JP2013534424A - 二本鎖rnaによるアンドロゲン受容体の特異的阻害に対する方法と化合物 - Google Patents

二本鎖rnaによるアンドロゲン受容体の特異的阻害に対する方法と化合物 Download PDF

Info

Publication number
JP2013534424A
JP2013534424A JP2013518760A JP2013518760A JP2013534424A JP 2013534424 A JP2013534424 A JP 2013534424A JP 2013518760 A JP2013518760 A JP 2013518760A JP 2013518760 A JP2013518760 A JP 2013518760A JP 2013534424 A JP2013534424 A JP 2013534424A
Authority
JP
Japan
Prior art keywords
strand
dsrna
nucleotides
isolated
target
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
JP2013518760A
Other languages
English (en)
Japanese (ja)
Other versions
JP2013534424A5 (enExample
Inventor
ボブ, デール ブラウン,
ヘンリク, ティー ドゥーデック,
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.)
Dicerna Pharmaceuticals Inc
Original Assignee
Dicerna 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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of JP2013534424A publication Critical patent/JP2013534424A/ja
Publication of JP2013534424A5 publication Critical patent/JP2013534424A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/1138Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/08Drugs for disorders of the urinary system of the prostate
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2013518760A 2010-07-06 2011-07-01 二本鎖rnaによるアンドロゲン受容体の特異的阻害に対する方法と化合物 Pending JP2013534424A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US36175910P 2010-07-06 2010-07-06
US61/361,759 2010-07-06
PCT/US2011/042810 WO2012006241A2 (en) 2010-07-06 2011-07-01 Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna

Publications (2)

Publication Number Publication Date
JP2013534424A true JP2013534424A (ja) 2013-09-05
JP2013534424A5 JP2013534424A5 (enExample) 2014-08-14

Family

ID=45441760

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2013518760A Pending JP2013534424A (ja) 2010-07-06 2011-07-01 二本鎖rnaによるアンドロゲン受容体の特異的阻害に対する方法と化合物

Country Status (6)

Country Link
US (2) US8927515B2 (enExample)
EP (1) EP2591104A4 (enExample)
JP (1) JP2013534424A (enExample)
AU (1) AU2011276363A1 (enExample)
CA (1) CA2804210A1 (enExample)
WO (1) WO2012006241A2 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019526260A (ja) * 2016-09-01 2019-09-19 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 化学修飾された一本鎖rna編集オリゴヌクレオチド
JP2022513662A (ja) * 2018-11-28 2022-02-09 バイオニア コーポレーション アンドロゲン受容体特異的配列を含む二本鎖オリゴヌクレオチド構造体、及びこれを含む脱毛予防及び発毛用組成物

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100048914A1 (en) 2008-03-14 2010-02-25 Angela Brodie Novel C-17-Heteroaryl Steroidal Cyp17 Inhibitors/Antiandrogens, In Vitro Biological Activities, Pharmacokinetics and Antitumor Activity
WO2009128936A2 (en) 2008-04-16 2009-10-22 The Johns Hopkins University Compositions and methods for treating or preventing prostate cancer and for detecting androgen receptor variants
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
JP2016501533A (ja) * 2012-12-14 2016-01-21 ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. 二本鎖rnaによるckap5の特異的阻害に関する方法および組成物
KR20150127720A (ko) 2013-03-14 2015-11-17 유니버시티 오브 매릴랜드, 발티모어 안드로겐 수용체 하향 조절제 및 그의 용도
ES2905257T3 (es) 2013-07-03 2022-04-07 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario
WO2015023710A1 (en) 2013-08-12 2015-02-19 Tokai Pharmaceuticals, Inc. Biomarkers for treatment of neoplastic disorders using androgen-targeted therapies
US10774387B2 (en) 2014-05-19 2020-09-15 The Johns Hopkins University Methods for identifying androgen receptor splice variants in subjects having castration resistant prostate cancer
CA2959336A1 (en) * 2014-08-25 2016-03-03 The Johns Hopkins University Methods and compositions related to prostate cancer therapeutics
DK3234134T3 (da) 2014-12-17 2020-07-27 Proqr Therapeutics Ii Bv Målrettet rna-redigering
CA2989384A1 (en) * 2015-07-30 2017-02-02 Bayer Cropscience Aktiengesellschaft Methods and compositions for the control of rust fungi by inhibiting expression of the hxt1 gene
MA45340A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques du récepteur des androgènes et leurs utilisations
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
KR102321426B1 (ko) * 2017-02-21 2021-11-05 올릭스 주식회사 남성형 탈모 표적 유전자의 발현을 억제하는 비대칭 siRNA
EP3755814B1 (en) * 2018-02-23 2025-04-09 Cornell University Assay for detection of androgen receptor variants
AU2019243946B2 (en) 2018-03-27 2025-03-06 Proqr Therapeutics Ii B.V. Nucleic acid molecules for pseudouridylation
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
WO2025160224A1 (en) * 2024-01-24 2025-07-31 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of androgen receptor (ar), compositions thereof, and methods of use
WO2025226946A1 (en) * 2024-04-24 2025-10-30 Cedric Francois Methods and compositions for the treatment of androgenic alopecia

