PL2539451T3 - Kompozycje do ukierunkowanego dostarczania siRNA - Google Patents

Kompozycje do ukierunkowanego dostarczania siRNA

Info

Publication number
PL2539451T3
PL2539451T3 PL11703711T PL11703711T PL2539451T3 PL 2539451 T3 PL2539451 T3 PL 2539451T3 PL 11703711 T PL11703711 T PL 11703711T PL 11703711 T PL11703711 T PL 11703711T PL 2539451 T3 PL2539451 T3 PL 2539451T3
Authority
PL
Poland
Prior art keywords
sirna
compositions
targeted delivery
targeted
delivery
Prior art date
Application number
PL11703711T
Other languages
English (en)
Inventor
Philipp Hadwiger
Torsten Hoffmann
Eric A Kitas
David L Lewis
Peter Mohr
David B Rozema
Wilma Thuer
Linda Christine Valis
Darren H Wakefield
Original Assignee
Arrowhead Res Corporation
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 Arrowhead Res Corporation filed Critical Arrowhead Res Corporation
Publication of PL2539451T3 publication Critical patent/PL2539451T3/pl

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/32—Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers, poly(meth)acrylates, or polyvinyl pyrrolidone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
PL11703711T 2010-02-24 2011-02-18 Kompozycje do ukierunkowanego dostarczania siRNA PL2539451T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US30749010P 2010-02-24 2010-02-24
PCT/EP2011/052393 WO2011104169A1 (en) 2010-02-24 2011-02-18 Compositions for targeted delivery of sirna
EP11703711.9A EP2539451B1 (en) 2010-02-24 2011-02-18 Compositions for targeted delivery of sirna

Publications (1)

Publication Number Publication Date
PL2539451T3 true PL2539451T3 (pl) 2016-08-31

Family

ID=43901424

Family Applications (1)

Application Number Title Priority Date Filing Date
PL11703711T PL2539451T3 (pl) 2010-02-24 2011-02-18 Kompozycje do ukierunkowanego dostarczania siRNA

Country Status (25)

Country Link
US (4) US8313772B2 (pl)
EP (2) EP3023495B1 (pl)
JP (2) JP6137834B2 (pl)
KR (1) KR101956623B1 (pl)
CN (1) CN102869774B (pl)
AP (1) AP3284A (pl)
AU (1) AU2011219941C1 (pl)
BR (1) BR112012021256A2 (pl)
CA (1) CA2788600C (pl)
DK (1) DK2539451T3 (pl)
EA (1) EA024534B1 (pl)
ES (1) ES2562817T3 (pl)
HR (1) HRP20160093T1 (pl)
HU (1) HUE026811T2 (pl)
IL (1) IL221396A (pl)
MX (1) MX2012009178A (pl)
NZ (1) NZ601737A (pl)
PL (1) PL2539451T3 (pl)
PT (1) PT2539451E (pl)
RS (1) RS54578B1 (pl)
SG (1) SG183374A1 (pl)
SI (1) SI2539451T1 (pl)
SM (1) SMT201600079B (pl)
WO (1) WO2011104169A1 (pl)
ZA (1) ZA201205685B (pl)

Families Citing this family (170)

