CN1604783A - 通过rna干扰治疗纤维化疾病的药物 - Google Patents

通过rna干扰治疗纤维化疾病的药物 Download PDF

Info

Publication number
CN1604783A
CN1604783A CNA028251636A CN02825163A CN1604783A CN 1604783 A CN1604783 A CN 1604783A CN A028251636 A CNA028251636 A CN A028251636A CN 02825163 A CN02825163 A CN 02825163A CN 1604783 A CN1604783 A CN 1604783A
Authority
CN
China
Prior art keywords
dsrna
chain
medicine
purposes
gene
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
CNA028251636A
Other languages
English (en)
Chinese (zh)
Inventor
R·克罗伊策尔
S·林默
D·舒潘
M·约翰
M·鲍尔
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.)
Alnylam Europe AG
Original Assignee
Ribopharma AG
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
Priority claimed from DE10160151A external-priority patent/DE10160151A1/de
Priority claimed from PCT/EP2002/000151 external-priority patent/WO2002055692A2/de
Application filed by Ribopharma AG filed Critical Ribopharma AG
Publication of CN1604783A publication Critical patent/CN1604783A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • 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
    • 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
    • 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
    • C12N15/1131Non-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 viruses
    • 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
    • C12N2310/14Type 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Endocrinology (AREA)
  • Pulmonology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CNA028251636A 2001-10-26 2002-10-25 通过rna干扰治疗纤维化疾病的药物 Pending CN1604783A (zh)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
DE10155280 2001-10-26
DE10155280.7 2001-10-26
DE10158411.3 2001-11-29
DE10158411 2001-11-29
DE10160151.4 2001-12-07
DE10160151A DE10160151A1 (de) 2001-01-09 2001-12-07 Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
EPPCT/EP02/00152 2002-01-09
PCT/EP2002/000151 WO2002055692A2 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eines zielgens und medikament zur therapie einer tumorerkrankung
EPPCT/EP02/00151 2002-01-09
PCT/EP2002/000152 WO2002055693A2 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eines zielgens

Publications (1)

Publication Number Publication Date
CN1604783A true CN1604783A (zh) 2005-04-06

Family

ID=39189390

Family Applications (1)

Application Number Title Priority Date Filing Date
CNA028251636A Pending CN1604783A (zh) 2001-10-26 2002-10-25 通过rna干扰治疗纤维化疾病的药物

Country Status (4)

Country Link
US (1) US20080070856A1 (enExample)
JP (1) JP2005512976A (enExample)
CN (1) CN1604783A (enExample)
WO (2) WO2003035083A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109432047A (zh) * 2018-10-29 2019-03-08 中国药科大学 一种逆转肺纤维化纳米制剂及其制备方法
CN112843253A (zh) * 2021-01-13 2021-05-28 上海交通大学 一种基因/药物复合脂质制剂及其制备方法和用途

