AU2017388753A1 - Synthetic guide molecules, compositions and methods relating thereto - Google Patents

Synthetic guide molecules, compositions and methods relating thereto Download PDF

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Publication number
AU2017388753A1
AU2017388753A1 AU2017388753A AU2017388753A AU2017388753A1 AU 2017388753 A1 AU2017388753 A1 AU 2017388753A1 AU 2017388753 A AU2017388753 A AU 2017388753A AU 2017388753 A AU2017388753 A AU 2017388753A AU 2017388753 A1 AU2017388753 A1 AU 2017388753A1
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Australia
Prior art keywords
linkage
independently
integer
guide molecule
inclusive
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Abandoned
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AU2017388753A
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English (en)
Inventor
Karin Zemski BERRY
Stacy CAPEHART
Bruce Eaton
Cecilia Fernandez
Jim Heil
Hariharan JAYARAM
Stephanie KING
Sam SACCOMANO
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Editas Medicine Inc
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Editas Medicine Inc
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Application filed by Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of AU2017388753A1 publication Critical patent/AU2017388753A1/en
Priority to AU2024205204A priority Critical patent/AU2024205204A1/en
Abandoned legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/319Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-position
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
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    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/53Methods for regulating/modulating their activity reducing unwanted side-effects
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00Production
    • C12N2330/30Production chemically synthesised

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Hydrogenated Pyridines (AREA)
  • Saccharide Compounds (AREA)
AU2017388753A 2016-12-30 2017-12-29 Synthetic guide molecules, compositions and methods relating thereto Abandoned AU2017388753A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024205204A AU2024205204A1 (en) 2016-12-30 2024-07-30 Synthetic guide molecules, compositions and methods relating thereto

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662441046P 2016-12-30 2016-12-30
US62/441,046 2016-12-30
US201762492001P 2017-04-28 2017-04-28
US62/492,001 2017-04-28
PCT/US2017/069019 WO2018126176A1 (en) 2016-12-30 2017-12-29 Synthetic guide molecules, compositions and methods relating thereto

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2024205204A Division AU2024205204A1 (en) 2016-12-30 2024-07-30 Synthetic guide molecules, compositions and methods relating thereto

Publications (1)

Publication Number Publication Date
AU2017388753A1 true AU2017388753A1 (en) 2019-07-11

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
AU2017388753A Abandoned AU2017388753A1 (en) 2016-12-30 2017-12-29 Synthetic guide molecules, compositions and methods relating thereto
AU2024205204A Pending AU2024205204A1 (en) 2016-12-30 2024-07-30 Synthetic guide molecules, compositions and methods relating thereto

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2024205204A Pending AU2024205204A1 (en) 2016-12-30 2024-07-30 Synthetic guide molecules, compositions and methods relating thereto

Country Status (9)

Country Link
US (1) US20230111575A1 (enExample)
EP (1) EP3565895A1 (enExample)
JP (3) JP7167029B2 (enExample)
KR (2) KR102758180B1 (enExample)
CN (2) CN118345072A (enExample)
AU (2) AU2017388753A1 (enExample)
CA (1) CA3048434A1 (enExample)
MX (1) MX420023B (enExample)
WO (1) WO2018126176A1 (enExample)

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US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
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WO2016172727A1 (en) 2015-04-24 2016-10-27 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
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Also Published As

Publication number Publication date
JP2025016776A (ja) 2025-02-04
CN110249052A (zh) 2019-09-17
US20230111575A1 (en) 2023-04-13
KR102618864B1 (ko) 2024-01-02
CN110249052B (zh) 2024-04-12
JP2022173349A (ja) 2022-11-18
JP7167029B2 (ja) 2022-11-08
MX420023B (es) 2025-01-14
AU2024205204A1 (en) 2024-09-05
WO2018126176A1 (en) 2018-07-05
MX2019007750A (es) 2019-10-15
CN118345072A (zh) 2024-07-16
CA3048434A1 (en) 2018-07-05
KR20190110554A (ko) 2019-09-30
KR20230175330A (ko) 2023-12-29
JP2020503049A (ja) 2020-01-30
EP3565895A1 (en) 2019-11-13
KR102758180B1 (ko) 2025-01-23
JP7610559B2 (ja) 2025-01-08

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