CA3099698A1 - Galnac conjugated modified oligonucleotide as mir-122 inhibitor having hcv antiviral activity with reduced hyperbilirubinemia side-effect - Google Patents

Galnac conjugated modified oligonucleotide as mir-122 inhibitor having hcv antiviral activity with reduced hyperbilirubinemia side-effect Download PDF

Info

Publication number
CA3099698A1
CA3099698A1 CA3099698A CA3099698A CA3099698A1 CA 3099698 A1 CA3099698 A1 CA 3099698A1 CA 3099698 A CA3099698 A CA 3099698A CA 3099698 A CA3099698 A CA 3099698A CA 3099698 A1 CA3099698 A1 CA 3099698A1
Authority
CA
Canada
Prior art keywords
hcv
compound
dose
treatment period
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.)
Pending
Application number
CA3099698A
Other languages
English (en)
French (fr)
Inventor
Charles R. ALLERSON
Steven S. NEBEN
Timothy Wright
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Regulus Therapeutics Inc
Original Assignee
Regulus Therapeutics 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 Regulus Therapeutics Inc filed Critical Regulus Therapeutics Inc
Publication of CA3099698A1 publication Critical patent/CA3099698A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (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)
  • Plural Heterocyclic Compounds (AREA)
CA3099698A 2018-05-08 2019-05-07 Galnac conjugated modified oligonucleotide as mir-122 inhibitor having hcv antiviral activity with reduced hyperbilirubinemia side-effect Pending CA3099698A1 (en)

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 (1)

Publication Number Publication Date
CA3099698A1 true CA3099698A1 (en) 2019-11-14

Family

ID=66625315

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3099698A Pending CA3099698A1 (en) 2018-05-08 2019-05-07 Galnac conjugated modified oligonucleotide as mir-122 inhibitor having hcv antiviral activity with reduced hyperbilirubinemia side-effect

Country Status (7)

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

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
CA3246334A1 (en) * 2022-03-25 2023-09-28 Shanghai Hansoh Biomedical Co., Ltd. BICYCLIC HETEROCYCLES AND THEIR LIGANDS FOR TARGETED ADMINISTRATION OF THERAPEUTIC AGENTS

Family Cites Families (61)

