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WO2005105995A3 - RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) - Google Patents

RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) Download PDF

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Publication number
WO2005105995A3
WO2005105995A3 PCT/US2005/006661 US2005006661W WO2005105995A3 WO 2005105995 A3 WO2005105995 A3 WO 2005105995A3 US 2005006661 W US2005006661 W US 2005006661W WO 2005105995 A3 WO2005105995 A3 WO 2005105995A3
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Prior art keywords
polyq
repeat
diseases
nucleic acid
conditions
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PCT/US2005/006661
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French (fr)
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WO2005105995A2 (en
Inventor
James Mcswiggen
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Sirna Therapeutics Inc
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Sirna Therapeutics Inc
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Priority claimed from US10/824,036 external-priority patent/US20050191638A1/en
Priority claimed from US10/826,966 external-priority patent/US20050032733A1/en
Priority claimed from PCT/US2004/013456 external-priority patent/WO2005041859A2/en
Priority claimed from PCT/US2004/016390 external-priority patent/WO2005019453A2/en
Priority claimed from US10/923,536 external-priority patent/US20070042983A1/en
Priority claimed from PCT/US2005/004270 external-priority patent/WO2005078097A2/en
Application filed by Sirna Therapeutics Inc filed Critical Sirna Therapeutics Inc
Priority to EP05724244A priority Critical patent/EP1735443A2/en
Publication of WO2005105995A2 publication Critical patent/WO2005105995A2/en
Publication of WO2005105995A3 publication Critical patent/WO2005105995A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention concerns compounds, compositions, and methods for the study, diagnosis, and treatment of diseases and conditions associated with polyglutamine repeat (polyQ) allelic variants that respond to the modulation of gene expression and/or activity. The present invention also concerns compounds, compositions, and methods relating to diseases and conditions associated with polyglutamine repeat (polyQ) allelic variants that respond to the modulation of expression and/or activity of genes involved in polyQ repeat gene expression pathways. or other cellular processes that mediate the maintenance or development of polyQ repeat diseases and conditions such as Huntington disease and related conditions such as progressive chorea, rigidity, dementia, and seizures, spinocerebellar ataxia, Spinal and bulbar muscular dystrophy (SBMA), dentatorubropallidoluysian atrophy (DIZPLA), and any other diseases or conditions that are related to or will respond to the levels of a repeat expansion (RE) protein in a cell or tissue, alone or in combination with other therapies. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), douse-stranded RN (dsRNA), micro-RNA (miRNA), and short hairpin RN (shRNA) molecules capable of mediating RN interference (RNAi) against the expression disease related genes or alleles having polyQ repeat sequences.
PCT/US2005/006661 2004-04-14 2005-03-01 RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) Ceased WO2005105995A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP05724244A EP1735443A2 (en) 2004-04-14 2005-03-01 RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US10/824,036 US20050191638A1 (en) 2002-02-20 2004-04-14 RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US10/824,036 2004-04-14
US10/826,966 US20050032733A1 (en) 2001-05-18 2004-04-16 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
US10/826,966 2004-04-16
PCT/US2004/013456 WO2005041859A2 (en) 2003-04-30 2004-04-30 Conjugates and compositions for cellular delivery.
USPCT/US04/13456 2004-04-30
USPCT/US04/16390 2004-05-24
PCT/US2004/016390 WO2005019453A2 (en) 2001-05-18 2004-05-24 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
US10/923,536 2004-08-20
US10/923,536 US20070042983A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
USPCT/US05/04270 2005-02-09
PCT/US2005/004270 WO2005078097A2 (en) 2004-02-10 2005-02-09 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING MULTIFUNCTIONAL SHORT INTERFERING NUCLEIC ACID (Multifunctional siNA)

Publications (2)

Publication Number Publication Date
WO2005105995A2 WO2005105995A2 (en) 2005-11-10
WO2005105995A3 true WO2005105995A3 (en) 2005-12-29

Family

ID=56290667

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2005/006661 Ceased WO2005105995A2 (en) 2004-04-14 2005-03-01 RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)

Country Status (3)

Country Link
EP (1) EP1735443A2 (en)
GB (1) GB2415961A (en)
WO (1) WO2005105995A2 (en)

Cited By (4)