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US7423142B2 (en) 2001-01-09 2008-09-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
US7745418B2 (en) 2001-10-12 2010-06-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting viral replication
DE10202419A1 (de) 2002-01-22 2003-08-07 Ribopharma Ag Verfahren zur Hemmung der Expression eines durch eine Chromosomen-Aberration entstandenen Zielgens
US7786290B2 (en) 2003-06-13 2010-08-31 Alnylam Pharmaceuticals, Inc. Double-stranded ribonucleic acid with increased effectiveness in an organism
EP1486564A1 (de) * 2003-06-13 2004-12-15 Ribopharma AG SiRNA mit erhöhter Stabilität in Serum
US20070004657A1 (en) * 2003-11-17 2007-01-04 Orna Mor Diagnosis and treatment of kidney fibrosis and other fibrotic diseases
US20080171051A1 (en) * 2003-11-26 2008-07-17 Patrick Gerard Johnston Cancer Treatment
JP4543189B2 (ja) * 2004-03-10 2010-09-15 学校法人日本医科大学 TGFβ1レセプターII型に対するRNAiとして作用するRNA配列
FR2898908A1 (fr) 2006-03-24 2007-09-28 Agronomique Inst Nat Rech Procede de preparation de cellules aviaires differenciees et genes impliques dans le maintien de la pluripotence
EP2323695B1 (en) 2008-08-19 2018-12-05 Nektar Therapeutics Complexes of small-interfering nucleic acids
MX2011002143A (es) * 2008-08-25 2011-07-20 Excaliard Pharmaceuticals Inc Oligonucleotidos antisentido dirigidos contra el factor de crecimiento del tejido conectivo y usos de los mismos.
US8946172B2 (en) * 2008-08-25 2015-02-03 Excaliard Pharmaceuticals, Inc. Method for reducing scarring during wound healing using antisense compounds directed to CTGF
US8796443B2 (en) * 2008-09-22 2014-08-05 Rxi Pharmaceuticals Corporation Reduced size self-delivering RNAi compounds
US8771692B2 (en) 2009-07-02 2014-07-08 Fibrogen, Inc. Methods for treatment of muscular dystrophy
US8916693B2 (en) 2009-09-17 2014-12-23 Nektar Therapeutics Monoconjugated chitosans as delivery agents for small interfering nucleic acids
WO2011056234A1 (en) 2009-11-06 2011-05-12 Fibrogen, Inc. Treatment for radiation-induced disorders
KR20180044433A (ko) * 2010-03-24 2018-05-02 알엑스아이 파마슈티칼스 코포레이션 진피 및 섬유증성 적응증에서의 rna 간섭
CA2794187C (en) 2010-03-24 2020-07-14 Rxi Pharmaceuticals Corporation Rna interference in ocular indications
AU2011261434B2 (en) * 2010-06-02 2015-11-26 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
AR083445A1 (es) 2010-10-14 2013-02-27 Univ Mie siARN CONTRA LA FIBROSIS
WO2012061811A2 (en) 2010-11-05 2012-05-10 Fibrogen, Inc. Treatment method for lung remodeling diseases
PT2670411T (pt) 2011-02-02 2019-06-18 Excaliard Pharmaceuticals Inc Compostos anti sentido visando um fator de crescimento do tecido conetivo (ctfg) para utilização num método de tratamento de queloides ou cicatrizes hipertróficas
WO2016098782A1 (ja) 2014-12-15 2016-06-23 株式会社ボナック TGF-β1発現抑制のための一本鎖核酸分子
ES2844899T3 (es) 2015-12-10 2021-07-23 Fibrogen Inc Métodos para el tratamiento de enfermedades de las neuronas motoras
KR102666000B1 (ko) * 2016-07-29 2024-05-14 서울대학교 산학협력단 cFLIP siRNA를 포함하는 인터페론 베타 저항성 암 질환 치료용 또는 감작용 조성물
US20200369759A1 (en) 2019-05-23 2020-11-26 Fibrogen, Inc. Methods of treatment of muscular dystrophies
WO2024262593A1 (ja) * 2023-06-22 2024-12-26 公益財団法人川崎市産業振興財団 核酸の血中安定性を向上させ、標的化効率を向上させる方法

