KR20220019685A - B형 간염 치료를 위한 조성물 및 방법 - Google Patents

B형 간염 치료를 위한 조성물 및 방법 Download PDF

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KR20220019685A
KR20220019685A KR1020217039921A KR20217039921A KR20220019685A KR 20220019685 A KR20220019685 A KR 20220019685A KR 1020217039921 A KR1020217039921 A KR 1020217039921A KR 20217039921 A KR20217039921 A KR 20217039921A KR 20220019685 A KR20220019685 A KR 20220019685A
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hbv
tada
cas9
protein
nucleic acid
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Korean (ko)
Inventor
엘레나 스메칼로바
마이클 페커
프랑신 그레고아르
루이스 바레라
주세페 키아라멜라
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빔 테라퓨틱스, 인크.
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • 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
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • 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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    • 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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    • C12N15/09—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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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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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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    • 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)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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]
    • C—CHEMISTRY; METALLURGY
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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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  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020217039921A 2019-05-10 2020-05-08 B형 간염 치료를 위한 조성물 및 방법 Pending KR20220019685A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
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

Publications (1)

Publication Number Publication Date
KR20220019685A true KR20220019685A (ko) 2022-02-17

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KR1020217039921A Pending KR20220019685A (ko) 2019-05-10 2020-05-08 B형 간염 치료를 위한 조성물 및 방법

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Country Link
US (1) US20230070861A1 (enExample)
EP (1) EP3965832A4 (enExample)
JP (2) JP7679309B2 (enExample)
KR (1) KR20220019685A (enExample)
CN (2) CN114929287B (enExample)
AU (1) AU2020276218A1 (enExample)
CA (1) CA3139324A1 (enExample)
WO (1) WO2020231863A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021222318A1 (en) 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
US12171813B2 (en) 2021-02-05 2024-12-24 Christiana Care Gene Editing Institute, 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
KR20240111821A (ko) * 2021-09-27 2024-07-17 빔 테라퓨틱스, 인크. B형 간염 바이러스 감염을 치료하기 위한 조성물 및 방법
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
TW202436622A (zh) * 2023-03-06 2024-09-16 美商英特利亞醫療公司 用於ｂ型肝炎病毒（ｈｂｖ）基因體編輯之組合物及方法
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112015031608A2 (pt) * 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
SG10201804975PA (en) * 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
EP3365447A1 (en) * 2015-10-21 2018-08-29 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating hepatitis b virus
GB2568182A (en) * 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
CN110520163A (zh) * 2017-01-05 2019-11-29 新泽西鲁特格斯州立大学 独立于dna双链断裂的靶向基因编辑平台及其用途
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
JP2020534795A (ja) * 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物

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US20230070861A1 (en) 2023-03-09
EP3965832A4 (en) 2024-02-07
JP2022532139A (ja) 2022-07-13
CN114929287A (zh) 2022-08-19
WO2020231863A1 (en) 2020-11-19
AU2020276218A1 (en) 2021-12-02
JP2025102768A (ja) 2025-07-08
JP7679309B2 (ja) 2025-05-19
CN114929287B (zh) 2025-06-03
EP3965832A1 (en) 2022-03-16
CA3139324A1 (en) 2020-11-19
CN120586100A (zh) 2025-09-05

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