DK3569711T3 - Ligandmodificerede dobbeltstrengede nukleinsyrer - Google Patents

Ligandmodificerede dobbeltstrengede nukleinsyrer Download PDF

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Publication number
DK3569711T3
DK3569711T3 DK19180844.3T DK19180844T DK3569711T3 DK 3569711 T3 DK3569711 T3 DK 3569711T3 DK 19180844 T DK19180844 T DK 19180844T DK 3569711 T3 DK3569711 T3 DK 3569711T3
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Denmark
Prior art keywords
nucleic acids
modified double
ligand modified
double string
string
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DK19180844.3T
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English (en)
Inventor
Bob Dale Brown
Weimin Wang
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Dicerna Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H1/00—Processes for the preparation of sugar derivatives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H99/00—Subject matter not provided for in other groups of this subclass
    • 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
    • C12N15/11—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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • 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
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • 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
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
DK19180844.3T 2014-12-15 2015-12-15 Ligandmodificerede dobbeltstrengede nukleinsyrer DK3569711T3 (da)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462092241P 2014-12-15 2014-12-15
US201462092238P 2014-12-15 2014-12-15
US201562187856P 2015-07-02 2015-07-02
US201562187848P 2015-07-02 2015-07-02
EP15817743.6A EP3234132B1 (en) 2014-12-15 2015-12-15 Ligand-modified double-stranded nucleic acids

Publications (1)

Publication Number Publication Date
DK3569711T3 true DK3569711T3 (da) 2021-02-22

Family

ID=56127491

Family Applications (2)

Application Number Title Priority Date Filing Date
DK19180844.3T DK3569711T3 (da) 2014-12-15 2015-12-15 Ligandmodificerede dobbeltstrengede nukleinsyrer
DK15817743.6T DK3234132T3 (da) 2014-12-15 2015-12-15 Ligand-modificerede dobbeltstrengede nukleinsyrer

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK15817743.6T DK3234132T3 (da) 2014-12-15 2015-12-15 Ligand-modificerede dobbeltstrengede nukleinsyrer

Country Status (8)

Country Link
US (2) US20170305956A1 (da)
EP (3) EP3569711B1 (da)
JP (1) JP7105065B2 (da)
AU (2) AU2015362630B2 (da)
CA (1) CA2970801C (da)
DK (2) DK3569711T3 (da)
ES (2) ES2858403T3 (da)
WO (1) WO2016100401A1 (da)

