JP7642531B2 - 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アミノ酸を置換する方法 - Google Patents

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アミノ酸を置換する方法 Download PDF

Info

Publication number
JP7642531B2
JP7642531B2 JP2021513763A JP2021513763A JP7642531B2 JP 7642531 B2 JP7642531 B2 JP 7642531B2 JP 2021513763 A JP2021513763 A JP 2021513763A JP 2021513763 A JP2021513763 A JP 2021513763A JP 7642531 B2 JP7642531 B2 JP 7642531B2
Authority
JP
Japan
Prior art keywords
amino acid
cas9
domain
polynucleotide
protein
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
JP2021513763A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021523737A5 (enExample
JP2021523737A (ja
Inventor
デイビッド ブライソン、
ジョン エヴァンス、
マイケル パッカー、
ヤンファン フ、
ニコル ゴーデッリ、
ジェイソン マイケル ゲールケ、
ジェイ． キース ジョン、
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 JP2021523737A publication Critical patent/JP2021523737A/ja
Publication of JP2021523737A5 publication Critical patent/JP2021523737A5/ja
Priority to JP2024183249A priority Critical patent/JP2025026840A/ja
Application granted granted Critical
Publication of JP7642531B2 publication Critical patent/JP7642531B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • 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
    • C12N15/87—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
    • 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
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2510/00—Genetically modified cells
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021513763A 2018-05-11 2019-05-11 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アミノ酸を置換する方法 Active JP7642531B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024183249A JP2025026840A (ja) 2018-05-11 2024-10-18 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アミノ酸を置換する方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862670539P 2018-05-11 2018-05-11
US201862670521P 2018-05-11 2018-05-11
US62/670,521 2018-05-11
US62/670,539 2018-05-11
US201862780890P 2018-12-17 2018-12-17
US62/780,890 2018-12-17
PCT/US2019/031897 WO2019217942A1 (en) 2018-05-11 2019-05-11 Methods of substituting pathogenic amino acids using programmable base editor systems

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024183249A Division JP2025026840A (ja) 2018-05-11 2024-10-18 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アミノ酸を置換する方法

Publications (3)

Publication Number Publication Date
JP2021523737A JP2021523737A (ja) 2021-09-09
JP2021523737A5 JP2021523737A5 (enExample) 2022-05-19
JP7642531B2 true JP7642531B2 (ja) 2025-03-10

Family

ID=68467606

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021513763A Active JP7642531B2 (ja) 2018-05-11 2019-05-11 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アミノ酸を置換する方法
JP2024183249A Pending JP2025026840A (ja) 2018-05-11 2024-10-18 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アミノ酸を置換する方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024183249A Pending JP2025026840A (ja) 2018-05-11 2024-10-18 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アミノ酸を置換する方法

Country Status (8)

Country Link
US (2) US12133884B2 (enExample)
EP (1) EP3799603A4 (enExample)
JP (2) JP7642531B2 (enExample)
KR (2) KR20250134703A (enExample)
CN (2) CN121555430A (enExample)
AU (2) AU2019265019B2 (enExample)
CA (1) CA3100019A1 (enExample)
WO (1) WO2019217942A1 (enExample)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
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
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN114096666B (zh) 2019-02-13 2025-03-18 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN119913134A (zh) * 2019-08-29 2025-05-02 比姆医疗股份有限公司 用于编辑突变以允许转录或表达的组合物和方法
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
KR20220116512A (ko) * 2019-12-17 2022-08-23 시그마-알드리치 컴퍼니., 엘엘씨 박테로이데스의 게놈편집
EP4093864A4 (en) 2020-01-24 2024-04-10 The General Hospital Corporation Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants
WO2021151085A2 (en) 2020-01-24 2021-07-29 The General Hospital Corporation Crispr-cas enzymes with enhanced on-target activity
CN116113692A (zh) * 2020-02-13 2023-05-12 比姆医疗股份有限公司 用于植入碱基编辑细胞的组合物和方法
EP4103705A4 (en) * 2020-02-14 2024-02-28 Ohio State Innovation Foundation Nucleobase editors and methods of use thereof
WO2021183504A1 (en) 2020-03-11 2021-09-16 North Carolina State University Compositions, methods, and systems for genome editing technology
WO2021222318A1 (en) 2020-04-28 2021-11-04 The Broad Institute, Inc. Targeted base editing of the ush2a gene
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
US20230279438A1 (en) * 2020-05-13 2023-09-07 Inserm (Institut National De La Santé Et La Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20240002834A1 (en) * 2020-12-01 2024-01-04 Iucf-Hyu (Industry-University Cooperation Foundation Hanyang University) Adenine base editor lacking cytosine editing activity and use thereof
EP4274902A4 (en) * 2021-01-05 2025-04-16 Revvity Discovery Limited Guide RNA designs and complexes for tracless V-CAS systems
US20240060088A1 (en) * 2021-01-05 2024-02-22 Horizon Discovery Limited Guide RNA Designs and Complexes for Type V Cas Systems
EP4314308A4 (en) * 2021-04-02 2026-05-20 Univ Shanghai Technology GENE THERAPY FOR THE TREATMENT OF BETA-HEMOGLOBINOPATHIES
CN117729931A (zh) * 2021-05-14 2024-03-19 比姆医疗股份有限公司 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法
JP7125727B1 (ja) * 2021-09-07 2022-08-25 国立大学法人千葉大学 核酸配列改変用組成物および核酸配列の標的部位を改変する方法
WO2023056139A1 (en) * 2021-10-01 2023-04-06 The Regents Of The University Of California Compositions and methods for treating a β-thalassemia disease
CN117187220A (zh) * 2022-03-08 2023-12-08 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
WO2024018056A1 (en) * 2022-07-22 2024-01-25 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 βeta-thalassemia
WO2025022290A1 (en) * 2023-07-21 2025-01-30 Crispr Therapeutics Ag Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene
CN120813698A (zh) * 2023-09-22 2025-10-17 北京大学现代农业研究院 改进的基因组编辑方法
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
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016528890A (ja) 2013-07-09 2016-09-23 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用

