JP2024533459A - リンパ球抗原７５（ｌｙ７５）、表面抗原分類１６４（ｃｄ１６４）、またはポリ（ａｄｐ－リボース）ポリメラーゼ１（ｐａｒｐ１）阻害剤を用いた未確定の潜在能をもつクローン性造血（ｃｈｉｐ）の治療方法 - Google Patents

リンパ球抗原７５（ｌｙ７５）、表面抗原分類１６４（ｃｄ１６４）、またはポリ（ａｄｐ－リボース）ポリメラーゼ１（ｐａｒｐ１）阻害剤を用いた未確定の潜在能をもつクローン性造血（ｃｈｉｐ）の治療方法 Download PDF

Info

Publication number
JP2024533459A
JP2024533459A JP2024515871A JP2024515871A JP2024533459A JP 2024533459 A JP2024533459 A JP 2024533459A JP 2024515871 A JP2024515871 A JP 2024515871A JP 2024515871 A JP2024515871 A JP 2024515871A JP 2024533459 A JP2024533459 A JP 2024533459A
Authority
JP
Japan
Prior art keywords
nucleic acid
acid molecule
subject
variant
parp1
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
JP2024515871A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024533459A5 (enExample
Inventor
ジョルゲンソン、エリック
ケスラー、マイケル
ダマスク、エイミー
オキーフ、ショーン
ホワイト、ライアン
グラス、デイビッド
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.)
Regeneron Pharmaceuticals Inc
Original Assignee
Regeneron 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 Regeneron Pharmaceuticals Inc filed Critical Regeneron Pharmaceuticals Inc
Publication of JP2024533459A publication Critical patent/JP2024533459A/ja
Publication of JP2024533459A5 publication Critical patent/JP2024533459A5/ja
Pending legal-status Critical Current

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
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C12Q1/6886—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/118—Prognosis of disease development
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/70—Mechanisms involved in disease identification
    • G01N2800/7023—(Hyper)proliferation

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Hospice & Palliative Care (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2024515871A 2021-09-13 2022-09-12 リンパ球抗原７５（ｌｙ７５）、表面抗原分類１６４（ｃｄ１６４）、またはポリ（ａｄｐ－リボース）ポリメラーゼ１（ｐａｒｐ１）阻害剤を用いた未確定の潜在能をもつクローン性造血（ｃｈｉｐ）の治療方法 Pending JP2024533459A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163243564P 2021-09-13 2021-09-13
US63/243,564 2021-09-13
US202163271171P 2021-10-24 2021-10-24
US63/271,171 2021-10-24
PCT/US2022/076298 WO2023039580A2 (en) 2021-09-13 2022-09-12 Methods of treating clonal hematopoiesis of indeterminate potential (chip) with lymphocyte antigen 75 (ly75), cluster of differentiation 164 (cd164), or poly(adp-ribose) polymerase 1 (parp1) inhibitors

Publications (2)

Publication Number Publication Date
JP2024533459A true JP2024533459A (ja) 2024-09-12
JP2024533459A5 JP2024533459A5 (enExample) 2025-09-17

Family

ID=83598663

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024515871A Pending JP2024533459A (ja) 2021-09-13 2022-09-12 リンパ球抗原７５（ｌｙ７５）、表面抗原分類１６４（ｃｄ１６４）、またはポリ（ａｄｐ－リボース）ポリメラーゼ１（ｐａｒｐ１）阻害剤を用いた未確定の潜在能をもつクローン性造血（ｃｈｉｐ）の治療方法

Country Status (6)

Country Link
US (1) US20230113866A1 (enExample)
EP (1) EP4401831A2 (enExample)
JP (1) JP2024533459A (enExample)
CA (1) CA3229899A1 (enExample)
MX (1) MX2024003132A (enExample)
WO (1) WO2023039580A2 (enExample)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060281672A1 (en) * 2002-12-06 2006-12-14 Hart Derek N J Dec-205 (ly 75)/dcl-1 intergenic splice variants associated with hodgkin's disease, and uses thereof
WO2014011465A2 (en) * 2012-07-13 2014-01-16 Albert Einstein College Of Medicine Of Yeshiva University Aptamer-targeted antigen delivery
GB201220010D0 (en) * 2012-11-07 2012-12-19 Oxford Biotherapeutics Ltd Therapeutic amd diagnostic target
WO2017156416A1 (en) * 2016-03-11 2017-09-14 The Brigham And Women's Hospital, Inc. Compositions and methods for treating chemotherapy resistant cancer
GB201703876D0 (en) * 2017-03-10 2017-04-26 Berlin-Chemie Ag Pharmaceutical combinations
EP4660324A3 (en) * 2020-02-24 2026-02-18 The Board of Trustees of the Leland Stanford Junior University Methods of analyzing cell free nucleic acids and applications thereof