Families Citing this family (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3204497B1 (en) 2014-10-10 2020-03-11 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
EP3506909B1 (en) 2016-09-02 2022-06-29 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
CA3037192A1 (en) * 2016-11-07 2018-05-11 nanoSUR LLC Post-transcriptionally chemically modified double strand rnas
WO2018102397A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2019006375A1 (en) * 2017-06-29 2019-01-03 Dicerna Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR INHIBITING HMGB1 EXPRESSION
HUE063026T2 (hu) * 2017-10-13 2023-12-28 Novo Nordisk Healthcare Ag Eljárások és készítmények LDHA expressziójának gátlására
CA3076924A1 (en) * 2017-10-18 2019-04-25 Dicerna Pharmaceuticals, Inc. Beta catenin nucleic acid inhibitor molecule
FI3684377T3 (fi) 2017-10-20 2023-01-31 Menetelmiä hepatiitti b -infektion hoitamiseksi
KR20200106513A (ko) 2018-01-05 2020-09-14 다이서나 파마수이티컬, 인크. 면역요법을 강화시키기 위하여 베타-카테닌 및 ido 발현의 감소
MX2020007582A (es) 2018-01-16 2020-09-03 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de aldehido deshidrogenasa mitocondrial 2 (aldh2).
KR20200127211A (ko) 2018-03-02 2020-11-10 다이서나 파마수이티컬, 인크. Gys2 발현을 억제하기 위한 조성물 및 방법
CN111971051A (zh) * 2018-04-13 2020-11-20 迪克纳制药公司 用增加tm的核苷酸修饰的双链核酸抑制剂分子
AU2019270152A1 (en) * 2018-05-16 2020-12-10 Alnylam Pharmaceuticals, Inc. Modified RNA agents with reduced off-target effect
CA3110014A1 (en) * 2018-08-27 2020-03-05 Sirnaomics, Inc. Products and compositions
BR112021005815A2 (pt) 2018-09-28 2021-06-29 Nutcracker Therapeutics, Inc. formulações de nanoparticulas lipídicas compreendendo compostos peptídicos catiônicos lipidados para liberação de ácido nucleico
SG11202106046XA (en) * 2018-12-12 2021-07-29 Dicerna Pharmaceuticals Inc Double-stranded nucleic acid inhibitor molecules containing a triloop
CA3125289A1 (en) * 2018-12-28 2020-07-02 Sirnaomics, Inc. Targeted delivery of therapeutic molecules
MX2021009754A (es) 2019-02-12 2021-09-08 Dicerna Pharmaceuticals Inc Metodos y composiciones para inhibir la expresion de cyp27a1.
MX2021011928A (es) 2019-03-29 2022-01-04 Dicerna Pharmaceuticals Inc Composiciones y metodos para el tratamiento de enfermedades o trastornos asociados con kras.
EP3947683A1 (en) * 2019-04-04 2022-02-09 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression in the central nervous system
EP3963072A1 (en) 2019-05-03 2022-03-09 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
CA3149775A1 (en) 2019-08-30 2021-03-04 Weimin Wang Ligand-2'-modified nucleic acids, synthesis thereof and intermediate compounds thereof
WO2021062251A2 (en) * 2019-09-25 2021-04-01 University Of Massachusetts Targeting rlim to modulate body weight and obesity
WO2021067744A1 (en) 2019-10-02 2021-04-08 Dicerna Pharmaceuticals, Inc. Chemical modifications of small interfering rna with minimal fluorine content
EP4048313A4 (en) * 2019-10-25 2023-05-31 Phitonex, Inc. POLYNUCLEOTIDE-LINKED BIOCONJUGATES AND METHODS OF PREPARING AND USING
TW202137987A (zh) 2019-12-24 2021-10-16 瑞士商赫孚孟拉羅股份公司 用於治療ｈｂｖ之靶向ｈｂｖ的治療性寡核苷酸及ｔｌｒ７促效劑之醫藥組合
WO2021130270A1 (en) 2019-12-24 2021-07-01 F. Hoffmann-La Roche Ag Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
AU2021207504A1 (en) 2020-01-15 2022-08-04 Dicerna Pharmaceuticals, Inc. 4'-O-methylene phosphonate nucleic acids and analogues thereof
WO2021153687A1 (ja) 2020-01-30 2021-08-05 エーザイ・アール・アンド・ディー・マネジメント株式会社 核酸複合体及びそれを含む医薬組成物
EP4121536A1 (en) 2020-03-18 2023-01-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting angptl3 expression
TW202221120A (zh) 2020-08-04 2022-06-01 美商黛瑟納製藥公司 用於治療代謝症候群之組成物及方法
EP4192478A2 (en) 2020-08-04 2023-06-14 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting plp1 expression
KR20230061389A (ko) 2020-08-04 2023-05-08 다이서나 파마수이티컬, 인크. 올리고뉴클레오티드의 전신 전달
JP7476422B2 (ja) 2020-08-05 2024-04-30 ディセルナ ファーマシューティカルズ インコーポレイテッド Ｌｐａ発現を阻害するための組成物及び方法