Family Cites Families (321)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH0329734A (ja) 1989-06-20 1991-02-07 Kubota Corp ラベルプリンタ
US7144731B2 (en) 1989-10-16 2006-12-05 Amgen Inc. SCF antibody compositions and methods of using the same
US20050276784A1 (en) 1990-08-27 2005-12-15 Solan-Kettering Institute For Cancer Research Ligand for the c-kit receptor and methods of use thereof
DE4205148A1 (de) 1991-05-25 1993-01-21 Boehringer Mannheim Gmbh Monoklonale antikoerper gegen c-kit
WO1994008039A1 (en) 1992-10-02 1994-04-14 Systemix, Inc. Method of enrichment for human hematopoietic stem cells using c-kit
US5821108A (en) 1995-04-07 1998-10-13 The Board Of Trustees Of The Leland Stanford Junior University Enrichment for a thymocyte subset having progenitor cell activity using c-kit as a selection marker
CN1173776C (zh) 1996-06-28 2004-11-03 卡钳技术有限公司 在微规模流体性设备里的高通过量的筛选分析系统
WO1999019500A1 (en) 1997-10-10 1999-04-22 The Government Of The United States Of America, Represented By The Secretary, Department Of Healt H And Human Services Complex of biotinylated viral vector and ligand for targeted gene delivery
AU4323599A (en) 1998-05-29 1999-12-13 Instituto Superiore Di Sanita Compositions and methods for use in affecting hematopoietic stem cell populations in mammals
EP1103814B1 (en) 1998-07-31 2005-12-21 Mitsubishi Chemical Corporation Method for assaying physiologically active component
US6989248B2 (en) 1999-05-06 2006-01-24 The Trustees Of Columbia University In The City Of New York Methods of use of compounds which inhibit the stem cell signaling pathway
JP2003514564A (ja) 1999-11-24 2003-04-22 エムシーエス マイクロ キャリア システムズ ゲーエムベーハー 核局在化シグナルまたはタンパク質導入領域の多量体を含むポリペプチド、および分子を細胞内へ移入するためのその使用法
US20030082155A1 (en) 1999-12-06 2003-05-01 Habener Joel F. Stem cells of the islets of langerhans and their use in treating diabetes mellitus
US7862810B2 (en) 2000-07-31 2011-01-04 New York Medical College Methods and compositions for the repair and/or regeneration of damaged myocardium
US20040003420A1 (en) 2000-11-10 2004-01-01 Ralf Kuhn Modified recombinase
US20050222030A1 (en) 2001-02-21 2005-10-06 Anthony Allison Modified annexin proteins and methods for preventing thrombosis
CA2439472A1 (en) 2001-02-27 2002-09-06 University Of Rochester Methods and compositions for modifying apolipoprotein b mrna editing
CN100516204C (zh) 2001-04-24 2009-07-22 味之素株式会社 干细胞及其分离方法
AU2002330714A1 (en) 2001-05-30 2003-01-02 Biomedical Center In silico screening for phenotype-associated expressed sequences
EP2277991A1 (en) 2001-08-21 2011-01-26 Ventana Medical Systems, Inc. Method and quantification assay for determining c-kit/SCF/pAKT status
US8021876B2 (en) 2001-11-15 2011-09-20 Children's Medical Center Corporation Methods of isolation, expansion and differentiation of fetal stem cells from chorionic villus, amniotic fluid, and placenta and therapeutic uses thereof
CN100455275C (zh) 2002-02-06 2009-01-28 祥丰医疗有限公司 包覆有可促进内皮细胞的黏附和分化的包衣的医疗装置
US20040161419A1 (en) 2002-04-19 2004-08-19 Strom Stephen C. Placental stem cells and uses thereof
AU2003241313A1 (en) 2002-04-23 2003-11-10 Roger Williams Hospital Compositions and methods for stem cell delivery
US20040005623A1 (en) 2002-06-10 2004-01-08 Longley B. Jack Method of determining tumor sensitivities to therapeutic drugs
AU2003245752A1 (en) 2002-06-28 2004-01-19 Bio Transplant, Inc. Process for promoting graft acceptance by depletion of hematopoietic stem cells
WO2004005531A2 (en) 2002-07-05 2004-01-15 Roger Williams Medical Center Targeting and tracking of cells to specific organs and tissues in vivo
US20050032122A1 (en) 2003-08-06 2005-02-10 Shiaw-Min Hwang Optimizing culture medium for CD34<+> hematopoietic cell expansion
US20050124896A1 (en) 2003-08-25 2005-06-09 Jacob Richter Method for protecting implantable sensors and protected implantable sensors
EA200600495A1 (ru) 2003-09-23 2006-10-27 Новартис Аг Комбинация ингибитора vegf рецептора с химиотерапевтическим агентом
TWI338714B (en) 2003-12-02 2011-03-11 Cathay General Hospital Method of isolation and enrichment of mesenchymal stem cells from amniotic fluid
EP1718735A1 (en) 2004-02-11 2006-11-08 Aldagen, Inc. Stem cell populations and methods of use
US20050187607A1 (en) 2004-02-20 2005-08-25 Akhtar Adil J. Drug delivery device
USRE46379E1 (en) 2004-04-08 2017-04-25 Cornell Research Foundation, Inc. Functional immunohistochemical cell cycle analysis as a prognostic indicator for cancer
WO2006031524A2 (en) 2004-09-10 2006-03-23 The Regents Of The Unversity Of Colorado Early detection of hemangiosarcoma and angiosarcoma
WO2006062946A2 (en) 2004-12-06 2006-06-15 University Of Florida Research Foundation, Inc. Incorporation of bone marrow derived stem cells in tumors
CA2593549C (en) 2005-01-07 2016-04-26 Wake Forest University Health Sciences Regeneration of pancreatic islets by amniotic fluid stem cell therapy
WO2006089048A2 (en) 2005-02-16 2006-08-24 Hemogenix, Inc. Methods of screening compounds to predict toxicity and residual proliferative and differentiation capacity of the lympho-hematopoietic system
US20060222634A1 (en) 2005-03-31 2006-10-05 Clarke Diana L Amnion-derived cell compositions, methods of making and uses thereof
CA2606081C (en) 2005-04-26 2013-09-17 Markus M. Heiss Combination of the application of antibodies for immunostimulation together with glucocorticoids
US20070041948A1 (en) 2005-07-20 2007-02-22 Seoul National University Industry Foundation Method for culturing and proliferating hematopoietic stem cells and progenitor cells using human endometrial cells
US20080274469A1 (en) 2005-10-21 2008-11-06 The Regents Of The University Of California C-Kit Oncogene Mutations in Melanoma
KR101353763B1 (ko) 2005-11-07 2014-01-21 에자이 알앤드디 매니지먼트 가부시키가이샤 혈관 신생 저해 물질과 ｃ―ｋｉｔ 키나아제 저해 물질의병용
