DE69715786T2 - Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle - Google Patents

Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle

Info

Publication number
DE69715786T2
DE69715786T2 DE69715786T DE69715786T DE69715786T2 DE 69715786 T2 DE69715786 T2 DE 69715786T2 DE 69715786 T DE69715786 T DE 69715786T DE 69715786 T DE69715786 T DE 69715786T DE 69715786 T2 DE69715786 T2 DE 69715786T2
Authority
DE
Germany
Prior art keywords
compositions
colipides
therapeutic molecules
cationic amphiphiles
intracellular administration
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.)
Expired - Fee Related
Application number
DE69715786T
Other languages
English (en)
Other versions
DE69715786D1 (de
Inventor
Allen J Fasbender
Michael J Welsh
Craig S Siegel
Edward R Lee
Chau-Dung Chang
John Marshall
Seng H Cheng
David J Harris
Simon J Eastman
Shirley C Hubbard
Mathieu B Lane
Eric A Rowe
Ronald K Scheule
Nelson S Yew
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.)
University of Iowa Research Foundation UIRF
Genzyme Corp
Original Assignee
University of Iowa Research Foundation UIRF
Genzyme Corp
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 University of Iowa Research Foundation UIRF, Genzyme Corp filed Critical University of Iowa Research Foundation UIRF
Application granted granted Critical
Publication of DE69715786D1 publication Critical patent/DE69715786D1/de
Publication of DE69715786T2 publication Critical patent/DE69715786T2/de
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Pulmonology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
DE69715786T 1996-06-03 1997-05-30 Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle Expired - Fee Related DE69715786T2 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/657,238 US5935936A (en) 1996-06-03 1996-06-03 Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules
PCT/US1997/009142 WO1997046223A1 (en) 1996-06-03 1997-05-30 Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules

Publications (2)

Publication Number Publication Date
DE69715786D1 DE69715786D1 (de) 2002-10-31
DE69715786T2 true DE69715786T2 (de) 2003-05-28

Family

ID=24636394

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69715786T Expired - Fee Related DE69715786T2 (de) 1996-06-03 1997-05-30 Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle

Country Status (8)

Country Link
US (1) US5935936A (de)
EP (1) EP0845981B1 (de)
JP (1) JPH11511757A (de)
AT (1) ATE224705T1 (de)
CA (1) CA2228444A1 (de)
DE (1) DE69715786T2 (de)
ES (1) ES2187791T3 (de)
WO (1) WO1997046223A1 (de)

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US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
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JP4252461B2 (ja) * 2002-03-26 2009-04-08 カウンシル オブ サイエンティフィク アンド インダストリアル リサーチ 治療分子の細胞内伝達のためのカチオン性両親媒質、その組成物、工程およびそれらの使用
US20040180438A1 (en) * 2002-04-26 2004-09-16 Pachuk Catherine J. Methods and compositions for silencing genes without inducing toxicity
EP1549352A4 (de) * 2002-05-06 2005-07-27 Nucleonics Inc Verfahren zur abgabe von nukleinsäuren
US7183263B2 (en) 2002-07-17 2007-02-27 University Of Utah Research Foundation Linear polyethylenimine-sterol conjugates for gene delivery
AU2003274906A1 (en) * 2002-07-31 2004-02-16 Nucleonics, Inc. Double stranded rna structures and constructs, and methods for generating and using the same
CA2508279A1 (en) * 2002-12-23 2004-07-22 Vical Incorporated Method for freeze-drying nucleic acid/block copolymer/cationic surfactant complexes
US8025899B2 (en) 2003-08-28 2011-09-27 Abbott Laboratories Solid pharmaceutical dosage form
US8377952B2 (en) 2003-08-28 2013-02-19 Abbott Laboratories Solid pharmaceutical dosage formulation
ATE470454T1 (de) 2004-09-13 2010-06-15 Genzyme Corp Multimere konstrukte
CN101094866A (zh) * 2004-11-04 2007-12-26 新英格兰医疗中心医院有限公司 G蛋白耦合受体激动剂和拮抗剂及其使用方法
DK2494993T3 (en) * 2007-05-04 2018-11-12 Marina Biotech Inc Amino acid lipids and uses thereof
EP3608330B1 (de) 2008-12-16 2022-11-09 Genzyme Corporation Synthetische zwischenprodukte zur verwendung zur herstellung von oligosaccharid-protein-konjugate
ES2713852T3 (es) 2009-12-01 2019-05-24 Translate Bio Inc Derivado de esteroides para la administración de ARNm en enfermedades genéticas humanas
IL280771B2 (en) 2011-06-08 2024-03-01 Shire Human Genetic Therapies Preparations of lipid nanoparticles and methods for administration of mRNA
EP2859102A4 (de) 2012-06-08 2016-05-11 Shire Human Genetic Therapies Nukleaseresistente polynukleotide und verwendungen davon
DK2970955T3 (en) 2013-03-14 2019-02-11 Translate Bio Inc METHODS FOR CLEANING MESSENGER RNA
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
US11224642B2 (en) 2013-10-22 2022-01-18 Translate Bio, Inc. MRNA therapy for argininosuccinate synthetase deficiency
EP3085376A4 (de) * 2013-12-21 2017-06-21 Nishizaki Bioinformation Research Institute Antikrebsmittel
EP3134506B1 (de) 2014-04-25 2019-08-07 Translate Bio, Inc. Verfahren zur reinigung von messenger-rna
CN106572974B (zh) 2014-07-15 2021-04-23 生命技术公司 用于将分子有效递送到细胞的具有脂质聚集体的组合物和方法
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
EP3624824A1 (de) 2017-05-16 2020-03-25 Translate Bio, Inc. Behandlung von zystischer fibrose durch verabreichung von codonoptimierter mrna, die cftr codiert
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Also Published As

Publication number Publication date
ES2187791T3 (es) 2003-06-16
EP0845981B1 (de) 2002-09-25
WO1997046223A1 (en) 1997-12-11
ATE224705T1 (de) 2002-10-15
DE69715786D1 (de) 2002-10-31
JPH11511757A (ja) 1999-10-12
EP0845981A1 (de) 1998-06-10
CA2228444A1 (en) 1997-12-11
US5935936A (en) 1999-08-10

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Legal Events

Date Code Title Description
8339 Ceased/non-payment of the annual fee