CN101448944B - Cns病症的治疗 - Google Patents

Cns病症的治疗 Download PDF

Info

Publication number
CN101448944B
CN101448944B CN200780017978.7A CN200780017978A CN101448944B CN 101448944 B CN101448944 B CN 101448944B CN 200780017978 A CN200780017978 A CN 200780017978A CN 101448944 B CN101448944 B CN 101448944B
Authority
CN
China
Prior art keywords
sirna
sina
gene
cns
application
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.)
Active
Application number
CN200780017978.7A
Other languages
English (en)
Chinese (zh)
Other versions
CN101448944A (zh
Inventor
安娜·伊萨贝尔·希门尼斯安东
安赫拉·塞斯托·亚格
玛·康塞普西翁·希门尼斯·戈麦斯
爱德华多·戈麦斯-阿塞沃古利翁
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.)
Sylentis SA
Original Assignee
Sylentis SA
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 Sylentis SA filed Critical Sylentis SA
Publication of CN101448944A publication Critical patent/CN101448944A/zh
Application granted granted Critical
Publication of CN101448944B publication Critical patent/CN101448944B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
    • A61K2039/541—Mucosal route
    • A61K2039/543—Mucosal route intranasal
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN200780017978.7A 2006-03-17 2007-03-16 Cns病症的治疗 Active CN101448944B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB0605337.5A GB0605337D0 (en) 2006-03-17 2006-03-17 Treatment of CNS conditions
GB0605337.5 2006-03-17
PCT/GB2007/050128 WO2007107789A2 (en) 2006-03-17 2007-03-16 Treatment of cns conditions

Publications (2)

Publication Number Publication Date
CN101448944A CN101448944A (zh) 2009-06-03
CN101448944B true CN101448944B (zh) 2014-02-12

Family

ID=36292900

Family Applications (1)

Application Number Title Priority Date Filing Date
CN200780017978.7A Active CN101448944B (zh) 2006-03-17 2007-03-16 Cns病症的治疗

Country Status (13)

