CN107922947A - 用于治疗肌萎缩性侧索硬化症(als)的mir‑155抑制剂 - Google Patents

用于治疗肌萎缩性侧索硬化症(als)的mir‑155抑制剂 Download PDF

Info

Publication number
CN107922947A
CN107922947A CN201680038698.3A CN201680038698A CN107922947A CN 107922947 A CN107922947 A CN 107922947A CN 201680038698 A CN201680038698 A CN 201680038698A CN 107922947 A CN107922947 A CN 107922947A
Authority
CN
China
Prior art keywords
mir
nucleotide
nucleic acid
inhibitor
locked nucleic
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
CN201680038698.3A
Other languages
English (en)
Chinese (zh)
Inventor
A.杰克逊
C.达尔比
H.L.韦纳
O.布托夫斯基
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.)
Meter La Gen Medical Treatment Limited-Liability Co
Massachusetts Eye and Ear
Original Assignee
Meter La Gen Medical Treatment Limited-Liability Co
Massachusetts Eye and Ear Infirmary
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 Meter La Gen Medical Treatment Limited-Liability Co, Massachusetts Eye and Ear Infirmary filed Critical Meter La Gen Medical Treatment Limited-Liability Co
Publication of CN107922947A publication Critical patent/CN107922947A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN201680038698.3A 2015-06-05 2016-06-03 用于治疗肌萎缩性侧索硬化症(als)的mir‑155抑制剂 Pending CN107922947A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562171743P 2015-06-05 2015-06-05
US62/171,743 2015-06-05
PCT/US2016/035794 WO2016196978A1 (en) 2015-06-05 2016-06-03 Mir-155 inhibitors for treating amyotrophic lateral sclerosis (als)

Publications (1)

Publication Number Publication Date
CN107922947A true CN107922947A (zh) 2018-04-17

Family

ID=57442041

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201680038698.3A Pending CN107922947A (zh) 2015-06-05 2016-06-03 用于治疗肌萎缩性侧索硬化症(als)的mir‑155抑制剂

Country Status (6)

Country Link
US (1) US20180161357A1 (enExample)
EP (1) EP3303589A4 (enExample)
JP (1) JP2018517704A (enExample)
CN (1) CN107922947A (enExample)
CA (1) CA2986913A1 (enExample)
WO (1) WO2016196978A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112587662A (zh) * 2020-12-22 2021-04-02 上海市徐汇区中心医院 一种miR-155/PEA15信号通路抑制剂在病理性心脏重构药物中的应用
CN114272378A (zh) * 2020-09-27 2022-04-05 四川大学华西医院 一种使cttnbp2nl功能缺失的试剂在制备治疗疾病的药物中的用途

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2014004516A (es) 2011-10-11 2015-01-16 Brigham & Womens Hospital Micro-arns en trastornos neurodegenerativos.
RU2718534C2 (ru) 2015-06-05 2020-04-08 Мираджен Терапьютикс, Инк. Ингибиторы mir-155 для лечения кожной t-клеточной лимфомы (ctcl)
CA3040687A1 (en) 2016-10-14 2018-04-19 Childrens' Medical Center Corporation Compositions and methods for treating diseases and disorders of the central nervous system
CN110913872B (zh) 2017-01-17 2023-08-04 儿童医疗中心有限公司 治疗溶酶体贮积疾病及病症的组合物和方法
US12000843B2 (en) 2017-01-17 2024-06-04 Children's Medical Center Corporation Compositions and methods for diagnosing and treating peroxisomal diseases
US12144822B2 (en) 2018-04-03 2024-11-19 Academia Sinica Mir-17˜92 as therapeutic or diagnostic target of motor neuron (MN) degeneration diseases
EP3841220A1 (en) * 2018-08-23 2021-06-30 Roche Innovation Center Copenhagen A/S Microrna-134 biomarker
WO2021067613A1 (en) * 2019-10-01 2021-04-08 Children's Medical Center Corporation Compositions and methods for treating amyotrophic lateral sclerosis

