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

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02Carboxylic 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
    • C07C237/04Carboxylic 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
    • C07C237/12Carboxylic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction 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)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Physics & Mathematics (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (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 (27)

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EP1912679A4 (de) * 2005-06-15 2009-07-29 Massachusetts Inst Technology Aminhaltige lipide und ihre verwendungen
MX363224B (es) * 2006-10-03 2019-03-15 Alnylam Pharmaceuticals Inc Formulaciones que contienen lipidos.
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
RS58405B1 (sr) 2009-12-01 2019-04-30 Translate Bio Inc Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima
EP2609135A4 (de) 2010-08-26 2015-05-20 Massachusetts Inst Technology Poly(beta-amino-alkohole), ihre herstellung und verwendungen davon
US20120136073A1 (en) 2010-11-15 2012-05-31 Life Technologies Corporation Amine-Containing Transfection Reagents and methods for making and using same
US8546338B2 (en) 2010-12-08 2013-10-01 Johnson & Johnson Consumer Companies, Inc. Self-assembling hydrogels based on dicephalic peptide amphiphiles
EP2691443B1 (de) 2011-03-28 2021-02-17 Massachusetts Institute of Technology Konjugierte lipomers und ihre verwendung
SMT201900445T1 (it) 2011-06-08 2019-09-09 Translate Bio Inc Composizione di nanoparticelle lipidiche e metodi per il rilascio di mrna
EA032088B1 (ru) 2011-10-27 2019-04-30 Массачусетс Инститьют Оф Текнолоджи Аминокислотные производные, функционализованные на n-конце, способные образовывать микросферы, инкапсулирующие лекарственное средство
EP3536787A1 (de) 2012-06-08 2019-09-11 Translate Bio, Inc. Nukleaseresistente polynukleotide und verwendungen davon
EP2882706A1 (de) 2012-08-13 2015-06-17 Massachusetts Institute of Technology Aminhaltige lipoide und ihre verwendungen
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
EP4446413A3 (de) 2013-03-14 2024-12-18 Translate Bio, Inc. Verfahren zur reinigung von messenger-rna
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
JP6525435B2 (ja) 2013-10-22 2019-06-12 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド メッセンジャーrnaの送達のための脂質製剤
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
EP3060303B1 (de) 2013-10-22 2018-11-14 Translate Bio, Inc. Mrna-therapie für argininosuccinat-synthase-mangel
EP3636742B1 (de) 2014-04-25 2025-11-05 Translate Bio, Inc. Verfahren zur reinigung von messenger-rna
EP3148552B1 (de) 2014-05-30 2019-07-31 Translate Bio, Inc. Biologisch abbaubare lipide zur freisetzung von nukleinsäuren
SG11201610670WA (en) 2014-06-24 2017-01-27 Shire Human Genetic Therapies 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
CA2990172A1 (en) 2015-06-19 2016-12-22 Massachusetts Institute Of Technology Alkenyl substituted 2,5-piperazinediones and their use in compositions for delivering an agent to a subject or cell
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
US11173190B2 (en) 2017-05-16 2021-11-16 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mRNA encoding CFTR
US11174500B2 (en) 2018-08-24 2021-11-16 Translate Bio, Inc. Methods for purification of messenger RNA
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding 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
EP0725651B1 (de) * 1993-10-29 1999-08-11 Smithkline Beecham Corporation Blutregulierende peptide
WO1995027705A1 (en) 1994-04-08 1995-10-19 Bracco International B.V. Aromatic amide compounds and metal chelates thereof
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
EP1261620A2 (de) * 2000-02-07 2002-12-04 Roche Diagnostics Corporation Kationische amphiphile zur verwendung bei der nucleinsäuretransfektin
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
GB0425555D0 (en) * 2004-11-19 2004-12-22 Glaxo Group Ltd Novel compounds
GB0425556D0 (en) * 2004-11-19 2004-12-22 Glaxo Group Ltd Novel compounds

Also Published As

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

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