06735059T PL 1850880 T3 PL1850880 T3 PL 1850880T3
Authority
PL
Poland
Prior art keywords
complement
treatment
related disorders
therapeutics useful
aptamer therapeutics
Prior art date
Application number
PL06735059T
Other languages
English (en)
Inventor
Claude Benedict
David Epstein
Charles Wilson
Dilara Grate
Jeffrey Kurz
Markus Kurz
Thomas Green Mccauley
James Rottman
Original Assignee
Archemix Llc
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 Archemix Llc filed Critical Archemix Llc
Publication of PL1850880T3 publication Critical patent/PL1850880T3/pl

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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
    • 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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • 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
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/12—Antidiuretics, e.g. drugs for diabetes insipidus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N2310/16—Aptamers
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • 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
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Animal Behavior & Ethology (AREA)
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  • Medicinal Chemistry (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Wood Science & Technology (AREA)
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  • Epidemiology (AREA)
  • Immunology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hematology (AREA)
  • Biochemistry (AREA)
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  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Urology & Nephrology (AREA)
  • Emergency Medicine (AREA)
  • Neurosurgery (AREA)
  • Vascular Medicine (AREA)
  • Obesity (AREA)
  • Endocrinology (AREA)
  • Hospice & Palliative Care (AREA)
PL06735059T 2005-02-14 2006-02-14 Środki terapeutyczne na bazie aptamerów przydatne w leczeniu zaburzeń układu dopełniacza PL1850880T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11/058,134 US7803931B2 (en) 2004-02-12 2005-02-14 Aptamer therapeutics useful in the treatment of complement-related disorders
EP06735059.5A EP1850880B1 (en) 2005-02-14 2006-02-14 Aptamer therapeutics useful in the treatment of complement-related disorders
PCT/US2006/005215 WO2006088888A2 (en) 2005-02-14 2006-02-14 Aptamer therapeutics useful in the treatment of complement-related disorders

Publications (1)

Publication Number Publication Date
PL1850880T3 true PL1850880T3 (pl) 2014-04-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PL06735059T PL1850880T3 (pl) 2005-02-14 2006-02-14 Środki terapeutyczne na bazie aptamerów przydatne w leczeniu zaburzeń układu dopełniacza

Country Status (21)

Country Link
US (8) US7803931B2 (pl)
EP (2) EP1850880B1 (pl)
JP (1) JP5253818B2 (pl)
KR (4) KR101622490B1 (pl)
CN (4) CN108148840B (pl)
AU (1) AU2006214437C1 (pl)
BR (1) BRPI0607002B1 (pl)
CA (4) CA3124030A1 (pl)
CY (1) CY1115059T1 (pl)
DK (1) DK1850880T3 (pl)
ES (1) ES2449047T3 (pl)
HK (1) HK1207661A1 (pl)
IL (2) IL185312A (pl)
MX (1) MX2007009807A (pl)
NZ (1) NZ560804A (pl)
PL (1) PL1850880T3 (pl)
PT (1) PT1850880E (pl)
RU (1) RU2406507C2 (pl)
SI (1) SI1850880T1 (pl)
WO (1) WO2006088888A2 (pl)
ZA (1) ZA200707191B (pl)

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6395888B1 (en) * 1996-02-01 2002-05-28 Gilead Sciences, Inc. High affinity nucleic acid ligands of complement system proteins
NZ592039A (en) * 2003-08-27 2013-03-28 Ophthotech Corp Combination therapy for the treatment of ocular neovascular disorders
US7803931B2 (en) 2004-02-12 2010-09-28 Archemix Corp. Aptamer therapeutics useful in the treatment of complement-related disorders
HUE038239T2 (hu) * 2004-02-12 2018-10-29 Archemix Llc Komplementrendszerrel összefüggõ rendellenességek kezelésében alkalmazható aptamer terapeutikumok
NZ571791A (en) * 2006-03-08 2012-03-30 Archemix Llc Complement binding aptamers and anti-C5 agents useful in the treatment of ocular disorders
AU2013204622B2 (en) * 2006-03-08 2016-04-21 Archemix Llc Complement Binding Aptamers and Anti-C5 Agents Useful in the Treatment of Ocular Disorders
AU2016204713B2 (en) * 2006-03-08 2017-08-10 Astellas Us Llc Complement Binding Aptamers and Anti-C5 Agents Useful in the Treatment of Ocular Disorders
CN101553244B (zh) * 2006-07-21 2013-01-02 普罗米克斯有限公司 内膜增生和相关病状的治疗
WO2008034123A2 (en) * 2006-09-15 2008-03-20 Enzon Pharmaceuticals, Inc. Polymeric conjugates containing positively-charged moieties
EP2562256A3 (en) * 2007-09-24 2013-06-19 Noxxon Pharma AG C5a binding nucleic acids
EP2340028B1 (en) * 2008-09-30 2018-06-27 The Trustees Of The University Of Pennsylvania Method of inhibiting biomaterial-induced procoagulant activity using complement inhibitors
CA2742802C (en) * 2008-11-10 2019-11-26 Alexion Pharmaceuticals, Inc. Methods and compositions for treating complement-associated disorders
WO2010108657A2 (en) * 2009-03-23 2010-09-30 Noxxon Pharma Ag C5a binding nucleic acids and the use thereof
KR102034203B1 (ko) * 2012-01-10 2019-10-18 아프타리온 바이오테크 아게 새로운 C5a 결합 핵산
EP3511339B1 (en) 2012-02-20 2025-04-09 IPC Research, LLC Polypeptides binding to human complement c5
MX357540B (es) * 2012-04-06 2018-07-13 Omeros Corp Composiciones y metodos para inhibir masp-1 y/o masp-2 y/o masp-3 para el tratamiento de la hemoglobinuria paroxistica nocturna.
