ATE224705T1 - 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
ATE224705T1
ATE224705T1 AT97929716T AT97929716T ATE224705T1 AT E224705 T1 ATE224705 T1 AT E224705T1 AT 97929716 T AT97929716 T AT 97929716T AT 97929716 T AT97929716 T AT 97929716T AT E224705 T1 ATE224705 T1 AT E224705T1
Authority
AT
Austria
Prior art keywords
colipides
amphiphils
compositions containing
therapeutic molecules
containing cationic
Prior art date
Application number
AT97929716T
Other languages
English (en)
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
Original Assignee
Genzyme Corp
Univ Iowa Res Found
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 Genzyme Corp, Univ Iowa Res Found filed Critical Genzyme Corp
Application granted granted Critical
Publication of ATE224705T1 publication Critical patent/ATE224705T1/de

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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Pulmonology (AREA)
  • Dispersion Chemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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)
AT97929716T 1996-06-03 1997-05-30 Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle ATE224705T1 (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 (1)

Publication Number Publication Date
ATE224705T1 true ATE224705T1 (de) 2002-10-15

Family

ID=24636394

Family Applications (1)

Application Number Title Priority Date Filing Date
AT97929716T ATE224705T1 (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)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5674908A (en) 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
US6989434B1 (en) 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
US5837533A (en) * 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
AU6949098A (en) * 1997-04-04 1998-10-30 Valentis, Inc. Improved methods of delivery using cationic lipids and helper lipids
US6235310B1 (en) 1997-04-04 2001-05-22 Valentis, Inc. Methods of delivery using cationic lipids and helper lipids
US6835395B1 (en) 1997-05-14 2004-12-28 The University Of British Columbia Composition containing small multilamellar oligodeoxynucleotide-containing lipid vesicles
WO1998051278A2 (en) 1997-05-14 1998-11-19 Inex Pharmaceuticals Corporation High efficiency encapsulation of charged therapeutic agents in lipid vesicles
ES2296419T3 (es) 1998-11-12 2008-04-16 Invitrogen Corporation Reactivos de transfeccion.
US6521431B1 (en) 1999-06-22 2003-02-18 Access Pharmaceuticals, Inc. Biodegradable cross-linkers having a polyacid connected to reactive groups for cross-linking polymer filaments
IL131074A0 (en) 1999-07-23 2001-03-19 Polygene Ltd A biodegradable polycation composition for delivery of an anionic macromolecule
US6677445B1 (en) * 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
EP1144609B1 (de) * 1999-08-27 2008-03-05 Novartis Vaccines and Diagnostics, Inc. Chimerische antisense-oligonukleotide und zelltransfektions-zusammensetzungen davon
US7696168B2 (en) * 2000-04-21 2010-04-13 Tufts Medical Center, Inc. G protein coupled receptor agonists and antagonists and methods of activating and inhibiting G protein coupled receptors using the same
EP2348047A1 (de) * 2000-04-21 2011-07-27 New England Medical Center G-Protein gekoppelte Rezeptor (GPCR)- agonisten und -antagonisten sowie Verfahren zur GPCR-Aktivierung/-Inhibierung
DE10026698A1 (de) 2000-05-30 2001-12-06 Basf Ag Selbstemulgierende Wirkstoffformulierung und Verwendung dieser Formulierung
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
WO2001097829A2 (en) * 2000-06-19 2001-12-27 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
ES2300379T3 (es) * 2000-12-23 2008-06-16 Novartis Vaccines And Diagnostics, Inc. Procedimiento de deteccion selectiva de transfeccion de oligonucleotidos.
IL140844A0 (en) * 2001-01-10 2002-02-10 Polygene Ltd Cationic polysaccharide compositions
US20030096414A1 (en) 2001-03-27 2003-05-22 Invitrogen Corporation Culture medium for cell growth and transfection
WO2002100317A2 (en) 2001-05-25 2002-12-19 The Trustees Of The University Of Pennsylvania Targeted particles and methods of using the same
WO2003035669A1 (en) * 2001-10-22 2003-05-01 Sterrenbeld Biotechnologie North America, Inc. Amphiphilic cationic lipids derived from cholesterol
EP1492876B1 (de) * 2002-03-26 2007-12-05 Council of Scientific and Industrial Research Katonische amphiphile zur intrazellulären verabreichung therapeutischer moleküle, zusammensetzung, verfahren sowie deren verwendung
US20040180438A1 (en) * 2002-04-26 2004-09-16 Pachuk Catherine J. Methods and compositions for silencing genes without inducing toxicity
EP2298358A1 (de) * 2002-05-06 2011-03-23 Alnylam Pharmaceuticals Inc. Methoden zum Nukleinsäuren-Transfer
US7183263B2 (en) * 2002-07-17 2007-02-27 University Of Utah Research Foundation Linear polyethylenimine-sterol conjugates for gene delivery