* 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
US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
KR20110128345A (ko) * 2009-03-13 2011-11-29 에젠, 인코포레이티드 생물학적 활성 ｒｎａ의 전달을 위한 조성물 및 방법
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
JP6137834B2 (ja) * 2010-02-24 2017-05-31 アローヘッド ファーマシューティカルズ インコーポレイテッド ｓｉＲＮＡの標的化送達のための組成物
WO2011133876A2 (en) * 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
PT3590949T (pt) 2010-10-01 2022-08-02 Modernatx Inc Ácidos ribonucleicos contendo n1-metilpseudouracilos e suas utilizações
BR112013013434A2 (pt) * 2010-12-17 2016-08-16 Arrowhead Res Corp metade que tem por alvo modulador fármaco cinético em cacho de galactose para sirna
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
BR122020024394B1 (pt) 2010-12-29 2021-05-11 F. Hoffmann-La Roche Ag conjugado e composição farmacêutica
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
EP2720540A4 (en) 2011-06-17 2015-06-03 Arrowhead Madison Inc DISUBSTITUTED MALE ANHYDRIDES HAVING MODIFIED CYCLE CLOSURE KINETICS
AU2012300476B2 (en) 2011-08-26 2016-03-24 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
EP2807216A4 (en) * 2011-08-26 2015-10-28 Arrowhead Res Corp POLY (VINYLESTER) POLYMERS FOR IN VIVO NUCLEIC ACID RELIEF
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
SG11201401196WA (en) 2011-10-03 2014-05-29 Moderna Therapeutics Inc Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
AU2012352180A1 (en) 2011-12-16 2014-07-31 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
EP2794879A4 (en) * 2011-12-23 2015-10-14 Egen Inc COMPOSITIONS AND METHODS FOR RELEASING BIOLOGICALLY ACTIVE RNA
WO2013102878A2 (en) 2012-01-05 2013-07-11 Department Of Biotechnology (Dbt) Fat1 gene in cancer and inflammation
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
EP3505176A1 (en) 2012-04-02 2019-07-03 Moderna Therapeutics, Inc. Modified polynucleotides for the production of secreted proteins
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
AU2012377385A1 (en) 2012-04-18 2014-01-23 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
EP2838544A4 (en) * 2012-04-18 2015-10-14 Arrowhead Res Corp POLY (ACRYLATE) POLYMERS FOR IN VIVO NUCLEIC ACID ADMINISTRATION
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
AR090906A1 (es) * 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y procedimientos para la administracion de oligonucleotidos
AR090905A1 (es) * 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
US10323279B2 (en) * 2012-08-14 2019-06-18 10X Genomics, Inc. Methods and systems for processing polynucleotides
US9801953B2 (en) 2012-10-15 2017-10-31 Emory University Nanoparticles carrying nucleic acid cassettes for expressing RNA
CA2889596C (en) 2012-11-15 2022-08-23 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
JP6144355B2 (ja) 2012-11-26 2017-06-07 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 化学修飾ｍＲＮＡ
EP2958998B1 (en) * 2013-02-22 2017-12-27 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) molecules containing a 2' internucleoside linkage
CN105247051A (zh) 2013-02-28 2016-01-13 箭头研究公司 治疗epas1相关疾病的有机组合物
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
CN105378085B (zh) 2013-05-01 2019-02-15 Ionis制药公司 用于调节hbv和ttr表达的组合物和方法
JP2016518842A (ja) 2013-05-01 2016-06-30 レグルス セラピューティクス インコーポレイテッド マイクロＲＮＡ化合物およびｍｉＲ−１２２をモジュレートする方法
TWI680767B (zh) 2013-05-01 2020-01-01 美商雷格勒斯治療公司 用於增強的細胞攝取之化合物及方法
JP6433001B2 (ja) 2013-08-07 2018-12-05 アローヘッド ファーマシューティカルズ インコーポレイテッド 腫瘍細胞へのＲＮＡｉトリガーのインビボ送達のためのポリ結合体
