UY37146A - Ligandos de direccionamiento para compuestos terapéuticos - Google Patents

Ligandos de direccionamiento para compuestos terapéuticos

Info

Publication number
UY37146A
UY37146A UY0001037146A UY37146A UY37146A UY 37146 A UY37146 A UY 37146A UY 0001037146 A UY0001037146 A UY 0001037146A UY 37146 A UY37146 A UY 37146A UY 37146 A UY37146 A UY 37146A
Authority
UY
Uruguay
Prior art keywords
compounds
targeting ligands
therapeutic compounds
therapeutic
expression
Prior art date
Application number
UY0001037146A
Other languages
English (en)
Spanish (es)
Inventor
H Wakefield Darren
Tao Pei
B Rozema David
V Blokhin Andrei
D Benson Jonathan
Zhen Li
Fred Fleitz
Original Assignee
Arrowhead Pharmaceuticals Inc
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 Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of UY37146A publication Critical patent/UY37146A/es

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
    • C12N15/11—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
    • A61K47/50—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
    • 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
    • A61K48/0033—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 non-polymeric
    • 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
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
UY0001037146A 2016-03-07 2017-03-07 Ligandos de direccionamiento para compuestos terapéuticos UY37146A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662304652P 2016-03-07 2016-03-07
US201662370754P 2016-08-04 2016-08-04
US201662426916P 2016-11-28 2016-11-28

Publications (1)

Publication Number Publication Date
UY37146A true UY37146A (es) 2017-09-29

Family

ID=59723428

Family Applications (1)

Application Number Title Priority Date Filing Date
UY0001037146A UY37146A (es) 2016-03-07 2017-03-07 Ligandos de direccionamiento para compuestos terapéuticos

Country Status (15)

