JP2022535717A - アンジオポエチン様7(angptl7)関連疾患の処置 - Google Patents

アンジオポエチン様7(angptl7)関連疾患の処置 Download PDF

Info

Publication number
JP2022535717A
JP2022535717A JP2021569951A JP2021569951A JP2022535717A JP 2022535717 A JP2022535717 A JP 2022535717A JP 2021569951 A JP2021569951 A JP 2021569951A JP 2021569951 A JP2021569951 A JP 2021569951A JP 2022535717 A JP2022535717 A JP 2022535717A
Authority
JP
Japan
Prior art keywords
sequence
sense strand
antisense strand
nucleoside
composition
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
JP2021569951A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022535717A5 (https=
JPWO2020242896A5 (https=
Inventor
ゴッテスマン,オムリ
ブルース,シャノン
バスケ,ポール
カジェス,ブライアン
ルイス,デイビッド
ロゼマ,デイビッド
Original Assignee
エンピリコ インク.
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 エンピリコ インク. filed Critical エンピリコ インク.
Publication of JP2022535717A publication Critical patent/JP2022535717A/ja
Publication of JP2022535717A5 publication Critical patent/JP2022535717A5/ja
Publication of JPWO2020242896A5 publication Critical patent/JPWO2020242896A5/ja
Priority to JP2025076474A priority Critical patent/JP2025128091A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1136Non-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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2021569951A 2019-05-24 2020-05-21 アンジオポエチン様7(angptl7)関連疾患の処置 Pending JP2022535717A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025076474A JP2025128091A (ja) 2019-05-24 2025-05-01 アンジオポエチン様7(angptl7)関連疾患の処置

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962852813P 2019-05-24 2019-05-24
US62/852,813 2019-05-24
US201962881906P 2019-08-01 2019-08-01
US62/881,906 2019-08-01
PCT/US2020/034063 WO2020242896A2 (en) 2019-05-24 2020-05-21 Treatment of angiopoietin like 7 (angptl7) related diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025076474A Division JP2025128091A (ja) 2019-05-24 2025-05-01 アンジオポエチン様7(angptl7)関連疾患の処置

Publications (3)

Publication Number Publication Date
JP2022535717A true JP2022535717A (ja) 2022-08-10
JP2022535717A5 JP2022535717A5 (https=) 2023-05-11
JPWO2020242896A5 JPWO2020242896A5 (https=) 2023-05-11

Family

ID=73553086

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021569951A Pending JP2022535717A (ja) 2019-05-24 2020-05-21 アンジオポエチン様7(angptl7)関連疾患の処置
JP2025076474A Pending JP2025128091A (ja) 2019-05-24 2025-05-01 アンジオポエチン様7(angptl7)関連疾患の処置

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025076474A Pending JP2025128091A (ja) 2019-05-24 2025-05-01 アンジオポエチン様7(angptl7)関連疾患の処置

Country Status (8)

Country Link
US (2) US10941404B2 (https=)
EP (1) EP3976059A4 (https=)
JP (2) JP2022535717A (https=)
KR (1) KR20220024153A (https=)
CN (2) CN120022289A (https=)
AU (1) AU2020285639A1 (https=)
CA (1) CA3140917A1 (https=)
WO (1) WO2020242896A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11845989B2 (en) 2019-01-23 2023-12-19 Regeneron Pharmaceuticals, Inc. Treatment of ophthalmic conditions with angiopoietin-like 7 (ANGPTL7) inhibitors
JP2022523301A (ja) 2019-01-23 2022-04-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アンジオポエチン様7(angptl7)阻害剤による眼疾患の治療
KR20220024153A (ko) 2019-05-24 2022-03-03 엠피리코 인크. 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
WO2022182768A1 (en) * 2021-02-26 2022-09-01 Regeneron Pharmaceuticals, Inc. Treatment of inflammation with glucocorticoids and angiopoietin-like 7 (angptl7) inhibitors
EP4352231A4 (en) * 2021-06-07 2026-02-11 Empirico Inc TREATMENT OF DISEASES ASSOCIATED WITH ANGPTL4
US20240409943A1 (en) * 2021-10-01 2024-12-12 Alnylam Pharmaceuticals, Inc. iRNA Compositions and Methods for Targeting ANGPTL7
CN118632622A (zh) 2021-12-08 2024-09-10 瑞泽恩制药公司 突变型肌纤蛋白疾病模型及其用途
US20250197859A1 (en) * 2022-03-28 2025-06-19 Empirico Inc. Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases
CN115624539A (zh) * 2022-12-16 2023-01-20 首都医科大学附属北京朝阳医院 一种脂质纳米颗粒及其制备方法和应用
WO2025042862A1 (en) * 2023-08-18 2025-02-27 The Johns Hopkins University Modulation of nuclear envelope protein expression for alleviation of nuclear pore complex injury cascades and tdp- 43 dysfunction in neurodegeneration
CN118604350A (zh) * 2024-05-17 2024-09-06 上海市第一人民医院 年龄相关性黄斑变性诊断的生物标志物、检测试剂盒及其应用

