JP7679309B2 - Ｂ型肝炎を治療するための組成物及び方法 - Google Patents

Ｂ型肝炎を治療するための組成物及び方法 Download PDF

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JP7679309B2
JP7679309B2 JP2021566276A JP2021566276A JP7679309B2 JP 7679309 B2 JP7679309 B2 JP 7679309B2 JP 2021566276 A JP2021566276 A JP 2021566276A JP 2021566276 A JP2021566276 A JP 2021566276A JP 7679309 B2 JP7679309 B2 JP 7679309B2
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hbv
cas9
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protein
gene
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JPWO2020231863A5 (enExample
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エレナ スメカロヴァ、
マイケル パッカー、
フランシーヌ グレゴワール、
ルイス バレラ、
ジュゼッペ チャラメラ、
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ビーム セラピューティクス インク．
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    • 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
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/1131—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 viruses
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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
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
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    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)

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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021566276A 2019-05-10 2020-05-08 Ｂ型肝炎を治療するための組成物及び方法 Active JP7679309B2 (ja)

Priority Applications (1)

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JP2025033734A JP2025102768A (ja) 2019-05-10 2025-03-04 Ｂ型肝炎を治療するための組成物及び方法

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US201962846422P 2019-05-10 2019-05-10
US62/846,422 2019-05-10
US201962927585P 2019-10-29 2019-10-29
US62/927,585 2019-10-29
PCT/US2020/032226 WO2020231863A1 (en) 2019-05-10 2020-05-08 Compositions and methods for treating hepatitis b

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JP2022532139A JP2022532139A (ja) 2022-07-13
JPWO2020231863A5 JPWO2020231863A5 (enExample) 2023-05-16
JP7679309B2 true JP7679309B2 (ja) 2025-05-19

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JP2025033734A Pending JP2025102768A (ja) 2019-05-10 2025-03-04 Ｂ型肝炎を治療するための組成物及び方法

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EP (1) EP3965832A4 (enExample)
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KR (1) KR20220019685A (enExample)
CN (2) CN120586100A (enExample)
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Families Citing this family (8)

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Publication number Priority date Publication date Assignee Title
US20230159913A1 (en) 2020-04-28 2023-05-25 The Broad Institute, Inc. Targeted base editing of the ush2a gene
WO2022170059A1 (en) 2021-02-05 2022-08-11 Christiana Care Health Services, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2022192634A1 (en) * 2021-03-12 2022-09-15 Massachusetts Institute Of Technology Compositions and methods for dominant antiviral therapy
KR20240087635A (ko) * 2021-07-22 2024-06-19 에멘도바이오 인코포레이티드 B형 간염 바이러스(hbv) 녹아웃
JP2024534639A (ja) * 2021-09-27 2024-09-20 ビーム セラピューティクス インク． Ｂ型肝炎ウイルス感染症を治療するための組成物及び方法
WO2024103049A1 (en) * 2022-11-11 2024-05-16 The United States Government As Represented By The Department Of Veterans Affairs Cd33 specific exosomes and uses thereof
WO2024186890A1 (en) * 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression

Citations (2)

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JP2017527256A (ja) 2013-12-12 2017-09-21 ザ・ブロード・インスティテュート・インコーポレイテッド ＨＢＶ及びウイルス性疾患及び障害のためのＣＲＩＳＰＲ−Ｃａｓ系及び組成物の送達、使用及び治療適用
WO2018129129A1 (en) 2017-01-05 2018-07-12 Rutgers, The State University Of New Jersey Targeted gene editing platform independent of dna double strand break and uses thereof

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CN107995927B (zh) * 2013-06-17 2021-07-30 布罗德研究所有限公司 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途
AU2016341919A1 (en) * 2015-10-21 2018-04-19 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating hepatitis b virus
KR20250103795A (ko) * 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2019005886A1 (en) * 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-CYTIDINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED EDITING OF NUCLEIC ACIDS
CN111801345A (zh) * 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017527256A (ja) 2013-12-12 2017-09-21 ザ・ブロード・インスティテュート・インコーポレイテッド ＨＢＶ及びウイルス性疾患及び障害のためのＣＲＩＳＰＲ−Ｃａｓ系及び組成物の送達、使用及び治療適用
WO2018129129A1 (en) 2017-01-05 2018-07-12 Rutgers, The State University Of New Jersey Targeted gene editing platform independent of dna double strand break and uses thereof

Non-Patent Citations (2)

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Title
A.Eid, et al.，Biochemical Journal，2018年，Vol.475，p.1955-1964
C.Peng, et al.，VIROLOGICA SINICA，2015年，Vol.30(5)，p.317-325

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EP3965832A4 (en) 2024-02-07
CA3139324A1 (en) 2020-11-19
EP3965832A1 (en) 2022-03-16
KR20220019685A (ko) 2022-02-17
CN114929287A (zh) 2022-08-19
CN114929287B (zh) 2025-06-03
JP2022532139A (ja) 2022-07-13
US20230070861A1 (en) 2023-03-09
AU2020276218A1 (en) 2021-12-02
CN120586100A (zh) 2025-09-05
WO2020231863A1 (en) 2020-11-19
JP2025102768A (ja) 2025-07-08

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