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
WO2015051214A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
NZ757749A (en) 2013-10-04 2022-07-01 Icahn School Med Mount Sinai Compositions and methods for inhibiting expression of the alas1 gene
US9994846B2 (en) 2013-10-25 2018-06-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
CA2928349A1 (en) * 2013-11-14 2015-05-21 Roche Innovation Center Copenhagen A/S Apob antisense conjugate compounds
EP3137476B1 (en) 2014-04-28 2019-10-09 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
ES2849600T3 (es) 2014-05-01 2021-08-19 Ionis Pharmaceuticals Inc Conjugados de oligonucleótidos antisentido modificados y su uso para modular la expresión de PKK
CN106459969B (zh) 2014-05-01 2019-11-15 Ionis制药公司 用于调节生长激素受体表达的组合物和方法
MX2016014102A (es) 2014-05-01 2017-05-03 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de similar a la angiopoyetina tipo 3.
PE20190626A1 (es) 2014-05-01 2019-04-26 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del factor b del complemento
RU2771104C2 (ru) 2014-05-14 2022-04-26 Таргиммьюн Терапьютикс Аг Улучшенные полиэтилениминовые полиэтиленгликолевые векторы
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2015188194A1 (en) * 2014-06-06 2015-12-10 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
BR112017001931A2 (pt) 2014-08-07 2017-11-28 Regulus Therapeutics Inc direcionamento de micrornas para distúrbios metabólicos
JOP20200115A1 (ar) * 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
US20170327524A1 (en) * 2014-10-10 2017-11-16 Hoffmann-La Roche, Inc. Galnac phosphoramidites, nucleic acid conjugates thereof and their use
US10351854B2 (en) * 2014-10-10 2019-07-16 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
WO2016061487A1 (en) * 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
US10517898B2 (en) 2014-11-20 2019-12-31 The Regents Of The University Of California Compositions and methods related to hematologic recovery
WO2016138132A1 (en) * 2015-02-25 2016-09-01 The Trustees Of Columbia University In The City Of New York Dipeptidyl peptidase-iv(dpp4) inhibitors, methods and compositions for suppressing adipose tissue inflammation
CA2979703A1 (en) 2015-03-17 2016-09-22 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of factor xii
WO2016196239A1 (en) 2015-05-29 2016-12-08 Arrowhead Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression of hif2alpha
MX2017016088A (es) 2015-06-15 2018-04-11 Mpeg La Llc Oligonucleotidos multi-conjugados definidos.
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
CN109477106B (zh) 2015-07-10 2022-10-04 Ionis制药公司 二酰基甘油酰基转移酶2(dgat2)的调节剂
ES2744437T3 (es) 2015-08-06 2020-02-25 Hoffmann La Roche Procedimientos para la preparación de derivados ácidos de GalNAc
WO2017027350A2 (en) 2015-08-07 2017-02-16 Arrowhead Pharmaceuticals, Inc. Rnai therapy for hepatitis b virus infection
JP6877414B2 (ja) 2015-09-24 2021-05-26 アイオニス・ファーマシューティカルズ・インコーポレイテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｋｒａｓ発現のモジュレーター
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
WO2017079745A1 (en) 2015-11-06 2017-05-11 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
US10557137B2 (en) 2015-11-06 2020-02-11 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
AR106683A1 (es) * 2015-11-16 2018-02-07 Hoffmann La Roche FOSFORAMIDITA DE AGRUPACIÓN DE GalNAc