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050080246A1 (en) * 2002-11-05 2005-04-14 Charles Allerson Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
WO2011051276A1 (en) * 2009-10-26 2011-05-05 Externautics S.P.A. Colon and rectal tumor markers and methods of use thereof

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US543227A (en) 1895-07-23 Core-drilling machinery
IE922292A1 (en) 1991-07-15 1993-01-27 Jolla Pharma Modified phosphorous intermediates for providing functional¹groups on the 5' end of oligonucleotides
US20060148743A1 (en) * 2001-05-18 2006-07-06 Vasant Jadhav RNA interference mediated inhibition of histone deacetylase (HDAC) gene expression using short interfering nucleic acid (siNA)
US20090137507A1 (en) * 2002-02-20 2009-05-28 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF ANGIOPOIETIN GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP2305812A3 (en) * 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US20040198640A1 (en) * 2003-04-02 2004-10-07 Dharmacon, Inc. Stabilized polynucleotides for use in RNA interference
US7601501B2 (en) * 2006-08-11 2009-10-13 The Scripps Research Institute Controlling osteogenesis by inhibition of osteogenic suppressors
JP2010505897A (ja) 2006-10-11 2010-02-25 マックス−プランク−ゲゼルシャフト・ツア・フェルデルング・デア・ヴィッセンシャフテン・エー・ファオ インフルエンザターゲット
US20110301228A1 (en) 2009-02-11 2011-12-08 The University Of North Carolina At Chapel Hill Gene therapy vector for treatment of steroid glaucoma
EP3028707A1 (en) 2009-05-01 2016-06-08 Ophthotech Corporation Methods for treating or preventing ophthalmological diseases
EP3260540A1 (en) * 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
EP2780454A2 (en) 2011-11-18 2014-09-24 Alnylam Pharmaceuticals, Inc. Modified rnai agents
WO2013120497A1 (en) * 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
US20150174203A1 (en) * 2012-05-30 2015-06-25 Icahn School Of Medicine At Mount Sinai Compositions And Methods For Modulating Pro-Inflammatory Immune Response
US9206423B2 (en) * 2012-12-30 2015-12-08 The Regents Of The University Of California Methods of modulating compliance of the trabecular meshwork
WO2014167529A1 (en) * 2013-04-10 2014-10-16 Institut De Cardiologie De Montreal Methods and compositions for preventing and treating atherosclerosis
CN103509101B (zh) * 2013-08-12 2016-11-02 中国科学院广州生物医药与健康研究院 一种扩增脐带血造血干细胞的细胞因子及其培养基
WO2015106128A2 (en) 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
JP6708630B2 (ja) * 2015-04-17 2020-06-10 国立大学法人 東京大学 角膜障害の予防又は治療剤
WO2016205488A1 (en) * 2015-06-17 2016-12-22 The California Institute For Biomedical Research Modified therapeutic agents and compositions thereof
US11696954B2 (en) 2017-04-28 2023-07-11 Exicure Operating Company Synthesis of spherical nucleic acids using lipophilic moieties
JP7420727B2 (ja) 2018-02-17 2024-01-23 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 トリアルキン結合剤及び使用方法
US11452738B2 (en) 2019-01-04 2022-09-27 Empirico Inc. Treatment of thymic stromal lymphopoietin (TSLP) related diseases by inhibition of long-form TSLP transcripts
JP2022523301A (ja) 2019-01-23 2022-04-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アンジオポエチン様7(angptl7)阻害剤による眼疾患の治療
US20220089664A1 (en) 2019-01-23 2022-03-24 Regeneron Pharmaceuticals, Inc. Treatment Of Ophthalmic Conditions With Angiopoietin-Like 7 (ANGPTL7) Inhibitors
KR20220024153A (ko) 2019-05-24 2022-03-03 엠피리코 인크. 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료
IL300283A (en) 2020-08-04 2023-04-01 Dicerna Pharmaceuticals Inc Systemic administration of oligonucleotides
WO2022072356A1 (en) 2020-09-29 2022-04-07 Empirico Inc. Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases
US20250197859A1 (en) 2022-03-28 2025-06-19 Empirico Inc. Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050080246A1 (en) * 2002-11-05 2005-04-14 Charles Allerson Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
WO2011051276A1 (en) * 2009-10-26 2011-05-05 Externautics S.P.A. Colon and rectal tumor markers and methods of use thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
IOVS, vol. 52, no. 14, JPN6024016831, 2011, pages 4622, ISSN: 0005605632 *