WO2004011624A2 (en) * 2002-07-31 2004-02-05 Nucleonics, Inc. Double stranded rna structures and constructs, and methods for generating and using the same
EP1578193A4 (de) * 2002-12-23 2011-06-15 Vical Inc Gefriertrocknungsverfahren für nukleinsäure-/blockcopolymer-/kationtensid-komplexe
US8377952B2 (en) 2003-08-28 2013-02-19 Abbott Laboratories Solid pharmaceutical dosage formulation
US8025899B2 (en) 2003-08-28 2011-09-27 Abbott Laboratories Solid pharmaceutical dosage form
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
LT2494993T (lt) 2007-05-04 2018-12-27 Marina Biotech, Inc. Aminorūgščių lipidai ir jų panaudojimas
SI2889043T1 (sl) 2008-12-16 2019-08-30 Genzyme Corporation Sintetične vmesne spojine za pripravo konjugatov oligosaharid-protein
ME03091B (de) 2009-12-01 2019-01-20 Translate Bio Inc Ausgabe von mrna zur vermehrung von proteinen und enzymen bei humangenetischen erkrankungen
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
CA2904151C (en) 2013-03-14 2023-09-12 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
KR20150128687A (ko) 2013-03-14 2015-11-18 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna의 정제 방법
EA201992208A1 (ru) 2013-10-22 2020-07-31 Транслейт Био, Инк. ЛЕЧЕНИЕ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
US9770458B2 (en) 2013-12-21 2017-09-26 Nishizaki Bioinformation Research Institute Anti-cancer agent containing a diacylphosphatidylethanolamine as an active ingredient
KR102739894B1 (ko) 2014-04-25 2024-12-05 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna 의 정제 방법
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
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
WO2020041793A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4394448A (en) * 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
US4544545A (en) * 1983-06-20 1985-10-01 Trustees University Of Massachusetts Liposomes containing modified cholesterol for organ targeting
JPS617292A (ja) * 1984-06-20 1986-01-13 Japan Atom Energy Res Inst 制癌剤を結合させたエストラサイト系化合物を製造する方法
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4971803A (en) * 1985-04-08 1990-11-20 California Institute Of Technology Lamellar vesicles formed of cholesterol derivatives
JPH02500360A (ja) * 1986-07-28 1990-02-08 リポソーム　テクノロジー，インコーポレイテッド 粘膜組織上での高い保持力を有するリポソーム
US5043164A (en) * 1989-01-17 1991-08-27 The University Of Tennessee Research Corporation Blood-stable, cholesterol-free liposomes
FR2645866B1 (fr) * 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US4958013A (en) * 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5004737A (en) * 1989-09-22 1991-04-02 Pacific Chemical Co., Ltd. Quaternary ammonium-substituted sterol derivatives
CA2037478A1 (en) * 1990-03-05 1991-09-06 Richard Gregory Diagnostic and treatment methods involving the cystic fibrosis transmembrane regulator
US5279833A (en) * 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) * 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
EP0490379A3 (de) * 1990-12-13 1992-06-24 BERLIN-CHEMIE Aktiengesellschaft Diaminosäurederivate und pharmazeutische Zusammensetzungen
US5283185A (en) * 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5334761A (en) * 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5705655A (en) * 1992-12-17 1998-01-06 Megabios Corporation Amphiphilic nitrogen containing imidazolinium derivative compounds and uses
CA2157594A1 (en) * 1993-03-10 1994-09-15 Leah L. Frye Steroid derivatives, pharmaceutical compositions containing them, and their use as antibiotics or disinfectants
US5578475A (en) * 1993-07-12 1996-11-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
AU691226B2 (en) * 1993-11-24 1998-05-14 Megabios Corporation Amphiphilic derivatives of guanidine
JPH09505593A (ja) * 1993-11-24 1997-06-03 メガバイオス・コーポレイション ピペラジンの両親媒性誘導体
ES2173944T3 (es) * 1993-12-20 2002-11-01 Biotech Tools Sa Compuesto capaz de introducir al menos una molecula en una celula.
WO1995021812A1 (en) * 1994-02-14 1995-08-17 Indiana University Foundation Aminophospholipid compositions and uses thereof
AU2116995A (en) * 1994-03-07 1995-09-25 University Of Medicine And Dentistry Of New Jersey Cyclic polycationic polymer-oligonucleotide conjugates and methods for preparing same
US5670347A (en) * 1994-05-11 1997-09-23 Amba Biosciences Llc Peptide-mediated gene transfer
US5908635A (en) * 1994-08-05 1999-06-01 The United States Of America As Represented By The Department Of Health And Human Services Method for the liposomal delivery of nucleic acids
US5837533A (en) * 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US6071890A (en) * 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
EP0748332B1 (de) * 1994-12-28 1999-11-10 Max-Delbrück-Centrum Für Molekulare Medizin Neues cholesterolderivat für den liposomalen gentransfer
FR2744453B1 (fr) * 1996-02-05 1998-07-10 Transgene Sa Composes lipopolyamines, compositions pharmaceutiques en comprenant, vecteurs de transfert d'acide nucleique et procede de preparation