US20090304625A1 (en) 2006-01-18 2009-12-10 Ahsan Husain Modulators of cardiac cell hypertrophy and hyperplasia
US20070254319A1 (en) 2006-04-07 2007-11-01 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Identification of a constitutively resistant cancer stem cell
TWI395754B (zh) 2006-04-24 2013-05-11 Amgen Inc 人類化之ｃ-ｋｉｔ抗體
US8568286B2 (en) 2006-06-14 2013-10-29 Cardiac Pacemakers, Inc. Methods to position therapeutic agents using a magnetic field
EP3255061B1 (en) 2006-11-03 2021-06-16 The Board of Trustees of the Leland Stanford Junior University Selective immunodepletion of endogenous stem cell niche for engraftment
WO2008115300A1 (en) 2006-12-01 2008-09-25 Apocell, Inc. C-kit phosphorylation in cancer
WO2008076143A1 (en) 2006-12-18 2008-06-26 Osi Pharmaceuticals, Inc. Combination of igfr inhibitor and anti-cancer agent
WO2008085951A2 (en) 2007-01-04 2008-07-17 Vivek Mittal Treatment of tumors by ablating bone marrow-derived endothelial progenitor cells
DE102007008650B4 (de) 2007-02-20 2012-06-06 Charité - Universitätsmedizin Berlin Zellen zur Therapie des Herzens
JP2010530214A (ja) 2007-03-01 2010-09-09 クライオ−セル インターナショナル インコーポレイテッド 子宮内膜細胞／月経血細胞の獲得、単離および凍結保存
US9200253B1 (en) 2007-08-06 2015-12-01 Anthrogenesis Corporation Method of producing erythrocytes
WO2009058983A2 (en) 2007-10-30 2009-05-07 University Of Louisville Research Foundation, Inc. Subpopulations of bone marrow-derived adherent stem cells and methods of use therefor
US20090169613A1 (en) 2007-11-09 2009-07-02 Reznik Boris N Targeting of tumor stem cells through selective silencing of boris expression
US20110262465A1 (en) 2008-10-22 2011-10-27 Genentech, Inc. Prostate stem cells and uses thereof
JP5923035B2 (ja) 2009-03-24 2016-05-24 バイオセプト インコーポレイティッド 細胞の捕捉および解析のデバイスおよび方法
WO2010136508A2 (en) 2009-05-28 2010-12-02 Glaxo Group Limited Stem cell targeting
EP2259065A1 (en) 2009-06-03 2010-12-08 Erasmus University Medical Center Rotterdam Methods, reagents and kits for flow cytometric immunophenotyping
WO2013166281A1 (en) 2012-05-02 2013-11-07 Cedars-Sinai Medical Center Bi-functional compositions for targeting cells to diseased tissues and methods of using same
US20110104787A1 (en) 2009-11-05 2011-05-05 President And Fellows Of Harvard College Fusion Peptides That Bind to and Modify Target Nucleic Acid Sequences
US9149544B2 (en) 2009-11-06 2015-10-06 The Penn State Research Foundation Bioconjugation of calcium phosphosilicate nanoparticles for selective targeting of cells in vivo
US8518716B2 (en) 2009-11-30 2013-08-27 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Soluble CD117 (sc-kit) for diagnosis of preeclampsia and eclampsia
WO2011073521A1 (en) 2009-12-15 2011-06-23 Petri Salven Methods for enriching adult-derived endothelial progenitor cells and uses thereof
US20130011380A1 (en) 2009-12-18 2013-01-10 Blau Helen M Use of Cytidine Deaminase-Related Agents to Promote Demethylation and Cell Reprogramming
RU2558931C2 (ru) 2010-01-12 2015-08-10 Нестек С.А. Способы предсказания ответа трижды негативного рака молочной железы на терапию
WO2012004778A1 (en) 2010-07-09 2012-01-12 Ecole Polytechnique Federale De Lausanne (Epfl) Method for in-vitro monitoring of neuronal disorders and use thereof
WO2012047951A2 (en) 2010-10-05 2012-04-12 The Brigham And Women's Hospital, Inc. Human lung stem cells and uses thereof
EP2640827B1 (en) 2010-11-16 2023-07-19 Ingeneron, Inc. Methods for preserving target cells
WO2012068243A2 (en) 2010-11-16 2012-05-24 Ingeneron, Inc. Methods for obtaining target cells
US9816998B2 (en) 2011-04-01 2017-11-14 Cornell University Circulating exosomes as diagnostic/prognostic indicators and therapeutic targets of melanoma and other cancers
WO2012142411A1 (en) 2011-04-15 2012-10-18 Clavis Pharma Asa Systems and methods for detecting hent1 expression in hematological disorders
CN103841848B (zh) 2011-05-11 2017-12-05 施特姆拉德有限公司 辐射防护装置及其方法
AR086044A1 (es) 2011-05-12 2013-11-13 Imclone Llc Anticuerpos que se unen especificamente a un dominio extracelular de c-kit y usos de los mismos
WO2012171112A1 (en) 2011-06-14 2012-12-20 Mcmaster University Isolated embryonic stem cells that express lineage markers and associated methods
CN102876666B (zh) 2011-07-15 2015-02-04 内蒙古弘泰医学科技有限公司 Rna胞体及其用途
CN103088008B (zh) 2011-10-31 2014-08-20 中国科学院微生物研究所 胞苷脱氨酶及其编码基因和它们的应用
EP2836226B1 (en) 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
KR101384360B1 (ko) 2012-05-04 2014-04-14 아주대학교산학협력단 Ｓｃｆ 또는 이의 수용체를 억제하는 물질을 포함하는 혈관 투과성 관련 질환의 치료 또는 예방용 조성물
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
WO2013188037A2 (en) 2012-06-11 2013-12-19 Agilent Technologies, Inc Method of adaptor-dimer subtraction using a crispr cas6 protein
AU2013274965B2 (en) 2012-06-14 2018-07-19 Erasmus University Medical Center Rotterdam Methods, reagents and kits for detecting minimal residual disease
PT2877490T (pt) 2012-06-27 2019-02-12 Univ Princeton Inteínas clivadas, conjugados e suas utilizações
EP2890780B8 (en) 2012-08-29 2020-08-19 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
EP2895170A4 (en) 2012-09-14 2016-07-20 Childrens Medical Center INHIBITION OF VIRAL INFECTION-RELATED ASTHMA WITH A C-KIT HEMMER
WO2014043131A1 (en) 2012-09-14 2014-03-20 The Regents Of The University Of California Lentiviral vector for stem cell gene therapy of sickle cell disease
CA2886607A1 (en) 2012-10-17 2014-04-24 Cedars-Sinai Medical Center Molecular signatures of ovarian cancer
US20150299315A1 (en) 2012-10-22 2015-10-22 Becton, Dickinson And Company Non-b-lineage cells capable of producing antibody
EP2914273B1 (en) 2012-10-26 2018-07-04 Cedars-Sinai Medical Center Therapeutic cells depleted of specific subpopulations of cells for use in tissue repair of regeneration