EP3722320A3 (en) 2012-10-25 2020-12-30 Bioverativ USA Inc. Anti-complement c1s antibodies and uses thereof
DK2914291T3 (da) 2012-11-02 2022-05-16 Bioverativ Usa Inc Anti-komplement-c1s-antistoffer og anvendelser deraf
RU2523450C1 (ru) * 2013-05-06 2014-07-20 Федеральное государственное бюджетное учреждение "Научно-исследовательский институт комплексных проблем сердечно-сосудистых заболеваний" Сибирского отделения Российской академии медицинских наук (ФГБУ "НИИ КПССЗ" СО РАМН) Способ профликатики ранней послеоперационной когнитивной дисфункции у пациентов после коронарного шунтирования в условиях искуственного кровообращения
DK4374873T3 (da) 2013-07-12 2025-11-03 Astellas Us Llc Middel til brug ved behandling eller forebyggelse af oftalmologiske tilstande
DE102013112915A1 (de) 2013-11-22 2015-05-28 Universitätsklinikum Hamburg-Eppendorf (UKE) DNA-Aptamere, die E- und P-Selektine spezifisch binden
EP3973994A1 (en) 2014-06-12 2022-03-30 RA Pharmaceuticals, Inc. Modulation of complement activity
GB201411150D0 (en) 2014-06-23 2014-08-06 Altermune Technologies Llc Novel aptamers and therapeutic uses thereof
US9937222B2 (en) 2015-01-28 2018-04-10 Ra Pharmaceuticals, Inc. Modulators of complement activity
CA2981321A1 (en) 2015-04-06 2016-10-13 True North Therapeutics, Inc. Humanized anti-c1s antibodies and methods of use thereof
RU2733720C2 (ru) 2015-12-16 2020-10-06 Ра Фармасьютикалз, Инк. Модуляторы активности комплемента
DE102016100039A1 (de) 2016-01-04 2017-07-06 Universitätsklinikum Hamburg-Eppendorf (UKE) α6-Integrin bindendes DNA-Aptamer
WO2017127761A1 (en) 2016-01-20 2017-07-27 Vitrisa Therapeutics, Inc. Compositions and methods for inhibiting factor d
CN110300520B (zh) 2016-10-12 2022-10-04 美国比奥维拉迪维股份有限公司 抗C1s抗体及其使用方法
KR20190093196A (ko) 2016-12-07 2019-08-08 라 파마슈티컬스 인코포레이티드 보체 활성의 조절인자
WO2018136827A1 (en) 2017-01-20 2018-07-26 Vitrisa Therapeutics, Inc. Stem-loop compositions and methods for inhibiting factor d
WO2019022986A1 (en) * 2017-07-24 2019-01-31 Vitrisa Therapeutics, Inc. NUCLEIC ACID COMPOSITIONS AND METHODS OF INHIBITING D-FACTOR
JP2022535411A (ja) 2019-06-04 2022-08-08 ラ ファーマシューティカルズ インコーポレイテッド 補体阻害剤による炎症性疾患治療
AU2023413061A1 (en) * 2022-12-23 2025-05-08 Astellas Us Llc Pharmaceutical compositions for delivery to the eye
WO2025176843A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof
WO2026027707A1 (en) 2024-08-02 2026-02-05 Sandoz Ag Pre-filled syringe containing avacincaptad formulation

Family Cites Families (113)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3710795A (en) * 1970-09-29 1973-01-16 Alza Corp Drug-delivery device with stretched, rate-controlling membrane
US5212071A (en) * 1988-04-01 1993-05-18 The Johns Hopkins University Nucleic acids encoding a human C3b/C4b receptor (CR1)
US5981481A (en) * 1974-12-06 1999-11-09 The Johns Hopkins University Human C3b/C4b receptor (CR1)
US4816567A (en) * 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US4959309A (en) * 1983-07-14 1990-09-25 Molecular Diagnostics, Inc. Fast photochemical method of labelling nucleic acids for detection purposes in hybridization assays
US4683195A (en) * 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
DE229046T1 (de) * 1985-03-30 1987-12-17 Marc Genf/Geneve Ballivet Verfahren zum erhalten von dns, rns, peptiden, polypeptiden oder proteinen durch dns-rekombinant-verfahren.