Country Link
US (4) US10246709B2 (enExample)
EP (1) EP3426261A4 (enExample)
JP (4) JP6983797B2 (enExample)
KR (3) KR102515329B1 (enExample)
CN (2) CN113797348A (enExample)
AU (3) AU2017229469B2 (enExample)
BR (1) BR112018068230B1 (enExample)
CA (1) CA3011946A1 (enExample)
IL (3) IL296377A (enExample)
JO (2) JOP20170057B1 (enExample)
MX (3) MX2018009854A (enExample)
NZ (2) NZ785880A (enExample)
TW (3) TWI727009B (enExample)
UY (1) UY37146A (enExample)
WO (1) WO2017156012A1 (enExample)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
US10246709B2 (en) * 2016-03-07 2019-04-02 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
EA201892285A1 (ru) 2016-06-06 2019-07-31 Эрроухэд Фармасьютикалз, Инк. Нуклеотиды, модифицированные циклофосфонатом по положению 5'
JOP20170161A1 (ar) 2016-08-04 2019-01-30 Arrowhead Pharmaceuticals Inc عوامل RNAi للعدوى بفيروس التهاب الكبد ب
CA3011668A1 (en) 2016-09-02 2018-03-08 Arrowhead Pharmaceuticals, Inc. Targeting ligands
CN110268060B (zh) 2017-01-10 2024-07-26 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
EP3573623A4 (en) 2017-01-30 2020-11-25 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF INHIBITION OF GENE EXPRESSION OF FACTOR XII
WO2019051402A1 (en) 2017-09-11 2019-03-14 Arrowhead Pharmaceuticals, Inc. RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)
EP3681513A4 (en) 2017-09-14 2021-09-22 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF ANGIOPOIETIN-LIKE 3 (ANGPTL3) AND METHOD OF USE
JP7473472B2 (ja) 2017-10-17 2024-04-23 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 アシアロ糖タンパク質受容体１の発現を阻害するためのＲＮＡｉ剤および組成物
MY209138A (en) * 2018-09-19 2025-06-24 Arrowhead Pharmaceuticals Inc Rnai agents for inhibiting expression of 17beta-hsd type 13- (hsd17b13), compositions thereof, and methods of use
CN111377985B (zh) * 2018-12-29 2023-11-10 苏州瑞博生物技术股份有限公司 化合物和缀合物及其制备方法和用途
MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).
US20220305117A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company Combination of hepatitis b virus (hbv) vaccines and hbv-targeting rnai
WO2020264055A1 (en) 2019-06-25 2020-12-30 Amgen Inc. Purification methods for carbohydrate-linked oligonucleotides
EP4025252A4 (en) 2019-09-03 2023-10-18 Arcturus Therapeutics, Inc. ASIALOGLYCOPROTEIN RECEPTOR-MEDIATED ADMINISTRATION OF THERAPEUTICALLY ACTIVE CONJUGATES
MX2022010980A (es) * 2020-03-04 2022-12-02 Verve Therapeutics Inc Composiciones y metodos para el suministro de arn dirigido.
MX2022011801A (es) * 2020-03-23 2022-10-07 Amgen Inc Anticuerpos monoclonales contra acidos nucleicos modificados quimicamente y usos de los mismos.
CA3176418A1 (en) 2020-03-26 2021-09-30 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of pnpla3, pharmaceutical compositions thereof, and methods of use
WO2021198958A1 (en) 2020-04-01 2021-10-07 Janssen Biopharma, Inc. Nucleic acid polymers
US20230321284A1 (en) * 2020-04-10 2023-10-12 St. Marianna University School Of Medicine Compound, contrast agent, and method for producing compound
WO2021254360A1 (zh) * 2020-06-16 2021-12-23 上海拓界生物医药科技有限公司 一种碳水化合物分子簇及其制备方法和医药用途
KR20230067648A (ko) 2020-09-15 2023-05-16 애로우헤드 파마슈티컬스 인코포레이티드 Z-aat 단백질 수준을 감소시키기 위한 방법
TW202233837A (zh) 2020-11-05 2022-09-01 美商安進公司 用靶向ＬＰＡ的ＲＮＡｉ構建體治療動脈粥樣硬化性心血管疾病之方法
MX2023007630A (es) 2020-12-23 2023-08-25 Flagship Pioneering Innovations Vi Llc Composiciones de trem modificadas y usos de las mismas.
EP4326334A1 (en) 2021-04-23 2024-02-28 GanNA Bio, Inc. Glycan modified nucleic acids, methods of preparation, and therapeutic uses
US11549112B1 (en) 2021-06-21 2023-01-10 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
CA3225632A1 (en) * 2021-07-02 2023-01-05 Tuojie Biotech (Shanghai) Co., Ltd. Nucleic acid ligand and conjugate thereof, and preparation method therefor and use thereof
CN114703184B (zh) * 2022-03-11 2024-06-18 厦门甘宝利生物医药有限公司 Lpa抑制剂及其用途
US20250283089A1 (en) 2022-04-08 2025-09-11 Arrowhead Pharmaceuticals, Inc. Treatment of a non-alcoholic fatty liver disease
JP2025515631A (ja) * 2022-05-02 2025-05-20 アムジェン インコーポレイテッド 炭水化物ターゲティング部分であるｎａｇ－２５、及びその中間体を製造する方法
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
EP4544043A1 (en) 2022-06-22 2025-04-30 Flagship Pioneering Innovations VI, LLC Compositions of modified trems and uses thereof
AU2024240837A1 (en) 2023-03-21 2025-09-11 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of complement factor b (cfb) pharmaceutical compositions thereof, and methods of use
GB202311324D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
GB202311334D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025021831A1 (en) 2023-07-24 2025-01-30 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025038661A1 (en) 2023-08-14 2025-02-20 Arrowhead Pharmaceuticals, Inc. Oligonucleotide synthesis methods
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025074157A1 (en) 2023-10-06 2025-04-10 Glaxosmithkline Intellectual Property (No.3) Limited Treatment of a non-alcoholic fatty liver disease
TW202523695A (zh) 2023-11-22 2025-06-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於治療非酒精性脂肪性肝病之方法及組成物
US20250333742A1 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease

