GB0507019D0 - 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)

Info

Publication number
GB0507019D0
GB0507019D0 GBGB0507019.8A GB0507019A GB0507019D0 GB 0507019 D0 GB0507019 D0 GB 0507019D0 GB 0507019 A GB0507019 A GB 0507019A GB 0507019 D0 GB0507019 D0 GB 0507019D0
Authority
GB
United Kingdom
Prior art keywords
polyq
polyglutamine
sina
nucleic acid
rna interference
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.)
Withdrawn
Application number
GBGB0507019.8A
Other versions
GB2415961A (en
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.)
Sirna Therapeutics Inc
Original Assignee
Sirna Therapeutics 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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=56290667&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=GB0507019(D0) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
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
Publication of GB0507019D0 publication Critical patent/GB0507019D0/en
Publication of GB2415961A publication Critical patent/GB2415961A/en
Withdrawn legal-status Critical Current

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    • 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
    • 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
    • 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
    • C12N2310/11—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
    • 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 N.A.
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/332—Abasic residue
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

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)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
GB0507019A 2004-04-14 2005-04-06 RNA interference (RNAi) of huntingtin (htt) gene Withdrawn GB2415961A (en)

Applications Claiming Priority (6)

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)
US10/826,966 US20050032733A1 (en) 2001-05-18 2004-04-16 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
PCT/US2004/013456 WO2005041859A2 (en) 2003-04-30 2004-04-30 Conjugates and compositions for cellular delivery.
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 US20070042983A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
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
GB0507019D0 true GB0507019D0 (en) 2005-05-11
GB2415961A GB2415961A (en) 2006-01-11

Family

ID=56290667

Family Applications (1)

Application Number Title Priority Date Filing Date
GB0507019A Withdrawn GB2415961A (en) 2004-04-14 2005-04-06 RNA interference (RNAi) of huntingtin (htt) gene

Country Status (3)

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

Families Citing this family (40)

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US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
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
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
CA2596588C (en) * 2005-01-31 2017-06-27 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
WO2006121960A2 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
PL3210633T3 (en) 2006-01-26 2019-12-31 Ionis Pharmaceuticals, Inc. 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
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
CA2662704A1 (en) * 2006-07-07 2008-01-10 University Of Massachusetts Rna silencing compositions and methods for the treatment of huntington's disease
NZ574807A (en) 2006-08-11 2011-01-28 Prosensa Technologies Bv Methods and means for treating dna repeat instability associated genetic disorders
US8258286B2 (en) 2007-04-26 2012-09-04 University Of Iowa Research Foundation Reduction of off-target RNA interference toxicity
US20090036395A1 (en) 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
EP2167135A2 (en) 2007-07-12 2010-03-31 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
CN103212085A (en) 2007-07-12 2013-07-24 普罗森那技术公司 Molecules for targeting compounds to various selected organs or tissues
CN101896186A (en) 2007-10-26 2010-11-24 莱顿教学医院 The mode and the method for antagonism disorder of muscle
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
WO2009099326A1 (en) 2008-02-08 2009-08-13 Prosensa Holding Bv Methods and means for treating dna repeat instability associated 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
WO2010014592A1 (en) 2008-07-29 2010-02-04 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
ES2593836T3 (en) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleotide comprising an inosine to treat DMD
CA2931725C (en) 2009-09-11 2021-10-19 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
EP2742056B2 (en) 2011-08-11 2020-06-10 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP4043039A1 (en) 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
RS56783B9 (en) 2012-12-05 2021-12-31 Alnylam Pharmaceuticals Inc 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
CN107075514A (en) 2014-05-20 2017-08-18 衣阿华大学研究基金会 The therapeutic compounds of Huntington's disease
PL3237618T3 (en) * 2014-12-24 2019-09-30 Uniqure Ip B.V. Rnai induced huntingtin gene suppression
US20180127803A1 (en) * 2015-05-10 2018-05-10 Quandx Inc. Ultra sensitive probes for detection of nucleic acid
IL257486B (en) 2015-08-25 2022-07-01 Alnylam Pharmaceuticals Inc Methods and compositions for treating a proprotein convertase subtilisin kexin (pcsk9) gene-associated disorder
MX2021008331A (en) 2019-01-09 2021-08-05 Univ De Coimbra Double stranded rna and uses thereof.
WO2021005223A1 (en) 2019-07-10 2021-01-14 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
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
BR112023019981A2 (en) * 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc COMPOSITIONS OF HUNTINGTINA IRNA AGENT (HTT) AND METHODS OF USE THEREOF

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WO2003070910A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) AND VEGF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
ATE529512T1 (en) * 2002-02-01 2011-11-15 Life Technologies Corp DOUBLE STRANDED OLIGONUCLEOTIDES
US20050096284A1 (en) * 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20040092465A1 (en) * 2002-11-11 2004-05-13 Isis Pharmaceuticals Inc. Modulation of huntingtin interacting protein 1 expression
JP3917034B2 (en) * 2002-07-23 2007-05-23 湖北工業株式会社 Optical connector and manufacturing method thereof
US20050106731A1 (en) * 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
EP2821085B1 (en) * 2003-09-12 2020-04-29 University of Massachusetts Rna interference for the treatment of gain-of-function disorders
CA2554212A1 (en) * 2004-02-10 2005-08-25 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using multifunctional short interfering nucleic acid (multifunctional sina)

Also Published As

Publication number Publication date
WO2005105995A3 (en) 2005-12-29
EP1735443A2 (en) 2006-12-27
WO2005105995A2 (en) 2005-11-10
GB2415961A (en) 2006-01-11

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