US4935363A (en) * 1987-03-30 1990-06-19 Board Of Regents, The University Of Texas System Sterol regulatory elements
WO1989006694A1 (en) 1988-01-15 1989-07-27 Trustees Of The University Of Pennsylvania Process for selection of proteinaceous substances which mimic growth-inducing molecules
US5256642A (en) * 1988-04-01 1993-10-26 The Johns Hopkins University Compositions of soluble complement receptor 1 (CR1) and a thrombolytic agent, and the methods of use thereof
US5244805A (en) * 1989-05-17 1993-09-14 University Of Georgia Research Foundation, Inc. Baculovirus expression vectors
US5070010A (en) * 1989-10-30 1991-12-03 Hoffman-La Roche Inc. Method for determining anti-viral transactivating activity
JPH05501060A (ja) 1990-03-21 1993-03-04 アイシス・ファーマシューティカルス・インコーポレーテッド Ｒｎａ模倣による遺伝子発現活性変調のための試薬および方法
US5707796A (en) * 1990-06-11 1998-01-13 Nexstar Pharmaceuticals, Inc. Method for selecting nucleic acids on the basis of structure
US5476766A (en) * 1990-06-11 1995-12-19 Nexstar Pharmaceuticals, Inc. Ligands of thrombin
US5705337A (en) 1990-06-11 1998-01-06 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: chemi-SELEX
US6395888B1 (en) 1996-02-01 2002-05-28 Gilead Sciences, Inc. High affinity nucleic acid ligands of complement system proteins
US5543293A (en) * 1990-06-11 1996-08-06 Nexstar Pharmaceuticals, Inc. DNA ligands of thrombin
US5496938A (en) * 1990-06-11 1996-03-05 Nexstar Pharmaceuticals, Inc. Nucleic acid ligands to HIV-RT and HIV-1 rev
US5674685A (en) * 1990-06-11 1997-10-07 Nexstar Pharmaceuticals, Inc. High affinity PDGF nucleic acid ligands
US5660985A (en) * 1990-06-11 1997-08-26 Nexstar Pharmaceuticals, Inc. High affinity nucleic acid ligands containing modified nucleotides
US5668264A (en) 1990-06-11 1997-09-16 Nexstar Pharmaceuticals, Inc. High affinity PDGF nucleic acid ligands
US5459015A (en) * 1990-06-11 1995-10-17 Nexstar Pharmaceuticals, Inc. High-affinity RNA ligands of basic fibroblast growth factor
US5503978A (en) * 1990-06-11 1996-04-02 University Research Corporation Method for identification of high affinity DNA ligands of HIV-1 reverse transcriptase
US6011020A (en) * 1990-06-11 2000-01-04 Nexstar Pharmaceuticals, Inc. Nucleic acid ligand complexes
US5472841A (en) * 1990-06-11 1995-12-05 Nexstar Pharmaceuticals, Inc. Methods for identifying nucleic acid ligands of human neutrophil elastase
US5648214A (en) * 1990-06-11 1997-07-15 University Research Corporation High-affinity oligonucleotide ligands to the tachykinin substance P
US5527894A (en) * 1990-06-11 1996-06-18 Nexstar Pharmacueticals, Inc. Ligands of HIV-1 tat protein
US5789157A (en) * 1990-06-11 1998-08-04 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: tissue selex
US5763177A (en) * 1990-06-11 1998-06-09 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: photoselection of nucleic acid ligands and solution selex
US5861254A (en) * 1997-01-31 1999-01-19 Nexstar Pharmaceuticals, Inc. Flow cell SELEX
US6147204A (en) * 1990-06-11 2000-11-14 Nexstar Pharmaceuticals, Inc. Nucleic acid ligand complexes
US5726017A (en) * 1990-06-11 1998-03-10 Nexstar Pharmaceuticals, Inc. High affinity HIV-1 gag nucleic acid ligands
US6140490A (en) * 1996-02-01 2000-10-31 Nexstar Pharmaceuticals, Inc. High affinity nucleic acid ligands of complement system proteins
US5270163A (en) * 1990-06-11 1993-12-14 University Research Corporation Methods for identifying nucleic acid ligands