Family Cites Families (97)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
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
US4751219A (en) 1985-02-05 1988-06-14 Nederlandse Centrale Organisatie Voor Toegepast-Natuur-Wetenschappelijk Onderzoek Synthetic glycolipides, a process for the preparation thereof and several uses for these synthetic glycolipides
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US6172045B1 (en) 1994-12-07 2001-01-09 Neorx Corporation Cluster clearing agents
US6908903B1 (en) 1994-12-07 2005-06-21 Aletheon Pharmaceuticals, Inc. Cluster clearing agents
EP0862439A4 (en) 1995-11-22 2001-01-10 O Paul O P Ts LIGANDS FOR INCREASING THE CELLULAR UPtake OF BIOMOLECULES
US20030119724A1 (en) 1995-11-22 2003-06-26 Ts`O Paul O.P. Ligands to enhance cellular uptake of biomolecules
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
EP0950665A4 (en) 1996-09-26 2004-05-06 Ajinomoto Kk PHYSIOLOGICALLY ACTIVE MODIFIED PROTEINS AND DRUG COMPOSITIONS CONTAINING THEM
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
ID29943A (id) * 1998-10-30 2001-10-25 Daiichi Seiyaku Co Senyawa dds dan metode pengukurannya
US6383812B1 (en) 1999-05-28 2002-05-07 Academia Sinica Anti liver disease drug R-YEEE and method of synthesizing branched galactose-terminal glycoproteins
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US8541548B2 (en) 1999-06-07 2013-09-24 Arrowhead Madison Inc. Compounds and methods for reversible modification of biologically active molecules
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
CA2404890C (en) 2000-03-30 2013-11-19 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
CN100406065C (zh) 2000-12-01 2008-07-30 细胞工厂治疗公司 糖基化/半乳糖基化肽、双官能接头和核苷酸单体/多聚体的缀合物以及相关的组合物和使用方法
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
DE60231868D1 (de) 2001-04-24 2009-05-20 Purdue Research Foundation Folat-mimetika und deren folatrezeptorbindende konjugate
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
EP3231445A1 (en) 2001-05-18 2017-10-18 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
WO2003008628A2 (en) 2001-07-20 2003-01-30 Ribozyme Pharmacuticals, Inc. Enzymatic nucleic acid peptide conjugates
US7439043B2 (en) 2001-10-10 2008-10-21 Neose Technologies, Inc. Galactosyl nucleotide sugars
US20100240730A1 (en) 2002-02-20 2010-09-23 Merck Sharp And Dohme Corp. RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA)
WO2004024757A2 (en) 2002-09-11 2004-03-25 Santaris Pharma A/S Modified pna molecules
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
CA2488224A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals, Inc. Irna conjugates
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
EP2660322A3 (en) 2003-04-17 2013-11-13 Alnylam Pharmaceuticals Inc. Modified iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
JPWO2004101619A1 (ja) 2003-05-15 2006-10-26 塩野義製薬株式会社 機能的糖ペプチドの合理的設計および合成
CA2576233C (en) 2004-08-10 2016-03-15 Alnylam Pharmaceuticals, Inc. Conjugate comprising an antagomir and a ligand
US20090203132A1 (en) 2004-09-09 2009-08-13 Swayze Eric E Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
EP1841867A1 (en) 2005-01-24 2007-10-10 Avaris AB COMPLEX CONTAINING SiRNA, ShRNA OR ANTISENSE MOLECULE AND FUNCTIONAL ENTITY, FOR IMPROVED SPECIFICITY AND DELIVERY
