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

    • 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
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712Cystic fibrosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (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)
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
CA2286739A1 (en) * 1997-04-04 1998-10-15 Jinkang Wang 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
DE69841002D1 (de) 1997-05-14 2009-09-03 Univ British Columbia Hochwirksame verkapselung von nukleinsäuren in lipidvesikeln
PT1129064E (pt) 1998-11-12 2008-01-31 Invitrogen Corp Reagentes de transfecção
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
PT1144609E (pt) * 1999-08-27 2008-05-28 Novartis Vaccines & Diagnostic Oligonucleótidos anti-sentido quiméricos e suas formulações para transfecção de células
US6677445B1 (en) * 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
EP2336169A1 (de) * 2000-04-21 2011-06-22 New England Medical Center Hospital G-Protein gekoppelte Rezeptor (GPCR)-Agonisten und -Antagonisten sowie Verfahren zur GPCR-Aktivierung/ -Inhibierung.
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
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
AU2001269923A1 (en) * 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
DE60133049T2 (de) * 2000-12-23 2009-02-19 Novartis Vaccines and Diagnostics, Inc., Emeryville Screeningverfahren für die oligonukleotidtransfektion
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
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
AU2003266014B2 (en) * 2002-05-06 2009-05-14 Alnylam Pharmaceuticals, Inc. Methods for delivery of nucleic acids
US7183263B2 (en) * 2002-07-17 2007-02-27 University Of Utah Research Foundation Linear polyethylenimine-sterol conjugates for gene delivery
EP1540004A4 (de) * 2002-07-31 2007-10-03 Nucleonics Inc Doppelstrang-rna-strukturen und -konstrukte sowie verfahren zur erzeugung und verwendung davon
JP4917263B2 (ja) * 2002-12-23 2012-04-18 バイカル インコーポレイテッド 核酸/ブロックコポリマー/陽イオン性界面活性剤複合体を凍結乾燥する方法
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
DK2229956T3 (da) 2004-09-13 2013-07-29 Genzyme Corp Multimere konstruktioner
RU2430113C2 (ru) * 2004-11-04 2011-09-27 Тафтс Медикал Сентер, Инк. Химерный полипептид, селективно взаимодействующий с родственным ему рецептором хемокина, фармацевтическая композиция и применение химерного полипептида (варианты)
EP2157982B1 (de) 2007-05-04 2014-12-17 Marina Biotech, Inc. Aminosäurelipide und ihre verwendungen
HUE044381T2 (hu) 2008-12-16 2019-10-28 Genzyme Corp Szintetikus intermedierek oligoszacharid-fehérje konjugátumok elõállításához
RS58405B1 (sr) 2009-12-01 2019-04-30 Translate Bio Inc Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima
SMT201900445T1 (it) 2011-06-08 2019-09-09 Translate Bio Inc Composizione di nanoparticelle lipidiche e metodi per il rilascio di mrna
EP3536787A1 (de) 2012-06-08 2019-09-11 Translate Bio, Inc. Nukleaseresistente polynukleotide und verwendungen davon
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
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
WO2015093588A1 (ja) * 2013-12-21 2015-06-25 Ktnバイオテック株式会社 抗癌剤
EP3636742B1 (de) 2014-04-25 2025-11-05 Translate Bio, Inc. Verfahren zur reinigung von messenger-rna
EP3169310A1 (de) 2014-07-15 2017-05-24 Life Technologies Corporation Zusammensetzungen mit lipidaggregaten und verfahren zur effizienten bereitstellung von molekülen an zellen
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 (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
EP0316345A1 (de) * 1986-07-28 1989-05-24 Liposome Technology, Inc. Liposome mit verbesserter bindung an schleimgewebe
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
DK0688333T3 (da) * 1993-03-10 1999-02-08 Magainin Pharma Steroidderivater, farmaceutiske præparater indeholdende disse og deres anvendelse som antibiotika eller desinfektionsmidler
US5578475A (en) * 1993-07-12 1996-11-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
CA2176715A1 (en) * 1993-11-24 1995-06-01 Megabios Corporation Amphiphilic derivatives of guanidine
KR960705798A (ko) * 1993-11-24 1996-11-08 벤자민 에프 맥그로우 피페라진의 양쪽친화성 유도체(amphiphilic derivatives of piperazine)
ES2173944T3 (es) * 1993-12-20 2002-11-01 Biotech Tools Sa Compuesto capaz de introducir al menos una molecula en una celula.
AU1919295A (en) * 1994-02-14 1995-08-29 Indiana University Foundation Aminophospholipid compositions and uses thereof
WO1995024222A1 (en) * 1994-03-07 1995-09-14 University Of Medicine & 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
WO1996020208A2 (de) * 1994-12-28 1996-07-04 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
CA2228444A1 (en) 1997-12-11
EP0845981A1 (de) 1998-06-10
ES2187791T3 (es) 2003-06-16
WO1997046223A1 (en) 1997-12-11
DE69715786T2 (de) 2003-05-28
DE69715786D1 (de) 2002-10-31
JPH11511757A (ja) 1999-10-12
EP0845981B1 (de) 2002-09-25
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
DE69807587D1 (de) Quinazolin-4-on AMPA Antagonisten
NO963850L (no) Heterosykliske forbindelser, deres fremstilling og anvendelse
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.
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