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101437942A (zh) * 2006-04-03 2009-05-20 桑塔里斯制药公司 包含抗微小rna反义寡核苷酸的药物组合物
CN101821391A (zh) * 2007-10-04 2010-09-01 桑塔里斯制药公司 微小聚体
US20110077288A1 (en) * 2008-03-07 2011-03-31 Santaris Pharma A/S Pharmaceutical Compositions for Treatment of MicroRNA Related Diseases
US20120283114A1 (en) * 2009-07-29 2012-11-08 Pharnext New diagnostic tools for alzheimer disease
WO2013134403A1 (en) * 2012-03-06 2013-09-12 The Washington University Method of treating neurodegenerative diseases with microrna regulators
CN104011210A (zh) * 2011-10-11 2014-08-27 布里格姆及妇女医院股份有限公司 神经退行性病症中的microRNA

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101407707B1 (ko) * 2006-04-03 2014-06-19 산타리스 팔마 에이/에스 Ａｎｔｉ-ｍｉｒｎａ 안티센스 올리고뉴클레오타이드를 함유하는 약학적 조성물

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101437942A (zh) * 2006-04-03 2009-05-20 桑塔里斯制药公司 包含抗微小rna反义寡核苷酸的药物组合物
CN101821391A (zh) * 2007-10-04 2010-09-01 桑塔里斯制药公司 微小聚体
US20110077288A1 (en) * 2008-03-07 2011-03-31 Santaris Pharma A/S Pharmaceutical Compositions for Treatment of MicroRNA Related Diseases
US20120283114A1 (en) * 2009-07-29 2012-11-08 Pharnext New diagnostic tools for alzheimer disease
CN104011210A (zh) * 2011-10-11 2014-08-27 布里格姆及妇女医院股份有限公司 神经退行性病症中的microRNA
WO2013134403A1 (en) * 2012-03-06 2013-09-12 The Washington University Method of treating neurodegenerative diseases with microrna regulators

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
KOVAL等: "Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice", 《HUM MOL GENET》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114272378A (zh) * 2020-09-27 2022-04-05 四川大学华西医院 一种使cttnbp2nl功能缺失的试剂在制备治疗疾病的药物中的用途
CN112587662A (zh) * 2020-12-22 2021-04-02 上海市徐汇区中心医院 一种miR-155/PEA15信号通路抑制剂在病理性心脏重构药物中的应用

Also Published As

Publication number Publication date
CA2986913A1 (en) 2016-12-08
EP3303589A1 (en) 2018-04-11
JP2018517704A (ja) 2018-07-05
WO2016196978A1 (en) 2016-12-08
US20180161357A1 (en) 2018-06-14
EP3303589A4 (en) 2019-01-02

Similar Documents

Publication Publication Date Title
CN107922947A (zh) 用于治疗肌萎缩性侧索硬化症(als)的mir‑155抑制剂
RU2566724C9 (ru) Композиции и способы модуляции smn2 сплайсинга у субъекта
JP7049262B2 (ja) 結合組織成長因子を標的とするｒｎａ複合体を用いた特発性肺胞線維症の治療
KR102825946B1 (ko) IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
JP2015518710A (ja) ヘモグロビン遺伝子ファミリー発現を調節するための組成物及び方法
KR102034619B1 (ko) 피부 t 세포 림프종(ctcl) 치료용 mir-155 억제제
JP2020525011A (ja) ２テイル自己デリバリー型ｓｉＲＮＡおよび関連方法
WO2023051822A1 (zh) 用于治疗与pcsk9相关疾病的靶向寡核苷酸
JP7318166B2 (ja) マイクロｒｎａ２２の阻害剤
EP3797780A1 (en) Compositions and methods for modulation of smn2 splicing in a subject
JP6049143B2 (ja) オリゴヌクレオチド、グルココルチコイド感受性増強剤、医薬組成物、及び発現ベクター
CN120957731A (zh) 用于治疗多囊肾病的修饰的寡核苷酸
WO2011133142A1 (en) Treatment of vhl-negative tumors
JP7788561B2 (ja) アンチセンスオリゴヌクレオチド及びその使用
WO2024187102A2 (en) Compositions and methods for modulating c3
US20240229031A9 (en) Microrna compositions and methods of use thereof for the treatment of nervous system dysfunction
AU2023385485A1 (en) Inhibitory nucleic acids and methods of use thereof
HK40090166A (en) Compositions and methods for modulation of smn2 splicing in a subject
JP2025033947A (ja) 筋炎治療薬のスクリーニング方法
CN115141848A (zh) 一种用于治疗亨廷顿病的rna递送系统
HK40049373A (en) Compositions and methods for modulation of smn2 splicing in a subject

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20180417

WD01 Invention patent application deemed withdrawn after publication