WO2014081712A2 (en) 2012-11-20 2014-05-30 Celgene Avilomics Research, Inc. Methods of treating a disease or disorder associated with bruton's tyrosine kinase
DK3363902T3 (da) 2012-12-06 2020-01-02 Sigma Aldrich Co Llc Crispr-baseret genommodifikation og -regulering
PT2931898E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições para manipulação de sequências com domínios funcionais
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
IL308158A (en) 2012-12-17 2023-12-01 Harvard College Rna-guided human genome engineering
US20210137991A1 (en) 2013-02-08 2021-05-13 Themba Inc. Gene editing for autoimmune disorders
US20160145348A1 (en) 2013-03-14 2016-05-26 Fred Hutchinson Cancer Research Center Compositions and methods to modify cells for therapeutic objectives
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
WO2014141306A2 (en) 2013-03-15 2014-09-18 Rajiv Gandhi Centre For Biotechnology Methods and materials for identifying therapeutic response in chronic myeloid leukemia
WO2014186686A2 (en) 2013-05-17 2014-11-20 Two Blades Foundation Targeted mutagenesis and genome engineering in plants using rna-guided cas nucleases
EP3603679B1 (en) 2013-06-04 2022-08-10 President and Fellows of Harvard College Rna-guided transcriptional regulation
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
WO2014197821A1 (en) 2013-06-06 2014-12-11 President And Fellows Of Harvard College Homeodomain fusion proteins and uses thereof
FR3008408B1 (fr) 2013-07-11 2018-03-09 Mc Saf Nouveaux conjugues anticorps-medicament et leur utilisation en therapie
PL3019619T3 (pl) 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
US20150044772A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. Crispr/cas system-based novel fusion protein and its applications in genome editing
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
CN111218447B (zh) 2013-11-07 2024-10-11 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP3835419A1 (en) 2013-12-12 2021-06-16 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
WO2015148512A1 (en) 2014-03-24 2015-10-01 Qt Holdings Corp Shaped articles including hydrogels and methods of manufacture and use thereof
US10369104B2 (en) 2014-04-01 2019-08-06 Children's Hospital Los Angeles Targeted polymerized nanoparticles for cancer treatment
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
ES2945588T3 (es) 2014-08-26 2023-07-04 Univ Leland Stanford Junior Injerto de células madre con una combinación de un agente dirigido a las células madre y modulación de señalización inmunoreguladora
US10450584B2 (en) 2014-08-28 2019-10-22 North Carolina State University Cas9 proteins and guiding features for DNA targeting and genome editing
EP2995948A1 (en) 2014-09-09 2016-03-16 Ecole Polytechnique Federale de Lausanne (EPFL) Methods and compounds useful in hematopoietic stem cell medicine
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
AU2015332533B2 (en) 2014-10-14 2018-08-30 Halozyme, Inc. Compositions of adenosine deaminase-2 (ADA2), variants thereof and methods of using same
EP3212221B1 (en) 2014-10-29 2023-12-06 Massachusetts Eye & Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
JP6153180B2 (ja) 2014-11-04 2017-06-28 国立大学法人神戸大学 脱塩基反応により標的化したｄｎａ配列に特異的に変異を導入する、ゲノム配列の改変方法、並びにそれに用いる分子複合体
WO2016083777A1 (en) 2014-11-26 2016-06-02 Isis Innovation Limited Detection of acute myeloid leukaemia (aml) leukaemic stem cells (lsc)
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
AU2015367378A1 (en) 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
WO2016135558A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
AU2016226077B2 (en) 2015-03-03 2021-12-23 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases with altered PAM specificity
AU2016232719A1 (en) 2015-03-18 2017-10-12 Memorial Sloan Kettering Cancer Center Compositions and methods for targeting CD99 in haematopoietic and lymphoid malignancies
HK1256681A1 (zh) 2015-04-06 2019-10-04 President And Fellows Of Harvard College 用於非清髓性预处理的组合物和方法
CA2985615A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating hiv infection and aids
CN106011104B (zh) 2015-05-21 2019-09-27 清华大学 利用拆分Cas系统进行基因编辑和表达调控方法
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
EP3957731A1 (en) 2015-07-15 2022-02-23 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US11008555B2 (en) 2015-09-17 2021-05-18 The Regents Of The University Of California Variant Cas9 polypeptides comprising internal insertions
WO2017059241A1 (en) 2015-10-02 2017-04-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Lentiviral protein delivery system for rna-guided genome editing
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
EP3371306B8 (en) 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US11866727B2 (en) 2015-11-06 2024-01-09 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1A
WO2017093804A2 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
IL313038A (en) 2016-03-14 2024-07-01 Editas Medicine Inc Crispr/cas-related methods and compositions for treating beta hemoglobinopathies
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
ES3030658T3 (en) 2016-03-30 2025-07-01 Intellia Therapeutics Inc Lipid nanoparticle formulations for crispr/cas components
JP2019103391A (ja) 2016-04-14 2019-06-27 国立大学法人 東京医科歯科大学 単球系統のみに分化するヒト単球前駆細胞およびその単離方法
EP4166161B1 (en) 2016-04-14 2026-03-04 Fred Hutchinson Cancer Center Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
CA3026110A1 (en) 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
FR3051555B1 (fr) 2016-05-20 2018-05-11 Horiba Abx Sas Detection de la maladie residuelle du myelome multiple
MX2018014577A (es) 2016-05-27 2019-05-20 Tg Therapeutics Inc Combinacion de anticuerpo anti-cd20, inhibidor selectivo de quinasa p13-delta, e inhibidor de btk para tratar trastornos proliferativos de celulas b.