SG11201804729RA (en) 2015-12-07 2018-07-30 Genzyme Corp Methods and compositions for treating a serpinc1-associated disorder
US20190233814A1 (en) 2015-12-18 2019-08-01 The Broad Institute, Inc. Novel crispr enzymes and systems
CN113797348A (zh) 2016-03-07 2021-12-17 箭头药业股份有限公司 用于治疗性化合物的靶向配体
US10407469B2 (en) 2016-04-01 2019-09-10 Northwestern University Programmable polypeptide and nucleic acid nanoparticles
MA45478A (fr) * 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
KR102468177B1 (ko) 2016-04-14 2022-11-16 에프. 호프만-라 로슈 아게 트리틸-모노-GalNAc 화합물 및 이의 용도
SG10202010311SA (en) 2016-04-19 2020-11-27 Broad Inst Inc Novel Crispr Enzymes and Systems
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
CN109689106B (zh) 2016-04-26 2023-01-24 微而广治疗有限公司 治疗虾类病毒感染的组合物和方法
CN114736256A (zh) 2016-06-06 2022-07-12 箭头药业股份有限公司 5’-环膦酸酯修饰核苷酸
PT3484524T (pt) 2016-07-15 2023-02-15 Ionis Pharmaceuticals Inc Composições e métodos para a modulação de smn2
WO2018017814A1 (en) 2016-07-20 2018-01-25 President And Fellows Of Harvard College Peptidoglycan glycosyltransferase inhibitors of sed proteins for treating bacterial infections
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
KR102403408B1 (ko) 2016-09-02 2022-05-30 애로우헤드 파마슈티컬스 인코포레이티드 표적화 리간드
US11202818B2 (en) 2016-09-14 2021-12-21 President And Fellows Of Harvard College Methods and compositions for modulating erythropoiesis
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
WO2018112033A1 (en) 2016-12-13 2018-06-21 President And Fellows Of Harvard College Methods and compositions for targeting tumor-infiltrating tregs
US20190314427A1 (en) 2016-12-16 2019-10-17 Evelo Biosciences, Inc. Methods of treating cancer using parabacteroides
WO2018112360A1 (en) 2016-12-16 2018-06-21 Evelo Biosciences, Inc. Combination therapies for treating cancer
WO2018112364A1 (en) 2016-12-16 2018-06-21 Evelo Biosciences, Inc. Combination therapies for treating melanoma
WO2018112365A2 (en) 2016-12-16 2018-06-21 Evelo Biosciences, Inc. Methods of treating colorectal cancer and melanoma using parabacteroides goldsteinii
JP7258773B2 (ja) 2017-01-10 2023-04-17 アローヘッド ファーマシューティカルズ インコーポレイテッド α－１アンチトリプシン（ＡＡＴ）ＲＮＡｉ剤、ＡＡＴ ＲＮＡｉ剤を含む組成物、及び使用方法。
WO2018136598A1 (en) 2017-01-18 2018-07-26 Evelo Biosciences, Inc. Methods of treating cancer
WO2018136617A2 (en) 2017-01-18 2018-07-26 Evelo Biosciences, Inc. Methods of treating cancer
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
SG11201909433XA (en) 2017-04-11 2019-11-28 Arbutus Biopharma Corp Targeted compositions
WO2018215049A1 (en) 2017-05-23 2018-11-29 F. Hoffmann-La Roche Ag Process for galnac oligonucleotide conjugates
US10590416B2 (en) 2017-07-06 2020-03-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of alpha-ENaC and methods of use
WO2019014491A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals, Inc. METHODS OF INHIBITING GENE EXPRESSION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE))
WO2019046401A1 (en) 2017-08-29 2019-03-07 Evelo Biosciences, Inc. TREATMENT OF CANCER USING BLAUTIA STRAIN
EP3681516A4 (en) 2017-09-11 2021-09-29 Arrowhead Pharmaceuticals, Inc. RNA INTERFERENCE AGENTS AND COMPOSITIONS INTENDED TO INHIBIT APOLIPOPROTEIN C-III (APOC3) EXPRESSION
CN117701562A (zh) 2017-09-14 2024-03-15 箭头药业股份有限公司 用于抑制血管生成素-样3(ANGPTL3)的表达的RNAi剂和组合物以及使用方法
EP3679141B1 (en) 2017-10-13 2023-06-07 Novo Nordisk Health Care AG Methods and compositions for inhibiting expression of ldha
AU2018352379A1 (en) 2017-10-17 2020-06-04 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of Asialoglycoprotein receptor 1
