ATE520306T1 - Endosomolytische polymere - Google Patents

Endosomolytische polymere

Info

Publication number
ATE520306T1
ATE520306T1 AT04758868T AT04758868T ATE520306T1 AT E520306 T1 ATE520306 T1 AT E520306T1 AT 04758868 T AT04758868 T AT 04758868T AT 04758868 T AT04758868 T AT 04758868T AT E520306 T1 ATE520306 T1 AT E520306T1
Authority
AT
Austria
Prior art keywords
endosomolytic polymers
polymers
endosomolytic
sensitive
endosomes
Prior art date
Application number
AT04758868T
Other languages
English (en)
Inventor
David Rozema
Darren Wakefield
Jon Wolff
James Hagstrom
Vladimir Trubetskoy
Vladimir Budker
Sean Monahan
Paul Slattum
Aaron Loomis
Original Assignee
Mirus Bio 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 Mirus Bio Corp filed Critical Mirus Bio Corp
Application granted granted Critical
Publication of ATE520306T1 publication Critical patent/ATE520306T1/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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

Landscapes

  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Polymerisation Methods In General (AREA)
  • Addition Polymer Or Copolymer, Post-Treatments, Or Chemical Modifications (AREA)
  • Hard Magnetic Materials (AREA)
AT04758868T 2003-04-04 2004-04-03 Endosomolytische polymere ATE520306T1 (de)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US46045503P 2003-04-04 2003-04-04
US10/816,081 US8217015B2 (en) 2003-04-04 2004-04-01 Endosomolytic polymers
PCT/US2004/010398 WO2004090107A2 (en) 2003-04-04 2004-04-03 Endosomolytic polymers

Publications (1)

Publication Number Publication Date
ATE520306T1 true ATE520306T1 (de) 2011-09-15

Family

ID=33101441

Family Applications (1)

Application Number Title Priority Date Filing Date
AT04758868T ATE520306T1 (de) 2003-04-04 2004-04-03 Endosomolytische polymere

Country Status (4)

Country Link
US (1) US8217015B2 (de)
EP (1) EP1610612B1 (de)
AT (1) ATE520306T1 (de)
WO (1) WO2004090107A2 (de)

Families Citing this family (34)

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US20080281041A1 (en) * 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US8137695B2 (en) * 2006-08-18 2012-03-20 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
US8344116B2 (en) * 2008-03-17 2013-01-01 Case Western Reserve University Polymers and complexes for delivery of nucleic acids to intracellular targets
WO2009126933A2 (en) 2008-04-11 2009-10-15 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
FR2941152B1 (fr) * 2009-01-20 2013-10-18 Centre Nat Rech Scient Vecteurs comprenant une macromolecule anionique et un lipide cationique pour l'administration de petits acides nucleiques.
EP2669290A1 (de) 2009-03-02 2013-12-04 Alnylam Pharmaceuticals Inc. Chemische Nukleinsäuremodifikationen
US20130079382A1 (en) 2009-10-12 2013-03-28 Larry J. Smith Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro
DK2539451T3 (da) * 2010-02-24 2016-04-04 Arrowhead Res Corp Sammensætninger til målrettet tilførsel af siRNA
WO2011133868A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Conformationally restricted dinucleotide monomers and oligonucleotides
US10913767B2 (en) 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
EP2601212A1 (de) 2010-08-04 2013-06-12 Cizzle Biotechnology Limited Verfahren und verbindungen zur diagnose und behandlung von
HK1200484A1 (zh) 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
JP2015523854A (ja) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法
US10017770B2 (en) * 2014-07-03 2018-07-10 Ut-Battelle, Llc TNT cloning system
CA2970795A1 (en) 2014-12-18 2016-06-23 Alnylam Pharmaceuticals, Inc. Reversir compounds
EP3919619A1 (de) 2015-07-17 2021-12-08 Alnylam Pharmaceuticals, Inc. Mehrfach gerichtete einheitlichen konjugate
WO2019217459A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
WO2020069055A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
JP2022532652A (ja) 2019-05-17 2022-07-15 アルニラム ファーマスーティカルズ インコーポレイテッド オリゴヌクレオチドの経口送達
EP4055165A1 (de) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr)-irna-zusammensetzungen und verfahren zur verwendung davon für die behandlung oder prävention ttr-assoziierter augenerkrankungen
CA3160329A1 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
EP4271695A2 (de) 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. Auf 2'-modifiziertem nukleosid basierende oligonukleotid-prodrugs
WO2022147214A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
TW202421169A (zh) 2021-07-21 2024-06-01 美商艾拉倫製藥股份有限公司 代謝疾患相關的標靶基因ｉＲＮＡ組成物及其使用方法
MX2024004437A (es) 2021-10-15 2024-07-01 Alnylam Pharmaceuticals Inc Composiciones de inhibidor de la angiotensina-receptor neprilisina (arni) de administracion extrahepatica y metodos de uso de las mismas.
EP4522742A2 (de) 2022-05-13 2025-03-19 Alnylam Pharmaceuticals, Inc. Einzelsträngige schleifenoligonukleotide
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
JP2025532985A (ja) 2022-09-30 2025-10-03 アルナイラム ファーマシューティカルズ， インコーポレイテッド 修飾二本鎖ｒｎａ剤
AU2024254919A1 (en) 2023-04-12 2025-10-30 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
WO2026034445A1 (ja) * 2024-08-05 2026-02-12 日本新薬株式会社 ホスホロジアミデートモルフォリノオリゴ核酸（ｐｍｏ）とポリアニオンとを含む、ｐｍｏ複合体