Also Published As

Publication number Publication date
WO2023039580A2 (en) 2023-03-16
US20230113866A1 (en) 2023-04-13
CA3229899A1 (en) 2023-03-16
EP4401831A2 (en) 2024-07-24
MX2024003132A (es) 2024-04-10
WO2023039580A3 (en) 2023-06-08

Similar Documents

Publication Publication Date Title
JP2024501627A (ja) インヒビンサブユニットβＥ（ＩＮＨＢＥ）阻害剤による代謝障害及び心血管疾患の治療方法
CN116249787A (zh) 用g蛋白偶联受体75(gpr75)抑制剂治疗肥胖症
CN116194484A (zh) 内质网氨肽酶2(erap2)介导的免疫应答的调节
US20230107173A1 (en) Methods Of Treating Chronic Kidney Disease (CKD) With Inhibitors Of Protective Loss-Of-Function Genes
JP2023539742A (ja) Ｐｃｓｋ９調節物質及びｌｄｌｒ調節物質による敗血症の治療
CA3167756A1 (en) Treatment of ophthalmic conditions with son of sevenless 2 (sos2) inhibitors
JP2023524824A (ja) Ｋｅｌｃｈドメイン含有７Ｂ（ＫＬＨＤＣ７Ｂ）バリアント及びその使用
JP2024533459A (ja) リンパ球抗原７５（ｌｙ７５）、表面抗原分類１６４（ｃｄ１６４）、またはポリ（ａｄｐ－リボース）ポリメラーゼ１（ｐａｒｐ１）阻害剤を用いた未確定の潜在能をもつクローン性造血（ｃｈｉｐ）の治療方法
JP2022525545A (ja) ステロール調節エレメント結合転写因子１（ｓｒｅｂｆ１）阻害剤による上昇した脂質レベルの治療
JP2024522148A (ja) インターフェロン誘導ヘリカーゼｃドメイン１（ｉｆｉｈ１）阻害剤による乾癬の治療
JP2024524387A (ja) マイトジェン活性化プロテインキナーゼキナーゼキナーゼ１５（ｍａｐ３ｋ１５）阻害剤による代謝障害の治療方法
JP2024521224A (ja) 神経原性座位Ｎｏｔｃｈホモログタンパク質３（ＮＯＴＣＨ３）剤による脳血管疾患の治療
JP2024517190A (ja) ホスホジエステラーゼ３ｂ（ｐｄｅ３ｂ）阻害剤による肝疾患を治療する方法
CN118488844A (zh) 用淋巴细胞抗原75(ly75)、分化簇164(cd164)或聚(adp-核糖)聚合酶1(parp1)抑制剂治疗不确定潜能的克隆性造血(chip)的方法
CN113423435A (zh) 使用花生四烯酸15-脂氧合酶(alox15)抑制剂治疗呼吸系统病症
US20250136996A1 (en) Treatment of smoking with cholinergic receptor nicotinic beta 3 subunit (chrnb3) inhibitors
US20230383351A1 (en) Rare Variants In Hematopoietic Stem Cells (HSC) And Hematopoietic Progenitor Cells (HPC) Associated With Somatic Alterations Of The Blood
JP2024526290A (ja) 溶質輸送体ファミリー２７メンバー３（ｓｌｃ２７ａ３）阻害剤による喘息の治療方法
CN118541479A (zh) 用卵黄样羧肽酶(cpvl)抑制剂治疗皮肤癌的方法
JP2024524526A (ja) α－Ｎ－アセチルガラクトサミニドα－２，６－シアリルトランスフェラーゼ５（ＳＴ６ＧＡＬＮＡＣ５）阻害剤による認知障害の治療
KR20240099232A (ko) Rho 구아닌 뉴클레오타이드 교환 인자 12[Rho Guanine Nucleotide Exchange Factor 12, ARHGEF12] 억제제를 이용한 녹내장 치료
AU2022303368A1 (en) Treatment of hypertension with solute carrier family 9 isoform a3 regulatory factor 2 (slc9a3r2) inhibitors
WO2025189037A2 (en) Treatment of aneurysms with apolipoprotein c3 (apoc3) inhibitors and gene editing
WO2026050616A1 (en) Rtel1 inhibitor for use in the treatment of cancer
CN118613584A (zh) 使用内质网氨基肽酶1(erap1)抑制剂治疗葡萄膜炎

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250908

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250908