[go: up one dir, main page]

WO2011078797A3 - Antisense oligonucleotides and uses threreof - Google Patents

Antisense oligonucleotides and uses threreof Download PDF

Info

Publication number
WO2011078797A3
WO2011078797A3 PCT/SG2010/000479 SG2010000479W WO2011078797A3 WO 2011078797 A3 WO2011078797 A3 WO 2011078797A3 SG 2010000479 W SG2010000479 W SG 2010000479W WO 2011078797 A3 WO2011078797 A3 WO 2011078797A3
Authority
WO
WIPO (PCT)
Prior art keywords
score
antisense oligonucleotides
threreof
mrna
antisense oligonucleotide
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/SG2010/000479
Other languages
French (fr)
Other versions
WO2011078797A2 (en
Inventor
Dwi Pramono Sarengat
Woon Chee Yee
Poh San Lai
Keng Boon Wee
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.)
Agency for Science Technology and Research Singapore
Singapore Health Services Pte Ltd
Original Assignee
Agency for Science Technology and Research Singapore
Singapore Health Services Pte Ltd
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 Agency for Science Technology and Research Singapore, Singapore Health Services Pte Ltd filed Critical Agency for Science Technology and Research Singapore
Publication of WO2011078797A2 publication Critical patent/WO2011078797A2/en
Publication of WO2011078797A3 publication Critical patent/WO2011078797A3/en
Anticipated expiration legal-status Critical
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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B15/00—ICT specially adapted for analysing two-dimensional or three-dimensional molecular structures, e.g. structural or functional relations or structure alignment
    • G16B15/10—Nucleic acid folding
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/20—Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
    • 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
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2320/33—Alteration of splicing
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B15/00—ICT specially adapted for analysing two-dimensional or three-dimensional molecular structures, e.g. structural or functional relations or structure alignment
    • G—PHYSICS
    • G16—INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR SPECIFIC APPLICATION FIELDS
    • G16B—BIOINFORMATICS, i.e. INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTED FOR GENETIC OR PROTEIN-RELATED DATA PROCESSING IN COMPUTATIONAL MOLECULAR BIOLOGY
    • G16B20/00—ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
    • G16B20/30—Detection of binding sites or motifs

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Spectroscopy & Molecular Physics (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Evolutionary Biology (AREA)
  • Medical Informatics (AREA)
  • Theoretical Computer Science (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Analytical Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention refers to an antisense oligonucleotide having a L3 score of about < 0.1; a L4_OR(5´) score of about < 0.15; a L4_OR(3´) score of about < 0.12; a PESE score of about > 70 %; and the antisense oligonucleotide binds to a target exon pre-mRNA starting in the first one third of the target exon pre-mRNA, counting from 5´ to 3' as well as method of a method of selecting at least one antisense oligonucleotide from a plurality of antisense oligonucleotides targeting a target sequence on one of a plurality of exons on a pre-mRNA.
PCT/SG2010/000479 2009-12-22 2010-12-22 Antisense oligonucleotides and uses threreof Ceased WO2011078797A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US28887709P 2009-12-22 2009-12-22
US61/288,877 2009-12-22

Publications (2)

Publication Number Publication Date
WO2011078797A2 WO2011078797A2 (en) 2011-06-30
WO2011078797A3 true WO2011078797A3 (en) 2011-11-10

Family

ID=44196374

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/SG2010/000479 Ceased WO2011078797A2 (en) 2009-12-22 2010-12-22 Antisense oligonucleotides and uses threreof

Country Status (1)

Country Link
WO (1) WO2011078797A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11053494B2 (en) 2014-08-09 2021-07-06 Research Institute At Nationwide Children's Hospital Methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2203173B1 (en) 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle 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
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
EP2870246B1 (en) * 2012-07-03 2019-09-11 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
MX388221B (en) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital RECOMBINANT ADENO-ASSOCIATED VIRUS DELIVERY OF U7snRNA POLYNUCLEOTIDE CONSTRUCTS TARGETING EXON 2.
SG10201912902TA (en) 2015-10-09 2020-02-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
MA45328A (en) 2016-04-01 2019-02-06 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
SG10201607303YA (en) * 2016-09-01 2018-04-27 Agency Science Tech & Res Antisense oligonucleotides to induce exon skipping
SG10201609048RA (en) 2016-10-28 2018-05-30 Agency Science Tech & Res Antisense oligonucleotides
KR20190104381A (en) 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 Nucleic Acid-Polypeptide Compositions and Methods of Inducing Exon Skipping
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
US11453879B2 (en) 2017-08-11 2022-09-27 Agency For Science, Technology And Research Method for screening splicing variants or events
TW202449155A (en) * 2018-07-27 2024-12-16 美商薩羅塔治療公司 Exon skipping oligomers for muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
MX2024000489A (en) 2021-07-09 2024-04-09 Dyne Therapeutics Inc Muscle targeting complexes and formulations for treating dystrophinopathies.
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
CN115440296A (en) * 2022-08-01 2022-12-06 赛业（广州）生物科技有限公司 A method, system, device and medium for predicting an ASO target