* Cited by examiner, † Cited by third party
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US9260716B2 (en) 2002-08-05 2016-02-16 University Of Iowa Research Foundation RNA interference suppression of neurodegenerative diseases and methods of use thereof
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9487779B2 (en) 2002-08-05 2016-11-08 University Of Iowa Research Foundation siRNA-mediated gene silencing
US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA

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US20050042646A1 (en) 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US7829694B2 (en) * 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
WO2008005562A2 (en) * 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2006083800A2 (en) * 2005-01-31 2006-08-10 University Of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
EP1885854B1 (en) 2005-05-06 2012-10-17 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
US20070299027A1 (en) 2006-01-26 2007-12-27 Gene Hung Compositions and their uses directed to huntingtin
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
SI2049664T1 (en) 2006-08-11 2012-04-30 Prosensa Technologies Bv Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders
WO2008134646A2 (en) * 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
AU2008260103B2 (en) 2007-05-31 2014-04-03 University Of Iowa Research Foundation Reduction of off-target RNA interference toxicity
CN101790385A (en) 2007-07-12 2010-07-28 普罗森那技术公司 Molecules for targeting compounds to various selected organs, tissues or tumor cells
EP2167136B1 (en) 2007-07-12 2016-04-20 BioMarin Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
RU2356498C1 (en) * 2007-09-26 2009-05-27 Государственное Учреждение Научный Центр Неврологии Российской Академии Медицинских Наук Neurodegenerative huntington's chorea diagnostic method
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
JP2011510678A (en) 2008-02-08 2011-04-07 プロセンサ ホールディング ビーブイ Methods and means for treating DNA repeat instability related genetic disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
EP2317847B1 (en) 2008-07-29 2019-04-17 The Board of Regents of The University of Texas System Selective inhibition of polyglutamine protein expression
NZ595955A (en) 2009-04-24 2012-10-26 Prosensa Technologies Bv Oligonucleotide comprising an inosine for treating dmd
NZ714887A (en) 2009-09-11 2019-07-26 Ionis Pharmaceuticals Inc Modulation of huntingtin expression
US8778904B2 (en) 2009-12-09 2014-07-15 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the CNS
WO2012109667A1 (en) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
WO2013022984A1 (en) 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
CN112251436A (en) 2012-01-27 2021-01-22 比奥马林技术公司 RNA-regulated oligonucleotides with improved properties for the treatment of duchenne muscular dystrophy and becker muscular dystrophy
KR102096014B1 (en) 2012-12-05 2020-04-03 알닐람 파마슈티칼스 인코포레이티드 PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
RU2552913C1 (en) * 2014-03-25 2015-06-10 Государственное бюджетное образовательное учреждение высшего профессионального образования "Нижегородская государственная медицинская академия" Министерства Здравоохранения Российской Федерации (ГБОУ ВПО НижГМА Минздрава России) Method of diagnosing huntington's disease
US9523093B2 (en) 2014-05-20 2016-12-20 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
HK1246344B (en) 2014-12-24 2020-01-10 Uniqure Ip B.V. Rnai induced huntingtin gene suppression
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EA201890571A1 (en) 2015-08-25 2018-10-31 Элнилэм Фармасьютикалз, Инк. METHODS AND COMPOSITIONS FOR THE TREATMENT OF VIOLATION ASSOCIATED WITH THE PROTROTYNCONVERTASE GENE SUBTILYSINE / CEXINOUS TYPE (PCSK9)
WO2020144611A1 (en) 2019-01-09 2020-07-16 Universidade De Coimbra Double stranded rna and uses thereof
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JP2023527684A (en) * 2020-05-11 2023-06-30 ジェネンテック， インコーポレイテッド Complement component C1S inhibitors for treating neurological disorders and related compositions, systems and methods of using same
AR125230A1 (en) 2021-03-29 2023-06-28 Alnylam Pharmaceuticals Inc COMPOSITIONS OF ANTI-HUNTINGTIN (HTT) RNAi AGENTS AND THEIR METHODS OF USE
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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9260716B2 (en) 2002-08-05 2016-02-16 University Of Iowa Research Foundation RNA interference suppression of neurodegenerative diseases and methods of use thereof
US9487779B2 (en) 2002-08-05 2016-11-08 University Of Iowa Research Foundation siRNA-mediated gene silencing
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA

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EP1735443A2 (en) 2006-12-27
GB2415961A (en) 2006-01-11
GB0507019D0 (en) 2005-05-11

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