ES2647772T3 - Oligonucleótidos de fosfodiésteres sintéticos y sus usos terapéuticos - Google Patents

Oligonucleótidos de fosfodiésteres sintéticos y sus usos terapéuticos Download PDF

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Publication number
ES2647772T3
ES2647772T3 ES09721382.1T ES09721382T ES2647772T3 ES 2647772 T3 ES2647772 T3 ES 2647772T3 ES 09721382 T ES09721382 T ES 09721382T ES 2647772 T3 ES2647772 T3 ES 2647772T3
Authority
ES
Spain
Prior art keywords
defibrotide
pharmaceutical formulation
oligonucleotides
used according
bases
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.)
Active
Application number
ES09721382.1T
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English (en)
Spanish (es)
Inventor
Aaron Cy Stein
Massimo Iacobelli
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.)
Gentium SRL
Original Assignee
Gentium SRL
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 Gentium SRL filed Critical Gentium SRL
Application granted granted Critical
Publication of ES2647772T3 publication Critical patent/ES2647772T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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
    • 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
    • C12N15/117—Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • 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
    • 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
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • A61P9/10—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
    • C12N2310/17—Immunomodulatory nucleic acids
    • 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
    • C12N2320/31—Combination therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
ES09721382.1T 2008-03-19 2009-03-13 Oligonucleótidos de fosfodiésteres sintéticos y sus usos terapéuticos Active ES2647772T3 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP08425175A EP2103689A1 (en) 2008-03-19 2008-03-19 Synthetic phosphodiester oligonucleotides and therapeutical uses thereof
EP08425175 2008-03-19
US5108808P 2008-05-07 2008-05-07
US51088P 2008-05-07
PCT/EP2009/053002 WO2009115465A1 (en) 2008-03-19 2009-03-13 Synthetic phosphodiester oligonucleotides and therapeutical uses thereof

Publications (1)

Publication Number Publication Date
ES2647772T3 true ES2647772T3 (es) 2017-12-26

Family

ID=39683815

Family Applications (1)

Application Number Title Priority Date Filing Date
ES09721382.1T Active ES2647772T3 (es) 2008-03-19 2009-03-13 Oligonucleótidos de fosfodiésteres sintéticos y sus usos terapéuticos

Country Status (11)

Country Link
US (1) US20110092576A1 (enExample)
EP (2) EP2103689A1 (enExample)
JP (1) JP2011515357A (enExample)
KR (1) KR20100124820A (enExample)
CN (2) CN101978059A (enExample)
AU (1) AU2009226906B2 (enExample)
CA (1) CA2712705C (enExample)
DK (1) DK2252688T3 (enExample)
ES (1) ES2647772T3 (enExample)
IL (1) IL207032A (enExample)
WO (1) WO2009115465A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2637672T3 (en) * 2010-11-12 2018-10-22 Gentium S R L DEFIBROTID FOR USE IN PROPHYLAXY AND / OR TREATMENT OF GRAPHIC VERSUS HOST DISEASE (GVHD)
KR102038357B1 (ko) 2012-06-22 2019-10-31 젠티엄 에스알엘 디파이브로타이드의 생물학적 활성 측정을 위한 유우글로불린에 기초한 방법
EP3026122A1 (en) 2014-11-27 2016-06-01 Gentium S.p.A. Cellular-based method for determining the potency of defibrotide
CN111132663A (zh) 2017-08-03 2020-05-08 爵士制药爱尔兰有限公司 包含高浓度核酸的制剂
AU2019252790A1 (en) 2018-04-12 2020-10-15 Jazz Pharmaceuticals, Inc. Defibrotide for the prevention and treatment of cytokine release syndrome and neurotoxicity associated with immunodepletion
US20220023533A1 (en) 2018-12-07 2022-01-27 Jazz Phrmaceticals Ireland Limited Subcutaneous delivery of high concentration formulations
WO2021174039A1 (en) 2020-02-28 2021-09-02 Jazz Pharmaceuticals Ireland Limited Delivery of low viscosity formulations
EP4135717A1 (en) 2020-04-17 2023-02-22 Jazz Pharmaceuticals Ireland Limited Defibrotide treatment for the prevention of organ rejection and injury
TW202308659A (zh) 2021-05-06 2023-03-01 愛爾蘭商爵士製藥愛爾蘭有限責任公司 用於急性呼吸窘迫症候群之治療及預防的去纖維蛋白多核苷酸

