EP4010004A4 - Zusammensetzungen und verfahren zur modulation von genexpression - Google Patents

Zusammensetzungen und verfahren zur modulation von genexpression Download PDF

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Publication number
EP4010004A4
EP4010004A4 EP20851042.0A EP20851042A EP4010004A4 EP 4010004 A4 EP4010004 A4 EP 4010004A4 EP 20851042 A EP20851042 A EP 20851042A EP 4010004 A4 EP4010004 A4 EP 4010004A4
Authority
EP
European Patent Office
Prior art keywords
compositions
methods
gene expression
modulating gene
modulating
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.)
Pending
Application number
EP20851042.0A
Other languages
English (en)
French (fr)
Other versions
EP4010004A2 (de
Inventor
Fyodor Urnov
John A. Stamatoyannopoulos
Joycelynn PEARL
Matthew WILKEN
Christie CIARLO
Shon GREEN
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.)
Altius Institute for Biomedical Sciences
Original Assignee
Altius Institute for Biomedical Sciences
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 Altius Institute for Biomedical Sciences filed Critical Altius Institute for Biomedical Sciences
Publication of EP4010004A2 publication Critical patent/EP4010004A2/de
Publication of EP4010004A4 publication Critical patent/EP4010004A4/de
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70521—CD28, CD152
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Peptides Or Proteins (AREA)
EP20851042.0A 2019-08-07 2020-08-06 Zusammensetzungen und verfahren zur modulation von genexpression Pending EP4010004A4 (de)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962884028P 2019-08-07 2019-08-07
US201962898434P 2019-09-10 2019-09-10
US201962937011P 2019-11-18 2019-11-18
PCT/US2020/045174 WO2021026336A2 (en) 2019-08-07 2020-08-06 Compositions and methods for modulation of gene expression

Publications (2)

Publication Number Publication Date
EP4010004A2 EP4010004A2 (de) 2022-06-15
EP4010004A4 true EP4010004A4 (de) 2023-09-13

Family

ID=74502663

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20851042.0A Pending EP4010004A4 (de) 2019-08-07 2020-08-06 Zusammensetzungen und verfahren zur modulation von genexpression

Country Status (2)

Country Link
EP (1) EP4010004A4 (de)
WO (1) WO2021026336A2 (de)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230045612A (ko) * 2020-08-14 2023-04-04 더 가버닝 카운슬 오브 더 유니버시티 오브 토론토 Krab 융합 억제제 및 유전자 발현을 억제하기 위한 방법 및 조성물
JP2024534523A (ja) 2021-09-21 2024-09-20 スクライブ・セラピューティクス・インコーポレイテッド 操作されたｃａｓｘリプレッサー系
CN114195902B (zh) * 2021-10-18 2023-06-20 翌圣生物科技(上海)股份有限公司 重组蛋白结构域增强tet酶及全基因组dna甲基化检测方法
CN114600837B (zh) * 2022-04-15 2023-05-02 润康生物医药(苏州)有限公司 一种粒细胞缺乏症动物模型及其构建方法以及ikzf1和cmyb在构建模型中的应用
EP4314267A1 (de) 2022-06-07 2024-02-07 Scribe Therapeutics Inc. Zusammensetzungen und verfahren zum targeting von pcsk9
WO2025034754A2 (en) * 2023-08-08 2025-02-13 Altius Institute For Biomedical Sciences Nucleic acid binding domains and methods of use thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012104729A1 (en) * 2011-02-02 2012-08-09 Ulla Bonas Modular dna-binding domains and methods of use
WO2014059173A2 (en) * 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
WO2017069958A2 (en) * 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6919204B2 (en) * 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
JP6144691B2 (ja) * 2011-11-16 2017-06-07 サンガモ セラピューティクス， インコーポレイテッド 修飾されたｄｎａ結合タンパク質およびその使用
PL2890780T3 (pl) * 2012-08-29 2021-02-08 Sangamo Therapeutics, Inc. Sposoby i kompozycje do leczenia zaburzeń genetycznych
PL3068881T3 (pl) * 2013-11-13 2019-08-30 Children's Medical Center Corporation Regulacja ekspresji genów, w której pośredniczą nukleazy
WO2016019144A2 (en) * 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CA3097648A1 (en) * 2018-04-18 2019-10-24 Altius Institute For Biomedical Sciences Animal pathogen-derived polypeptides and uses thereof for genetic engineering

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012104729A1 (en) * 2011-02-02 2012-08-09 Ulla Bonas Modular dna-binding domains and methods of use
WO2014059173A2 (en) * 2012-10-10 2014-04-17 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
WO2017069958A2 (en) * 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology

Also Published As

Publication number Publication date
WO2021026336A2 (en) 2021-02-11
EP4010004A2 (de) 2022-06-15
WO2021026336A3 (en) 2021-03-11

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