WO2003066104A2 - Composition pharmaceutique ameliorant le transfert de gene in vivo - Google Patents

Composition pharmaceutique ameliorant le transfert de gene in vivo Download PDF

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Publication number
WO2003066104A2
WO2003066104A2 PCT/FR2003/000407 FR0300407W WO03066104A2 WO 2003066104 A2 WO2003066104 A2 WO 2003066104A2 FR 0300407 W FR0300407 W FR 0300407W WO 03066104 A2 WO03066104 A2 WO 03066104A2
Authority
WO
WIPO (PCT)
Prior art keywords
composition according
nucleic acid
pharmaceutical composition
compound
general formula
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.)
Ceased
Application number
PCT/FR2003/000407
Other languages
English (en)
French (fr)
Other versions
WO2003066104A3 (fr
Inventor
Bruno Pitard
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.)
Institut National de la Sante et de la Recherche Medicale INSERM
Original Assignee
Institut National de la Sante et de la Recherche Medicale INSERM
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 Institut National de la Sante et de la Recherche Medicale INSERM filed Critical Institut National de la Sante et de la Recherche Medicale INSERM
Priority to EP03718854A priority Critical patent/EP1471944B1/fr
Priority to JP2003565527A priority patent/JP4613011B2/ja
Priority to DE60305443T priority patent/DE60305443T2/de
Priority to US10/502,973 priority patent/US7709452B2/en
Priority to CA2473212A priority patent/CA2473212C/fr
Priority to AU2003222896A priority patent/AU2003222896A1/en
Publication of WO2003066104A2 publication Critical patent/WO2003066104A2/fr
Publication of WO2003066104A3 publication Critical patent/WO2003066104A3/fr
Anticipated expiration legal-status Critical
Priority to US12/659,618 priority patent/US8367631B2/en
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • A61K31/74—Synthetic polymeric materials
    • A61K31/765—Polymers containing oxygen
    • A61K31/77—Polymers containing oxygen of oxiranes
    • 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
    • A61K31/74—Synthetic polymeric materials
    • A61K31/785—Polymers containing nitrogen
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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

Definitions

  • GFP Green-Fluorescent Protein

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
PCT/FR2003/000407 2002-02-08 2003-02-07 Composition pharmaceutique ameliorant le transfert de gene in vivo Ceased WO2003066104A2 (fr)

Priority Applications (7)

Application Number Priority Date Filing Date Title
EP03718854A EP1471944B1 (fr) 2002-02-08 2003-02-07 Composition pharmaceutique ameliorant le transfert de gene in vivo
JP2003565527A JP4613011B2 (ja) 2002-02-08 2003-02-07 ｉｎｖｉｖｏ遺伝子伝達を改善する医薬組成物
DE60305443T DE60305443T2 (de) 2002-02-08 2003-02-07 Pharmazeutische zusammnesetzung zur verbesserten in-vivo genübertragung
US10/502,973 US7709452B2 (en) 2002-02-08 2003-02-07 Pharmaceutical composition which improves in vivo gene transfer
CA2473212A CA2473212C (fr) 2002-02-08 2003-02-07 Composition pharmaceutique ameliorant le transfert de gene in vivo
AU2003222896A AU2003222896A1 (en) 2002-02-08 2003-02-07 Pharmaceutical composition which improves in vivo gene transfer
US12/659,618 US8367631B2 (en) 2002-02-08 2010-03-15 Pharmaceutical composition which improves in vivo gene transfer

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
FR02/01584 2002-02-08
FR0201584A FR2835749B1 (fr) 2002-02-08 2002-02-08 Composition pharmaceutique ameliorant le transfert de gene in vivo

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US10502973 A-371-Of-International 2003-02-07
US12/659,618 Continuation US8367631B2 (en) 2002-02-08 2010-03-15 Pharmaceutical composition which improves in vivo gene transfer

Publications (2)

Publication Number Publication Date
WO2003066104A2 true WO2003066104A2 (fr) 2003-08-14
WO2003066104A3 WO2003066104A3 (fr) 2004-03-25