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0737071A1 (en) * 1993-06-15 1996-10-16 Il- Yang Pharm. Co., Ltd. Anti-sense oligodeoxynucleotide to fibrogenic cytokines and use thereof
DE19631919C2 (de) * 1996-08-07 1998-07-16 Deutsches Krebsforsch Anti-Sinn-RNA mit Sekundärstruktur
US6242569B1 (en) * 1997-02-05 2001-06-05 Tularik, Inc. Regulators of apoptosis
DE19713393C2 (de) * 1997-04-01 2002-12-05 Apotech Res & Dev Ltd Flip-Gen und Flip-Protein
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
WO1999062927A1 (en) * 1998-06-05 1999-12-09 Human Genome Sciences, Inc. Connective tissue growth factor-4
EP1117443B1 (en) * 1998-10-08 2005-11-02 Stichting voor de Technische Wetenschappen Peptide-based carrier devices for stellate cells
AU765133B2 (en) * 1998-11-06 2003-09-11 Fibrogen, Inc. Connective tissue growth factor (CTGF) and methods of use
KR100664625B1 (ko) * 1998-12-14 2007-01-04 유니버시티 오브 마이애미 결합 조직 성장인자 단편, 그의 제조 방법 및 그의 용도
DE19956568A1 (de) * 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
JP2003509030A (ja) * 1999-09-17 2003-03-11 アイシス・ファーマシューティカルス・インコーポレーテッド トランスフォーミング成長因子−β発現のアンチセンスオリゴヌクレオチド調節
GB9927444D0 (en) * 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
KR20080023768A (ko) * 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
KR100909681B1 (ko) * 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109432047A (zh) * 2018-10-29 2019-03-08 中国药科大学 一种逆转肺纤维化纳米制剂及其制备方法
CN112843253A (zh) * 2021-01-13 2021-05-28 上海交通大学 一种基因/药物复合脂质制剂及其制备方法和用途

Also Published As

Publication number Publication date
WO2003035083A1 (de) 2003-05-01
JP2005512976A (ja) 2005-05-12
US20080070856A1 (en) 2008-03-20
WO2003035868A1 (de) 2003-05-01

Similar Documents

Publication Publication Date Title
CN1604783A (zh) 通过rna干扰治疗纤维化疾病的药物
JP6999590B2 (ja) 細胞内貫通能を持ってrna干渉を誘導する核酸分子およびその用途
US8106180B2 (en) Methods and products for expression of micro RNAs
US20050119202A1 (en) Medicament to treat a fibrotic disease
CN1241646C (zh) 子宫内膜功能或相关功能的改善
CN1622994A (zh) TGF-β特异性共价闭合反义分子
CN1650010A (zh) 抑制靶基因表达的方法和治疗肿瘤疾病的药物
JP6789513B2 (ja) 有用エビ類の卵成熟抑制を解除する方法
CN115161321B (zh) ssc-miR-30c-3p在制备抗PDCoV增殖药物中的应用
CN1458281A (zh) 严重急性呼吸道综合症病原冠状病毒rna干扰制剂
CN1615157A (zh) 抑制器官移植排异反应的组合物及其使用方法
CN1922330A (zh) 用于抗SARS冠状病毒治疗的RNAi物质
CN115068611B (zh) ssc-miR-374b-3p在制备抗PDCoV增殖药物中的应用
JP5567555B2 (ja) 頭頸部腫瘍増殖抑制剤
WO2022270071A1 (ja) 肝線維化抑制のための剤及び医薬組成物
CN109988767A (zh) 一种抑制rip3基因表达的小干扰rna制备及其应用
WO2013051459A1 (ja) RNA干渉によるmRNA発現の抑制を促進する促進剤およびその用途
CN115851902B (zh) Rp11-544d21.2的制药用途、在制备抗心衰药物方面的用途及药物与制备方法
CN114533877A (zh) Bag3抑制剂在制备治疗瘢痕产品中的用途
CN1842349A (zh) 使用无针注射器进行皮肤疾病基因治疗
CN1176098C (zh) 新的三螺旋形成寡核苷酸及其在抗乙肝病毒中的应用
CN1427008A (zh) 设计与选择天然的siRNA作为基因药物的方法及药物配方
CN116585326B (zh) 科罗索酸在制备抑制流行性乙型脑炎病毒药物中的用途
CN1225557C (zh) 一种以微生物克隆载体生产治疗血管疾病基因药物血管内皮生长因子-2裸dna的方法
CN1824326A (zh) 抗病毒药物组合物

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C12 Rejection of a patent application after its publication
RJ01 Rejection of invention patent application after publication