JP7693552B2 - アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法 - Google Patents

アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法 Download PDF

Info

Publication number
JP7693552B2
JP7693552B2 JP2021546894A JP2021546894A JP7693552B2 JP 7693552 B2 JP7693552 B2 JP 7693552B2 JP 2021546894 A JP2021546894 A JP 2021546894A JP 2021546894 A JP2021546894 A JP 2021546894A JP 7693552 B2 JP7693552 B2 JP 7693552B2
Authority
JP
Japan
Prior art keywords
cas9
domain
sequence
protein
target
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2021546894A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022520081A5 (https=
JPWO2020168132A5 (https=
JP2022520081A (ja
Inventor
ニコル ゴーデッリ、
マイケル パッカー、
イアン スレイメイカー、
イ ユ、
ベルンド ゼッツェ、
ジェイソン マイケル ゲールケ、
ナタリー ペトロシャン、
アンジェリカ メッサーナ、
イボンヌ アラティン、
フランシーヌ グレゴワール、
ジェネシス ラング、
シャウナ バーコヴィッチ、
デイビッド エー． ボーン、
スン―ジュ イ、
Original Assignee
ビーム セラピューティクス インク．
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 ビーム セラピューティクス インク． filed Critical ビーム セラピューティクス インク．
Publication of JP2022520081A publication Critical patent/JP2022520081A/ja
Publication of JP2022520081A5 publication Critical patent/JP2022520081A5/ja
Publication of JPWO2020168132A5 publication Critical patent/JPWO2020168132A5/ja
Priority to JP2025093886A priority Critical patent/JP2025143279A/ja
Application granted granted Critical
Publication of JP7693552B2 publication Critical patent/JP7693552B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/11—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
    • 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)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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)
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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]
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Plant Substances (AREA)
JP2021546894A 2019-02-13 2020-02-13 アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法 Active JP7693552B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025093886A JP2025143279A (ja) 2019-02-13 2025-06-05 アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法

Applications Claiming Priority (33)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US201962805238P 2019-02-13 2019-02-13
US201962805277P 2019-02-13 2019-02-13
US62/805,277 2019-02-13
US62/805,238 2019-02-13
US62/805,271 2019-02-13
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US62/852,228 2019-05-23
US62/852,224 2019-05-23
US201962873140P 2019-07-11 2019-07-11
US201962873144P 2019-07-11 2019-07-11
US201962873138P 2019-07-11 2019-07-11
US62/873,138 2019-07-11
US62/873,140 2019-07-11
US62/873,144 2019-07-11
US201962876354P 2019-07-19 2019-07-19
US62/876,354 2019-07-19
US201962888867P 2019-08-19 2019-08-19
US62/888,867 2019-08-19
US201962912992P 2019-10-09 2019-10-09
US62/912,992 2019-10-09
US201962931747P 2019-11-06 2019-11-06
US201962931722P 2019-11-06 2019-11-06
US62/931,722 2019-11-06
US62/931,747 2019-11-06
US201962941523P 2019-11-27 2019-11-27
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
US62/941,523 2019-11-27
US202062966526P 2020-01-27 2020-01-27
US62/966,526 2020-01-27
PCT/US2020/018192 WO2020168132A1 (en) 2019-02-13 2020-02-13 Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025093886A Division JP2025143279A (ja) 2019-02-13 2025-06-05 アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法

Publications (4)

Publication Number Publication Date
JP2022520081A JP2022520081A (ja) 2022-03-28
JP2022520081A5 JP2022520081A5 (https=) 2023-02-17
JPWO2020168132A5 JPWO2020168132A5 (https=) 2023-02-17
JP7693552B2 true JP7693552B2 (ja) 2025-06-17

Family

ID=72043868

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021546894A Active JP7693552B2 (ja) 2019-02-13 2020-02-13 アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法
JP2025093886A Pending JP2025143279A (ja) 2019-02-13 2025-06-05 アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025093886A Pending JP2025143279A (ja) 2019-02-13 2025-06-05 アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法

Country Status (8)

Country Link
US (3) US20220170027A1 (https=)
EP (1) EP3924479A4 (https=)
JP (2) JP7693552B2 (https=)
KR (1) KR20210139265A (https=)
CN (1) CN114072496A (https=)
AU (1) AU2020221366A1 (https=)
CA (1) CA3129158A1 (https=)
WO (1) WO2020168132A1 (https=)