US5654151A (en) * 1990-06-11 1997-08-05 Nexstar Pharmaceuticals, Inc. High affinity HIV Nucleocapsid nucleic acid ligands
US5580737A (en) * 1990-06-11 1996-12-03 Nexstar Pharmaceuticals, Inc. High-affinity nucleic acid ligands that discriminate between theophylline and caffeine
US5635615A (en) * 1990-06-11 1997-06-03 Nexstar Pharmaceuticals, Inc. High affinity HIV nucleocapsid nucleic acid ligands
US5683867A (en) * 1990-06-11 1997-11-04 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: blended SELEX
US5763173A (en) * 1990-06-11 1998-06-09 Nexstar Pharmaceuticals, Inc. Nucleic acid ligand inhibitors to DNA polymerases
ES2259800T3 (es) * 1990-06-11 2006-10-16 Gilead Sciences, Inc. Procedimientos de uso de ligandos de acido nucleico.
US5567588A (en) * 1990-06-11 1996-10-22 University Research Corporation Systematic evolution of ligands by exponential enrichment: Solution SELEX
US5637459A (en) 1990-06-11 1997-06-10 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: chimeric selex
JPH06503715A (ja) 1990-09-21 1994-04-28 フレッド ハッチンソン キャンサー リサーチ センター タンパク質配列特異的オリゴヌクレオチド配列
ES2061416T3 (es) * 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
JPH06508022A (ja) 1991-02-21 1994-09-14 ギリアド　サイエンシズ，インコーポレイテッド 生体分子に特異的なアプタマーおよび生産方法
WO1992014842A1 (en) 1991-02-21 1992-09-03 Gilead Sciences, Inc. Aptamers specific for thrombin and methods of use
WO1994004679A1 (en) * 1991-06-14 1994-03-03 Genentech, Inc. Method for making humanized antibodies
US5252216A (en) * 1992-03-24 1993-10-12 Smithkline Beecham Corporation Protein purification
US5456909A (en) * 1992-08-07 1995-10-10 T Cell Sciences, Inc. Glycoform fractions of recombinant soluble complement receptor 1 (sCR1) having extended half-lives in vivo
US5338671A (en) 1992-10-07 1994-08-16 Eastman Kodak Company DNA amplification with thermostable DNA polymerase and polymerase inhibiting antibody
AU5442194A (en) 1992-10-14 1994-05-09 University Research Corporation Method for selecting nucleic acids on the basis of structure
US5262564A (en) * 1992-10-30 1993-11-16 Octamer, Inc. Sulfinic acid adducts of organo nitroso compounds useful as retroviral inactivating agents anti-retroviral agents and anti-tumor agents
EP0682526B1 (en) * 1993-02-12 2002-09-04 Avant Immunotherapeutics, Inc. PULMONARY ADMINISTRATION OF THE SOLUBLE COMPLEM RECEPTOR TYPE 1 sCR1
US5428149A (en) * 1993-06-14 1995-06-27 Washington State University Research Foundation Method for palladium catalyzed carbon-carbon coulping and products
RU2073518C1 (ru) 1993-06-17 1997-02-20 Совместное русско-американское акционерное общество закрытого типа "Неофарм" Средство, восстанавливающее функцию сетчатой оболочки глаза
WO1995001797A1 (en) * 1993-07-09 1995-01-19 Smithkline Beecham Corporation Protein purification
US5817635A (en) * 1993-08-09 1998-10-06 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
JPH09502354A (ja) 1993-09-08 1997-03-11 ネクスター ファーマスーティカルズ，インコーポレイテッド 核酸リガンドと、同リガンドを製造するための改良された方法
EP1889910A3 (en) 1993-09-17 2009-09-02 SomaLogic, Inc. Systematic evolution of ligands by exponential enrichment: photoselection of nucleic acid ligands and solution selex
US5919455A (en) * 1993-10-27 1999-07-06 Enzon, Inc. Non-antigenic branched polymer conjugates
US6074642A (en) * 1994-05-02 2000-06-13 Alexion Pharmaceuticals, Inc. Use of antibodies specific to human complement component C5 for the treatment of glomerulonephritis
