AU2021261374A1 - Compositions and methods using snRNA components - Google Patents

Compositions and methods using snRNA components Download PDF

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Publication number
AU2021261374A1
AU2021261374A1 AU2021261374A AU2021261374A AU2021261374A1 AU 2021261374 A1 AU2021261374 A1 AU 2021261374A1 AU 2021261374 A AU2021261374 A AU 2021261374A AU 2021261374 A AU2021261374 A AU 2021261374A AU 2021261374 A1 AU2021261374 A1 AU 2021261374A1
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Australia
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bases
engineered polynucleotide
nucleotides
rna
engineered
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Pending
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AU2021261374A
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English (en)
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Susan BYRNE
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Shape Therapeutics Inc
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Shape Therapeutics Inc
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Publication of AU2021261374A1 publication Critical patent/AU2021261374A1/en
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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
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • 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
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • 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
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses

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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)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Steroid Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AU2021261374A 2020-04-22 2021-04-22 Compositions and methods using snRNA components Pending AU2021261374A1 (en)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US202063013774P 2020-04-22 2020-04-22
US63/013,774 2020-04-22
US202063030118P 2020-05-26 2020-05-26
US63/030,118 2020-05-26
US202063086434P 2020-10-01 2020-10-01
US63/086,434 2020-10-01
US202063112486P 2020-11-11 2020-11-11
US63/112,486 2020-11-11
US202063119878P 2020-12-01 2020-12-01
US63/119,878 2020-12-01
US202163153817P 2021-02-25 2021-02-25
US63/153,817 2021-02-25
PCT/US2021/028618 WO2021216853A1 (en) 2020-04-22 2021-04-22 Compositions and methods using snrna components

Publications (1)

Publication Number Publication Date
AU2021261374A1 true AU2021261374A1 (en) 2022-10-13

Family

ID=78270014

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021261374A Pending AU2021261374A1 (en) 2020-04-22 2021-04-22 Compositions and methods using snRNA components

Country Status (7)

Country Link
US (1) US20240011028A9 (https=)
EP (1) EP4139453A1 (https=)
JP (1) JP2023523237A (https=)
CN (1) CN115777020A (https=)
AU (1) AU2021261374A1 (https=)
CA (1) CA3173012A1 (https=)
WO (1) WO2021216853A1 (https=)

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GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
WO2021113270A1 (en) 2019-12-02 2021-06-10 Shape Therapeutics Inc. Therapeutic editing
MX2022006991A (es) * 2019-12-09 2022-08-25 Astellas Pharma Inc Arn guía antisentido con región funcional añadida para editar arn blanco.
WO2022103852A1 (en) * 2020-11-11 2022-05-19 Shape Therapeutics Inc. Rna-editing compositions and methods of use
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
US20250084408A1 (en) * 2021-10-27 2025-03-13 Shape Therapeutics Inc. Engineered rnas
EP4441219A2 (en) * 2021-12-01 2024-10-09 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides for rna editing targeting lrrk2
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
JP2025507958A (ja) 2022-03-04 2025-03-21 ロックアネイビオ, インコーポレイテッド 操作された核内低分子RNA(snRNA)を含む組成物および方法
WO2023183825A2 (en) * 2022-03-21 2023-09-28 Ptc Therapeutics Gt, Inc. Gene therapy of ush2a-associated diseases
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
EP4555086A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucleotides for adar-mediated rna editing and use thereof
CN120112640A (zh) * 2022-08-24 2025-06-06 塑造治疗公司 用于提高rna有效载荷转录的工程化构建体
CN120129747A (zh) * 2022-10-11 2025-06-10 广州瑞风生物科技有限公司 snRNA核酸分子及其应用
WO2024081411A1 (en) * 2022-10-13 2024-04-18 Shape Therapeutics Inc. Engineered constructs for enhanced stability or localization of rna payloads
AU2023364059A1 (en) * 2022-10-18 2025-05-08 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising programmable snrnas for rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
AR131145A1 (es) 2022-11-24 2025-02-19 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de hemocromatosis hfe hereditaria
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CA3276262A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
WO2024137991A2 (en) * 2022-12-23 2024-06-27 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
JP2026504808A (ja) * 2023-01-18 2026-02-10 アプター バイオ,インク. 神経変性疾患の処置のためのポリヌクレオチド組成物および方法
EP4652278A1 (en) * 2023-01-18 2025-11-26 Aptah Bio, Inc. Polynucleotide compositions and methods for treatment of cancer
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
EP4669753A1 (en) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
WO2024206175A1 (en) 2023-03-24 2024-10-03 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
AU2024246572A1 (en) 2023-03-27 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024226860A2 (en) * 2023-04-26 2024-10-31 Shape Therapeutics Inc. Small nuclear rna processing hairpins for small rna payloads
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WO2025190385A1 (zh) * 2024-03-14 2025-09-18 上海拓界生物医药科技有限公司 靶向LRRK2的RNAi剂及其医药用途
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Also Published As

Publication number Publication date
EP4139453A1 (en) 2023-03-01
WO2021216853A1 (en) 2021-10-28
CA3173012A1 (en) 2021-10-28
JP2023523237A (ja) 2023-06-02
US20240011028A9 (en) 2024-01-11
US20230203483A1 (en) 2023-06-29
CN115777020A (zh) 2023-03-10

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