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US178761A (en) * 1876-06-13 Improvement in refrigerating-cans
US2006A (en) * 1841-03-16 Clamp for crimping leather
US1325962A (en) * 1919-12-23 Geared lifting-jack
US876235A (en) * 1904-11-07 1908-01-07 Leonard G Quackenboss Adjustable flower-box.
US1276497A (en) * 1917-07-18 1918-08-20 Wencel H Kirchman Dirigible balloon.
IT1043823B (it) 1970-11-03 1980-02-29 Prephar Procedimento per l estrazione di acidi nucleici da organi animali
DE2154279A1 (de) 1970-11-03 1972-05-25 Crinos Industria Farmaco Medikamente für das fibrinolytische System
US3899481A (en) 1970-11-03 1975-08-12 Crinos Industria Farmaco Process for the controlled partial degradation of deoxyribonucleic acid extracted from animal organs
US4853221A (en) * 1980-11-13 1989-08-01 Warner-Lambert Company Method for treating non-small cell lung cancer, head and neck cancers and breast cancer
IT1170215B (it) * 1983-09-12 1987-06-03 Crinos Industria Farmaco Composizione farmaceutica per il trattamento di stati di insufficienza renale acuta
IT1170214B (it) 1983-09-12 1987-06-03 Crinos Industria Farmaco Composizione farmaceutica per la cura delle arteriopatie periferiche
IT1206341B (it) 1984-02-16 1989-04-14 Crinos Industria Farmaco Composizione farmaceutica per il trattamento dell'ischemia acuta del miocardio.
US4694134A (en) 1985-05-28 1987-09-15 Ajax Magnethermic Corporation Apparatus for overheating edges of skelp for the production of compression welded pipe
IT1190313B (it) * 1986-04-17 1988-02-16 Crinos Industria Farmaco Procedimento per l'ottenimento di polidesossiribonucleotidi chimicamente definiti e riproducibili e prodotto farmacologicamente attivo risultante
US5223609A (en) 1986-04-17 1993-06-29 Crinos Industria Farmacobiologica S.P.A. Process for obtaining chemically defined and reproducible polydeoxyribonucleotides
US4753221A (en) * 1986-10-22 1988-06-28 Intravascular Surgical Instruments, Inc. Blood pumping catheter and method of use
IT1231509B (it) 1989-09-07 1991-12-07 Crinos Industria Farmaco Composizione farmceutica ad uso topico per la terapia della fragilita' capillare.
US5166617A (en) * 1991-01-11 1992-11-24 Varian Associates, Inc. High power NMR probe
US6699985B2 (en) 1991-08-21 2004-03-02 Arsinur Burcoglu Method of treating HIV infection and related secondary infections thereof
US5624912A (en) 1991-08-21 1997-04-29 Burcoglu; Arsinur Method of treating HIV infection and related secondary infections with defibrotide
US5977083A (en) 1991-08-21 1999-11-02 Burcoglu; Arsinur Method for using polynucleotides, oligonucleotides and derivatives thereof to treat various disease states
IT1252174B (it) * 1991-12-09 1995-06-05 Crinos Industria Farmaco Oligodesossimibonucleotidi ad attivita' antiischemica e procedimenti per il loro ottenimento
US5578716A (en) * 1993-12-01 1996-11-26 Mcgill University DNA methyltransferase antisense oligonucleotides
US5856444A (en) * 1994-11-30 1999-01-05 Chugai Seiyaku Kabushiki Kaisha Thrombocytopoiesis stimulating factor
US6573372B2 (en) * 1999-01-07 2003-06-03 Heska Corporation Feline immunoglobulin E molecules and compositions there of
ES2251134T3 (es) * 1999-06-08 2006-04-16 Gentium S.P.A. Uso de complejos entre liposomas cationicos y polidesoxirribonucleotidos como medicamentos.
EP1147777A1 (en) * 2000-04-18 2001-10-24 Crinos Industria Farmacobiologica S.p.A. Combination of defibrotide and G-CSF and its use to activate haematopoietic progenitors
DE10046547A1 (de) 2000-09-19 2002-03-28 Innovat Ges Fuer Sondermaschb Vorrichtung zum induktiven Aufheizen von Werkstücken
CA2426540A1 (en) * 2000-10-20 2002-07-25 Biocardia, Inc. Leukocyte expression profiling
US7235358B2 (en) * 2001-06-08 2007-06-26 Expression Diagnostics, Inc. Methods and compositions for diagnosing and monitoring transplant rejection
AU2002353783A1 (en) * 2001-09-24 2003-04-07 The Government Of The United States Of America As Represented By The Secretary Of The Department Of SUPPRESSORS OF CpG OLIGONUCLEOTIDES AND METHODS OF USE
US6965025B2 (en) * 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
EP1325962A1 (en) * 2001-12-17 2003-07-09 Gentium S.p.A. A method for determining the biological activity of defibrotide
US20050215498A1 (en) * 2002-05-31 2005-09-29 Guenther Eissner Method for the protection of endothelial and epithclial cells during chemotherapy
US20050196382A1 (en) * 2002-09-13 2005-09-08 Replicor, Inc. Antiviral oligonucleotides targeting viral families
US7301265B2 (en) * 2003-05-22 2007-11-27 Seiko Epson Corporation Light source unit, method of manufacturing light source unit, and projector
ITMI20031714A1 (it) * 2003-09-05 2005-03-06 Gentium Spa Formazioni ad azione antitumorale.
US7138558B2 (en) * 2004-11-01 2006-11-21 Uop Llc Direct return of oxygenate recycle stream in olefin production process
KR20070121001A (ko) 2005-03-03 2007-12-26 젠티엄 에스피에이 신혈관 형성-의존성 종양 치료용 약학적 제제
WO2006119619A1 (en) * 2005-05-06 2006-11-16 Replicor Inc. Oligonucleotides inhibiting cell proliferation
GB2462413B (en) * 2008-08-04 2011-03-02 Rolls Royce Plc An actuator arrangement

Also Published As

Publication number Publication date
EP2252688A1 (en) 2010-11-24
EP2252688B1 (en) 2017-08-16
CA2712705A1 (en) 2009-09-24
CA2712705C (en) 2017-08-15
IL207032A (en) 2016-08-31
CN101978059A (zh) 2011-02-16
KR20100124820A (ko) 2010-11-29
WO2009115465A1 (en) 2009-09-24
IL207032A0 (en) 2010-12-30
CN106361763A (zh) 2017-02-01
AU2009226906A1 (en) 2009-09-24
EP2103689A1 (en) 2009-09-23
DK2252688T3 (da) 2017-11-13
JP2011515357A (ja) 2011-05-19
AU2009226906B2 (en) 2015-05-07
US20110092576A1 (en) 2011-04-21

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