EP0767657A4 (en) 1994-06-22 1999-01-20 Nexstar Pharmaceuticals Inc NOVEL PROCESS FOR THE PRODUCTION OF KNOWN AND NEW NUCLEOSIDES, MODIFIED IN THE 2 'POSITION BY INTRAMOLECULAR NUCLEOPHILIC DISPLACEMENT
US5681702A (en) 1994-08-30 1997-10-28 Chiron Corporation Reduction of nonspecific hybridization by using novel base-pairing schemes
US5932462A (en) * 1995-01-10 1999-08-03 Shearwater Polymers, Inc. Multiarmed, monofunctional, polymer for coupling to molecules and surfaces
US5723750A (en) * 1995-01-12 1998-03-03 Vanderbilt University Transgenic plants expressing disassembly deficient viral coat proteins
US6013443A (en) * 1995-05-03 2000-01-11 Nexstar Pharmaceuticals, Inc. Systematic evolution of ligands by exponential enrichment: tissue SELEX
JP4531132B2 (ja) * 1995-06-02 2010-08-25 ギリード・サイエンシズ・インコーポレーテッド 増殖因子に対する高親和性オリゴヌクレオチドリガンド
US6229002B1 (en) * 1995-06-07 2001-05-08 Nexstar Pharmaceuticlas, Inc. Platelet derived growth factor (PDGF) nucleic acid ligand complexes
US6020130A (en) * 1995-06-07 2000-02-01 Nexstar Pharmaceuticals, Inc. Nucleic acid ligands that bind to and inhibit DNA polymerases
US6111095A (en) 1995-06-07 2000-08-29 Merck & Co., Inc. Capped synthetic RNA, analogs, and aptamers
ATE468396T1 (de) 1996-02-01 2010-06-15 Gilead Sciences Inc Nukleinsäureliganden mit hoher affinität für das c1q protein des komplementsystems
US5668564A (en) * 1996-02-20 1997-09-16 R.A. Miller Industries, Inc. Combined AM/FM/cellular telephone antenna system
AU2994997A (en) 1996-05-07 1997-11-26 T Cell Sciences, Inc. Aptamers for complement protein c3b
RU2177950C2 (ru) 1996-10-25 2002-01-10 Нексстар Фармасьютикалз, Инк. Комплексы нуклеиновых кислот - лигандов сосудистого эндотелиального фактора роста (vegf)
US6051698A (en) * 1997-06-06 2000-04-18 Janjic; Nebojsa Vascular endothelial growth factor (VEGF) nucleic acid ligand complexes
US6278039B1 (en) * 1997-05-28 2001-08-21 Axys Pharmaceuticals, Inc. C. elegans deletion mutants
US7419663B2 (en) * 1998-03-20 2008-09-02 Genentech, Inc. Treatment of complement-associated disorders
US6251588B1 (en) * 1998-02-10 2001-06-26 Agilent Technologies, Inc. Method for evaluating oligonucleotide probe sequences
DK1140173T4 (da) 1998-12-22 2013-06-10 Genentech Inc Vaskulære endothelcellevækstfaktorantagonister og anvendelser deraf
US6329145B1 (en) 1999-02-09 2001-12-11 Gilead Science, Inc. Determining non-nucleic acid molecule binding to target by competition with nucleic acid ligand
US20020102581A1 (en) 1999-02-19 2002-08-01 Hageman Gregory S. Diagnostics and therapeutics for ocular disorders
US7306799B2 (en) * 1999-06-08 2007-12-11 Regeneron Pharmaceuticals, Inc. Use of VEGF inhibitors for treatment of eye disorders
US6573299B1 (en) * 1999-09-20 2003-06-03 Advanced Medical Instruments Method and compositions for treatment of the aging eye
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
AU2001263251A1 (en) 2000-05-17 2001-11-26 Gilead Sciences, Inc. Method for treatment of tumors using nucleic acid ligands to pdgf
EP1383782A1 (en) 2001-03-26 2004-01-28 Sirna Therpeutics, Inc. Oligonucleotide mediated inhibition of hepatitis b virus and hepatitis c virus replication
WO2003051458A1 (en) 2001-12-14 2003-06-26 Alcon, Inc. Superoxide dismutase mimics for the treatment of ocular disorders and diseases
RU2222345C2 (ru) 2002-01-21 2004-01-27 Волчек Игорь Анатольевич Фармацевтическая композиция цитокинового и иммуномодулирующего действия