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
CN109641051A (zh) 2016-06-17 2019-04-16 美真达治疗公司 用于耗尽细胞的组合物和方法
US10111966B2 (en) 2016-06-17 2018-10-30 Magenta Therapeutics, Inc. Methods for the depletion of CD117+ cells
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
US12350349B2 (en) 2016-08-03 2025-07-08 Washington University Gene editing of CAR-T cells for the treatment of T cell malignancies with chimeric antigen receptors
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
WO2018039438A1 (en) * 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN110023489A (zh) 2016-08-29 2019-07-16 Pdi医药发展国际股份公司 提供治疗活性细胞产物
WO2018041973A1 (en) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
US11590098B2 (en) 2016-09-06 2023-02-28 Mayo Foundation For Medical Education And Research Methods of treating triple-negative breast cancer using compositions of antibodies and carrier proteins
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US20190365806A1 (en) 2016-11-02 2019-12-05 Universität Basel Immunologically discernible cell surface variants for use in cell therapy
WO2018085516A2 (en) 2016-11-02 2018-05-11 Aal Scientifics, Inc. Non-mesenchymal human lung stem cells and methods of their use for treating respiratory diseases
US11180778B2 (en) 2016-11-11 2021-11-23 The Regents Of The University Of California Variant RNA-guided polypeptides and methods of use
CN107043779B (zh) 2016-12-01 2020-05-12 中国农业科学院作物科学研究所 一种CRISPR/nCas9介导的定点碱基替换在植物中的应用
EP3551757A1 (en) 2016-12-08 2019-10-16 Intellia Therapeutics, Inc. Modified guide rnas
JP7383478B2 (ja) 2016-12-22 2023-11-20 インテリア セラピューティクス，インコーポレイテッド アルファ１アンチトリプシン欠乏症を治療するための組成物および方法
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR102569848B1 (ko) 2016-12-23 2023-08-25 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Pcsk9의 유전자 편집
EP3401834B1 (en) 2016-12-26 2022-08-17 Shenzhen Goodix Technology Co., Ltd. Fingerprint recognition system
EP3565608A4 (en) 2017-01-05 2020-12-16 Rutgers, The State University of New Jersey TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES
SG11201906319XA (en) 2017-01-20 2019-08-27 Magenta Therapeutics Inc Compositions and methods for the depletion of cd137+ cells
IL301075B2 (en) 2017-01-30 2024-05-01 Univ Leland Stanford Junior A genotoxic matching regimen for stem cell transplantation
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
WO2018152197A1 (en) 2017-02-15 2018-08-23 Massachusetts Institute Of Technology Dna writers, molecular recorders and uses thereof
KR102709603B1 (ko) 2017-02-28 2024-09-27 보르 바이오파마 인크. 계통 특이적 단백질의 억제를 위한 조성물 및 방법
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
US12485186B2 (en) 2017-03-31 2025-12-02 The Children's Medical Center Corporation Antibody-mediated conditioning with immunosuppression to enable allogeneic transplantation
CN106916852B (zh) 2017-04-13 2020-12-04 上海科技大学 一种碱基编辑系统及其构建和应用方法
PL3609540T3 (pl) 2017-04-14 2023-03-27 Bolt Biotherapeutics, Inc. Sposób syntezy immunokoniugatu
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
KR102678809B1 (ko) 2017-05-18 2024-06-26 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법 및 조성물
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CA3063733A1 (en) 2017-05-25 2018-11-29 The General Hospital Corporation Base editors with improved precision and specificity
IT201700062176A1 (it) 2017-06-07 2018-12-07 Univ Degli Studi Milano Metodo per l’isolamento di sottopopolazioni di progenitori cellulari cardiaci e relativi usi in campo medico
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
CN111328290A (zh) 2017-06-26 2020-06-23 博德研究所 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法
US10392616B2 (en) 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
US20200140896A1 (en) 2017-06-30 2020-05-07 Novartis Ag Methods for the treatment of disease with gene editing systems
CA3070242A1 (en) 2017-07-18 2019-01-24 Calimmune, Inc. Compositions and methods for treating beta-hemoglobinopathies
EP3658589B1 (en) 2017-07-26 2023-09-27 Forty Seven, Inc. Anti-sirp-alpha antibodies and related methods
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
WO2019040650A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
US10849924B2 (en) 2017-09-26 2020-12-01 New York Medical College Compositions and methods for restoring endothelial glycocalyx
IL316453A (en) 2017-10-06 2024-12-01 Univ Oregon Health & Science RNA editing agents and methods
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
CN111565719A (zh) 2017-10-17 2020-08-21 瑞真药业公司 用于治疗食管癌的crac通道调节剂
WO2019084053A1 (en) 2017-10-24 2019-05-02 Magenta Therapeutics, Inc. COMPOSITIONS AND METHODS FOR DEPLOYING CD117 + CELLS
EP3700568A4 (en) 2017-10-24 2021-11-10 Magenta Therapeutics, Inc. COMPOSITIONS AND METHODS FOR DEPLOYING CD117 + CELLS
US10882915B2 (en) 2017-10-24 2021-01-05 Magenta Therapeutics, Inc. Compositions and methods for the depletion of CD117+ cells
WO2019082124A1 (en) 2017-10-26 2019-05-02 Rhizen Pharmaceuticals Sa COMPOSITION AND METHOD FOR THE TREATMENT OF LARGE B-CELL DIFFUSED LYMPHOMA
MA50849A (fr) 2017-10-26 2020-09-02 Vertex Pharma Substances et procédés pour le traitement d'hémoglobinopathies
EA202090682A1 (ru) 2017-10-30 2020-10-15 Ризен Фармасьютикалз Са Модуляторы кальциевых каналов, активируемых высвобождением кальция, для лечения гематологических и солидных раковых заболеваний
CN111770776A (zh) 2017-12-06 2020-10-13 瑞真药业公司 治疗外周t细胞淋巴瘤和皮肤t细胞淋巴瘤的组合物和方法
WO2019113437A1 (en) 2017-12-08 2019-06-13 The Board Of Trustees Of The Leland Stanford Junior University Hematopoietic stem cell engraftment
AU2018383712B2 (en) 2017-12-11 2025-09-04 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