Family

ID=27620039

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/FR2003/000407 Ceased WO2003066104A2 (fr) 2002-02-08 2003-02-07 Composition pharmaceutique ameliorant le transfert de gene in vivo

Country Status (10)

Country Link
US (2) US7709452B2 (https=)
EP (1) EP1471944B1 (https=)
JP (1) JP4613011B2 (https=)
AT (1) ATE326986T1 (https=)
AU (1) AU2003222896A1 (https=)
CA (1) CA2473212C (https=)
DE (1) DE60305443T2 (https=)
ES (1) ES2265569T3 (https=)
FR (1) FR2835749B1 (https=)
WO (1) WO2003066104A2 (https=)

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2868953A1 (fr) * 2004-04-16 2005-10-21 Inst Nat Sante Rech Med Composition pour le transfert intracellulaire d'un acide nucleique.
DE102006051623A1 (de) 2006-11-02 2008-07-31 Horn, Peter A. Verfahren und Stoffgemisch zur Verbesserung eines in vivo Genfransfers
WO2013128424A1 (en) 2012-03-02 2013-09-06 Institut National De La Sante Et De La Recherche Medicale Vaccine for prophylaxis or treatment of an allergen-driven and/or a chronic airways disease
WO2013186394A1 (en) 2012-06-15 2013-12-19 INSERM (Institut National de la Santé et de la Recherche Médicale) Polypeptides for use in the prophylactic treatment of allergic asthma
WO2015151048A1 (en) * 2014-04-01 2015-10-08 Institut National De La Sante Et De La Recherche Medicale (Inserm) Capped and uncapped rna molecules and block copolymers for intracellular delivery of rna
WO2019170584A1 (en) 2018-03-05 2019-09-12 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and composition for the treatment of allergic asthma
WO2022229227A1 (en) 2021-04-28 2022-11-03 Ospedale San Raffaele S.R.L. Compositions for improving the transduction of cells by viral vectors
WO2023209225A1 (en) 2022-04-29 2023-11-02 Ospedale San Raffaele S.R.L. Gene therapy

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2076257A4 (en) * 2006-09-15 2014-04-16 Belrose Pharma Inc POLYMER CONJUGATES WITH POSITIVELY LOADED PARTS
US8790664B2 (en) 2008-09-05 2014-07-29 Institut National De La Sante Et De La Recherche Medicale (Inserm) Multimodular assembly useful for intracellular delivery
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
RU2013120302A (ru) 2010-10-01 2014-11-20 Модерна Терапьютикс, Инк. Сконструированные нуклеиновые кислоты и способы их применения
EP2691101A2 (en) 2011-03-31 2014-02-05 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
RU2648950C2 (ru) 2011-10-03 2018-04-02 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
CN104114572A (zh) 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
WO2013106558A1 (en) 2012-01-10 2013-07-18 Merial Limited Lipid based adjuvants for dna - plasmid vaccines
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2014081507A1 (en) 2012-11-26 2014-05-30 Moderna Therapeutics, Inc. Terminally modified rna
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
JP2016538829A (ja) 2013-10-03 2016-12-15 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 低密度リポタンパク質受容体をコードするポリヌクレオチド
EP3225246A1 (en) 2016-03-31 2017-10-04 Klinikum der Universität München Synthetic compound for improving efficiency of transfection
AU2020234003B2 (en) * 2019-03-11 2026-02-12 Evaxion A/S Nucleic acid vaccination using neo-epitope encoding constructs
EP4704818A1 (en) 2023-03-30 2026-03-11 Vivadju Pvax14 nucleic acid in combination with all-trans retinoic acid (atra) and a checkpoint inhibitor for the treatment of cancer
EP4704856A1 (en) 2023-03-30 2026-03-11 Vivadju Pvax14 nucleic acid formulated with nano-vehicle in combination with all-trans retinoic acid (atra) for activating immune pathways
WO2025129128A1 (en) 2023-12-15 2025-06-19 Vivasor, Inc. Compositions and methods for non-viral delivery of therapeutic compounds

