CA3222212A1 - Compositions and methods for delivering therapeutic oligonucleotides to the central nervous system - Google Patents

Compositions and methods for delivering therapeutic oligonucleotides to the central nervous system Download PDF

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Publication number
CA3222212A1
CA3222212A1 CA3222212A CA3222212A CA3222212A1 CA 3222212 A1 CA3222212 A1 CA 3222212A1 CA 3222212 A CA3222212 A CA 3222212A CA 3222212 A CA3222212 A CA 3222212A CA 3222212 A1 CA3222212 A1 CA 3222212A1
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CA
Canada
Prior art keywords
formula
ribonucleoside
internucleoside linkage
represented
sirna molecule
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.)
Pending
Application number
CA3222212A
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English (en)
French (fr)
Inventor
Matthew Hassler
Garth A. Kinberger
Bruno Miguel Da Cruz Godinho
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.)
Atalanta Therapeutics Inc
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Atalanta Therapeutics Inc
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Application filed by Atalanta Therapeutics Inc filed Critical Atalanta Therapeutics Inc
Publication of CA3222212A1 publication Critical patent/CA3222212A1/en
Pending legal-status Critical Current

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    • 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
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/52—Physical structure branched
    • C—CHEMISTRY; METALLURGY
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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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    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
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  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3222212A 2021-06-02 2022-06-02 Compositions and methods for delivering therapeutic oligonucleotides to the central nervous system Pending CA3222212A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163195993P 2021-06-02 2021-06-02
US63/195,993 2021-06-02
PCT/US2022/032014 WO2022256565A2 (en) 2021-06-02 2022-06-02 Compositions and methods for delivering therapeutic oligonucleotides to the central nervous system

Publications (1)

Publication Number Publication Date
CA3222212A1 true CA3222212A1 (en) 2022-12-08

Family

ID=84324589

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3222212A Pending CA3222212A1 (en) 2021-06-02 2022-06-02 Compositions and methods for delivering therapeutic oligonucleotides to the central nervous system

Country Status (9)

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US (1) US20240287526A1 (enExample)
EP (1) EP4346845A4 (enExample)
JP (1) JP2024521897A (enExample)
KR (1) KR20240038930A (enExample)
CN (1) CN117795069A (enExample)
AU (1) AU2022286420A1 (enExample)
CA (1) CA3222212A1 (enExample)
IL (1) IL308994A (enExample)
WO (1) WO2022256565A2 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024073595A2 (en) * 2022-09-28 2024-04-04 Atalanta Therapeutics, Inc. Compositions and methods for treatment of huntington's disease
CN118202055A (zh) * 2023-02-17 2024-06-14 苏州时安生物技术有限公司 一种抑制SCN9A基因表达的siRNA、其药物组合物及用途
CN121241143A (zh) * 2023-03-23 2025-12-30 阿塔兰塔治疗公司 用于改善的基因沉默的组合物和方法
WO2025111497A1 (en) * 2023-11-21 2025-05-30 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating acute activation
WO2025193786A1 (en) * 2024-03-12 2025-09-18 University Of Massachusetts Oligonucleotides for prnp modulation

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004042024A2 (en) * 2002-11-01 2004-05-21 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF HIF-1 ALPHA
WO2011017297A2 (en) * 2009-08-03 2011-02-10 The University Of North Carolina At Chapel Hill Biodegradable delivery system complexes for the delivery of bioactive compounds
JP5775581B2 (ja) * 2010-08-20 2015-09-09 レプリコール インコーポレーティッド オリゴヌクレオチドキレート錯体
CN102659495B (zh) * 2012-04-25 2014-02-26 天津大学 利用寡核苷酸拆分手性药物的方法
RU2668136C2 (ru) * 2012-05-10 2018-09-26 Эйдинкс, Инк. Композиции для доставки активных ингредиентов
AR091065A1 (es) * 2012-05-18 2014-12-30 Replicor Inc Una formulacion farmaceutica que comprende un quelato de oligonucleotidos antiviral para el tratamiento de una infeccion antiviral
WO2015125147A1 (en) * 2014-02-20 2015-08-27 B. G. Negev Technologies And Applications Ltd Anionic polyplexes for use in the delivery of nucleic acids
WO2017132669A1 (en) * 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
US20200339982A1 (en) * 2017-12-22 2020-10-29 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorodithioate internucleoside linkage

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Publication number Publication date
AU2022286420A1 (en) 2024-01-04
IL308994A (en) 2024-01-01
EP4346845A2 (en) 2024-04-10
KR20240038930A (ko) 2024-03-26
WO2022256565A3 (en) 2023-01-05
JP2024521897A (ja) 2024-06-04
WO2022256565A2 (en) 2022-12-08
EP4346845A4 (en) 2026-07-15
US20240287526A1 (en) 2024-08-29
CN117795069A (zh) 2024-03-29

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