AR114014A1 (es) 2017-12-22 2020-07-08 Inst Genetics & Developmental Biology Cas Sistema de edición de bases y método basado en proteína cpf1
US20230193242A1 (en) 2017-12-22 2023-06-22 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
GB201802201D0 (en) 2018-02-09 2018-03-28 Ultrahuman Five Ltd Binding agents
CA3093289A1 (en) 2018-03-07 2019-09-12 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2019178280A1 (en) 2018-03-13 2019-09-19 Amgen Inc. Sequential digestion of polypeptides for mass spectrometric analysis
WO2019178151A1 (en) 2018-03-13 2019-09-19 Amgen Inc. Methods for the preparation of trypsin-resistant polypeptides for mass spectrometric analysis
IL277234B2 (en) 2018-03-14 2026-03-01 Editas Medicine Inc Systems and methods for treating hemoglobin diseases
EP3765617A1 (en) 2018-03-14 2021-01-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US20210363518A1 (en) 2018-03-19 2021-11-25 University Of Massachusetts Modified guide rnas for crispr genome editing
AU2019256287B2 (en) 2018-04-17 2026-02-05 The General Hospital Corporation Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
KR102959497B1 (ko) 2018-05-11 2026-05-04 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 돌연변이를 억제하는 방법
KR102853088B1 (ko) 2018-05-11 2025-09-02 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법
JP7577648B2 (ja) 2018-05-14 2024-11-05 センバ インコーポレイテッド 自己免疫疾患のための遺伝子編集
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
JP2021526525A (ja) 2018-06-07 2021-10-07 マジェンタ セラピューティクス インコーポレイテッドＭａｇｅｎｔａ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． 抗体薬物コンジュゲート（ａｄｃｓ）を用いた治療方法
KR102894715B1 (ko) 2018-08-03 2025-12-03 빔 테라퓨틱스, 인크. 핵산 표적 서열을 변형시키기 위한 다중-이펙터 핵염기 편집기 및 이를 이용하는 방법
WO2020041751A1 (en) 2018-08-23 2020-02-27 The Broad Institute, Inc. Cas9 variants having non-canonical pam specificities and uses thereof
JP2021534769A (ja) 2018-08-31 2021-12-16 エーエルエックス オンコロジー インコーポレイテッド デコイポリペプチド
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
GB201815670D0 (en) 2018-09-26 2018-11-07 Univ Oxford Innovation Ltd Protein editing
CN109295186B (zh) 2018-09-30 2023-10-03 中山大学 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用
CN113302493A (zh) 2018-10-01 2021-08-24 小利兰·斯坦福大学托管委员会 同时解析多种抗体同种型的高特异性和灵敏度免疫吸附诊断测定
KR102716514B1 (ko) 2018-10-10 2024-10-15 주식회사 노벨티노빌리티 신규 항-c-KIT 항체
US20200376135A1 (en) 2018-10-23 2020-12-03 Magenta Therapeutics, Inc. Fc silenced antibody drug conjugates (adcs) and uses thereof
CN113330026A (zh) 2018-10-23 2021-08-31 美真达治疗公司 Fc沉默的抗体药物缀合物(ADC)及其用途
AU2019374055B2 (en) 2018-10-30 2026-06-11 Regeneron Pharmaceuticals, Inc. Methods for allogeneic hematopoietic stem cell transplantation
UA128431C2 (uk) 2018-11-26 2024-07-10 Форті Севен, Інк. ГУМАНІЗОВАНЕ АНТИТІЛО ДО c-Kit
KR20210094609A (ko) 2018-11-28 2021-07-29 포티 세븐, 인코포레이티드 절제 레짐에 대해 저항성인 유전적으로 변형된 hspc
EP3887521A1 (en) 2018-11-29 2021-10-06 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US12000775B2 (en) 2018-12-14 2024-06-04 Amgen Inc. System suitability method for use with protein concentration determination by slope
AU2020216484A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced off-target deamination and methods of using same to modify a nucleobase target sequence
JP7646552B2 (ja) 2019-01-31 2025-03-17 ビーム セラピューティクス インク． 低減された非標的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エディターの特徴づけのためのアッセイ
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
EP3924479A4 (en) 2019-02-13 2023-01-25 Beam Therapeutics, Inc. ADENOSINE DEAMINASE BASE EDITORS AND METHODS OF USING THEM TO MODIFY A NUCLEOBASE IN A TARGET SEQUENCE
CN114096666B (zh) 2019-02-13 2025-03-18 比姆医疗股份有限公司 治疗血红素病变的组合物和方法
US20220127594A1 (en) 2019-02-13 2022-04-28 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US20220098593A1 (en) 2019-02-13 2022-03-31 Beam Therapeutics Inc. Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
CN114040970B (zh) 2019-02-13 2024-09-27 比姆医疗股份有限公司 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法，包括遗传性疾病的治疗
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CA3134319A1 (en) 2019-04-24 2020-10-29 Rahul Palchaudhuri Anti-cd117 antibodies and uses thereof
WO2020216947A1 (en) 2019-04-24 2020-10-29 Heidelberg Pharma Research Gmbh Amatoxin antibody-drug conjugates and uses thereof
WO2020219778A2 (en) 2019-04-24 2020-10-29 Magenta Therapeutics, Inc. Anti-cd117 antibody-drug conjugates and uses thereof
JP2022530442A (ja) 2019-04-24 2022-06-29 マジェンタ セラピューティクス インコーポレイテッド アントラサイクリン抗体薬物複合体およびその使用
JP2022529727A (ja) 2019-04-24 2022-06-23 マジェンタ セラピューティクス インコーポレイテッド Ａｎｔｉ－ｃｄ１１７ とその使用
JP2022530438A (ja) 2019-04-24 2022-06-29 マジェンタ セラピューティクス インコーポレイテッド 抗ｃｄ１１７抗体薬物複合体およびその使用
EP3958878A4 (en) 2019-04-24 2022-12-28 Magenta Therapeutics, Inc. CONDITIONING METHODS FOR GENE THERAPY
EP3958879A4 (en) 2019-04-25 2023-05-10 Actinium Pharmaceuticals, Inc. Compositions and methods of immunodepletion for the treatment of malignant and non-malignant hematological diseases
CN110042124A (zh) 2019-04-25 2019-07-23 国家卫生健康委科学技术研究所 基因组碱基编辑增加人红细胞中胎儿血红蛋白水平的试剂盒及应用
JP7679311B2 (ja) 2019-05-21 2025-05-19 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いた一塩基多型編集法
KR20220012328A (ko) 2019-05-24 2022-02-03 포티 세븐, 인코포레이티드 c-kit 및 CD47에 대한 면역요법제의 병용 투여를 위한 요법
EP3983405A4 (en) 2019-06-12 2023-06-07 Ontario Institute for Cancer Research (OICR) NICOTINAMIDE PHOSPHORIBOSYLTRANSFERASE (NAMPT) INHIBITING CONJUGATES AND USES THEREOF
KR102258713B1 (ko) 2019-07-31 2021-05-31 한양대학교 산학협력단 사이토신 염기교정용 조성물 및 이의 용도
US20220288118A1 (en) 2019-07-31 2022-09-15 Forty Seven, Inc. Depletion regimes for engineered t-cell or nk-cell therapy
AU2020336969B2 (en) 2019-08-29 2024-12-05 Beam Therapeutics Inc. Compositions and methods for non-toxic conditioning