KR20230043877A (ko) 2020-08-05 2023-03-31 에프. 호프만-라 로슈 아게 B형 간염 환자의 올리고뉴클레오티드 치료
IL301940A (en) 2020-10-08 2023-06-01 Dicerna Pharmaceuticals Inc Selective delivery of oligonucleotides to glial cells
TW202233839A (zh) 2020-11-13 2022-09-01 美商戴瑟納製藥股份有限公司 用於抑制ａｌｄｈ２表現之化學修飾
GB202020534D0 (en) * 2020-12-23 2021-02-03 Argonaute Rna Ltd Conjugate
WO2022136466A1 (en) * 2020-12-23 2022-06-30 Argonaute RNA Limited Treatment of cardiovascular disease
AR124713A1 (es) * 2021-01-28 2023-04-26 Dicerna Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central
KR20230170732A (ko) 2021-04-12 2023-12-19 베링거 인겔하임 인터내셔날 게엠베하 케토헥소키나제 (khk)를 억제하기 위한 조성물 및 방법
US20220364098A1 (en) 2021-04-14 2022-11-17 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating pnpla3 expression
EP4326872A2 (en) 2021-04-19 2024-02-28 Novo Nordisk A/S Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
US11655473B2 (en) 2021-05-28 2023-05-23 Novo Nordisk A/S Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (MARC1) expression
US20230138928A1 (en) 2021-08-05 2023-05-04 Sanegene Bio Usa Inc. 1'-alkyl modified ribose derivatives and methods of use
WO2023016999A2 (en) * 2021-08-09 2023-02-16 Cargene Therapeutics Pte. Ltd. Compounds
WO2023027759A1 (en) 2021-08-25 2023-03-02 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting αlpha-1 antitrypsin expression
IL311486A (en) 2021-09-22 2024-05-01 Sanegene Bio Usa Inc 2'-Alkyl or 3'-Alkyl Modified Ribose Derivatives for Use in Intracorporeal Oligonucleotide Administration
AR127260A1 (es) * 2021-10-05 2024-01-03 Dicerna Pharmaceuticals Inc Composiciones y métodos para inhibir el receptor de melanocortina 2 y la expresión del citocromo p450 11b
AU2022361238A1 (en) 2021-10-05 2024-04-11 Sanegene Bio Usa Inc. Polyhydroxylated cyclopentane derivatives and methods of use
AU2022384619A1 (en) 2021-11-11 2024-04-11 F. Hoffmann-La Roche Ag Pharmaceutical combinations for treatment of hbv
WO2023102469A2 (en) 2021-12-01 2023-06-08 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating apoc3 expression
WO2023109935A1 (zh) * 2021-12-16 2023-06-22 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
WO2023164464A1 (en) 2022-02-22 2023-08-31 Sanegene Bio Usa Inc. 5'-modified carbocyclic ribonucleotide derivatives and methods of use
WO2023177866A1 (en) 2022-03-18 2023-09-21 Dicerna Pharmaceuticals, Inc. Decarboxylative acetoxylation using mn(ii) or mn(iii) reagent for synthesis of 4'-acetoxy- nucleoside and use thereof for synthesis of corresponding 4'-(dimethoxyphosphoryl)methoxy- nucleotide
TW202345873A (zh) 2022-04-15 2023-12-01 美商戴瑟納製藥股份有限公司 調節ｓｃａｐ活性之組合物及方法
US20240002857A1 (en) 2022-05-09 2024-01-04 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
TW202400792A (zh) 2022-05-12 2024-01-01 美商戴瑟納製藥股份有限公司 用於抑制ｍａｐｔ表現之組合物及方法
WO2023220351A1 (en) 2022-05-13 2023-11-16 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting snca expression
TW202400193A (zh) 2022-06-24 2024-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法
WO2024015796A1 (en) 2022-07-11 2024-01-18 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
US20240052348A1 (en) 2022-08-05 2024-02-15 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
WO2024040041A1 (en) 2022-08-15 2024-02-22 Dicerna Pharmaceuticals, Inc. Regulation of activity of rnai molecules
WO2024079076A1 (en) 2022-10-11 2024-04-18 Boehringer Ingelheim International Gmbh Methods for the treatment of nash with advanced fibrosis and/or cirrhosis
US20240139230A1 (en) 2022-10-11 2024-05-02 Boehringer Ingelheim International Gmbh Dosage regimen for the treatment of nash
US20240175026A1 (en) 2022-10-14 2024-05-30 Sanegene Bio Usa Inc. Small interfering rna targeting c3 and uses thereof
WO2024097821A1 (en) * 2022-11-02 2024-05-10 Sanegene Bio Usa Inc. Nucleotide-based enhancement agent for rna delivery and therapy
WO2024107993A1 (en) 2022-11-16 2024-05-23 Dicerna Pharmaceuticals, Inc. Stat3 targeting oligonucleotides and uses thereof