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050164970A1 (en) * 2003-12-22 2005-07-28 University Of Kansas Medical Center Method for treating prostate cancer using siRNA duplex for androgen receptor
JP2007215481A (ja) * 2006-02-16 2007-08-30 Ehime Univ アンドロゲン受容体遺伝子に特異的なsiRNA
US20080014584A1 (en) * 2006-06-01 2008-01-17 Mayo Foundation For Medical Education And Research Modulating androgen receptor activity

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050170371A1 (en) * 2001-05-18 2005-08-04 Sirna Therapeutics, Inc. RNA interference mediated inhibition of 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA)
DK2284266T3 (da) * 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
US20060257851A1 (en) * 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
JP5243789B2 (ja) * 2004-03-15 2013-07-24 シティ・オブ・ホープ 二本鎖rnaによる遺伝子発現の特異的阻害のための方法及び組成物
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050164970A1 (en) * 2003-12-22 2005-07-28 University Of Kansas Medical Center Method for treating prostate cancer using siRNA duplex for androgen receptor
JP2007215481A (ja) * 2006-02-16 2007-08-30 Ehime Univ アンドロゲン受容体遺伝子に特異的なsiRNA
US20080014584A1 (en) * 2006-06-01 2008-01-17 Mayo Foundation For Medical Education And Research Modulating androgen receptor activity

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JPN6015029963; Biochem. Biophys. Res. Commun., 2010, Vol. 391, pp. 1075-1079 *
JPN6015029964; PLOS one, 2007, Issue 10, e1006 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019526260A (ja) * 2016-09-01 2019-09-19 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 化学修飾された一本鎖rna編集オリゴヌクレオチド
JP7244922B2 (ja) 2016-09-01 2023-03-23 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ 化学修飾された一本鎖rna編集オリゴヌクレオチド
JP2022513662A (ja) * 2018-11-28 2022-02-09 バイオニア コーポレーション アンドロゲン受容体特異的配列を含む二本鎖オリゴヌクレオチド構造体、及びこれを含む脱毛予防及び発毛用組成物
JP2023113845A (ja) * 2018-11-28 2023-08-16 バイオニア コーポレーション アンドロゲン受容体特異的配列を含む二本鎖オリゴヌクレオチド構造体、及びこれを含む脱毛予防及び発毛用組成物
JP7625033B2 (ja) 2018-11-28 2025-01-31 バイオニア コーポレーション アンドロゲン受容体特異的配列を含む二本鎖オリゴヌクレオチド構造体、及びこれを含む脱毛予防及び発毛用組成物

Also Published As

Publication number Publication date
US9732347B2 (en) 2017-08-15
CA2804210A1 (en) 2012-01-12
US8927515B2 (en) 2015-01-06
WO2012006241A2 (en) 2012-01-12
AU2011276363A1 (en) 2013-01-10
EP2591104A2 (en) 2013-05-15
US20130131149A1 (en) 2013-05-23
EP2591104A4 (en) 2014-09-03
US20150197756A1 (en) 2015-07-16
WO2012006241A3 (en) 2012-04-19

Similar Documents

Publication Publication Date Title
US8927515B2 (en) Methods and compositions for the specific inhibition of androgen receptor by double-stranded RNA
JP5976643B2 (ja) 二本鎖rnaによるベータ−カテニンの特異的阻害に対する方法と組成物
JP6010458B2 (ja) 非対称二本鎖rnaによる特異的阻害のための、方法および組成物
JP2015529467A (ja) 二本鎖rnaによるmycの特異的阻害に関する方法および組成物
JP2012528882A (ja) ペプチドダイサー基質剤およびその特異的な遺伝子発現阻害のための方法
US9670488B2 (en) Methods and compositions for the specific inhibition of met by double stranded RNA
WO2014093746A2 (en) Methods and compositions for the specific inhibition of ckap5 by double-stranded rna
US10011834B2 (en) Methods and compositions for the specific inhibition of HIF-1α by double-stranded RNA
HK40059743A (en) Methods and compositions for the specific inhibition of beta-catenin by double-stranded rna
HK1227054B (en) Methods and compositions for the specific inhibition of beta-catenin by double-stranded rna

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20140625

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20140625

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20150727

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20160125