CN113301909B - Htt阻遏物及其应用 - Google Patents

Htt阻遏物及其应用 Download PDF

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Publication number
CN113301909B
CN113301909B CN202080009478.4A CN202080009478A CN113301909B CN 113301909 B CN113301909 B CN 113301909B CN 202080009478 A CN202080009478 A CN 202080009478A CN 113301909 B CN113301909 B CN 113301909B
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CN
China
Prior art keywords
zfp
protein
arg
mhtt
sequence
Prior art date
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Active
Application number
CN202080009478.4A
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English (en)
Chinese (zh)
Other versions
CN113301909A (zh
Inventor
G·凯瑞
M·齐奥克
V·乔
B·费利斯
S·弗洛里奇
D·克拉缇
J·米勒
D·帕斯乔恩
E·瑞巴
B·泽特勒
L·张
H·S·张
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.)
Takeda Pharmaceutical Co Ltd
Sangamo Therapeutics Inc
Original Assignee
Takeda Pharmaceutical Co Ltd
Sangamo Therapeutics Inc
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Publication of CN113301909A publication Critical patent/CN113301909A/zh
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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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • 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
    • C07K2319/80—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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202080009478.4A 2019-01-15 2020-01-15 Htt阻遏物及其应用 Active CN113301909B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962792701P 2019-01-15 2019-01-15
US62/792,701 2019-01-15
PCT/US2020/013661 WO2020150338A1 (en) 2019-01-15 2020-01-15 Htt repressors and uses thereof

Publications (2)

Publication Number Publication Date
CN113301909A CN113301909A (zh) 2021-08-24
CN113301909B true CN113301909B (zh) 2025-01-28

Family

ID=71614037

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080009478.4A Active CN113301909B (zh) 2019-01-15 2020-01-15 Htt阻遏物及其应用

Country Status (5)

Country Link
US (1) US20220064237A1 (https=)
EP (1) EP3911349A4 (https=)
JP (1) JP7542912B2 (https=)
CN (1) CN113301909B (https=)
WO (1) WO2020150338A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019204457A1 (en) 2018-04-18 2019-10-24 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (htt)
CN115487315B (zh) * 2022-04-20 2023-08-04 暨南大学 治疗亨廷顿病的药物
WO2025004001A1 (en) * 2023-06-30 2025-01-02 Takeda Pharmaceutical Company Limited Htt repressors and uses thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2769262A1 (en) * 2009-07-28 2011-02-10 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7888121B2 (en) * 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) * 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
JP5266210B2 (ja) * 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
CN108285491B (zh) 2012-02-29 2021-08-10 桑格摩生物科学股份有限公司 治疗亨廷顿氏病的方法和组合物
WO2015070212A1 (en) * 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
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
RU2691102C2 (ru) * 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
WO2017053753A1 (en) 2015-09-23 2017-03-30 Sangamo Biosciences, Inc. Htt repressors and uses thereof
GB2544270A (en) * 2015-11-05 2017-05-17 Fundació Centre De Regulació Genòmica Nucleic acids, peptides and methods
WO2019204457A1 (en) * 2018-04-18 2019-10-24 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (htt)

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2769262A1 (en) * 2009-07-28 2011-02-10 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders

Also Published As

Publication number Publication date
EP3911349A1 (en) 2021-11-24
WO2020150338A1 (en) 2020-07-23
JP7542912B2 (ja) 2024-09-02
EP3911349A4 (en) 2023-01-18
US20220064237A1 (en) 2022-03-03
JP2022517988A (ja) 2022-03-11
CN113301909A (zh) 2021-08-24

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Effective date of registration: 20211206

Address after: California, USA

Applicant after: Sangamo Therapeutics, Inc.

Applicant after: TAKEDA PHARMACEUTICAL Co.,Ltd.

Address before: California, USA

Applicant before: Sangamo Therapeutics, Inc.

Applicant before: Shire Human Genetic Therapies, Inc.

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SE01 Entry into force of request for substantive examination
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