CN110997917B (zh) 2017-12-01 2024-04-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
EP3719128A4 (en) 2017-12-01 2021-10-27 Suzhou Ribo Life Science Co., Ltd. DOUBLE STRANDED OLIGONUCLEOTIDE, COMPOSITION AND CONJUGATE WITH DOUBLE STRANDED OLIGONUCLEOTIDE, METHOD OF MANUFACTURING THEREOF AND USE THEREOF
EA202091513A1 (ru) 2017-12-19 2020-09-09 Янссен Сайенсиз Айрлэнд Анлимитед Компани Вакцины против вируса гепатита b (hbv) и их применение
CN110959011B (zh) 2017-12-29 2023-03-28 苏州瑞博生物技术股份有限公司 缀合物及其制备方法和用途
TW201934129A (zh) 2018-01-15 2019-09-01 美商Ionis製藥公司 Ｄｎｍ２表現之調節劑
EP3746131A1 (en) * 2018-01-29 2020-12-09 F. Hoffmann-La Roche AG Process for the preparation of galnac oligonucleotide conjugates
AU2019218987A1 (en) 2018-02-12 2020-07-23 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
WO2019169143A1 (en) 2018-02-28 2019-09-06 Evelo Biosciences, Inc. Compositions and methods for treating cancer using turicibacter sanguinis
WO2019169138A1 (en) 2018-02-28 2019-09-06 Evelo Biosciences, Inc. Compositions and methods for treating cancer using paraclostridium benzoelyticum
WO2019169168A1 (en) 2018-02-28 2019-09-06 Evelo Biosciences, Inc. Compositions and methods for treating cancer using agathobaculum
WO2019169160A1 (en) 2018-02-28 2019-09-06 Evelo Biosciences, Inc. Compositions and methods for treating cancer using ruminococcus gnavus
US20210113628A1 (en) 2018-03-12 2021-04-22 Sqz Biotechnologies Company Intracellular delivery of biomolecules to modify immune response
KR20200140322A (ko) 2018-04-05 2020-12-15 사일런스 테라퓨틱스 게엠베하 안티센스 가닥의 5' 단부에 비닐포스포네이트를 갖는 siRNA
KR20210018267A (ko) 2018-05-07 2021-02-17 알닐람 파마슈티칼스 인코포레이티드 간외 전달
CA3099698A1 (en) 2018-05-08 2019-11-14 Charles R. ALLERSON Galnac conjugated modified oligonucleotide as mir-122 inhibitor having hcv antiviral activity with reduced hyperbilirubinemia side-effect
SG11202010012PA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing fxi expression
TW202019441A (zh) 2018-07-20 2020-06-01 美商雷格勒斯治療公司 用於經口遞送寡核苷酸之方法
US20210292768A1 (en) 2018-08-08 2021-09-23 Arcturus Therapeutics, Inc. Compositions and agents against nonalcoholic steatohepatitis
US11918600B2 (en) 2018-08-21 2024-03-05 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof
US11273137B2 (en) 2018-09-04 2022-03-15 Board Of Trustees Of Michigan State University Methods and compositions to prevent and treat disorders associated with mutations in the ODC1 gene
TW202023573A (zh) 2018-09-19 2020-07-01 美商Ionis製藥公司 Pnpla3表現之調節劑
US11896674B2 (en) 2018-09-30 2024-02-13 Suzhou Ribo Life Science Co., Ltd. SiRNA conjugate, preparation method therefor and use thereof
CN112423794A (zh) * 2018-12-28 2021-02-26 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
WO2020135581A1 (zh) * 2018-12-28 2020-07-02 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
MX2021009495A (es) 2019-02-07 2021-09-08 Arrowhead Pharmaceuticals Inc Agentes de iarn para infeccion por el virus de la hepatitis b.
EP4112125A1 (en) 2019-03-12 2023-01-04 President and Fellows of Harvard College Methods and compositions for treating cancer
WO2020214976A1 (en) 2019-04-18 2020-10-22 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
WO2020214974A1 (en) 2019-04-18 2020-10-22 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
US20220226269A1 (en) 2019-06-12 2022-07-21 President And Fellows Of Harvard College Methods and compositions for modulation of an interspecies gut bacterial pathway for levodopa metabolism