Family Cites Families (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
EP0500799B1 (en) 1989-11-16 1998-01-14 Duke University Particle mediated transformation of animal skin tissue cells
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
DE69123979T2 (de) 1990-10-12 1997-04-30 Max Planck Gesellschaft Abgeänderte ribozyme
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
WO1994018987A1 (en) 1993-02-19 1994-09-01 Nippon Shinyaku Co., Ltd. Drug composition containing nucleic acid copolymer
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
US5858988A (en) 1993-02-24 1999-01-12 Wang; Jui H. Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use
TW360548B (en) 1993-04-08 1999-06-11 Powderject Res Ltd Products for therapeutic use
US5994316A (en) 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US6218108B1 (en) 1997-05-16 2001-04-17 Research Corporation Technologies, Inc. Nucleoside analogs with polycyclic aromatic groups attached, methods of synthesis and uses therefor
US6042820A (en) 1996-12-20 2000-03-28 Connaught Laboratories Limited Biodegradable copolymer containing α-hydroxy acid and α-amino acid units
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6472375B1 (en) 1998-04-16 2002-10-29 John Wayne Cancer Institute DNA vaccine and methods for its use
US20050054598A1 (en) 2002-02-20 2005-03-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition hairless (HR) gene expression using short interfering nucleic acid (siNA)
US8729036B2 (en) 2002-08-07 2014-05-20 University Of Massachusetts Compositions for RNA interference and methods of use thereof
JP2006522158A (ja) 2003-04-03 2006-09-28 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 ｉＲＮＡ複合体
JP4912873B2 (ja) 2003-04-09 2012-04-11 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 ｉＲＮＡ複合体
US20050042641A1 (en) 2003-05-27 2005-02-24 Cold Spring Harbor Laboratory In vivo high throughput selection of RNAi probes
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
AU2005222965B8 (en) 2004-03-15 2010-07-01 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20080213891A1 (en) 2004-07-21 2008-09-04 Alnylam Pharmaceuticals, Inc. RNAi Agents Comprising Universal Nucleobases
US20070161591A1 (en) 2005-08-18 2007-07-12 University Of Massachusetts Methods and compositions for treating neurological disease
US20090018097A1 (en) 2005-09-02 2009-01-15 Mdrna, Inc Modification of double-stranded ribonucleic acid molecules
JP4644619B2 (ja) 2006-03-27 2011-03-02 富士通株式会社 基地局装置、端末および帯域制御方法
GB0610636D0 (en) 2006-05-30 2006-07-05 Univ London Materials and complexes for the delivery of biologically-active material to cells
CA2930393C (en) * 2007-12-04 2022-11-29 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
US20100331389A1 (en) * 2008-09-22 2010-12-30 Bob Dale Brown Compositions and methods for the specific inhibition of gene expression by dsRNA containing modified nucleotides
EP3587434A1 (en) * 2008-09-23 2020-01-01 Alnylam Pharmaceuticals Inc. Chemical modifications of monomers and oligonucleotides with click components for conjugation with ligands
CA3151965A1 (en) * 2008-12-18 2010-07-15 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
CN102753187A (zh) * 2009-06-03 2012-10-24 戴瑟纳制药公司 肽-dicer底物试剂及其特异性抑制基因表达的方法
WO2011111874A1 (ja) * 2010-03-11 2011-09-15 独立行政法人理化学研究所 細胞膜透過性ダンベル型rnaおよびその製造方法
WO2011130458A2 (en) * 2010-04-13 2011-10-20 John Rossi Rna aptamers against baff-r as cell-type specific delivery agents and methods for their use
WO2011132672A1 (ja) * 2010-04-19 2011-10-27 独立行政法人理化学研究所 機能性核酸の安定化法
WO2012030683A2 (en) * 2010-08-31 2012-03-08 Merck Sharp & Dohme Corp. Novel single chemical entities and methods for delivery of oligonucleotides
CA2822161C (en) * 2010-12-29 2018-05-29 Philipp Hadwiger Small molecule conjugates for intracellular delivery of nucleic acids
KR20220045091A (ko) 2011-11-18 2022-04-12 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴（ＴＴＲ） 관련 질병을 치료하기 위한 ＲＮＡｉ 제제, 조성 및 그의 사용방법
WO2014005596A1 (en) * 2012-07-03 2014-01-09 Aarhus Universitet Modified payload molecules and their interactions and uses
DK3019200T3 (da) 2013-07-11 2022-06-20 Alnylam Pharmaceuticals Inc Oligonukleotid-ligand-konjugater og fremgangsmåde til fremstiling deraf
EP3077511A4 (en) * 2013-12-06 2017-07-05 Dicerna Pharmaceuticals Inc. Methods and compositions for the specific inhibition of transthyretin (ttr) by double-stranded rna
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

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