* 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
JP2000501414A (ja) 1995-11-22 2000-02-08 ザ・ジョンズ・ホプキンス・ユニバーシティー 生体分子の細胞取り込みを高めるリガンド
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
AU3041701A (en) 1999-12-23 2001-07-09 Exiqon A/S Therapeutic uses of lna-modified oligonucleotides
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
WO2002043771A2 (en) 2000-12-01 2002-06-06 Cell Works Inc. Conjugates of glycosylated/galactosylated peptide
CN1344803A (zh) 2001-09-27 2002-04-17 吴昌 核酸靶分子选择及扩增方法
JP4597976B2 (ja) 2003-04-17 2010-12-15 アルナイラム ファーマシューティカルズ インコーポレイテッド 修飾iRNA剤
EP2530157B1 (en) 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
AU2004275876B2 (en) 2003-09-25 2011-03-31 Coley Pharmaceutical Gmbh Nucleic acid-lipophilic conjugates
JP4642775B2 (ja) 2003-12-23 2011-03-02 サンタリス ファーマ アー/エス Bcl−2のモデュレーションのためのオリゴマー化合物
WO2006078278A2 (en) 2004-04-27 2006-07-27 Alnylam Pharmaceuticals, Inc. Single-stranded and double-stranded oligonucleotides comprising a 2-arylpropyl moiety
PT1747023E (pt) 2004-05-04 2011-04-11 Univ Leland Stanford Junior Métodos e composições para reduzir quantidades de genoma viral numa célula alvo
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
US20070099196A1 (en) 2004-12-29 2007-05-03 Sakari Kauppinen Novel oligonucleotide compositions and probe sequences useful for detection and analysis of micrornas and their target mRNAs
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
JP2008527993A (ja) 2005-01-24 2008-07-31 アヴァリス・アクチエボラーグ 特異性及び送達の改善のためのsiRNA分子、shRNA分子又はアンチセンス分子及び機能的実体を含む複合体
EP1919512B1 (en) 2005-08-10 2014-11-12 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides for use in modulating micro rna and uses thereof
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
DK1931780T3 (en) 2005-08-29 2016-01-25 Regulus Therapeutics Inc Antisense-forbindelser med forøget anti-microrna-aktivitet
WO2007027775A2 (en) 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Methods for use in modulating mir-122a
US8129515B2 (en) 2006-01-27 2012-03-06 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
EP2261333B1 (en) 2006-04-03 2016-03-30 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-miRNA antisense oligonucleotides
DK2666859T3 (en) 2006-04-03 2019-04-08 Roche Innovation Ct Copenhagen As PHARMACEUTICAL COMPOSITION CONTAINING ANTI-miRNA ANTISENSE OLIGONUCLEOTIDES
WO2008132234A2 (en) 2007-05-01 2008-11-06 Santaris Pharma A/S Rna antagonist compounds for the modulation of beta-catenin
ES2463665T3 (es) 2007-10-04 2014-05-28 Stella Aps Tratamiento de combinación para el tratamiento de infección por virus de la hepatitis C
KR20100110298A (ko) 2007-11-26 2010-10-12 산타리스 팔마 에이/에스 안드로겐 수용체를 표적화하는 lna 길항제
CN102006890A (zh) 2007-12-04 2011-04-06 阿尔尼拉姆医药品有限公司 靶向脂质
WO2009091972A2 (en) 2008-01-18 2009-07-23 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
WO2010076248A1 (en) 2008-12-31 2010-07-08 Santaris Pharma A/S Use of lna apob antisense oligomers for the treatment of acute coronary syndromes
JP5773535B2 (ja) 2009-04-24 2015-09-02 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 インターフェロンに非応答性のhcv患者の治療のための医薬組成物
AU2010258875A1 (en) 2009-06-08 2012-01-19 Miragen Therapeutics Chemical modification motifs for miRNA inhibitors and mimetics
EP2512491B1 (en) 2009-10-16 2017-10-11 Glaxo Group Limited Hbv antisense inhibitors
EP3023495B1 (en) 2010-02-24 2019-05-08 Arrowhead Pharmaceuticals, Inc. Compositions for targeted delivery of sirna
EP2553019A1 (en) 2010-03-26 2013-02-06 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
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
ES2605990T3 (es) 2010-12-29 2017-03-17 F. Hoffmann-La Roche Ag Conjugados de molécula pequeña para la administración intracelular de ácidos nucleicos
WO2012148952A1 (en) 2011-04-25 2012-11-01 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-21 activity
EP2722388B1 (en) 2011-06-16 2017-08-09 Nippon Steel & Sumikin Eco-Tech Corporation Nucleic acid probe for assaying nucleic acids
KR20140051271A (ko) 2011-06-23 2014-04-30 스텔라 에이피에스 Hcv 조합 치료
US20140213632A1 (en) 2011-06-30 2014-07-31 Stella Aps HCV Combination Therapy
JP2014520772A (ja) 2011-06-30 2014-08-25 ステラ・アンパルトセルスカブ Hcv併用療法
EP3640332A1 (en) * 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
AU2012334214A1 (en) 2011-11-07 2014-05-22 Roche Innovation Center Copenhagen A/S Prognostic method for checking efficacy of micro RNA-122 inhibitors in HCV+ patients
WO2013068348A1 (en) 2011-11-07 2013-05-16 Santaris Pharma A/S Lna oligomers for improvement in hepatic function
CN104685056A (zh) 2012-06-21 2015-06-03 米拉根医疗股份有限公司 包含锁核酸基序的基于寡核苷酸的抑制剂
EP2900822B1 (en) 2012-09-26 2021-11-24 Guangdong Maijinjia Biotechnology Co., Ltd Oligomers with improved off-target profile
SG10201804331TA (en) 2012-11-15 2018-07-30 Roche Innovation Ct Copenhagen As Oligonucleotide conjugates
US20150368642A1 (en) 2013-01-30 2015-12-24 Hoffmann-La Roche Inc. Lna oligonucleotide carbohydrate conjugates
WO2014118272A1 (en) * 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
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
EP2992095B1 (en) 2013-05-01 2019-01-09 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-122
CA2921162A1 (en) 2013-05-01 2014-11-06 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use

Also Published As

Publication number Publication date
EP3790972A1 (en) 2021-03-17
WO2019217369A1 (en) 2019-11-14
CN112400020A (zh) 2021-02-23
JP7279081B2 (ja) 2023-05-22
US20210170034A1 (en) 2021-06-10
TW202016302A (zh) 2020-05-01
US12005120B2 (en) 2024-06-11
CN112400020B (zh) 2024-11-19
TWI854980B (zh) 2024-09-11
JP2021522802A (ja) 2021-09-02

Similar Documents

Publication Publication Date Title
US20210108205A1 (en) MicroRNA Compounds and Methods for Modulating MIR-122
JP5773535B2 (ja) インターフェロンに非応答性のhcv患者の治療のための医薬組成物
JP2019518037A (ja) B型肝炎感染症治療用のPAPD5又はPAPD7のmRNA減少用核酸分子
CN112274647A (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
WO2013068348A1 (en) Lna oligomers for improvement in hepatic function
US12005120B2 (en) Galnac conjugated modified oligonucleotides as miR-122 inhibitor having HCV antiviral activity with reduced hyperbilirubinemia side-effect
EP4077671A1 (en) Use of saraf inhibitors for treating hepatitis b virus infection
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
WO2021122921A1 (en) Use of cops3 inhibitors for treating hepatitis b virus infection
NZ732101B2 (en) Microrna compounds and methods for modulating mir-122
HK40078455A (en) Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
HK40044543A (en) Oligomers and oligomer conjugates

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20240507

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250409

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250409

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20251104

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD

Year of fee payment: 7

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260409

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260409