BR112022006205A2 - Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método - Google Patents

Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método

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Publication number
BR112022006205A2
BR112022006205A2 BR112022006205A BR112022006205A BR112022006205A2 BR 112022006205 A2 BR112022006205 A2 BR 112022006205A2 BR 112022006205 A BR112022006205 A BR 112022006205A BR 112022006205 A BR112022006205 A BR 112022006205A BR 112022006205 A2 BR112022006205 A2 BR 112022006205A2
Authority
BR
Brazil
Prior art keywords
oligonucleotide
composition
nucleic acid
target
disease
Prior art date
Application number
BR112022006205A
Other languages
English (en)
Portuguese (pt)
Inventor
Monian Prashant
Shakti Shivalila Chikdu
Marappan Subramanian
Vargeese Chandra
Kandasamy Pachamuthu
Lu Genliang
Yu Hui
Charles Donnell Butler David
Henrique Apponi Luciano
Shimizu Mamoru
Michelle Standley Stephany
John Boulay David
Guzior Hoss Andrew
Desai Jigar
David Godfrey Jack
Iwamoto Naoki
Yang Hailin
Original Assignee
Wave Life Sciences 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 Wave Life Sciences Ltd filed Critical Wave Life Sciences Ltd
Publication of BR112022006205A2 publication Critical patent/BR112022006205A2/pt

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    • 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
    • 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
    • CCHEMISTRY; METALLURGY
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    • 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
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • 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
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • 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
    • C12N2310/314Phosphoramidates
    • 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
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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
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
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    • C12N2310/33Chemical structure of the base
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen

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  • Health & Medical Sciences (AREA)
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  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112022006205A 2019-10-06 2020-10-06 Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método BR112022006205A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962911334P 2019-10-06 2019-10-06
US202062959917P 2020-01-11 2020-01-11
US202063022559P 2020-05-10 2020-05-10
US202063069696P 2020-08-24 2020-08-24
PCT/US2020/054436 WO2021071858A1 (en) 2019-10-06 2020-10-06 Oligonucleotide compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
BR112022006205A2 true BR112022006205A2 (pt) 2022-07-19

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Family Applications (1)

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BR112022006205A BR112022006205A2 (pt) 2019-10-06 2020-10-06 Oligonucleotídeo; composição farmacêutica; composição de oligonucleotídeo; fosforamidita; método de preparação de um oligonucleotídeo ou composição; método para caracterizar um oligonucleotídeo ou uma composição; método para modificar uma adenosina alvo em um ácido nucleico alvo; método para desaminar uma adenosina alvo em um ácido nucleico alvo; método para prevenção ou tratamento de uma afecção, distúrbio ou doença passível de uma mutação de g para a; e composto, oligonucleotídeo, composição ou método

Country Status (11)

Country Link
US (1) US20230220384A1 (https=)
EP (1) EP4022059A4 (https=)
JP (2) JP7766589B2 (https=)
KR (1) KR20220076508A (https=)
CN (1) CN114585737A (https=)
AU (1) AU2020363391A1 (https=)
BR (1) BR112022006205A2 (https=)
CA (1) CA3154768A1 (https=)
IL (1) IL291933A (https=)
MX (1) MX2022004101A (https=)
WO (1) WO2021071858A1 (https=)

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EP4022059A4 (en) 2023-11-01
JP2025165976A (ja) 2025-11-05
AU2020363391A1 (en) 2022-03-24
JP7766589B2 (ja) 2025-11-10
US20230220384A1 (en) 2023-07-13
CA3154768A1 (en) 2021-04-15
KR20220076508A (ko) 2022-06-08
IL291933A (en) 2022-06-01
JP2022551124A (ja) 2022-12-07
CN114585737A (zh) 2022-06-03
WO2021071858A1 (en) 2021-04-15
MX2022004101A (es) 2022-04-26
EP4022059A1 (en) 2022-07-06

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