US8658211B2 (en) 2006-08-18 2014-02-25 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
EP2061443A4 (en) 2006-08-18 2013-07-24 Arrowhead Res Corp POLYCONJUGATE FOR IN VIVO ADMINISTRATION OF POLYNUCLEOTIDES
EP2609934A1 (en) 2007-02-16 2013-07-03 KTB Tumorforschungsgesellschaft mbH Receptor And Antigen Targeted Prodrug
US8877917B2 (en) 2007-04-23 2014-11-04 Alnylam Pharmaceuticals, Inc. Glycoconjugates of RNA interference agents
AU2008340355B2 (en) 2007-12-04 2015-01-22 Tekmira Pharmaceuticals Corporation Targeting lipids
AU2008340354B2 (en) 2007-12-04 2014-04-17 Alnylam Pharmaceuticals, Inc. Folate-iRNA conjugates
JP2011511004A (ja) 2008-01-31 2011-04-07 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 ＰＣＳＫ９遺伝子を標的とするｄｓＲＮＡを送達するための最適化された方法
WO2009120878A2 (en) 2008-03-26 2009-10-01 Alnylam Pharmaceuticals, Inc. Non-natural ribonucleotides, and methods of use thereof
JP5788312B2 (ja) 2008-04-11 2015-09-30 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達
US8962580B2 (en) 2008-09-23 2015-02-24 Alnylam Pharmaceuticals, Inc. Chemical modifications of monomers and oligonucleotides with cycloaddition
MX2011004268A (es) 2008-10-20 2011-06-01 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de transtiretina.
US8987435B2 (en) 2008-10-24 2015-03-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
ES2993911T3 (en) 2008-11-10 2025-01-13 Arbutus Biopharma Corp Novel lipids and compositions for the delivery of therapeutics
CA2760776C (en) 2009-05-05 2019-07-09 Alnylam Pharmaceuticals, Inc. Lipid compositions for the delivery of therapeutic agents
DK3431076T3 (da) 2009-06-10 2021-12-20 Arbutus Biopharma Corp Forbedret lipidformulering
WO2011005980A1 (en) * 2009-07-09 2011-01-13 Intradigm Corporation Novel heterobifunctional polyethylene glycol reagents, their preparation and uses thereof
WO2011038356A2 (en) 2009-09-25 2011-03-31 Johns Hopkins University Novel liver-targeting agents and their synthesis
TWI391144B (zh) 2009-10-26 2013-04-01 Iner Aec Executive Yuan 一種定量肝殘餘功能的檢驗方法與其新穎肝受體造影檢驗藥劑
TWI388338B (zh) 2009-10-26 2013-03-11 Iner Aec Executive Yuan 對聚合醣鏈進行放射標誌以作為肝受體造影劑之方法
WO2011072290A2 (en) 2009-12-11 2011-06-16 The Regents Of The University Of Michigan Targeted dendrimer-drug conjugates
ES2562817T3 (es) 2010-02-24 2016-03-08 Arrowhead Research Corporation Composiciones para el suministro dirigido de ARNip
JP2013528665A (ja) 2010-03-26 2013-07-11 メルサナ セラピューティックス， インコーポレイテッド ポリヌクレオチドの送達のための修飾ポリマー、その製造方法、およびその使用方法
US20130236968A1 (en) 2010-06-21 2013-09-12 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
JP2013541334A (ja) 2010-09-15 2013-11-14 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 修飾されたｉＲＮＡ剤
WO2012068187A1 (en) * 2010-11-19 2012-05-24 Merck Sharp & Dohme Corp. Poly(amide) polymers for the delivery of oligonucleotides
US8501930B2 (en) * 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
CA2816155C (en) * 2010-12-17 2020-10-27 Arrowhead Research Corporation Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
KR20130108655A (ko) 2010-12-29 2013-10-04 에프. 호프만-라 로슈 아게 핵산의 세포내 전달을 위한 소분자 접합체
