AU2021209699A1 - Zinc finger protein transcription factors for repressing tau expression - Google Patents

Zinc finger protein transcription factors for repressing tau expression Download PDF

Info

Publication number
AU2021209699A1
AU2021209699A1 AU2021209699A AU2021209699A AU2021209699A1 AU 2021209699 A1 AU2021209699 A1 AU 2021209699A1 AU 2021209699 A AU2021209699 A AU 2021209699A AU 2021209699 A AU2021209699 A AU 2021209699A AU 2021209699 A1 AU2021209699 A1 AU 2021209699A1
Authority
AU
Australia
Prior art keywords
zfp
fusion protein
cell
dna
tau
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
AU2021209699A
Other languages
English (en)
Inventor
Asa HATAMI
Bryan Zeitler
Lei Zhang
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.)
Sangamo Therapeutics Inc
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 AU2021209699A1 publication Critical patent/AU2021209699A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • 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
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Management, Administration, Business Operations System, And Electronic Commerce (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2021209699A 2020-01-22 2021-01-22 Zinc finger protein transcription factors for repressing tau expression Pending AU2021209699A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062964501P 2020-01-22 2020-01-22
US62/964,501 2020-01-22
PCT/US2021/014780 WO2021151012A1 (en) 2020-01-22 2021-01-22 Zinc finger protein transcription factors for repressing tau expression

Publications (1)

Publication Number Publication Date
AU2021209699A1 true AU2021209699A1 (en) 2022-07-21

Family

ID=74661487

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021209699A Pending AU2021209699A1 (en) 2020-01-22 2021-01-22 Zinc finger protein transcription factors for repressing tau expression

Country Status (14)

Country Link
US (1) US20230242602A1 (https=)
EP (1) EP4093754A1 (https=)
JP (1) JP2023511908A (https=)
KR (1) KR20220131273A (https=)
CN (1) CN115210251A (https=)
AR (1) AR121118A1 (https=)
AU (1) AU2021209699A1 (https=)
BR (1) BR112022014160A2 (https=)
CA (1) CA3168805A1 (https=)
IL (1) IL294535A (https=)
MX (1) MX2022008953A (https=)
TW (1) TW202134288A (https=)
UY (1) UY39036A (https=)
WO (1) WO2021151012A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110214184B (zh) 2016-12-01 2024-07-02 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
KR20210070344A (ko) 2018-10-02 2021-06-14 상가모 테라퓨틱스, 인코포레이티드 타우 단백질의 조정을 위한 방법 및 조성물
KR20220071248A (ko) * 2019-10-02 2022-05-31 상가모 테라퓨틱스, 인코포레이티드 알파-시누클레인 발현을 억제시키기 위한 아연 핑거 단백질 전사 인자
WO2025193848A1 (en) * 2024-03-13 2025-09-18 Sangamo Therapeutics, Inc. Delivery of tau repressors using the blood-brain barrier penetrant capsid

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69534629D1 (de) 1994-01-18 2005-12-29 Scripps Research Inst Derivate von zinkfingerproteinen und methoden
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
ATE524545T1 (de) 1994-08-20 2011-09-15 Gendaq Ltd Verbesserungen bei oder im zusammenhang mit bindeproteinen zur erkennung von dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
DE69942334D1 (de) 1998-03-02 2010-06-17 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
WO2001053480A1 (en) 2000-01-24 2001-07-26 Sangamo Biosciences, Inc. Nucleic acid binding polypeptides characterized by flexible linkers connected nucleic acid binding modules
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US9394531B2 (en) 2008-05-28 2016-07-19 Sangamo Biosciences, Inc. Compositions for linking DNA-binding domains and cleavage domains
PT2566972T (pt) 2010-05-03 2020-03-02 Sangamo Biosciences Inc Composições para a ligação de módulos de dedo de zinco
AU2014312295C1 (en) 2013-08-28 2020-08-13 Sangamo Therapeutics, Inc. Compositions for linking DNA-binding domains and cleavage domains
CN106459894B (zh) 2014-03-18 2020-02-18 桑格摩生物科学股份有限公司 用于调控锌指蛋白表达的方法和组合物
WO2017011556A1 (en) * 2015-07-13 2017-01-19 The General Hospital Corporation Rare phosphorylated high molecular weight (hmw) tau species that are involved in neuronal uptake and propagation and applications thereof
EP3411056A4 (en) 2016-02-02 2019-10-02 Sangamo Therapeutics, Inc. COMPOUNDS FOR NETWORKING DNA BINDING DOMAINS AND SPLITTING DOMAINS
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
CN110214184B (zh) * 2016-12-01 2024-07-02 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
CA3115158A1 (en) 2018-10-02 2020-04-09 Sangamo Therapeutics, Inc. Engineered genetic modulators
KR20210070344A (ko) 2018-10-02 2021-06-14 상가모 테라퓨틱스, 인코포레이티드 타우 단백질의 조정을 위한 방법 및 조성물

Also Published As

Publication number Publication date
CN115210251A (zh) 2022-10-18
JP2023511908A (ja) 2023-03-23
KR20220131273A (ko) 2022-09-27
TW202134288A (zh) 2021-09-16
EP4093754A1 (en) 2022-11-30
IL294535A (en) 2022-09-01
AR121118A1 (es) 2022-04-20
BR112022014160A2 (pt) 2022-09-13
UY39036A (es) 2021-08-31
MX2022008953A (es) 2022-08-15
CA3168805A1 (en) 2021-07-29
WO2021151012A1 (en) 2021-07-29
US20230242602A1 (en) 2023-08-03

Similar Documents

Publication Publication Date Title
US11060113B2 (en) Gene therapies for lysosomal disorders
US20230242602A1 (en) Zinc finger protein transcription factors for repressing tau expression
WO2023240236A1 (en) Compositions and methods for the treatment of spinal muscular atrophy related disorders
US20250066433A1 (en) Modulators of Chromosome 9 Open Reading Frame 72 Gene Expression and Uses Thereof
JP2025539750A (ja) 前頭側頭型認知症の遺伝子治療
EP4041271A1 (en) Zinc finger protein transcription factors for treatment of prion disease
TWI888417B (zh) 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之鋅指蛋白轉錄因子
EP4698233A2 (en) Zinc finger protein transcription factors for repressing nav1.7 expression
US20240018203A1 (en) Novel zinc finger protein transcription factors for repressing alpha-synuclein expression
JP2023522852A (ja) Ｉｄｓ遺伝子導入のためのアデノ随伴ウイルス組成物及びその使用方法
WO2025193848A1 (en) Delivery of tau repressors using the blood-brain barrier penetrant capsid
RU2828216C2 (ru) Модуляторы экспрессии гена открытой рамки считывания 72 хромосомы 9 и их применения