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)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (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)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Communicable Diseases (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (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
WO2006138380A2 (en) 2005-06-15 2006-12-28 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
CA2927045A1 (en) * 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
EP3269395A1 (de) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalkohol-lipoide und verwendungen davon
EP3318248B1 (de) 2009-12-01 2019-04-10 Translate Bio, Inc. Ausgabe von mrna zur vermehrung von proteinen und enzymen bei humangenetischen erkrankungen
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
CN103380113B (zh) * 2010-11-15 2018-03-30 生命科技公司 含胺的转染试剂及其制备和使用方法
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
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
PE20181541A1 (es) 2011-10-27 2018-09-26 Massachusetts Inst Technology Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
RS57739B1 (sr) 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
JP6586075B2 (ja) 2013-03-14 2019-10-02 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
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
JP6506749B2 (ja) 2013-10-22 2019-04-24 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 フェニルケトン尿症のためのｍＲＮＡ療法
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
CN106413811A (zh) 2013-10-22 2017-02-15 夏尔人类遗传性治疗公司 精氨基琥珀酸合成酶缺乏症的mrna疗法
US9850269B2 (en) 2014-04-25 2017-12-26 Translate Bio, Inc. Methods for purification of messenger RNA
JP6557722B2 (ja) 2014-05-30 2019-08-07 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 核酸の送達のための生分解性脂質
KR102559979B1 (ko) 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
US9840479B2 (en) 2014-07-02 2017-12-12 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
WO2016205691A1 (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
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
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.
KR102950141B1 (ko) 2018-08-24 2026-04-07 트랜슬레이트 바이오 인코포레이티드 전령 rna의 정제 방법
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
EP4031662A1 (de) 2019-09-20 2022-07-27 Translate Bio, Inc. Mrna-codierendes technisches cftr

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5565290A (en) * 1991-07-30 1996-10-15 Fuji Photo Film Co., Ltd. Desensitizing solution for offset printing
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
DE69420044T2 (de) 1993-10-29 2000-03-23 Smithkline Beecham Corp., King Of Prussia Blutregulierende peptide
JP3878208B2 (ja) 1994-04-08 2007-02-07 ブラッコ インターナショナル ベスローテン フェンノートシャップ 芳香族アミド化合物およびそれらのキレート
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
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
JP4991567B2 (ja) 2012-08-01
US20080058279A1 (en) 2008-03-06
US7425645B2 (en) 2008-09-16
EP1848684A1 (de) 2007-10-31
US7569720B2 (en) 2009-08-04
US20080293829A1 (en) 2008-11-27
GB0502482D0 (en) 2005-03-16
ES2357140T3 (es) 2011-04-19
EP1848684B1 (de) 2010-12-08
JP2009525259A (ja) 2009-07-09
DE602006018709D1 (de) 2011-01-20
WO2006082088A1 (en) 2006-08-10

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