GB2637278B - Compositions and methods for gene editing - Google Patents

Compositions and methods for gene editing

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Publication number
GB2637278B
GB2637278B GB2506591.3A GB202506591A GB2637278B GB 2637278 B GB2637278 B GB 2637278B GB 202506591 A GB202506591 A GB 202506591A GB 2637278 B GB2637278 B GB 2637278B
Authority
GB
United Kingdom
Prior art keywords
compositions
methods
gene editing
editing
gene
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
GB2506591.3A
Other languages
English (en)
Other versions
GB202506591D0 (en
GB2637278A (en
Inventor
Gilbert Luke
Weissman Jonathan
Nunez James
Chen Jin
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of California Berkeley
University of California, San Diego
Original Assignee
University of California Berkeley
University of California, San Diego
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 University of California Berkeley, University of California, San Diego filed Critical University of California Berkeley
Publication of GB202506591D0 publication Critical patent/GB202506591D0/en
Publication of GB2637278A publication Critical patent/GB2637278A/en
Application granted granted Critical
Publication of GB2637278B publication Critical patent/GB2637278B/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • 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
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/60—Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
GB2506591.3A 2020-06-02 2021-06-01 Compositions and methods for gene editing Active GB2637278B (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063033397P 2020-06-02 2020-06-02
US202063114850P 2020-11-17 2020-11-17
US202163171698P 2021-04-07 2021-04-07
GB2219607.5A GB2612212B (en) 2020-06-02 2021-06-01 Compositions and methods for gene editing

Publications (3)

Publication Number Publication Date
GB202506591D0 GB202506591D0 (en) 2025-06-11
GB2637278A GB2637278A (en) 2025-07-16
GB2637278B true GB2637278B (en) 2025-11-05

Family

ID=78831704

Family Applications (2)

Application Number Title Priority Date Filing Date
GB2506591.3A Active GB2637278B (en) 2020-06-02 2021-06-01 Compositions and methods for gene editing
GB2219607.5A Active GB2612212B (en) 2020-06-02 2021-06-01 Compositions and methods for gene editing

Family Applications After (1)

Application Number Title Priority Date Filing Date
GB2219607.5A Active GB2612212B (en) 2020-06-02 2021-06-01 Compositions and methods for gene editing

Country Status (10)

Country Link
US (1) US20240309345A1 (enExample)
EP (1) EP4158009A4 (enExample)
JP (1) JP2023529611A (enExample)
KR (1) KR20230031832A (enExample)
AU (2) AU2021283185A1 (enExample)
CA (1) CA3184884A1 (enExample)
GB (2) GB2637278B (enExample)
IL (1) IL298434A (enExample)
MX (1) MX2022015277A (enExample)
WO (1) WO2021247570A2 (enExample)