Also Published As

Publication number Publication date
EP3976059A4 (en) 2024-09-11
US12378556B2 (en) 2025-08-05
AU2020285639A1 (en) 2021-12-23
WO2020242896A3 (en) 2021-02-04
KR20220024153A (ko) 2022-03-03
EP3976059A2 (en) 2022-04-06
US10941404B2 (en) 2021-03-09
CN114375194A (zh) 2022-04-19
US20210163950A1 (en) 2021-06-03
CA3140917A1 (en) 2020-12-03
CN114375194B (zh) 2024-12-13
CN120022289A (zh) 2025-05-23
WO2020242896A2 (en) 2020-12-03
US20200399640A1 (en) 2020-12-24
JP2025128091A (ja) 2025-09-02

Similar Documents

Publication Publication Date Title
US12378556B2 (en) Treatment of angiopoietin like 7 (ANGPTL7) related diseases
US20260091053A1 (en) Treatment of thymic stromal lymphopoietin (tslp) related diseases by inhibition of long-form tslp transcripts
KR102095699B1 (ko) 트랜스티레틴(TTR) 관련 질병을 치료하기 위한 RNAi 제제, 조성 및 그의 사용방법
CN108064154B (zh) 用于抑制hao1(羟酸氧化酶1(乙醇酸盐氧化酶))基因表达的组合物及方法
JP6025567B2 (ja) 膜結合転写因子ペプチダーゼ、部位1(mbtps1)に対する天然アンチセンス転写物の阻害によるmbtps1関連性疾患の治療
US20230357774A1 (en) Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases
JP5886757B2 (ja) インターフェロン調節因子8(irf8)に対する天然アンチセンス転写物の阻害によるインターフェロン調節因子8(irf8)関連疾患の治療
JP5981850B2 (ja) RNaseH1に対する天然アンチセンス転写物の阻害によるRNaseH1関連疾患の治療
JP2019206564A (ja) 脳由来神経栄養因子(bdnf)関連疾病の、bdnfに対する天然アンチセンス転写物の抑制による治療
US20250228883A1 (en) Process to Inhibit or Eliminate Eosinophilic Diseases of the Airway and Related Conditions
CA3005249A1 (en) Compositions and methods for treatment of kidney diseases
JP2022515744A (ja) Kcnt1関連障害の治療のための組成物及び方法
CN104302768A (zh) 治疗β-联蛋白相关疾病的有机组合物
KR20230076823A (ko) 시신경 위축의 치료
US20250197859A1 (en) Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases
HK40068617B (zh) 血管生成素样7(angptl7)相关疾病的治疗
HK40068617A (en) Treatment of angiopoietin like 7 (angptl7) related diseases
CN115551519A (zh) 用于治疗神经系统疾病的补体组分c1s抑制剂以及相关的组合物、系统和使用它们的方法
WO2025137143A2 (en) Compositions for modulating cd33 expression
CN121358862A (zh) 用于治疗肾脏疾病的组合物和方法
HK40084771B (zh) 用於抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
CN115605592A (zh) 用于治疗神经系统疾病的补体组分c1r抑制剂以及相关的组合物、系统和使用它们的方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220124

RD12 Notification of acceptance of power of sub attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7432

Effective date: 20230111

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20230111

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230427

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230427

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240507

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240805

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240830

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241107

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250127

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20250528