AU2020297008A1 (en) 2019-06-18 2022-02-17 Janssen Sciences Ireland Unlimited Company Combination of hepatitis B virus (HBV) vaccines and HBV-targeting RNAi
AU2020295796A1 (en) 2019-06-18 2022-02-10 Janssen Sciences Ireland Unlimited Company Combination of hepatitis B virus (HBV) vaccines and HBV-targeting rnai
CN110218728A (zh) 2019-06-28 2019-09-10 厦门甘宝利生物医药有限公司 一种新化合物及其应用
CA3144467A1 (en) * 2019-07-30 2021-02-04 Jonathan Miles Brown Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity
WO2021022110A1 (en) 2019-08-01 2021-02-04 Evelo Biosciences, Inc. Inducing immune effects using bacteria of the genus bifidobacterium
JP2022552249A (ja) 2019-10-14 2022-12-15 アストラゼネカ・アクチエボラーグ Ｐｎｐｌａ３発現のモジュレーター
US20230054711A1 (en) * 2019-12-31 2023-02-23 Xiamen University Multimerization delivery system for intracellular delivery of molecule
CA3163848A1 (en) 2020-01-06 2021-07-15 Dong In Lee Composition for preventing or treating macular degeneration, containing cell permeable nucleic acid complex as active ingredient
US20220064638A1 (en) 2020-02-28 2022-03-03 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
CN115485385A (zh) 2020-03-04 2022-12-16 瑞泽恩制药公司 用于使肿瘤细胞对免疫疗法敏感的方法和组合物
WO2021178612A1 (en) 2020-03-05 2021-09-10 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
TW202202153A (zh) 2020-03-26 2022-01-16 美商艾羅海德製藥公司 用於抑制ＰＮＰＬＡ３表現之ＲＮＡｉ劑、其醫藥組成物及使用方法
EP4157264A1 (en) 2020-05-27 2023-04-05 The Regents of the University of California Compositions and methods for transdifferentiating cells
AU2021297475A1 (en) 2020-06-22 2023-01-05 Janssen Pharmaceuticals, Inc. Compositions and methods for treatment of Hepatitis D Virus infection
MX2023002853A (es) 2020-09-11 2023-03-31 Arrowhead Pharmaceuticals Inc Agentes de arni para inhibir la expresion de dux4, composiciones de dichos agentes, y metodos de uso.
AU2021340710A1 (en) 2020-09-11 2023-04-06 Arrowhead Pharmaceuticals, Inc. Skeletal muscle delivery platforms and methods of use
AU2021342158A1 (en) 2020-09-11 2023-04-13 Arrowhead Pharmaceuticals, Inc. Lipid conjugates for the delivery of therapeutic agents
WO2022056454A2 (en) 2020-09-14 2022-03-17 President And Fellows Of Harvard College Methods and compositions for treating hpv-positive cancers
US20230340124A1 (en) 2020-09-16 2023-10-26 President And Fellows Of Harvard College Methods of treating an individual that has failed an anti-pd-1/anti-pd-l1 therapy
US11447521B2 (en) 2020-11-18 2022-09-20 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
WO2022133230A1 (en) 2020-12-18 2022-06-23 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
WO2022152869A1 (en) 2021-01-15 2022-07-21 Janssen Sciences Ireland Unlimited Company Use of oligonucleotides for individuals with hepatic impairment
CN114767704A (zh) * 2021-01-21 2022-07-22 圣诺制药公司 一种能够靶向乙型肝炎病毒的药物构造及药物组合物
US11629349B2 (en) 2021-06-21 2023-04-18 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
WO2023281434A1 (en) 2021-07-09 2023-01-12 Janssen Pharmaceuticals, Inc. Use of oligonucleotides for individuals with renal impairment
WO2023084510A1 (en) 2021-11-09 2023-05-19 Viaqua Therapeutics Ltd. Compositions for aquaculturing
WO2023150181A1 (en) 2022-02-01 2023-08-10 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
WO2023245060A2 (en) 2022-06-15 2023-12-21 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of superoxide dismutase 1 (sod1), compositions thereof, and methods of use