GB201912681D0 (en) 2019-09-04 2019-10-16 Eth Zuerich Bispecific binding agent that binds to cd117/c-kit and cd3
AU2020344547A1 (en) 2019-09-09 2022-03-24 Beam Therapeutics Inc. Novel nucleobase editors and methods of using same
ES2960527T3 (es) 2019-09-13 2024-03-05 Miltenyi Biotec Bv & Co Kg Método para la generación de una composición celular de células progenitoras dopaminérgicas del mesencéfalo ventral
US20220324957A1 (en) 2019-09-16 2022-10-13 Opsidio, LLC Anti-stem cell factor antibodies and methods of use thereof in renal disease
AU2020466994B2 (en) 2019-09-17 2026-05-28 Rutgers, The State University Of New Jersey Highly efficient DNA base editors mediated by RNA-aptamer recruitment for targeted genome modification and uses thereof
CN112125915A (zh) 2019-09-18 2020-12-25 四川百利药业有限责任公司 一种喜树碱衍生物及其偶联物
US20220380757A1 (en) 2019-09-30 2022-12-01 The Children's Medical Center Corporation Bcl11a guide and base editor delivery
CN114828842A (zh) 2019-10-21 2022-07-29 理森制药股份公司 用于治疗急性髓系白血病的包含dhodh抑制剂的组合物
NL2024163B1 (en) 2019-11-05 2021-07-20 Univ Of Salamanca Means and methods for multiparameter cytometry-based leukocyte subsetting.
AU2020383622A1 (en) 2019-11-15 2022-06-16 Secretome Therapeutics, Inc. Immortalized cardiac stem cells for cardiac repair
EP3825330A1 (en) 2019-11-19 2021-05-26 International-Drug-Development-Biotech Anti-cd117 antibodies and methods of use thereof
KR102772859B1 (ko) 2019-11-25 2025-02-27 주식회사 노벨티노빌리티 c-kit에 대한 항체 및 이의 용도
WO2021123397A1 (en) 2019-12-20 2021-06-24 Biogemma IMPROVING EFFICIENCY OF BASE EDITING USING TypeV CRISPR ENZYMES
US20230235309A1 (en) 2020-02-05 2023-07-27 The Broad Institute, Inc. Adenine base editors and uses thereof
WO2021158999A1 (en) 2020-02-05 2021-08-12 The Broad Institute, Inc. Gene editing methods for treating spinal muscular atrophy
CN116113692A (zh) 2020-02-13 2023-05-12 比姆医疗股份有限公司 用于植入碱基编辑细胞的组合物和方法
BR112022017732A2 (pt) 2020-03-04 2023-01-17 Suzhou Qi Biodesign Biotechnology Company Ltd Sistema aperfeiçoado de edição de base de citosina
GB202003129D0 (en) 2020-03-04 2020-04-15 Hansa Biopharma AB Conditioning regimen for transplant of stem cells
EP4121462A4 (en) 2020-03-16 2024-04-24 Magenta Therapeutics, Inc. BISPECIFIC T CELL BINDING PROTEINS
WO2021188840A1 (en) 2020-03-19 2021-09-23 Rewrite Therapeutics, Inc. Methods and compositions for directed genome editing
EP4132951A4 (en) 2020-04-09 2025-06-11 Verve Therapeutics, Inc. CHEMICALLY MODIFIED GENE EDITING GUIDE RNAS WITH CAS12B
US20230121577A1 (en) 2020-04-15 2023-04-20 The Regents Of The University Of California Compositions and Methods for Treatment
US20230279438A1 (en) 2020-05-13 2023-09-07 Inserm (Institut National De La Santé Et La Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
JP7820823B2 (ja) 2020-05-27 2026-02-26 ウニヴェルジテート・チューリッヒ 骨髄性細胞およびミクログリアにおいて特異的に治療タンパク質を発現するウイルスベクター
BR112022024930A2 (pt) 2020-06-08 2022-12-27 Baili Bio Chengdu Pharmaceutical Co Ltd Fármaco de camptotecina possuindo unidade de conexão hidrofílica de alta estabilidade e conjugado do mesmo
EP4179079A1 (en) 2020-07-10 2023-05-17 Horizon Discovery Limited Method for producing genetically modified cells
WO2022011458A1 (en) 2020-07-13 2022-01-20 Ontario Institute For Cancer Research (Oicr) Nicotinamide phosphoribosyltransferase (nampt) inhibitor-conjugates and uses thereof
JP7890972B2 (ja) 2020-07-15 2026-07-15 ライフ エディット セラピューティクス，インコーポレイティド ウラシル安定化タンパク質並びにその活性断片及びバリアント並びに使用方法
WO2022036179A2 (en) 2020-08-14 2022-02-17 Orchard Therapeutics (Europe) Limited Compositions and methods for treating or preventing hereditary angioedema
WO2022040596A1 (en) 2020-08-20 2022-02-24 Ambrx, Inc. Antibody-tlr agonist conjugates, methods and uses thereof
MX2023002318A (es) 2020-08-29 2023-05-19 argenx BV Metodo de tratamiento de pacientes con sensibilidad reducida a un inhibidor de bcl-2.
EP4211235A2 (en) 2020-09-11 2023-07-19 Lifeedit Therapeutics, Inc. Dna modifying enzymes and active fragments and variants thereof and methods of use
CA3192224A1 (en) 2020-09-11 2022-03-17 Jyun-Liang LIN Base editing enzymes
US20230383277A1 (en) 2020-10-14 2023-11-30 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
WO2022081828A1 (en) 2020-10-16 2022-04-21 The Board Of Trustees Of The Leland Stanford Junior University Hematopoietic stem cell engraftment with a combination of agents
IT202000028688A1 (it) 2020-11-27 2022-05-27 Consiglio Nazionale Ricerche Varianti della citidina deaminasi per l’editazione di basi
WO2022143694A1 (zh) 2020-12-28 2022-07-07 华东师范大学 一种针对细胞中单个或多个基因进行基因编辑的方法、产品和应用
WO2022150706A2 (en) 2021-01-08 2022-07-14 The General Hospital Corporation Genome editing approaches to treat spinal muscular atrophy
US20240360414A1 (en) 2021-01-15 2024-10-31 St. Jude Children's Research Hospital, Inc. Systems and Methods for Base Editing Of HBG1/2 Gene Promoter and Fetal Hemoglobin Induction
WO2023279118A2 (en) 2021-07-02 2023-01-05 University Of Maryland, College Park Cytidine deaminases and methods of genome editing using the same
EP4396207A4 (en) 2021-09-03 2025-07-16 Univ Chicago POLYPEPTIDES AND METHODS FOR MODIFYING NUCLEIC ACIDS
GB2702733A (en) 2021-09-22 2026-06-24 Verve Therapeutics Inc Gene editing of PCSK9 or ANGPTL3 and compositions and methods of using same for treatment of disease
US20230105319A1 (en) 2021-09-24 2023-04-06 Crispr Therapeutics Ag PRODRUG INCORPORATED sgRNA SYNTHESIS
WO2023125814A1 (zh) 2021-12-29 2023-07-06 华东师范大学 腺嘌呤脱氨酶及其应用
WO2023155901A1 (en) 2022-02-17 2023-08-24 Correctsequence Therapeutics Mutant cytidine deaminases with improved editing precision
EP4540378A1 (en) 2022-06-20 2025-04-23 CRISPR Therapeutics AG Base editing proteins and uses thereof
JP2025520697A (ja) 2022-06-23 2025-07-03 ビーエーエスエフ アグリカルチュラル ソリューションズ ユーエス エルエルシー 多様化塩基編集