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5470568A (en) * 1992-02-13 1995-11-28 Arch Development Corporation Methods and compositions of a polymer (poloxamer) for cell repair
GB9216082D0 (en) * 1992-07-28 1992-09-09 Univ Nottingham Lymphatic delivery composition
US5656611A (en) * 1994-11-18 1997-08-12 Supratek Pharma Inc. Polynucleotide compositions
US6359054B1 (en) * 1994-11-18 2002-03-19 Supratek Pharma Inc. Polynucleotide compositions for intramuscular administration
US6221959B1 (en) * 1994-11-18 2001-04-24 Supratek Pharma, Inc. Polynucleotide compositions
US6040295A (en) * 1995-01-13 2000-03-21 Genemedicine, Inc. Formulated nucleic acid compositions and methods of administering the same for gene therapy
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
GB9904627D0 (en) * 1999-03-02 1999-04-21 Danbiosyst Uk Polymer compositions for polynucleotide delivery
WO2001005913A1 (en) 1999-07-16 2001-01-25 Reatech Phosphor addition in gasification
AU6389000A (en) * 1999-07-28 2001-02-19 Valentis, Inc. Sonoporation of tumors
AU2001238485A1 (en) * 2000-02-18 2001-08-27 The Immune Response Corporation Methods and compositions for gene delivery
CA2401239A1 (en) * 2000-03-03 2001-09-13 Valentis, Inc. Improved poloxamer and poloxamine compositions for nucleic acid delivery
WO2002061040A2 (en) * 2000-10-20 2002-08-08 Valentis, Inc. Gene delivery formulations and methods for treatment of ischemic conditions

Cited By (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2868953A1 (fr) * 2004-04-16 2005-10-21 Inst Nat Sante Rech Med Composition pour le transfert intracellulaire d'un acide nucleique.
WO2005099763A3 (fr) * 2004-04-16 2006-04-13 Inst Nat Sante Rech Med Composition pour le transfert intracellulaire d’un acide nucleique
DE102006051623A1 (de) 2006-11-02 2008-07-31 Horn, Peter A. Verfahren und Stoffgemisch zur Verbesserung eines in vivo Genfransfers
WO2013128424A1 (en) 2012-03-02 2013-09-06 Institut National De La Sante Et De La Recherche Medicale Vaccine for prophylaxis or treatment of an allergen-driven and/or a chronic airways disease
WO2013186394A1 (en) 2012-06-15 2013-12-19 INSERM (Institut National de la Santé et de la Recherche Médicale) Polypeptides for use in the prophylactic treatment of allergic asthma
WO2015151048A1 (en) * 2014-04-01 2015-10-08 Institut National De La Sante Et De La Recherche Medicale (Inserm) Capped and uncapped rna molecules and block copolymers for intracellular delivery of rna
US11179478B2 (en) 2014-04-01 2021-11-23 Institut National De La Sante Et De La Recherche Medicale (Inserm) Capped and uncapped RNA molecules and block copolymers for intracellular delivery of RNA
WO2019170584A1 (en) 2018-03-05 2019-09-12 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and composition for the treatment of allergic asthma
WO2022229227A1 (en) 2021-04-28 2022-11-03 Ospedale San Raffaele S.R.L. Compositions for improving the transduction of cells by viral vectors
WO2023209225A1 (en) 2022-04-29 2023-11-02 Ospedale San Raffaele S.R.L. Gene therapy

Also Published As

Publication number Publication date
DE60305443D1 (de) 2006-06-29
FR2835749B1 (fr) 2006-04-14
AU2003222896A8 (en) 2003-09-02
CA2473212C (fr) 2013-04-23
CA2473212A1 (fr) 2003-08-14
DE60305443T2 (de) 2007-05-10
AU2003222896A1 (en) 2003-09-02
EP1471944B1 (fr) 2006-05-24
US20050152870A1 (en) 2005-07-14
US20100179212A1 (en) 2010-07-15
JP4613011B2 (ja) 2011-01-12
WO2003066104A3 (fr) 2004-03-25
ATE326986T1 (de) 2006-06-15
US8367631B2 (en) 2013-02-05
JP2005522433A (ja) 2005-07-28
FR2835749A1 (fr) 2003-08-15
ES2265569T3 (es) 2007-02-16
US7709452B2 (en) 2010-05-04
EP1471944A2 (fr) 2004-11-03

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