Family Cites Families (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4587044A (en) * 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5166320A (en) * 1987-04-22 1992-11-24 University Of Connecticut Carrier system and method for the introduction of genes into mammalian cells
US4904582A (en) * 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5138045A (en) * 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
NZ244306A (en) * 1991-09-30 1995-07-26 Boehringer Ingelheim Int Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation
AU682308C (en) * 1992-04-03 2006-08-17 Regents Of The University Of California, The Self-assembling polynucleotide delivery system
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US5574142A (en) * 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
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
JP2690276B2 (ja) * 1995-01-10 1997-12-10 科学技術振興事業団 静電結合型高分子ミセル薬物担体とその薬剤
CN1120707C (zh) * 1995-11-22 2003-09-10 约翰斯·霍普金斯大学 增强生物分子的细胞摄取的配体
US8217015B2 (en) * 2003-04-04 2012-07-10 Arrowhead Madison Inc. Endosomolytic polymers
US20020165183A1 (en) * 1999-11-29 2002-11-07 Hans Herweijer Methods for genetic immunization
US5994316A (en) * 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
US6020457A (en) * 1996-09-30 2000-02-01 Dendritech Inc. Disulfide-containing dendritic polymers
GB9623051D0 (en) * 1996-11-06 1997-01-08 Schacht Etienne H Delivery of DNA to target cells in biological systems
DE19652586A1 (de) 1996-12-17 1998-06-18 Biotechnolog Forschung Gmbh Dhc-Peptid und Mittel
CA2294988C (en) * 1997-07-01 2015-11-24 Isis Pharmaceuticals Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
US5877309A (en) * 1997-08-13 1999-03-02 Isis Pharmaceuticals, Inc. Antisense oligonucleotides against JNK
US6300319B1 (en) * 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US8038984B2 (en) * 1998-06-20 2011-10-18 Washington University Membrane-permeant peptide complexes for treatment of sepsis
US20050112470A1 (en) * 1998-06-26 2005-05-26 Johnson Controls Technology Company Alloy for battery grids
US6773920B1 (en) * 1999-03-31 2004-08-10 Invitrogen Corporation Delivery of functional protein sequences by translocating polypeptides
US8211468B2 (en) * 1999-06-07 2012-07-03 Arrowhead Madison Inc. Endosomolytic polymers
DK1102785T3 (da) 1999-06-07 2013-05-13 Arrowhead Res Corp Sammensætninger til lægemiddeltilførsel ved anvendelse af pH-følsomme molekyler
US7019113B2 (en) * 1999-06-07 2006-03-28 Mirus Bio Corporation Reversible modification of membrane interaction
US20080281041A1 (en) * 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US7442764B2 (en) * 1999-06-07 2008-10-28 Mirns Bio Corporation Reversible modification of amine-containing compounds
US20040072785A1 (en) * 1999-11-23 2004-04-15 Wolff Jon A. Intravascular delivery of non-viral nucleic acid
US7098030B2 (en) * 1999-12-31 2006-08-29 Mirus Bio Corporation Polyampholytes for delivering polyions to a cell
US7491805B2 (en) * 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7833992B2 (en) * 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US20030072794A1 (en) * 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
JP2002122707A (ja) 2000-10-13 2002-04-26 Canon Inc 非球面マイクロ構造体、及びその作製方法
US20050148530A1 (en) * 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
EP1575976A4 (en) * 2001-11-02 2006-08-23 Insert Therapeutics Inc METHODS AND COMPOSITIONS FOR THE THERAPEUTIC USE OF RNA INTERFERENCE
US7176303B2 (en) * 2003-11-06 2007-02-13 Isis Pharmaceuticals, Inc. Modulation of STAT5 expression
US7220400B2 (en) * 2002-05-24 2007-05-22 Mirus Bio Corporation Compositions for delivering nucleic acids to cells
EP1658304A4 (en) * 2002-09-28 2009-01-14 Massachusetts Inst Technology THERAPY ANTIGRIPPALE
US20040204377A1 (en) * 2002-11-26 2004-10-14 University Of Massachusetts Delivery of siRNAs
US7682626B2 (en) * 2003-02-07 2010-03-23 Roche Madison Inc. Polyvinylethers for delivery of polynucleotides to mammalian cells
US7816337B2 (en) 2003-02-18 2010-10-19 Roche Madison Inc. Reversible attachment of a membrane active polymer to a polynucleotide
WO2004087931A1 (en) * 2003-04-03 2004-10-14 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US8454566B2 (en) 2003-07-10 2013-06-04 Medtronic Minimed, Inc. Methods and compositions for the inhibition of biofilms on medical devices
US20060040882A1 (en) * 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US20060008907A1 (en) * 2004-06-09 2006-01-12 The Curators Of The University Of Missouri Control of gene expression via light activated RNA interference
EP2199298A1 (en) * 2004-11-17 2010-06-23 Protiva Biotherapeutics Inc. Sirna silencing of Apolipoprotein B
US7923206B2 (en) * 2004-11-22 2011-04-12 Dharmacon, Inc. Method of determining a cellular response to a biological agent
WO2006078217A1 (en) 2005-01-24 2006-07-27 Avaris Ab COMPLEX CONTAINING SiRNA, ShRNA OR ANTISENSE MOLECULE AND FUNCTIONAL ENTITY, FOR IMPROVED SPECIFICITY AND DELIVERY
AU2006231452B2 (en) * 2005-04-01 2011-05-26 Intezyne Technologies, Inc. Polymeric micelles for drug delivery
US9139850B2 (en) * 2005-05-19 2015-09-22 L'oreal Vectorization of dsRNA by cationic particles and topical use
JP2007112768A (ja) * 2005-10-24 2007-05-10 Kyoto Univ 肝指向性リポソーム組成物
US8017109B2 (en) * 2006-08-18 2011-09-13 Roche Madison Inc. Endosomolytic poly(acrylate) polymers
WO2008022309A2 (en) * 2006-08-18 2008-02-21 F. Hoffmann-La Roche Ag Polyconjugates for in vivo delivery of polynucleotides
JP6137834B2 (ja) * 2010-02-24 2017-05-31 アローヘッド ファーマシューティカルズ インコーポレイテッド ｓｉＲＮＡの標的化送達のための組成物