CR20190207A (es) 2011-04-21 2019-06-26 Ionis Pharmaceuticals Inc MODULACIÓN DE LA EXPRESIÓN DEL VIRUS DE LA HEPATITIS B (VHB) (Divisional 2013-0551)
SI2726613T1 (sl) * 2011-06-30 2018-10-30 Arrowhead Pharmaceuticals, Inc. Sestavki in postopki za zaviranje izražanja gena virusa hepatitisa B
KR20140051357A (ko) 2011-08-26 2014-04-30 애로우헤드 리서치 코오포레이션 Ｉｎ Ｖｉｖｏ 핵산 전달용 폴리(비닐 에스테르) 고분자
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
JP2015515530A (ja) * 2012-04-18 2015-05-28 アローヘッド リサーチ コーポレイション インビボ核酸送達のためのポリ（アクリラート）ポリマー
TWI595885B (zh) 2012-05-02 2017-08-21 喜納製藥公司 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法
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
EP3453762B1 (en) 2012-05-02 2021-04-21 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
US9150602B2 (en) * 2012-07-24 2015-10-06 Atomic Energy Council, Institute Of Nuclear Energy Research Precursor used for labeling hepatorcyte receptor and containing trisaccharide and diamide demercaptide ligand, method for preparing the same, radiotracer and pharmaceutical composition of the same
US10086081B2 (en) 2012-08-06 2018-10-02 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated RNA agents and process for their preparation
WO2014076196A1 (en) 2012-11-15 2014-05-22 Santaris Pharma A/S Anti apob antisense conjugate compounds
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
CA2893801A1 (en) 2013-01-30 2014-08-07 F. Hoffmann-La Roche Ag Lna oligonucleotide carbohydrate conjugates
US9309513B2 (en) 2013-05-01 2016-04-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-122
RU2670614C9 (ru) 2013-05-01 2018-11-23 Ионис Фармасьютикалз, Инк. Композиции и способы модулирования экспрессии hbv и ttr
EP3019200B1 (en) 2013-07-11 2022-03-23 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
EP3610884B1 (en) 2013-11-06 2021-08-18 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
JP6667453B2 (ja) 2014-05-01 2020-03-18 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 成長ホルモン受容体発現を調節するための組成物及び方法
US20170145424A1 (en) 2014-06-06 2017-05-25 Ionis 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
WO2016001123A1 (en) 2014-07-01 2016-01-07 Gima Tt S.P.A. Package for smoking articles
WO2016044267A1 (en) 2014-09-15 2016-03-24 Qubole, Inc. Systems and methods for providing metadata-aware background caching in data analysis
RU2017114156A (ru) 2014-10-10 2018-11-15 Ф. Хоффманн-Ля Рош Аг N-ацетилгалактозаминовые (galnac) фосфорамитиды, их конъюгаты с нуклеиновыми кислотами и их применение
EP3271482A4 (en) * 2015-03-17 2019-02-13 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR INHIBITING THE GENE EXPRESSION OF FACTOR XII
WO2016149020A1 (en) 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
CN108271387B (zh) * 2015-08-07 2023-06-27 箭头药业股份有限公司 乙型肝炎病毒感染的RNAi疗法
JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
US10246709B2 (en) * 2016-03-07 2019-04-02 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
CA3011668A1 (en) 2016-09-02 2018-03-08 Arrowhead Pharmaceuticals, Inc. Targeting ligands
CN110268060B (zh) * 2017-01-10 2024-07-26 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
EP3573623A4 (en) 2017-01-30 2020-11-25 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF INHIBITION OF GENE EXPRESSION OF FACTOR XII

Also Published As

Publication number Publication date
CN109069529A (zh) 2018-12-21
US10246709B2 (en) 2019-04-02
US20170253875A1 (en) 2017-09-07
TW201735953A (zh) 2017-10-16
AU2025213612A1 (en) 2025-08-28
BR112018068230B1 (pt) 2024-02-27
TW202203972A (zh) 2022-02-01
BR112018068230A2 (pt) 2019-07-30
KR102515329B1 (ko) 2023-03-29
JP7485642B2 (ja) 2024-05-16
JOP20210231A1 (ar) 2023-01-30
JP2022023242A (ja) 2022-02-07
WO2017156012A1 (en) 2017-09-14
IL288341B2 (en) 2023-02-01
US20250243485A1 (en) 2025-07-31
AU2017229469B2 (en) 2023-02-23
JP2019511491A (ja) 2019-04-25
IL261598A (en) 2018-10-31
CA3011946A1 (en) 2017-09-14
TWI727009B (zh) 2021-05-11
KR102348425B1 (ko) 2022-01-10
KR102830443B1 (ko) 2025-07-07
JP2024097862A (ja) 2024-07-19
TW202330035A (zh) 2023-08-01
EP3426261A4 (en) 2020-03-25
JOP20170057B1 (ar) 2022-03-14
CN113797348A (zh) 2021-12-17
IL296377A (en) 2022-11-01
NZ785880A (en) 2025-09-26
MX2018009854A (es) 2018-11-09
MX2023006334A (es) 2023-06-12
AU2023203102B2 (en) 2025-05-15
AU2017229469A1 (en) 2018-08-09
TWI783434B (zh) 2022-11-11
IL288341B (en) 2022-10-01
IL288341A (en) 2022-01-01
CN109069529B (zh) 2021-08-20
US12116573B2 (en) 2024-10-15
US20190211333A1 (en) 2019-07-11
IL261598B (en) 2021-12-01
JP2025120251A (ja) 2025-08-15
KR20230048446A (ko) 2023-04-11
US20210189394A1 (en) 2021-06-24
JP6983797B2 (ja) 2021-12-17
EP3426261A1 (en) 2019-01-16
TWI842117B (zh) 2024-05-11
NZ744357A (en) 2025-09-26
KR20180121925A (ko) 2018-11-09
MX2023006332A (es) 2023-06-12
AU2023203102A1 (en) 2023-06-08
KR20220005633A (ko) 2022-01-13

Similar Documents

Publication Publication Date Title
UY37146A (es) Ligandos de direccionamiento para compuestos terapéuticos
UY37145A (es) Ligandos de direccionamiento
AR121312A2 (es) AGENTES DE ARNi, COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR)
CO2018003678A2 (es) Composiciones y métodos para inhibir la expresión génica de lpa
CY1119060T1 (el) Ρυθμιστες ενζυμων μεθυλικης τροποποιησης, συνθεσεις και χρησεις αυτων
MX2024004433A (es) Composiciones dirigidas.
CL2020001797A1 (es) Compuestos, composiciones y métodos para tratar enfermedades que involucren tejidos con enfermedades ácidas o hipóxicas.
CO2018009275A2 (es) Derivados pirrolidina espiro-condensados como inhibidores de enzimas desubiquitinantes (dub)
UY40064A (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO
CU20170172A7 (es) 4,6-diaminoquinolin-3-carbonitrilos sustituidos útiles como agentes analgésicos, antipiréticos o antiinflamatorios y antineoplásicos
AR090906A1 (es) Conjugados que contienen tetragalnac y procedimientos para la administracion de oligonucleotidos
CR20190301A (es) Compuestos de amino-triazolopiridina y su uso en el tratamiento del cáncer
CL2015000829A1 (es) Inhibidores de histona desmetilasas
UY37272A (es) Piridinas sustituidas con heteroarilo y métodos de uso
MX2018004114A (es) Compuestos terapeuticos y metodos.
BR112019000693A2 (pt) composições de muc1- car e métodos para uso
MX2019005898A (es) Composiciones de arni de serpina 1 y sus métodos de uso.
CU20160006A7 (es) Derivados de piperidinil-indol como inhibidores de factor de complemento b
CU24097B1 (es) Compuestos piridiloxi-indoles sustituidos como inhibidores del vegf-r2 útiles para el tratamiento de enfermedades proliferativas
DOP2014000033A (es) Aminoquinazolinas como inhibidores de quinasa
EA201692318A1 (ru) Композиции и способы для ингибирования экспрессии гена альфа-1-антитрипсина
MX386008B (es) Compuestos citotóxicos y antimitóticos, y métodos para utilizarlos.
NI201500078A (es) Composiciones que comprenden vortioxetina y donepezilo.
CR20150148A (es) Azaindolinas
UY33650A (es) Compuestos heterocíclicos

Legal Events

Date Code Title Description
110 Patent granted

Effective date: 20240611