AU2003208871A1 (en) 2002-02-18 2003-09-04 Lynkeus Bio Tech Gmbh Novel retina specific human proteins wdr17, net01, protein kinase a203, protein kinase mak a194, protein a105, protein a106 and c12orf3variants
US20040249130A1 (en) * 2002-06-18 2004-12-09 Martin Stanton Aptamer-toxin molecules and methods for using same
US20040022727A1 (en) * 2002-06-18 2004-02-05 Martin Stanton Aptamer-toxin molecules and methods for using same
WO2004041160A2 (en) 2002-10-30 2004-05-21 University Of Kentucky Research Foundation Methods and animal model for analyzing age-related macular degeneration
US7816497B2 (en) 2002-10-30 2010-10-19 University Of Kentucky Compositions and methods for inhibiting drusen complement components C3a and C5a for the treatment of age-related macular degeneration
US20040180360A1 (en) * 2002-11-21 2004-09-16 Charles Wilson Multivalent aptamer therapeutics with improved pharmacodynamic properties and methods of making and using the same
AU2003297682A1 (en) * 2002-12-03 2004-06-23 Archemix Corporation Method for in vitro selection of 2'-substituted nucleic acids
US20050037394A1 (en) * 2002-12-03 2005-02-17 Keefe Anthony D. Method for in vitro selection of 2'-substituted nucleic acids
AU2004206955A1 (en) 2003-01-21 2004-08-05 Archemix Corp. Aptamer therapeutics useful in ocular pharmacotherapy
CL2004001996A1 (es) 2003-08-08 2005-05-06 Eyetech Pharmaceuticals Inc Aptameros anti-vegf (factor de crecimiento endotelial vascular) con bloqueo nucleotidico 5'-5' o 3'-3' invertido, composicion que lo contiene, util para trastornos de neovascularizacion.
NZ592039A (en) 2003-08-27 2013-03-28 Ophthotech Corp Combination therapy for the treatment of ocular neovascular disorders
US8298532B2 (en) 2004-01-16 2012-10-30 Regeneron Pharmaceuticals, Inc. Fusion polypeptides capable of activating receptors
HUE038239T2 (hu) * 2004-02-12 2018-10-29 Archemix Llc Komplementrendszerrel összefüggõ rendellenességek kezelésében alkalmazható aptamer terapeutikumok
US7803931B2 (en) 2004-02-12 2010-09-28 Archemix Corp. Aptamer therapeutics useful in the treatment of complement-related disorders
WO2005084412A2 (en) * 2004-03-05 2005-09-15 Archemix Corp. Controlled modulation of the pharmacokinetics and biodistribution of aptamer therapeutics
US20070059336A1 (en) 2004-04-30 2007-03-15 Allergan, Inc. Anti-angiogenic sustained release intraocular implants and related methods
US7919094B2 (en) 2004-06-10 2011-04-05 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
US20080033053A1 (en) 2004-07-22 2008-02-07 Wolfgang Curt D Cross-Reference To Related Applications
EP2436391A3 (en) 2004-11-02 2012-07-04 Archemix LLC Stabilized aptamers to platelet derived growth factor and their use as oncology therapeutics
CA2613442C (en) * 2005-06-30 2016-08-23 Archemix Corp. Materials and methods for the generation of fully 2'-modified nucleic acid transcripts
NZ571791A (en) * 2006-03-08 2012-03-30 Archemix Llc Complement binding aptamers and anti-C5 agents useful in the treatment of ocular disorders
JP6587934B2 (ja) 2012-03-28 2019-10-09 ソマロジック・インコーポレーテッド Ｐｄｇｆおよびｖｅｇｆ結合アプタマー、および、ｐｄｇｆおよびｖｅｇｆが介在する病気の治療へのそれらの使用
JO3405B1 (ar) 2013-01-09 2019-10-20 Regeneron Pharma الأجسام المضادة لمضاد مستقبل عامل النمو المشتق من الصفائح الدموية - بيتا واستخداماتها
AU2017217677A1 (en) * 2016-02-08 2018-07-26 Vitrisa Therapeutics, Inc. Compositions with improved intravitreal half-life and uses thereof

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