SG11202103314QA - Engineered genetic modulators - Google Patents

Engineered genetic modulators

Info

Publication number
SG11202103314QA
SG11202103314QA SG11202103314QA SG11202103314QA SG11202103314QA SG 11202103314Q A SG11202103314Q A SG 11202103314QA SG 11202103314Q A SG11202103314Q A SG 11202103314QA SG 11202103314Q A SG11202103314Q A SG 11202103314QA SG 11202103314Q A SG11202103314Q A SG 11202103314QA
Authority
SG
Singapore
Prior art keywords
engineered genetic
genetic modulators
modulators
engineered
genetic
Prior art date
Application number
SG11202103314QA
Other languages
English (en)
Inventor
Jeffrey C Miller
Bryan Zeitler
Original Assignee
Sangamo Therapeutics Inc
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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of SG11202103314QA publication Critical patent/SG11202103314QA/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1003Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007Methyltransferases (general) (2.1.1.)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1025Acyltransferases (2.3)
    • C12N9/1029Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
SG11202103314QA 2018-10-02 2019-10-02 Engineered genetic modulators SG11202103314QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862740156P 2018-10-02 2018-10-02
PCT/US2019/054347 WO2020072684A1 (fr) 2018-10-02 2019-10-02 Modulateurs génétiques modifiés

Publications (1)

Publication Number Publication Date
SG11202103314QA true SG11202103314QA (en) 2021-04-29

Family

ID=70051837

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202103314QA SG11202103314QA (en) 2018-10-02 2019-10-02 Engineered genetic modulators

Country Status (10)

Country Link
US (1) US20200109406A1 (fr)
EP (1) EP3861130A4 (fr)
JP (1) JP2022504075A (fr)
KR (1) KR20210069692A (fr)
CN (1) CN113195002A (fr)
AU (1) AU2019354743A1 (fr)
CA (1) CA3115158A1 (fr)
IL (1) IL281950A (fr)
SG (1) SG11202103314QA (fr)
WO (1) WO2020072684A1 (fr)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163628A2 (fr) 2012-04-27 2013-10-31 Duke University Correction génétique de gènes ayant subi une mutation
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Rnaガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
EP4089175A1 (fr) 2015-10-13 2022-11-16 Duke University Ingénierie génomique avec systèmes crispr de type i dans des cellules eucaryotes
CN110214184A (zh) 2016-12-01 2019-09-06 桑格摩生物治疗股份有限公司 τ蛋白调节剂以及用于其递送的方法和组合物
JP2021521788A (ja) 2018-04-19 2021-08-30 ザ・リージエンツ・オブ・ザ・ユニバーシテイー・オブ・カリフオルニア ゲノム編集のための組成物および方法
WO2021151012A1 (fr) 2020-01-22 2021-07-29 Sangamo Therapeutics, Inc. Facteurs de transcription de protéines à doigt de zinc pour réprimer l'expression de la protéine tau
BR112022026885A2 (pt) * 2020-07-09 2023-01-24 Modalis Therapeutics Corp Método para tratar doença de alzheimer por direcionamento de gene mapt
CN116917335A (zh) * 2020-10-02 2023-10-20 桑格摩生物治疗股份有限公司 用于阻遏α-突触核蛋白(SYNUCLEIN)表达的新颖锌指蛋白转录因子
WO2023184108A1 (fr) * 2022-03-28 2023-10-05 Huigene Therapeutics Co., Ltd. Système crispr-cas13 pour le traitement de maladies associées à ube3a
WO2023184107A1 (fr) * 2022-03-28 2023-10-05 Huigene Therapeutics Co., Ltd. Système crispr-cas13 pour le traitement de maladies associées à mecp2
WO2024032678A1 (fr) * 2022-08-11 2024-02-15 益杰立科(上海)生物科技有限公司 Procédé d'édition d'épigénome de cibles et son utilisation
WO2024032677A1 (fr) * 2022-08-11 2024-02-15 益杰立科(上海)生物科技有限公司 Procédé d'édition épigénétique d'un site cible et son utilisation
WO2024032679A1 (fr) * 2022-08-11 2024-02-15 益杰立科(上海)生物科技有限公司 Procédé et utilisation destinés à une cible d'édition apparente

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2049663T1 (sl) * 2006-08-11 2015-12-31 Dow Agrosciences Llc Homologna rekombinacija, posredovana z nukleazo s cinkovim prstom
JP5898179B2 (ja) * 2010-05-03 2016-04-06 サンガモ バイオサイエンシーズ, インコーポレイテッド ジンクフィンガーモジュールを連結するための組成物
MX359327B (es) * 2012-02-29 2018-09-25 Sangamo Biosciences Inc Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
WO2017197141A2 (fr) * 2016-05-13 2017-11-16 Sangamo Therapeutics, Inc. Traitement ciblé de l'alopécie androgénique
WO2018039471A2 (fr) * 2016-08-25 2018-03-01 Trustees Of Boston University Régulateurs transcriptionnels et épigénétiques synthétiques basés sur des protéines de doigt de zinc orthogonales modifiées
AU2017324462B2 (en) * 2016-09-07 2024-03-21 Sangamo Therapeutics, Inc. Modulation of liver genes
US11371023B2 (en) * 2016-11-22 2022-06-28 Wisconsin Alumni Research Foundation Artificial transcription factors and uses thereof
CN110214184A (zh) * 2016-12-01 2019-09-06 桑格摩生物治疗股份有限公司 τ蛋白调节剂以及用于其递送的方法和组合物

Also Published As

Publication number Publication date
IL281950A (en) 2021-05-31
CA3115158A1 (fr) 2020-04-09
US20200109406A1 (en) 2020-04-09
EP3861130A1 (fr) 2021-08-11
WO2020072684A1 (fr) 2020-04-09
EP3861130A4 (fr) 2022-08-03
AU2019354743A1 (en) 2021-05-06
JP2022504075A (ja) 2022-01-13
KR20210069692A (ko) 2021-06-11
CN113195002A (zh) 2021-07-30

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