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6727355B2 (en) * 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
WO2006000057A1 (en) * 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US7618814B2 (en) * 2002-11-14 2009-11-17 Rosetta Genomics Ltd. Microrna-related nucleic acids and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6727355B2 (en) * 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
US7618814B2 (en) * 2002-11-14 2009-11-17 Rosetta Genomics Ltd. Microrna-related nucleic acids and uses thereof
WO2006000057A1 (en) * 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
AARTSMA-RUS A. ET AL.: "Functional Analysis of 114 Exon-Intemal AONs for Targeted DMD Exon Skipping: Indication for Steric Hindrance of SR Protein Binding Sites", OLIGONUCLEOTIDES, vol. 15, 2005, pages 284 - 297 *
AARTSMA-RUS A. ET AL.: "Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms", MOLECULAR THERAPY, vol. 17, no. 3, March 2009 (2009-03-01), pages 548 - 553 *
ARECHAVALA-GOMEZA V. ET AL.: "Comparative Analysis of Antisense Oligonucleotid Sequences for Targeted Skipping of Exon 51 During Dystrophin Pre- mRNA Splicing in Human Muscle", HUMAN GENE THERAPY, vol. 18, 2007, pages 798 - 810 *
HARDING P.L. ET AL.: "The Influence of Antisense Oligonucleotide Length on Dystrophin Exon Skipping", MOLECULAR THERAPY, vol. 15, no. 1, 2007, pages 157 - 166 *
POPPLEWELL L.J. ET AL.: "Design of Phosphorodiamidate Morpholino Oligomers (PMOs) for the Induction of Exon Skipping of the Human DMD Gene", MOLECULAR THERAPY, vol. 17, no. 3, March 2009 (2009-03-01), pages 554 - 561, XP002573471 *
WEE K. B. ET AL.: "Dynamics of Co-Transcriptional Pre-mRNA Folding Influences the Induction of Dystrophin Exon Skipping by Antisense Oligonucleotides", PLOS ONE, vol. 3, no. 3, 2008, pages E18144 *
WILTON S.D. ET AL.: "Antisense Oligonucleotide-induced Exon Skipping Across the Human Dystrophin Gene Transcript", MOLECULAR THERAPY, vol. 15, no. 7, 2007, pages 1288 - 1296 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11053494B2 (en) 2014-08-09 2021-07-06 Research Institute At Nationwide Children's Hospital Methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene

Also Published As

Publication number Publication date
WO2011078797A2 (en) 2011-06-30

Similar Documents

Publication Publication Date Title
WO2011078797A3 (en) Antisense oligonucleotides and uses threreof
EP4385568A3 (en) Compositions and methods for inhibiting expression of cd274/pd-l1 gene
HK1221978A1 (en) Tmprss6 irna compositions and methods of use thereof
EP4585686A3 (en) Modified double-stranded rna agents
WO2011133871A3 (en) 5&#39;-end derivatives
WO2011139911A3 (en) Lipid formulated single stranded rna
WO2009129465A3 (en) Compositions and methods for inhibiting expression of xbp-1 gene
WO2011123621A3 (en) 2&#39; and 5&#39; modified monomers and oligonucleotides
EP2194129A3 (en) Pharmaceutical composition comprising anti-miRNA antisense oligonucleotides
EP2666859A3 (en) Pharmaceutical composition comprising anti-miRNA antisense oligonucleotides
WO2009054551A3 (en) Lipid-modified double-stranded rna having potent rna interference effect
WO2008061537A3 (en) Oligonucleotides for modulating target rna activity
WO2008019142A3 (en) Oligonucleotide systems for targeted intracellular delivery
WO2008151639A3 (en) Oligonucleotides for modulation of target rna activity
EP1799859A4 (en) ANTISENSE OLIGONUCLEOTIDES IMPROVED
WO2007115168A3 (en) Compositions and methods for inhibiting expression of eg5 gene
NZ585327A (en) Lna antagonists targeting the androgen receptor
EP1766010A4 (en) ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON JUMPING AND METHODS OF USE
WO2007134161A8 (en) Compositions and methods for inhibiting expression of the pcsk9 gene
WO2011005860A3 (en) 5&#39; phosphate mimics
WO2009043353A3 (en) Micromirs
WO2010048585A3 (en) Oligomeric compounds and methods
WO2008151631A3 (en) Use of short oligonucleotides for reagent redundancy experiments in rna functional analysis
WO2008121604A3 (en) Compositions and methods for inhibiting expression of a gene from the ebola
WO2008008719A3 (en) Compositions and methods for inhibiting expression of the myc gene

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 10839911

Country of ref document: EP

Kind code of ref document: A1

NENP Non-entry into the national phase

Ref country code: DE

122 Ep: pct application non-entry in european phase

Ref document number: 10839911

Country of ref document: EP

Kind code of ref document: A2