Also Published As

Publication number Publication date
EP0845981B1 (de) 2002-09-25
JPH11511757A (ja) 1999-10-12
EP0845981A1 (de) 1998-06-10
WO1997046223A1 (en) 1997-12-11
DE69715786T2 (de) 2003-05-28
DE69715786D1 (de) 2002-10-31
ES2187791T3 (es) 2003-06-16
CA2228444A1 (en) 1997-12-11
US5935936A (en) 1999-08-10

Similar Documents

Publication Publication Date Title
ATE224705T1 (de) Zusammensetzungen mit kationischen amphiphilen und colipiden zur intrazellulären verabreichung therapeutischer moleküle
ATE203019T1 (de) Neue (r)-5-carbamoyl-8-fluor-3-n,n- disubstituierte-amino-3,4-dihydro-2h-1- benzopyrane
BG104315A (en) Prostaglandine agonists and their application for the treatment of osteal diseases
DE69627690D1 (de) Liposomale formulierungen von mitoxantron
NO986100D0 (no) Androgene reseptormodulerende forbindelser og fremgangsmÕter
DE69709407D1 (de) Zytotoxischer aminozucker und verwandte zuckerderivate von indolopyrrolocarbazolen
CR9879A (es) Moduladores de ccr5 (divisional de la solicitud n° 6392)
YU40701A (sh) Piperidini kao modulatori ccr5
ATE284690T1 (de) Antithrombotische diamine
HUT76511A (en) Benzoyl derivatives, preparation and use thereot
NO306779B1 (no) Naftalamider, anvendelse derav og farmasöytisk preparat
ATE223399T1 (de) Quinazolin-4-on ampa antagonisten
NO963850L (no) Heterosykliske forbindelser, deres fremstilling og anvendelse
AUPR118000A0 (en) Therapeutic molecules and methods
ATE297396T1 (de) Neue atropisomere von 2,3-disubstituierten-(5,6)- heteroarylkondensierten-pyrimidin-4-onen
ATE188873T1 (de) Tris-platin-komplexe
MX9403088A (es) Composiciones farmaceuticas que contienen deriva- dos de indol y metodos de uso.
AU7525398A (en) Inhibitors for urokinase receptor
ES2161913T3 (es) Complejos cationicos trinucleares de platino que tienen una actividad antitumoral y composiciones farmaceuticas que los contienen.
NO990322L (no) Farmas°ytisk blanding inneholdende en 5HT2c-antagonist og en D2-antagonist

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties