JP7279081B2 - Mir-122を調節するためのマイクロrna化合物及び方法 - Google Patents

Mir-122を調節するためのマイクロrna化合物及び方法 Download PDF

Info

Publication number
JP7279081B2
JP7279081B2 JP2020561923A JP2020561923A JP7279081B2 JP 7279081 B2 JP7279081 B2 JP 7279081B2 JP 2020561923 A JP2020561923 A JP 2020561923A JP 2020561923 A JP2020561923 A JP 2020561923A JP 7279081 B2 JP7279081 B2 JP 7279081B2
Authority
JP
Japan
Prior art keywords
composition
hcv
treatment period
days
certain embodiments
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2020561923A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021522802A (ja
JP2021522802A5 (https=
JPWO2019217369A5 (https=
Inventor
チャールズ アール. アラーソン
スティーブン エス. ネベン
ティモシー ライト
Original Assignee
レグルス セラピューティクス インコーポレイテッド
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 レグルス セラピューティクス インコーポレイテッド filed Critical レグルス セラピューティクス インコーポレイテッド
Publication of JP2021522802A publication Critical patent/JP2021522802A/ja
Publication of JP2021522802A5 publication Critical patent/JP2021522802A5/ja
Publication of JPWO2019217369A5 publication Critical patent/JPWO2019217369A5/ja
Application granted granted Critical
Publication of JP7279081B2 publication Critical patent/JP7279081B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/439Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring forming part of a bridged ring system, e.g. quinuclidine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A61K31/7072Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Virology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oncology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Communicable Diseases (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2020561923A 2018-05-08 2019-05-07 Mir-122を調節するためのマイクロrna化合物及び方法 Active JP7279081B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862668467P 2018-05-08 2018-05-08
US62/668,467 2018-05-08
PCT/US2019/031044 WO2019217369A1 (en) 2018-05-08 2019-05-07 Galnac conjugated modified oligonucleotide as mir-122 inhibitor having hcv antiviral activity with reduced hyperbilirubinemia side-effect

Publications (4)

Publication Number Publication Date
JP2021522802A JP2021522802A (ja) 2021-09-02
JP2021522802A5 JP2021522802A5 (https=) 2022-05-13
JPWO2019217369A5 JPWO2019217369A5 (https=) 2022-05-13
JP7279081B2 true JP7279081B2 (ja) 2023-05-22

Family

ID=66625315

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020561923A Active JP7279081B2 (ja) 2018-05-08 2019-05-07 Mir-122を調節するためのマイクロrna化合物及び方法

Country Status (7)

Country Link
US (1) US12005120B2 (https=)
EP (1) EP3790972A1 (https=)
JP (1) JP7279081B2 (https=)
CN (1) CN112400020B (https=)
CA (1) CA3099698A1 (https=)
TW (1) TWI854980B (https=)
WO (1) WO2019217369A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113718024A (zh) * 2020-05-26 2021-11-30 深圳市血液中心 血浆miRNA标志物及其应用
KR20240166519A (ko) * 2022-03-25 2024-11-26 한서 바이오 엘엘씨 치료제의 표적화된 전달을 위한 비사이클릭 헤테로사이클 및 이의 리간드

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2992095A2 (en) 2013-05-01 2016-03-09 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-122

Family Cites Families (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH10503364A (ja) 1994-05-10 1998-03-31 ザ ジェネラル ホスピタル コーポレーション C型肝炎ウイルスのアンチセンス阻害
AU1039397A (en) 1995-11-22 1997-06-27 Johns Hopkins University, The Ligands to enhance cellular uptake of biomolecules
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
IL148916A0 (en) 1999-10-04 2002-09-12 Exiqon As Design of high affinity rnase h recruiting oligonucleotide
US20020068709A1 (en) 1999-12-23 2002-06-06 Henrik Orum Therapeutic uses of LNA-modified oligonucleotides
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US6906182B2 (en) 2000-12-01 2005-06-14 Cell Works Therapeutics, Inc. Conjugates of glycosylated/galactosylated peptide, bifunctional linker, and nucleotidic monomers/polymers, and related compositions and method of use
CN1344803A (zh) 2001-09-27 2002-04-17 吴昌 核酸靶分子选择及扩增方法
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
CA2536139A1 (en) 2003-09-25 2005-04-07 Coley Pharmaceutical Group, Inc. Nucleic acid-lipophilic conjugates
RU2377301C2 (ru) 2003-12-23 2009-12-27 Сантарис Фарма А/С ОЛИГОМЕРНОЕ СОЕДИНЕНИЕ, ПОНИЖАЮЩЕЕ ЭКСПРЕССИЮ ЧЕЛОВЕЧЕСКОГО ГЕНА Bcl-2, КОНЪЮГАТ, ФАРМАЦЕВТИЧЕСКАЯ КОМПОЗИЦИЯ И ПРИМЕНЕНИЕ ОЛИГОМЕРНОГО СОЕДИНЕНИЯ ДЛЯ ЛЕЧЕНИЯ РАКА
AU2005325262B2 (en) 2004-04-27 2011-08-11 Alnylam Pharmaceuticals, Inc. Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
ES2361458T5 (es) 2004-05-04 2016-04-20 The Board Of Trustees Of The Leland Stanford Junior University Procedimientos y composiciones para reducir las cantidades de genoma vírico de VHC en una célula diana
CA2574603C (en) 2004-08-04 2014-11-04 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a ligand tethered to a modified or non-natural nucleobase
US20090203132A1 (en) 2004-09-09 2009-08-13 Swayze Eric E Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
WO2006069584A2 (en) 2004-12-29 2006-07-06 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF microRNAs AND THEIR TARGET mRNAs
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20120122801A1 (en) 2005-01-05 2012-05-17 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
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
WO2007021896A2 (en) 2005-08-10 2007-02-22 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides for use in modulating micro rna and uses thereof
CA2620856C (en) 2005-08-29 2017-11-28 Isis Pharmaceuticals, Inc. Methods for use in modulating mir-122a
WO2007027894A2 (en) 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Antisense compounds having enhanced anti-microrna activity
EP1984499B1 (en) 2006-01-27 2015-05-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
EP2666859B1 (en) 2006-04-03 2019-01-02 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
CA2648132C (en) 2006-04-03 2019-05-28 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
EP2152879B1 (en) 2007-05-01 2012-11-14 Santaris Pharma A/S Rna antagonist compounds for the modulation of beta-catenin
EP2623599B1 (en) 2007-10-04 2019-01-02 Roche Innovation Center Copenhagen A/S Micromirs
JP5872162B2 (ja) 2007-11-26 2016-03-01 エンゾン ファーマシューティカルズ,インコーポレーテッド アンドロゲン受容体を標的化するlnaアンタゴニスト
WO2009082607A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
US20110257244A1 (en) 2008-01-18 2011-10-20 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and small molecules for use in reducing micro rna activity levels and uses thereof
EP2268811A1 (en) 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
JP5793423B2 (ja) 2008-12-31 2015-10-14 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブRoche Innovation Center Copenhagen A/S 急性冠症候群の治療のためのLNAApoBアンチセンスオリゴマーの使用
ES2599979T3 (es) 2009-04-24 2017-02-06 Roche Innovation Center Copenhagen A/S Composiciones farmacéuticas para el tratamiento de pacientes de VHC que no responden al interferón
CA2765129A1 (en) 2009-06-08 2010-12-16 Miragen Therapeutics Chemical modification motifs for mirna inhibitors and mimetics
KR20120095397A (ko) 2009-10-16 2012-08-28 글락소 그룹 리미티드 Hbv 안티센스 억제제
DK2539451T3 (da) 2010-02-24 2016-04-04 Arrowhead Res Corp Sammensætninger til målrettet tilførsel af siRNA
US8349308B2 (en) 2010-03-26 2013-01-08 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
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
WO2012007477A1 (en) 2010-07-12 2012-01-19 Santaris Pharma A/S Anti hcv oligomers
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
EP2652134B1 (en) 2010-12-17 2017-03-01 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
ES2605990T3 (es) 2010-12-29 2017-03-17 F. Hoffmann-La Roche Ag Conjugados de molécula pequeña para la administración intracelular de ácidos nucleicos
EP3211082B1 (en) 2011-04-25 2021-02-17 Sanofi Microrna compounds and methods for modulating mir-21 activity
US20140248611A1 (en) 2011-06-16 2014-09-04 Nippon Steel & Sumikin Eco-Tech Corporation Nucleic acid probe for assaying nucleic acids
US20140127159A1 (en) * 2011-06-23 2014-05-08 Stella Aps HCV Combination Therapy
KR20140064765A (ko) 2011-06-30 2014-05-28 스텔라 에이피에스 Hcv 조합 치료
EP2726611A1 (en) 2011-06-30 2014-05-07 Stella ApS Hcv combination therapy
EP3453761A1 (en) * 2011-08-29 2019-03-13 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
WO2013068348A1 (en) 2011-11-07 2013-05-16 Santaris Pharma A/S Lna oligomers for improvement in hepatic function
EP2776590B1 (en) 2011-11-07 2016-10-26 Roche Innovation Center Copenhagen A/S Prognostic method for checking efficacy of micro rna-122 inhibitors in hcv+ patients
US9388408B2 (en) 2012-06-21 2016-07-12 MiRagen Therapeutics, Inc. Oligonucleotide-based inhibitors comprising locked nucleic acid motif
CN104955950B (zh) 2012-09-26 2019-04-26 米尔克斯治疗学公司 具有改善的脱靶特征谱的寡核苷酸
US10077443B2 (en) 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
CN104955952A (zh) 2013-01-30 2015-09-30 弗·哈夫曼-拉罗切有限公司 Lna寡核苷酸碳水化合物缀合物
CN105164261B (zh) 2013-05-01 2022-03-18 莱古路斯治疗法股份有限公司 用于增强的细胞摄取的化合物和方法
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2992095A2 (en) 2013-05-01 2016-03-09 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-122

Also Published As

Publication number Publication date
CN112400020A (zh) 2021-02-23
JP2021522802A (ja) 2021-09-02
CA3099698A1 (en) 2019-11-14
WO2019217369A1 (en) 2019-11-14
TWI854980B (zh) 2024-09-11
CN112400020B (zh) 2024-11-19
EP3790972A1 (en) 2021-03-17
TW202016302A (zh) 2020-05-01
US12005120B2 (en) 2024-06-11
US20210170034A1 (en) 2021-06-10

Similar Documents

Publication Publication Date Title
US20210108205A1 (en) MicroRNA Compounds and Methods for Modulating MIR-122
CN106470688B (zh) 寡聚物和寡聚物缀合物
CN114846140A (zh) 用于治疗hbv的靶向hbv的治疗性寡核苷酸和tlr7激动剂的药物组合
JP7279081B2 (ja) Mir-122を調節するためのマイクロrna化合物及び方法
EP4081217A1 (en) Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
US20220098581A1 (en) Methods for Oral Delivery of Oligonucleotides
NZ732101B2 (en) Microrna compounds and methods for modulating mir-122
HK40044543B (zh) 寡聚物和寡聚物缀合物
HK40044543A (en) Oligomers and oligomer conjugates
HK40078455A (en) Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220502

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220502

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230418

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230510

R150 Certificate of patent or registration of utility model

Ref document number: 7279081

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150