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3765615T3 (pl) 2018-03-14 2023-11-13 Arbor Biotechnologies, Inc. Nowe enzymy i układy crispr ukierunkowane na dna
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
US12522812B2 (en) 2018-05-16 2026-01-13 Arbor Biotechnologies, Inc. CRISPR-associated systems and components
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
KR20210139265A (ko) * 2019-02-13 2021-11-22 빔 테라퓨틱스, 인크. 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기 및 이의 사용 방법
US20220235347A1 (en) 2019-02-13 2022-07-28 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
US12569438B2 (en) 2019-07-29 2026-03-10 Georgia Tech Research Corporation Nanomaterials containing constrained lipids and uses thereof
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
US12435035B2 (en) 2020-01-09 2025-10-07 Guide Therapeutics, Llc Nanomaterials
CA3170326A1 (en) * 2020-02-13 2021-08-19 Beam Therapeutics Inc. Compositions and methods for engraftment of base edited cells
KR20230009379A (ko) * 2020-03-31 2023-01-17 아버 바이오테크놀로지스, 인크. Cas12i2 변이체 폴리펩타이드를 포함하는 조성물 및 이의 용도
CA3194019A1 (en) * 2020-09-04 2022-03-10 National University Corporation Kobe University Miniaturized cytidine deaminase-containing complex for modifying double-stranded dna
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20240018550A1 (en) * 2020-12-01 2024-01-18 Iucf-Hyu (Industry-University Cooperation Foundation Hanyang University) Adenine base editor having increased thymine-cytosine sequence-specific cytosine editing activity, and use thereof
WO2022119294A1 (ko) * 2020-12-01 2022-06-09 한양대학교 산학협력단 사이토신 교정 활성이 제거된 아데닌 염기교정 유전자가위 및 이의 용도
KR20230131863A (ko) 2021-01-20 2023-09-14 빔 테라퓨틱스, 인크. 나노물질
IL303845A (en) 2021-01-20 2023-08-01 Beam Therapeutics Inc Nanomaterials include a degradable property
CN115704015B (zh) * 2021-08-12 2025-08-12 清华大学 基于腺嘌呤和胞嘧啶双碱基编辑器的靶向诱变系统
CN115772512B (zh) * 2021-09-07 2025-12-23 华东师范大学 腺嘌呤脱氨酶、包含其的腺嘌呤碱基编辑器及其应用
WO2023052366A1 (en) 2021-09-28 2023-04-06 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-hemoglobinopathies
CN119286831A (zh) * 2021-10-21 2025-01-10 复旦大学 碱基编辑器及其构建方法与应用
WO2023099591A1 (en) 2021-12-01 2023-06-08 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer
EP4452925A1 (en) 2021-12-20 2024-10-30 Beam Therapeutics Inc. Nanomaterial comprising diamines
EP4452942A1 (en) 2021-12-20 2024-10-30 Beam Therapeutics Inc. Nanomaterials comprising disulfides
EP4452930A1 (en) 2021-12-20 2024-10-30 Beam Therapeutics Inc. Ionizable amine and ester lipids and lipid nanoparticles
CA3241488A1 (en) 2021-12-20 2023-06-29 Beam Therapeutics Inc. Ionizable amine lipids and lipid nanoparticles
WO2023121971A1 (en) 2021-12-20 2023-06-29 Beam Therapeutics Inc. Nanomaterials comprising tetravalent lipid compounds
US20250161492A1 (en) 2022-01-25 2025-05-22 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of beta-thalassemia
WO2023169454A1 (zh) * 2022-03-08 2023-09-14 中国科学院遗传与发育生物学研究所 腺嘌呤脱氨酶及其在碱基编辑中的用途
CN117925585A (zh) * 2022-04-07 2024-04-26 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
CN120456933A (zh) 2022-04-28 2025-08-08 布罗德研究所股份有限公司 编码碱基编辑器的aav载体及其用途
CN114686456B (zh) * 2022-05-10 2023-02-17 中山大学 基于双分子脱氨酶互补的碱基编辑系统及其应用
EP4522647A1 (en) 2022-05-10 2025-03-19 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from beta-thalassemia
WO2023217904A1 (en) 2022-05-10 2023-11-16 Institut National de la Santé et de la Recherche Médicale Syncitin-1 fusion proteins and uses thereof for cargo delivery into target cells
CN119923466A (zh) * 2022-06-15 2025-05-02 比姆医疗股份有限公司 用于减少补体激活的组合物和方法
EP4543469A2 (en) * 2022-06-27 2025-04-30 Beam Therapeutics Inc. Compositions and methods for non-genotoxic cell conditioning
WO2024019936A1 (en) 2022-07-20 2024-01-25 Beam Therapeutics Inc. Nanomaterials comprising triols
EP4558633A1 (en) 2022-07-22 2025-05-28 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from beta-thalassemia
WO2024086596A1 (en) * 2022-10-18 2024-04-25 4M Genomics Inc. Polypeptide fusions or conjugates for gene editing
JP2026506554A (ja) 2023-02-06 2026-02-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 多重塩基編集による遺伝子改変造血幹細胞の濃縮
EP4705461A2 (en) * 2023-05-03 2026-03-11 Beam Therapeutics Inc. Compositions and methods for treating stargardt disease
WO2024230760A1 (zh) * 2023-05-09 2024-11-14 北京齐禾生科生物科技有限公司 一种可作用于dna的腺苷脱氨酶及其应用
CN118871578A (zh) 2023-05-24 2024-10-29 锐正基因(苏州)有限公司 用于碱基编辑的脱氨酶及其变体
WO2025038821A1 (en) * 2023-08-16 2025-02-20 Chroma Medicine, Inc. Methods and compositions for multiplex epigenetic editing
WO2025059520A1 (en) * 2023-09-15 2025-03-20 Beam Therapeutics Inc. Compositions and methods for base editing a phenylalanine hydroxylase polynucleotide
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
CN120290534A (zh) * 2024-01-10 2025-07-11 上海数因信科智能科技有限公司 腺嘌呤脱氨酶、包含其的碱基编辑器融合蛋白和碱基编辑器系统及其应用
CN117568313B (zh) * 2024-01-15 2024-04-26 上海贝斯昂科生物科技有限公司 基因编辑组合物及其用途
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025202473A1 (en) * 2024-03-28 2025-10-02 Revvity Discovery Limited A nucleic acid deaminase, a base editor and uses thereof
US20250332260A1 (en) 2024-04-10 2025-10-30 Garuda Therapeutics, Inc. Immune compatible cells for allogeneic cell therapies to cover global, ethnic, or disease-specific populations
WO2025228273A1 (zh) * 2024-04-28 2025-11-06 广州瑞风生物科技有限公司 脱氨酶、单碱基编辑器及其应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
CA2982966C (en) * 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
WO2017048969A1 (en) * 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
WO2018089664A1 (en) * 2016-11-11 2018-05-17 The Regents Of The University Of California Variant rna-guided polypeptides and methods of use
EP3625342B1 (en) * 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US11168322B2 (en) * 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US11946040B2 (en) * 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
US20220235347A1 (en) * 2019-02-13 2022-07-28 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
KR20210139265A (ko) * 2019-02-13 2021-11-22 빔 테라퓨틱스, 인크. 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기 및 이의 사용 방법
KR20210126680A (ko) * 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법
US20220387622A1 (en) * 2019-05-21 2022-12-08 Beam Therapeutics Inc. Methods of editing a single nucleotide polymorphism using programmable base editor systems

