AU2022306377A1 - Compositions and methods for efficient genome editing - Google Patents

Compositions and methods for efficient genome editing Download PDF

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Publication number
AU2022306377A1
AU2022306377A1 AU2022306377A AU2022306377A AU2022306377A1 AU 2022306377 A1 AU2022306377 A1 AU 2022306377A1 AU 2022306377 A AU2022306377 A AU 2022306377A AU 2022306377 A AU2022306377 A AU 2022306377A AU 2022306377 A1 AU2022306377 A1 AU 2022306377A1
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AU
Australia
Prior art keywords
seq
sequence
prime editing
domain
editing composition
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Pending
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AU2022306377A
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English (en)
Inventor
Luis Barrera
Michael Packer
Holly A. REES
Ian SLAYMAKER
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Prime Medicine Inc
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Prime Medicine Inc
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Application filed by Prime Medicine Inc filed Critical Prime Medicine Inc
Publication of AU2022306377A1 publication Critical patent/AU2022306377A1/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
AU2022306377A 2021-07-06 2022-06-29 Compositions and methods for efficient genome editing Pending AU2022306377A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163218744P 2021-07-06 2021-07-06
US63/218,744 2021-07-06
US202163219623P 2021-07-08 2021-07-08
US63/219,623 2021-07-08
PCT/US2022/035613 WO2023283092A1 (en) 2021-07-06 2022-06-29 Compositions and methods for efficient genome editing

Publications (1)

Publication Number Publication Date
AU2022306377A1 true AU2022306377A1 (en) 2024-01-25

Family

ID=84800962

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2022306377A Pending AU2022306377A1 (en) 2021-07-06 2022-06-29 Compositions and methods for efficient genome editing

Country Status (6)

Country Link
US (1) US20240228988A1 (https=)
EP (1) EP4367227A4 (https=)
JP (1) JP2024525665A (https=)
AU (1) AU2022306377A1 (https=)
CA (1) CA3224970A1 (https=)
WO (1) WO2023283092A1 (https=)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
CA3174483A1 (en) 2020-03-04 2021-09-10 Flagship Pioneering Innovations Vi, Llc Improved methods and compositions for modulating a genome
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
JP2024533311A (ja) 2021-09-08 2024-09-12 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー ゲノムを調節するための方法及び組成物
KR20240099164A (ko) 2021-09-08 2024-06-28 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Pah-조절 조성물 및 방법
JP2024534945A (ja) * 2021-09-10 2024-09-26 アジレント・テクノロジーズ・インク 化学修飾を有するプライム編集のためのガイドｒｎａ
EP4444362A4 (en) 2021-12-10 2026-04-01 Flagship Pioneering Innovations Vi Llc CFTR COMPOSITIONS AND MODULATION METHODS
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
EP4665865A1 (en) 2023-02-17 2025-12-24 Anjarium Biosciences AG Methods of making dna molecules and compositions and uses thereof
WO2024178144A1 (en) * 2023-02-22 2024-08-29 Prime Medicine, Inc. Methods and compositions for editing nucleotide sequences
EP4720304A2 (en) * 2023-05-31 2026-04-08 University of Massachusetts Improved modular prime editing with modified effectors and templates
WO2024259051A1 (en) * 2023-06-14 2024-12-19 The Children's Medical Center Corporation Systems and methods for modifying a polynucleotide
WO2025038881A1 (en) * 2023-08-16 2025-02-20 Beam Therapeutics Inc. Prime editing of single base mutations in sickle cell disease
WO2025076306A1 (en) * 2023-10-06 2025-04-10 University Of Massachusetts Prime editors having improved prime editing efficiency
US20250354138A1 (en) 2024-03-15 2025-11-20 Beam Therapeutics Inc. Prime editing of single base mutations in alpha-1 antitrypsin deficiency
WO2025226946A1 (en) * 2024-04-24 2025-10-30 Cedric Francois Methods and compositions for the treatment of androgenic alopecia
WO2025231071A1 (en) * 2024-05-01 2025-11-06 Beam Therapeutics Inc. Compositions and methods for cell conditioning

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019186348A1 (en) * 2018-03-25 2019-10-03 GeneTether, Inc Modified nucleic acid editing systems for tethering donor dna
WO2020033083A1 (en) * 2018-08-10 2020-02-13 Cornell University Optimized base editors enable efficient editing in cells, organoids and mice
KR20210121113A (ko) * 2019-01-31 2021-10-07 빔 테라퓨틱스, 인크. 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석
CN116355966A (zh) * 2019-02-02 2023-06-30 上海科技大学 一种融合蛋白在遗传编辑的用途
JP7657726B2 (ja) * 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
WO2021042047A1 (en) * 2019-08-30 2021-03-04 The General Hospital Corporation C-to-g transversion dna base editors
EP4081635A4 (en) * 2019-12-26 2024-03-27 Agency for Science, Technology and Research Nucleobase editors

Also Published As

Publication number Publication date
US20240228988A1 (en) 2024-07-11
JP2024525665A (ja) 2024-07-12
EP4367227A4 (en) 2025-04-30
WO2023283092A1 (en) 2023-01-12
CA3224970A1 (en) 2023-01-12
EP4367227A1 (en) 2024-05-15

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