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AU2022318664A1 (en) 2021-07-30 2024-02-29 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
US20240254483A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
IL311610A (en) 2021-09-21 2024-05-01 Scribe Therapeutics Inc Engineered CASX suppressor systems
CN119836432A (zh) * 2022-05-01 2025-04-15 恩科洛玛生物公司 用于pcsk9表达的表观遗传调节的组合物和方法
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
EP4544058A1 (en) * 2022-06-23 2025-04-30 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of ciita expression
KR20250059560A (ko) * 2022-06-23 2025-05-02 크로마 메디슨, 인크. 후성유전학적 편집을 위한 조성물 및 방법
WO2023250148A1 (en) * 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic editing
WO2023250490A1 (en) * 2022-06-23 2023-12-28 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of trac expression
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
CA3260110A1 (en) 2022-06-24 2023-12-28 European Molecular Biology Laboratory MODULAR TOOL BASED ON CRISPR FOR THE SPECIFIC INTRODUCTION OF EPIGENETIC MODIFICATIONS ON TARGET LOCI
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
JP2025526073A (ja) * 2022-08-11 2025-08-07 エピジェニック・セラピューティクス・プライベイト・リミテッド エピジェネティック編集標的部位のための方法およびその使用
WO2024032681A1 (zh) * 2022-08-11 2024-02-15 益杰立科(上海)生物科技有限公司 一种表观编辑靶点的方法及用途
WO2024040254A2 (en) * 2022-08-19 2024-02-22 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
WO2024044574A1 (en) * 2022-08-23 2024-02-29 The Regents Of The University Of California Compositions and methods for reducing ionizing radiation-induced hematopoietic stem cell damage
JP2025531268A (ja) 2022-09-19 2025-09-19 チューン セラピューティクス インコーポレイテッド Ｔ細胞機能を調節するための組成物、システム、および方法
KR20250110209A (ko) * 2022-09-23 2025-07-18 엔크로마 바이오, 인크. Hbv 유전자 발현의 에피제네틱 조절을 위한 조성물 및 방법
WO2024081879A1 (en) * 2022-10-14 2024-04-18 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of cd247 expression
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
JP2026506083A (ja) 2023-02-17 2026-02-20 ホワイトヘッド・インスティテュート・フォー・バイオメディカル・リサーチ エピジェネティック修飾を行うための組成物および方法
WO2024238321A1 (en) * 2023-05-12 2024-11-21 Mammoth Biosciences, Inc. Compositions and methods for targeted epigenetic modification
JP2026523341A (ja) 2023-05-15 2026-07-14 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
TW202503061A (zh) * 2023-06-16 2025-01-16 大陸商益杰立科（上海）生物科技有限公司 靶向ｂ型肝炎病毒基因的表觀編輯工具
CN121909284A (zh) 2023-07-31 2026-04-21 图恩疗法股份有限公司 用于调节il-2基因表达的组合物和方法
EP4754248A1 (en) 2023-07-31 2026-06-10 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
WO2025251019A1 (en) * 2024-05-31 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic editing
WO2025260068A1 (en) 2024-06-14 2025-12-18 Tune Therapeutics, Inc. Lipid nanoparticle formulation for delivery of nucleic acids to cells
WO2026015647A1 (en) 2024-07-09 2026-01-15 Tune Therapeutics, Inc. Compositions, systems, and methods for cell differentiation using targeted gene activation of dll4 and/or vcam1
WO2026064753A1 (en) 2024-09-23 2026-03-26 Tune Therapeutics, Inc. Dna methyltransferase-like protein (dnmt3l) or dna methyltransferase 3a (dnmt3a) repressor systems for epigenetic editing
WO2026102058A1 (en) 2024-11-06 2026-05-15 Tune Therapeutics, Inc. Methods for treating chronic hepatitis b infection and related formulations
WO2026161895A1 (en) 2025-01-27 2026-07-30 Tune Therapeutics, Inc. Compositions, systems, and methods for epigenetic regulation of lipoprotein a (lpa) gene expression

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US20190127713A1 (en) * 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2019204766A1 (en) * 2018-04-19 2019-10-24 The Regents Of The University Of California Compositions and methods for gene editing

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US11286493B2 (en) * 2016-05-27 2022-03-29 The Regents Of The University Of California Methods and compositions for targeting RNA polymerases and non-coding RNA biogenesis to specific loci

Patent Citations (2)

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Publication number Priority date Publication date Assignee Title
US20190127713A1 (en) * 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2019204766A1 (en) * 2018-04-19 2019-10-24 The Regents Of The University Of California Compositions and methods for gene editing

Also Published As

Publication number Publication date
WO2021247570A2 (en) 2021-12-09
GB202506591D0 (en) 2025-06-11
AU2021283185A1 (en) 2023-01-05
KR20230031832A (ko) 2023-03-07
GB2612212A (en) 2023-04-26
JP2023529611A (ja) 2023-07-11
US20240309345A1 (en) 2024-09-19
IL298434A (en) 2023-01-01
AU2026203640A1 (en) 2026-07-23
GB2637278A (en) 2025-07-16
GB2612212B (en) 2025-06-18
CA3184884A1 (en) 2021-12-09
EP4158009A2 (en) 2023-04-05
EP4158009A4 (en) 2024-10-02
WO2021247570A8 (en) 2023-01-05
GB202219607D0 (en) 2023-02-08
WO2021247570A3 (en) 2022-03-24
MX2022015277A (es) 2023-02-13

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