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof

Also Published As

Publication number Publication date
EP3924479A4 (en) 2023-01-25
CN114072496A (zh) 2022-02-18
US20220170027A1 (en) 2022-06-02
AU2020221366A1 (en) 2021-08-26
EP3924479A1 (en) 2021-12-22
US11155803B2 (en) 2021-10-26
US20210130805A1 (en) 2021-05-06
JP2022520081A (ja) 2022-03-28
WO2020168132A1 (en) 2020-08-20
JP2025143279A (ja) 2025-10-01
CA3129158A1 (en) 2020-08-20
KR20210139265A (ko) 2021-11-22
WO2020168132A9 (en) 2020-10-08
US20240132867A1 (en) 2024-04-25

Similar Documents

Publication Publication Date Title
JP7693552B2 (ja) アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法
JP7779988B2 (ja) 低減されたオフターゲット脱アミノ化を有する核酸塩基エディターおよび核酸塩基標的配列を改変するためのその使用方法
JP7753096B2 (ja) 遺伝子疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いた疾患関連遺伝子のスプライスアクセプター部位破壊
JP7586601B2 (ja) 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法
AU2019316094B2 (en) Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
JP7558929B2 (ja) プログラム可能塩基エディターシステムを用いて病原性変異を抑制する方法
JP7596259B2 (ja) プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法
JP7781742B2 (ja) 転写または発現を可能にするように変異を編集するための組成物および方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230209

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230209

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240220

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240520

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240716

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241016

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241203

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250303

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250507

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250605

R150 Certificate of patent or registration of utility model

Ref document number: 7693552

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150