Country Link
US (1) US8871729B2 (enExample)
EP (1) EP2004823B1 (enExample)
JP (2) JP5781722B2 (enExample)
KR (2) KR101669944B1 (enExample)
CN (1) CN101448944B (enExample)
AU (1) AU2007228570B2 (enExample)
CA (1) CA2645120C (enExample)
ES (1) ES2582649T3 (enExample)
GB (1) GB0605337D0 (enExample)
IL (1) IL194114A (enExample)
MX (1) MX2008011731A (enExample)
RU (1) RU2426544C2 (enExample)
WO (1) WO2007107789A2 (enExample)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2145014B1 (en) * 2007-04-05 2012-12-12 The J. David Gladstone Institutes Agents that reduce neuronal overexcitation
US20110288155A1 (en) 2008-12-18 2011-11-24 Elena Feinstein Sirna compounds and methods of use thereof
RU2562861C2 (ru) * 2009-09-11 2015-09-10 Айсис Фармасьютикалс, Инк. Модуляция экспрессии гентингтина
AU2010324658A1 (en) 2009-11-26 2012-05-03 Quark Pharmaceuticals, Inc. siRNA compounds comprising terminal substitutions
US20110135613A1 (en) 2009-12-03 2011-06-09 The J. David Gladstone Institutes Methods for treating apolipoprotein e4-associated disorders
US8778904B2 (en) 2009-12-09 2014-07-15 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the CNS
AU2011219029B2 (en) * 2010-02-26 2017-02-02 Columbia University Methods and compositions for the detection and treatment of cancer involving miRNAs and miRNA inhibitors and targets
EP2380595A1 (en) 2010-04-19 2011-10-26 Nlife Therapeutics S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
DK2560687T3 (en) 2010-04-19 2017-09-18 Nlife Therapeutics S L Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types
US9260471B2 (en) 2010-10-29 2016-02-16 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
EP2649181B1 (en) 2010-12-06 2016-04-27 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising positional modifications
US9949923B2 (en) * 2011-03-15 2018-04-24 Optinose As Nasal delivery
WO2013053954A1 (en) * 2011-10-14 2013-04-18 Centre National De La Recherche Scientifique Method of diagnosis, prognostic or treatment of neurodegenerative diseases
US9486540B2 (en) * 2012-03-09 2016-11-08 Northeastern University Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders
WO2013148260A1 (en) 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
US9611473B2 (en) 2012-09-12 2017-04-04 Quark Pharmaceuticals, Inc. Double-stranded nucleic acid compounds
AU2013336581A1 (en) 2012-10-26 2015-06-11 Nlife Therapeutics, S.L. Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
ES2807379T3 (es) * 2013-03-14 2021-02-22 Ionis Pharmaceuticals Inc Composiciones y métodos para regular la expresión de Tau
JP2016523980A (ja) * 2013-07-11 2016-08-12 ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク タウ発現を抑制するマイクロｒｎａ
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
US20160208247A1 (en) 2013-07-31 2016-07-21 Qbi Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
JP2017505756A (ja) * 2013-12-13 2017-02-23 ザ ジェネラル ホスピタル コーポレイション 可溶性高分子量（ｈｍｗ）タウ種およびその用途
WO2016151523A1 (en) * 2015-03-25 2016-09-29 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
JOP20200228A1 (ar) * 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
AU2019233612A1 (en) 2018-03-13 2020-09-10 Janssen Pharmaceutica Nv Modified oligonucleotides for use in treatment of tauopathies
MX2022011516A (es) * 2020-03-18 2023-01-04 Univ Massachusetts Oligonucleotidos para la modulacion de mapt.
AU2021246024A1 (en) * 2020-03-30 2022-10-27 Alnylam Pharmaceuticals, Inc. Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof
CN113817728A (zh) * 2020-06-19 2021-12-21 四川大学华西医院 一种有效干扰Tau蛋白表达的重组慢病毒及其应用
AU2023269281A1 (en) * 2022-05-12 2024-10-10 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting mapt expression
CA3272944A1 (en) * 2022-12-29 2024-07-04 Voyager Therapeutics Inc MAPPING COMPOSITIONS AND REGULATION METHODS

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2116460A1 (en) 1993-03-02 1994-09-03 Mitsubishi Chemical Corporation Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being
US6410046B1 (en) * 1996-11-19 2002-06-25 Intrabrain International Nv Administering pharmaceuticals to the mammalian central nervous system
WO2005003350A2 (en) 2003-06-27 2005-01-13 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA)
JP2004532849A (ja) 2001-04-20 2004-10-28 カイロン コーポレイション 中枢神経系へのポリヌクレオチド薬剤の送達
US20050159376A1 (en) 2002-02-20 2005-07-21 Slrna Therapeutics, Inc. RNA interference mediated inhibition 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA)
IL143379A (en) 2001-05-24 2013-11-28 Yissum Res Dev Co Oligonucleotide against human ache isoform r and its uses
GB2406568B (en) 2002-02-20 2005-09-28 Sirna Therapeutics Inc RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
CA2482904A1 (en) 2002-04-18 2003-10-23 Lynkeus Biotech Gmbh Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification
US20030225031A1 (en) 2002-05-21 2003-12-04 Quay Steven C. Administration of acetylcholinesterase inhibitors to the cerebral spinal fluid
US20040254146A1 (en) 2002-05-21 2004-12-16 Nastech Pharmaceutical Company Inc. Carboxylate salts of galantamine and their pharmaceutical use
US20050042646A1 (en) 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20050106731A1 (en) 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
ATE513843T1 (de) 2002-09-25 2011-07-15 Univ Massachusetts Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna
AU2003279004B2 (en) 2002-09-28 2009-10-08 Massachusetts Institute Of Technology Influenza therapeutic
ES2334125T3 (es) * 2002-11-04 2010-03-05 University Of Massachusetts Interferencia de arn especifico de alelos.
US7829694B2 (en) 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
EP1635763B1 (en) * 2003-06-09 2012-08-08 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease
WO2005044976A2 (en) 2003-06-20 2005-05-19 Isis Pharmaceuticals, Inc. Oligomeric compounds for use in gene modulation
US20070185042A1 (en) 2003-08-08 2007-08-09 The Brigham And Women's Hospital, Inc. President And Fellows Of Harvard College Sirna based methods for treating alzheimer's disease
WO2005045037A2 (en) 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP1735443A2 (en) 2004-04-14 2006-12-27 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
US20050265927A1 (en) 2004-05-17 2005-12-01 Yale University Intranasal delivery of nucleic acid molecules
WO2006041922A2 (en) 2004-10-08 2006-04-20 Dara Biosciences, Inc. Agents and methods for administration to the central nervous system