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016528890A (ja) 2013-07-09 2016-09-23 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用

Also Published As

Publication number Publication date
WO2019217942A1 (en) 2019-11-14
KR102852347B1 (ko) 2025-09-01
AU2019265019B2 (en) 2025-11-06
EP3799603A1 (en) 2021-04-07
KR20250134703A (ko) 2025-09-11
AU2026200834A1 (en) 2026-02-26
JP2021523737A (ja) 2021-09-09
CA3100019A1 (en) 2019-11-14
US20220401530A1 (en) 2022-12-22
CN112534054A (zh) 2021-03-19
US12133884B2 (en) 2024-11-05
US20250108098A1 (en) 2025-04-03
JP2025026840A (ja) 2025-02-26
EP3799603A4 (en) 2022-03-02
CN121555430A (zh) 2026-02-24
AU2019265019A1 (en) 2020-11-26
CN112534054B (zh) 2025-10-28
KR20210023831A (ko) 2021-03-04

Similar Documents

Publication Publication Date Title
US20250108098A1 (en) Methods of substituting pathogenic amino acids using programmable base editor systems
CN112601816B (zh) 使用可编程碱基编辑器系统遏止病原性突变的方法
CN112469446B (zh) 利用可编程碱基编辑器系统编辑单核苷酸多态性的方法
JP7717684B2 (ja) 新規核酸塩基エディター及びその使用方法
JP7646554B2 (ja) アルファ－１アンチトリプシン不全を治療するための組成物および方法
WO2021041885A2 (en) Compositions and methods for editing a mutation to permit transcription or expression
WO2023086953A1 (en) Compositions and methods for the treatment of hereditary angioedema (hae)
CN117729931A (zh) 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法
KR20240111821A (ko) B형 간염 바이러스 감염을 치료하기 위한 조성물 및 방법

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220511

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220511

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230420

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230905

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231204

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240304

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240618

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241018

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20241115

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: 20250128

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250226

R150 Certificate of patent or registration of utility model

Ref document number: 7642531

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150