EP3183347A4 - Oligonucléotides modulateurs d'épissage et leurs procédés d'utilisation - Google Patents

Oligonucléotides modulateurs d'épissage et leurs procédés d'utilisation Download PDF

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Publication number
EP3183347A4
EP3183347A4 EP15833669.3A EP15833669A EP3183347A4 EP 3183347 A4 EP3183347 A4 EP 3183347A4 EP 15833669 A EP15833669 A EP 15833669A EP 3183347 A4 EP3183347 A4 EP 3183347A4
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EP
European Patent Office
Prior art keywords
methods
splice modulating
modulating oligonucleotides
oligonucleotides
splice
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Withdrawn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
EP15833669.3A
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German (de)
English (en)
Other versions
EP3183347A2 (fr
Inventor
Melanie TALLENT
Nicole LYKENS
Gordon LUTZ
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.)
Lifesplice Pharma LLC
Original Assignee
Lifesplice Pharma LLC
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 Lifesplice Pharma LLC filed Critical Lifesplice Pharma LLC
Publication of EP3183347A2 publication Critical patent/EP3183347A2/fr
Publication of EP3183347A4 publication Critical patent/EP3183347A4/fr
Withdrawn legal-status Critical Current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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • 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
    • C12N2310/32—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
EP15833669.3A 2015-10-17 2015-10-17 Oligonucléotides modulateurs d'épissage et leurs procédés d'utilisation Withdrawn EP3183347A4 (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/IB2015/001917 WO2016027168A2 (fr) 2014-08-20 2015-10-17 Oligonucléotides modulateurs d'épissage et leurs procédés d'utilisation

Publications (2)

Publication Number Publication Date
EP3183347A2 EP3183347A2 (fr) 2017-06-28
EP3183347A4 true EP3183347A4 (fr) 2018-04-18

Family

ID=60451288

Family Applications (1)

Application Number Title Priority Date Filing Date
EP15833669.3A Withdrawn EP3183347A4 (fr) 2015-10-17 2015-10-17 Oligonucléotides modulateurs d'épissage et leurs procédés d'utilisation

Country Status (2)

Country Link
EP (1) EP3183347A4 (fr)
WO (1) WO2016027168A2 (fr)

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JP5645840B2 (ja) 2008-12-02 2014-12-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ リン原子修飾核酸の合成方法
WO2011005761A1 (fr) 2009-07-06 2011-01-13 Ontorii, Inc Nouveaux précurseurs d'acide nucléique et leurs méthodes d'utilisation
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP2872485B1 (fr) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Groupe auxiliaire asymétrique
RU2677639C2 (ru) 2012-07-13 2019-01-18 Шин Ниппон Биомедикал Лэбораториз, Лтд. Хиральный адъювант нуклеиновой кислоты
WO2014012081A2 (fr) 2012-07-13 2014-01-16 Ontorii, Inc. Contrôle chiral
EP3095461A4 (fr) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Adjuvant d'acide nucléique chiral possédant une activité d'induction d'immunité, et activateur d'induction d'immunité
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
WO2015108048A1 (fr) 2014-01-15 2015-07-23 株式会社新日本科学 Adjuvant d'acide nucléique chiral ayant un effet antitumoral et agent antitumoral
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3201339A4 (fr) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Augmentation ciblée de la production de gènes nucléaires
US10196639B2 (en) 2015-10-09 2019-02-05 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
WO2017106377A1 (fr) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Oligomères antisens destinés au traitement du retard mental dominant autosomique 5 et du syndrome de dravet
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
GB2587457B (en) * 2017-08-04 2022-06-01 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
PL3673080T3 (pl) 2017-08-25 2024-03-11 Stoke Therapeutics, Inc. Oligomery antysensowne do leczenia stanów i chorób
BR112020022512A2 (pt) 2018-05-04 2021-05-04 Stoke Therapeutics, Inc. métodos e composições para tratamento de doença de armazenamento de éster de colesteril
JP2023525799A (ja) 2020-05-11 2023-06-19 ストーク セラピューティクス，インク． 状態及び疾患の治療のためのｏｐａ１アンチセンスオリゴマー

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US4902505A (en) 1986-07-30 1990-02-20 Alkermes Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US5166320A (en) 1987-04-22 1992-11-24 University Of Connecticut Carrier system and method for the introduction of genes into mammalian cells
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5538848A (en) 1994-11-16 1996-07-23 Applied Biosystems Division, Perkin-Elmer Corp. Method for detecting nucleic acid amplification using self-quenching fluorescence probe
AU4966997A (en) 1996-11-18 1998-06-10 Takeshi Imanishi Novel nucleotide analogues
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
US20050112633A1 (en) * 2003-09-17 2005-05-26 Armour Christopher D. Alternatively spliced isoforms of sodium channel, voltage gated, type VIII, alpha (SCN8A)

Non-Patent Citations (4)

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Title
JOHN BAUMAN ET AL: "Therapeutic Potential of Splice-Switching Oligonucleotides", OLIGONUCLEOTIDES, vol. 19, no. 1, 1 March 2009 (2009-03-01), pages 1 - 13, XP055053156, ISSN: 1545-4576, DOI: 10.1089/oli.2008.0161 *
KAVITHA SIVA ET AL: "Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases", NUCLEIC ACID THERAPEUTICS, vol. 24, no. 1, 1 February 2014 (2014-02-01), US, pages 69 - 86, XP055327023, ISSN: 2159-3337, DOI: 10.1089/nat.2013.0461 *
MEGAN OLIVA ET AL: "Sodium channels and the neurobiology of epilepsy : Sodium Channels and Epilepsy", EPILEPSIA, vol. 53, no. 11, 20 August 2012 (2012-08-20), NEW YORK, US, pages 1849 - 1859, XP055449109, ISSN: 0013-9580, DOI: 10.1111/j.1528-1167.2012.03631.x *
See also references of WO2016027168A2 *

Also Published As

Publication number Publication date
EP3183347A2 (fr) 2017-06-28
WO2016027168A3 (fr) 2016-04-21
WO2016027168A2 (fr) 2016-02-25

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