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 (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080281041A1 (en) * 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
CN101500548A (zh) * 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
US8344116B2 (en) * 2008-03-17 2013-01-01 Case Western Reserve University Polymers and complexes for delivery of nucleic acids to intracellular targets
US8575123B2 (en) 2008-04-11 2013-11-05 Tekmira Pharmaceuticals Corporation 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.
US8975389B2 (en) 2009-03-02 2015-03-10 Alnylam Pharmaceuticals, Inc. Nucleic acid chemical modifications
AU2010306940A1 (en) 2009-10-12 2012-06-07 Smith, Larry Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
PH12012501596A1 (en) * 2010-02-24 2017-02-03 Arrowhead Res Corporation Compositions for targeted delivery of sirna
WO2011133876A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
WO2011133868A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Conformationally restricted dinucleotide monomers and oligonucleotides
CN103328500B (zh) 2010-08-04 2018-01-26 西兹尔生物技术有限公司 用于癌症的诊断和治疗的方法和化合物
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
US20170369872A1 (en) 2014-12-18 2017-12-28 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
CA3205381A1 (en) 2015-07-17 2017-01-26 Alnylam Pharmaceuticals, Inc. Multi-targeted single entity conjugates
CA3098623A1 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
CA3114396A1 (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
WO2020236600A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
WO2021092371A2 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
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
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
US20240336914A1 (en) 2020-12-31 2024-10-10 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
US20240343746A1 (en) 2020-12-31 2024-10-17 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
CN114966007A (zh) * 2021-02-26 2022-08-30 科美诊断技术股份有限公司 一种用于均相化学发光检测的微球组合物
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
IL309897A (en) 2021-07-21 2024-03-01 Alnylam Pharmaceuticals Inc Target gene IRNA compositions associated with a metabolic disorder and methods of using them
EP4416284A1 (de) 2021-10-15 2024-08-21 Alnylam Pharmaceuticals, Inc. Extrahepatische irna-zusammensetzungen zur abgabe und verfahren zur verwendung davon
EP4522742A2 (de) 2022-05-13 2025-03-19 Alnylam Pharmaceuticals, Inc. Einzelsträngige schleifenoligonukleotide
EP4547683A2 (de) 2022-06-30 2025-05-07 Alnylam Pharmaceuticals, Inc. Oligonukleotid-prodrugs auf basis von mit cyclischem disulfid modifiziertem phosphat
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
JP2026514045A (ja) 2023-04-12 2026-05-01 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 二本鎖ｒｎａ剤の肝外デリバリー
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

Family Cites Families (13)

* Cited by examiner, † Cited by third party
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
EP1102785B1 (de) * 1999-06-07 2013-02-13 Arrowhead Research Corporation Zusammensetzungen zum drogentransport mit ph sensitiven molekülen
US7019113B2 (en) 1999-06-07 2006-03-28 Mirus Bio Corporation Reversible modification of membrane interaction
AU2764801A (en) 2000-01-07 2001-07-24 University Of Washington Enhanced transport of agents 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
US20030224055A1 (en) * 2002-05-28 2003-12-04 Lewis David L. Compositions and processes for inhibiting gene expression using polynucleotides
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
EP1610612A2 (de) 2006-01-04
EP1610612A4 (de) 2006-05-24
US20040198687A1 (en) 2004-10-07
US8217015B2 (en) 2012-07-10
WO2004090107A3 (en) 2005-10-13
WO2004090107A2 (en) 2004-10-21
EP1610612B1 (de) 2011-08-17

Similar Documents

Publication Publication Date Title
EP1610612A4 (de) Endosomolytische polymere
MY157962A (en) Polymeric electrolyte, method for production thereof, and electrochemical element
WO2007106868A3 (en) Cell culture devices having ultrathin porous membrane and uses thereof
ATE544189T1 (de) Festpolymerelektrolyt und herstellungsverfahren dafür
UA101647C2 (ru) Материал для электрохимического устройства
MX2007003167A (es) Asministracion de polinucleotidos.
WO2008150967A3 (en) A lead-acid battery separator having enhanced stiffness
DE602006005857D1 (de) Sekundäre Batterie mit eingespritztem Harzgehäuse, dass seichte Abteilungen für die Unterbringung von Elektrodenplattengruppen hat
ATE494012T1 (de) Stabilisierte polymer-thiol-reagenzien
PE20190853A1 (es) Esferas polimericas con impresion molecular para extraccion de litio, mercurio y escandio
DE112009002507B8 (de) Verstärkte brennstoffzellen-elektrolytmembran, membran-elektroden-anordnung und polymerelektrolytbrennstoffzelle, diese enthaltend und herstellungsverfahren dazu
WO2006071410A3 (en) Apparatus and system having dry gene silencing compositions
DE602005024180D1 (de) Erzeugungssystem für brennstoffzellen des polymer-elektrolyttyps
DE602004032515D1 (de) Membranaktive polymere
GB2443594A (en) Microbial fuel cell with flexible substrate and micro-pillar structure
WO2006058046A3 (en) Apparatus and system having dry gene silencing pools
WO2007035604A3 (en) Scaffold carrier cartridge
WO2006080009A3 (en) Implantable bioreactors and uses thereof
WO2007093451A3 (en) Multimodal imaging using a three compartment polymer nanoparticle with cell specificity
WO2009070636A3 (en) Engineered cells, imaging reporter gene/probe systems, and methods of imaging
DE602004031330D1 (de) Protonenleitfähiger film, prozess zu seiner herstellung und brennstoffzelle mit dem protonenleitfähigen film
ATE519772T1 (de) Vorrichtung und system mit trockener kontrollgen- verstummungszusammensetzung
WO2008069940A3 (en) Multi-microrna methods and compositions
HK1129025A2 (zh) 一种用於从头发护理设备发出离子的装置
WO2008049583A3 (de) Verfahren zur selektiven lokalisierung von wirkstoffen an und in mitochondrien und entsprechende wirkstoffe

Legal Events

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