ATE490954T1 - Estergebundene geminitensidverbindungen zur verwendung in der gentherapie - Google Patents

Estergebundene geminitensidverbindungen zur verwendung in der gentherapie

Info

Publication number
ATE490954T1
ATE490954T1 AT06701592T AT06701592T ATE490954T1 AT E490954 T1 ATE490954 T1 AT E490954T1 AT 06701592 T AT06701592 T AT 06701592T AT 06701592 T AT06701592 T AT 06701592T AT E490954 T1 ATE490954 T1 AT E490954T1
Authority
AT
Austria
Prior art keywords
ester
gemini surfactant
surfactant compounds
gene therapy
linked
Prior art date
Application number
AT06701592T
Other languages
English (en)
Inventor
Mariano Javier Castro
Christopher Kitson
Mark Ladlow
Alpesh Patel
Original Assignee
Glaxo Group Ltd
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 Glaxo Group Ltd filed Critical Glaxo Group Ltd
Application granted granted Critical
Publication of ATE490954T1 publication Critical patent/ATE490954T1/de

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/12—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by carboxyl groups
    • 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
    • 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
    • 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
    • A61P31/04—Antibacterial agents
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Oncology (AREA)
  • Microbiology (AREA)
  • Communicable Diseases (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
AT06701592T 2005-02-07 2006-02-03 Estergebundene geminitensidverbindungen zur verwendung in der gentherapie ATE490954T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB0502482.3A GB0502482D0 (en) 2005-02-07 2005-02-07 Novel compounds
PCT/EP2006/000998 WO2006082088A1 (en) 2005-02-07 2006-02-03 Ester-linked gemini surfactant compounds for use in gene therapy

Publications (1)

Publication Number Publication Date
ATE490954T1 true ATE490954T1 (de) 2010-12-15

Family

ID=34355904

Family Applications (1)

Application Number Title Priority Date Filing Date
AT06701592T ATE490954T1 (de) 2005-02-07 2006-02-03 Estergebundene geminitensidverbindungen zur verwendung in der gentherapie

Country Status (8)

Country Link
US (2) US7425645B2 (de)
EP (1) EP1848684B1 (de)
JP (1) JP4991567B2 (de)
AT (1) ATE490954T1 (de)
DE (1) DE602006018709D1 (de)
ES (1) ES2357140T3 (de)
GB (1) GB0502482D0 (de)
WO (1) WO2006082088A1 (de)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2476756A1 (de) 2005-06-15 2012-07-18 Massachusetts Institute of Technology Aminhaltige Lipide und ihre Verwendungen
WO2008042973A2 (en) 2006-10-03 2008-04-10 Alnylam Pharmaceuticals, Inc. Lipid containing formulations
MX2011004859A (es) 2008-11-07 2011-08-03 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
PL3338765T3 (pl) 2009-12-01 2019-06-28 Translate Bio, Inc. Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
JP2014508102A (ja) 2010-11-15 2014-04-03 ライフ テクノロジーズ コーポレーション アミン含有トランスフェクション試薬ならびにそれを生成および使用するための方法
US8546338B2 (en) 2010-12-08 2013-10-01 Johnson & Johnson Consumer Companies, Inc. Self-assembling hydrogels based on dicephalic peptide amphiphiles
US9238716B2 (en) 2011-03-28 2016-01-19 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
KR20150000461A (ko) 2011-10-27 2015-01-02 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, ｎ-말단 상에 관능화된 아미노산 유도체
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
EP2882706A1 (de) 2012-08-13 2015-06-17 Massachusetts Institute of Technology Aminhaltige lipoide und ihre verwendungen
EP2968586B1 (de) 2013-03-14 2018-07-25 Translate Bio, Inc. Cftr-mrna-zusammensetzungen und zugehörige verfahren sowie verwendungen
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
US11224642B2 (en) 2013-10-22 2022-01-18 Translate Bio, Inc. MRNA therapy for argininosuccinate synthetase deficiency
WO2015061467A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Lipid formulations for delivery of messenger rna
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
JP6571679B2 (ja) 2014-04-25 2019-09-04 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの精製方法
US10022455B2 (en) 2014-05-30 2018-07-17 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
CA2952824C (en) 2014-06-24 2023-02-21 Shire Human Genetic Therapies, Inc. Stereochemically enriched compositions for delivery of nucleic acids
WO2016004202A1 (en) 2014-07-02 2016-01-07 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
ES2949540T3 (es) 2015-06-19 2023-09-29 Massachusetts Inst Technology 2,5-piperazinadionas sustituidas con alquenilo y su uso en composiciones para suministrar un agente a un sujeto o una célula
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
CN118421617A (zh) 2018-08-24 2024-08-02 川斯勒佰尔公司 用于纯化信使rna的方法
EP3883592A1 (de) 2018-11-21 2021-09-29 Translate Bio, Inc. Behandlung von zystischer fibrose durch verabreichung von vernebelter mrna, die cftr codiert
WO2021055609A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH05104878A (ja) * 1991-10-17 1993-04-27 Fuji Photo Film Co Ltd オフセツト印刷用不感脂化処理液
US5565290A (en) 1991-07-30 1996-10-15 Fuji Photo Film Co., Ltd. Desensitizing solution for offset printing
DE69420044T2 (de) 1993-10-29 2000-03-23 Smithkline Beecham Corp., King Of Prussia Blutregulierende peptide
EP0702677A1 (de) 1994-04-08 1996-03-27 BRACCO International B.V. Aromatische amidverbindungen und seine metallchelate
AU3986097A (en) * 1996-08-27 1998-03-19 University Of Akron, The Lipophilic polyamine esters for the site specific delivery of nitric oxide in pharmaceutical use
GB9914045D0 (en) * 1999-06-16 1999-08-18 Smithkline Beecham Plc Novel compounds
US20030229037A1 (en) * 2000-02-07 2003-12-11 Ulrich Massing Novel cationic amphiphiles
WO2004087656A1 (de) 2003-04-03 2004-10-14 Schering Aktiengesellschaft Konjugate 2,4-ethano- und 2,4-propano-überbrückter 3,6,9-triaza-nonansäure-3n, 6n, 9,9n-tetraessigsäure- und entsprechender phosphorsäuremethylen-derivate und deren abkömmlinge mit biomolekülen, verfahren zu deren herstellung und deren verwendung zur herstellung
GB0425556D0 (en) * 2004-11-19 2004-12-22 Glaxo Group Ltd Novel compounds
GB0425555D0 (en) * 2004-11-19 2004-12-22 Glaxo Group Ltd Novel compounds

Also Published As

Publication number Publication date
JP4991567B2 (ja) 2012-08-01
DE602006018709D1 (de) 2011-01-20
WO2006082088A1 (en) 2006-08-10
EP1848684B1 (de) 2010-12-08
US7425645B2 (en) 2008-09-16
US20080293829A1 (en) 2008-11-27
GB0502482D0 (en) 2005-03-16
EP1848684A1 (de) 2007-10-31
ES2357140T3 (es) 2011-04-19
US7569720B2 (en) 2009-08-04
JP2009525259A (ja) 2009-07-09
US20080058279A1 (en) 2008-03-06

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