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DK2539451T3 (da) 2016-04-04
JP2016074671A (ja) 2016-05-12
AP2012006453A0 (en) 2012-08-31
CA2788600A1 (en) 2011-09-01
PT2539451E (pt) 2016-03-28
US20150202298A1 (en) 2015-07-23
AU2011219941A1 (en) 2012-08-16
US9011919B2 (en) 2015-04-21
IL221396A (en) 2016-11-30
US20110207799A1 (en) 2011-08-25
EP3023495A1 (en) 2016-05-25
HUE026811T2 (en) 2016-07-28
US20170204408A9 (en) 2017-07-20
JP2013520460A (ja) 2013-06-06
SMT201600079B (it) 2016-04-29
MX2012009178A (es) 2012-11-30
EA024534B1 (ru) 2016-09-30
JP6137834B2 (ja) 2017-05-31
WO2011104169A1 (en) 2011-09-01
EA201290602A1 (ru) 2013-04-30
EP2539451B1 (en) 2016-01-06
AU2011219941C1 (en) 2015-05-07
CN102869774A (zh) 2013-01-09
US9345775B2 (en) 2016-05-24
CN102869774B (zh) 2018-02-09
CA2788600C (en) 2019-12-03
SI2539451T1 (sl) 2016-04-29
US8313772B2 (en) 2012-11-20
US10316316B2 (en) 2019-06-11
ZA201205685B (en) 2013-09-25
AP3284A (en) 2015-05-31
EP3023495B1 (en) 2019-05-08
HRP20160093T1 (hr) 2016-02-26
BR112012021256A2 (pt) 2018-03-06
IL221396A0 (en) 2012-10-31
KR20130012112A (ko) 2013-02-01
US20160222384A1 (en) 2016-08-04
KR101956623B1 (ko) 2019-03-12
ES2562817T3 (es) 2016-03-08
AU2011219941B2 (en) 2014-10-23
NZ601737A (en) 2013-06-28
JP6290848B2 (ja) 2018-03-07

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