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Dzitoyeva S et al.Intra-abdominal injection of double-stranded RNA into anesthetized adult Drosophila triggers RNA interference in the central nervous system.《Mol Psychiatry》.2001,第6卷(第6期),665-670. *
吴云成 等.RNA 干扰及其在神经变性性疾病研究中的应用.《中国神经科学杂志》.2004,第20卷(第4期),321-326. *

Also Published As

Publication number Publication date
EP2004823A2 (en) 2008-12-24
EP2004823B1 (en) 2016-05-25
US20090176728A1 (en) 2009-07-09
JP5923466B2 (ja) 2016-05-24
AU2007228570B2 (en) 2013-09-19
GB0605337D0 (en) 2006-04-26
CA2645120A1 (en) 2007-09-27
RU2008141162A (ru) 2010-04-27
ES2582649T3 (es) 2016-09-14
IL194114A0 (en) 2011-08-01
IL194114A (en) 2012-06-28
JP2009530257A (ja) 2009-08-27
AU2007228570A8 (en) 2008-10-23
JP5781722B2 (ja) 2015-09-24
MX2008011731A (es) 2009-03-05
WO2007107789A2 (en) 2007-09-27
JP2013234197A (ja) 2013-11-21
CN101448944A (zh) 2009-06-03
AU2007228570A1 (en) 2007-09-27
CA2645120C (en) 2015-02-24
US8871729B2 (en) 2014-10-28
HK1126812A1 (zh) 2009-09-11
RU2426544C2 (ru) 2011-08-20
KR101605144B1 (ko) 2016-03-21
WO2007107789A3 (en) 2008-05-08
KR20140076634A (ko) 2014-06-20
KR20080111063A (ko) 2008-12-22
KR101669944B1 (ko) 2016-10-28

Similar Documents

Publication Publication Date Title
CN101448944B (zh) Cns病症的治疗
US11499155B2 (en) Dynamin 2 inhibitor for the treatment of centronuclear myopathies
EP1567539B1 (en) Allele-specific rna interference
JP5766900B2 (ja) 眼内圧の上昇によって特徴付けられる眼の疾患のｓｉＲＮＡによる治療
US7732421B2 (en) RNAI-mediated inhibition of tumor necrosis factor α-related conditions
US9982266B2 (en) Methods and pharmaceutical compositions for treatment of cystic fibrosis
JP2011517404A (ja) ＲＴＰ８０１を阻害するための新規なｓｉＲＮＡ化合物
CN120500343A (zh) 用于在治疗与雄激素受体(ar)的功能获得和/或ar辅激活物的过表达相关的疾病中使用的lsd1抑制剂和prmt6抑制剂
JP2008511302A (ja) Ｐ２ｘ７レセプター発現を阻害する方法及び組成物
HK1126812B (en) Treatment of cns conditions
US7741469B2 (en) Compositions for treating hearing loss and methods of use thereof
WO2025253282A1 (en) Small interfering nucletides (sioligo) for the treatment of cole-carpenter syndrome (ccs)
WO2025072604A1 (en) Rna-editing gene therapy approaches for treating myotonic dystrophy type 1 (dm1)
WO2024036343A2 (en) Synergistic nucleic acid based therapeutics and methods of use for treating genetic disorders

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C14 Grant of patent or utility model
GR01 Patent grant