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WO2012027713A3 - Compositions and methods for inhibition of snca - Google Patents

Compositions and methods for inhibition of snca Download PDF

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Publication number
WO2012027713A3
WO2012027713A3 PCT/US2011/049427 US2011049427W WO2012027713A3 WO 2012027713 A3 WO2012027713 A3 WO 2012027713A3 US 2011049427 W US2011049427 W US 2011049427W WO 2012027713 A3 WO2012027713 A3 WO 2012027713A3
Authority
WO
WIPO (PCT)
Prior art keywords
snca
methods
inhibition
compositions
dsrna
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.)
Ceased
Application number
PCT/US2011/049427
Other languages
French (fr)
Other versions
WO2012027713A2 (en
Inventor
Gregory Hinkle
David Bumcrot
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 Pharmaceuticals Inc
Original Assignee
Alnylam 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of WO2012027713A2 publication Critical patent/WO2012027713A2/en
Anticipated expiration legal-status Critical
Publication of WO2012027713A3 publication Critical patent/WO2012027713A3/en
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting alpha- synuclein (SNCA) gene expression and methods of using the dsRNA to inhibit expression of SNCA.
PCT/US2011/049427 2010-08-26 2011-08-26 Compositions and methods for inhibition of snca Ceased WO2012027713A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US37743710P 2010-08-26 2010-08-26
US61/377,437 2010-08-26

Publications (2)

Publication Number Publication Date
WO2012027713A2 WO2012027713A2 (en) 2012-03-01
WO2012027713A3 true WO2012027713A3 (en) 2014-05-30

Family

ID=45724098

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/049427 Ceased WO2012027713A2 (en) 2010-08-26 2011-08-26 Compositions and methods for inhibition of snca

Country Status (1)

Country Link
WO (1) WO2012027713A2 (en)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2011329777B2 (en) 2010-11-17 2016-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
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
BR112020006661A2 (en) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. gene therapies for liposomal disorders
CN112501208A (en) * 2017-10-03 2021-03-16 普利维尔治疗公司 Gene therapy for lysosomal disorders
US11802294B2 (en) 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
TWI809004B (en) * 2017-11-09 2023-07-21 美商Ionis製藥公司 Compounds and methods for reducing snca expression
CA3088112A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
KR102839166B1 (en) 2018-01-12 2025-07-28 브리스톨-마이어스 스큅 컴퍼니 Antisense oligonucleotides targeting alpha-synuclein and uses thereof
JP2021511027A (en) * 2018-01-12 2021-05-06 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブＲｏｃｈｅ Ｉｎｎｏｖａｔｉｏｎ Ｃｅｎｔｅｒ Ｃｏｐｅｎｈａｇｅｎ Ａ／Ｓ Alpha-synuclein antisense oligonucleotide and its use
PH12021550254A1 (en) * 2018-08-03 2023-02-06 Genzyme Corp Variant rnai against alpha-synuclein
WO2020210713A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN115777021A (en) * 2020-03-18 2023-03-10 马萨诸塞大学 Oligonucleotides for SNCA regulation
WO2022072447A1 (en) * 2020-10-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
WO2025052316A1 (en) * 2023-09-07 2025-03-13 Janssen Pharmaceutica Nv SNCA siRNA AND USES THEREOF
WO2025151407A1 (en) * 2024-01-08 2025-07-17 Denali Therapeutics Inc. Compositions and methods for modulating alpha-synuclein expression
WO2025160429A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
DE102024104395A1 (en) 2024-02-16 2025-08-21 Hochschule Bielefeld - University of Applied Sciences and Arts, abgekürzt HSBI, Körperschaft des öffentlichen Rechts Polyelectrolyte complex and its preparation

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090099069A1 (en) * 2004-12-01 2009-04-16 Whitehead Institute For Biomedical Research Modulators of alpha-synuclein toxicity
US7579458B2 (en) * 2002-11-14 2009-08-25 Dharmacon, Inc. siRNA targeting synuclein, alpha (SNCA-1)
US20090269744A1 (en) * 2005-10-28 2009-10-29 Exonhit Therapeutics Sa Cancer detection method
US20100204306A1 (en) * 2007-12-14 2010-08-12 Alnylam Pharmaceuticals, Inc. Method of Treating Neurodegenerative Disease

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7579458B2 (en) * 2002-11-14 2009-08-25 Dharmacon, Inc. siRNA targeting synuclein, alpha (SNCA-1)
US20090099069A1 (en) * 2004-12-01 2009-04-16 Whitehead Institute For Biomedical Research Modulators of alpha-synuclein toxicity
US20090269744A1 (en) * 2005-10-28 2009-10-29 Exonhit Therapeutics Sa Cancer detection method
US20100204306A1 (en) * 2007-12-14 2010-08-12 Alnylam Pharmaceuticals, Inc. Method of Treating Neurodegenerative Disease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SORTIRIOU ET AL.: "A single nucleotide polymorphism in the 3'UTR of the SNCA gene encoding alpha-synuclein is a new potential susceptibiity locus for parkinson disease.", NEUROSCI LETT, vol. 461, no. 2, 16 September 2009 (2009-09-16), pages 196 - 201 *

Also Published As

Publication number Publication date
WO2012027713A2 (en) 2012-03-01

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