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Publication number Priority date Publication date Assignee Title
US4542225A (en) * 1984-08-29 1985-09-17 Dana-Farber Cancer Institute, Inc. Acid-cleavable compound
DE3534273A1 (de) * 1985-09-26 1987-04-02 Basf Ag Verfahren zur herstellung von vinylamin-einheiten enthaltenden wasserloeslichen copolymerisaten und deren verwendung als nass- und trockenverfestigungsmittel fuer papier
US5629184A (en) * 1995-01-25 1997-05-13 Amgen Inc. Cationic copolymers of vinylamine and vinyl alcohol for the delivery of oligonucleotides
US20020022264A1 (en) * 1995-05-26 2002-02-21 Sullivan Sean M. Delivery vehicles comprising stable lipid/nucleic acid complexes
GB9623051D0 (en) * 1996-11-06 1997-01-08 Schacht Etienne H Delivery of DNA to target cells in biological systems
US7396919B1 (en) * 1998-07-17 2008-07-08 Mirus Bio Corporation Charge reversal of polyion complexes
WO2000075164A1 (en) * 1999-06-07 2000-12-14 Mirus Corporation COMPOSITIONS AND METHODS FOR DRUG DELIVERY USING pH SENSITIVE MOLECULES
US7019113B2 (en) * 1999-06-07 2006-03-28 Mirus Bio Corporation Reversible modification of membrane interaction
US7737108B1 (en) 2000-01-07 2010-06-15 University Of Washington Enhanced transport using membrane disruptive agents
AU4801501A (en) * 2000-05-26 2001-11-29 Transgene S.A. Complex for transferring an anionic substance of interest into a cell
US7208314B2 (en) * 2002-02-26 2007-04-24 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
EP1507874A4 (de) * 2002-05-28 2006-06-28 Mirus Bio Corp Zusammensetzungen und verfahren zur hemmung der genexpression mit polynukleotiden
US7682626B2 (en) * 2003-02-07 2010-03-23 Roche Madison Inc. Polyvinylethers for delivery of polynucleotides to mammalian cells

Also Published As

Publication number Publication date
EP1610612B1 (de) 2011-08-17
EP1610612A2 (de) 2006-01-04
WO2004090107A3 (en) 2005-10-13
EP1610612A4 (de) 2006-05-24
US20040198687A1 (en) 2004-10-07
WO2004090107A2 (en) 2004-10-21
US8217015B2 (en) 2012-07-10

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