TWI888417B - 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之鋅指蛋白轉錄因子 - Google Patents

用於抑制α-突觸核蛋白(SYNUCLEIN)表現之鋅指蛋白轉錄因子 Download PDF

Info

Publication number
TWI888417B
TWI888417B TW109134486A TW109134486A TWI888417B TW I888417 B TWI888417 B TW I888417B TW 109134486 A TW109134486 A TW 109134486A TW 109134486 A TW109134486 A TW 109134486A TW I888417 B TWI888417 B TW I888417B
Authority
TW
Taiwan
Prior art keywords
zfp
fusion protein
cdicgrkfa
tgekpfa
htkih
Prior art date
Application number
TW109134486A
Other languages
English (en)
Chinese (zh)
Other versions
TW202126681A (zh
Inventor
阿薩 哈塔米
布萊恩 齊特勒
雷 張
戴維 伊曼紐爾 帕肖恩
Original Assignee
美商聖加莫治療股份有限公司
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 美商聖加莫治療股份有限公司 filed Critical 美商聖加莫治療股份有限公司
Publication of TW202126681A publication Critical patent/TW202126681A/zh
Application granted granted Critical
Publication of TWI888417B publication Critical patent/TWI888417B/zh

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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/14071—Demonstrated in vivo effect
    • 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)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Psychiatry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Toxicology (AREA)
  • Psychology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW109134486A 2019-10-02 2020-10-05 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之鋅指蛋白轉錄因子 TWI888417B (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962909496P 2019-10-02 2019-10-02
US62/909,496 2019-10-02
US202062959153P 2020-01-09 2020-01-09
US62/959,153 2020-01-09

Publications (2)

Publication Number Publication Date
TW202126681A TW202126681A (zh) 2021-07-16
TWI888417B true TWI888417B (zh) 2025-07-01

Family

ID=73014653

Family Applications (2)

Application Number Title Priority Date Filing Date
TW109134486A TWI888417B (zh) 2019-10-02 2020-10-05 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之鋅指蛋白轉錄因子
TW114122441A TW202539725A (zh) 2019-10-02 2020-10-05 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之鋅指蛋白轉錄因子

Family Applications After (1)

Application Number Title Priority Date Filing Date
TW114122441A TW202539725A (zh) 2019-10-02 2020-10-05 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之鋅指蛋白轉錄因子

Country Status (10)

Country Link
US (1) US20220324928A1 (enExample)
EP (1) EP4041754A1 (enExample)
JP (1) JP2022551286A (enExample)
KR (1) KR20220071248A (enExample)
CN (1) CN114466867A (enExample)
AU (1) AU2020356962B2 (enExample)
CA (1) CA3156258A1 (enExample)
IL (1) IL291827A (enExample)
TW (2) TWI888417B (enExample)
WO (1) WO2021067871A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4731643A2 (en) * 2023-06-26 2026-04-29 Navega Therapeutics, Inc. Compositions and methods for reducing pain and inflammation through epigenetic modulation
WO2025264967A1 (en) * 2024-06-21 2025-12-26 The Johns Hopkins University Agents and/or compositions useful for modulating cis-regulatory elements in synucleinopathies and methods for identifying agents and compositions thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019152433A1 (en) * 2018-01-30 2019-08-08 Parkinson's Institute Crispr-based downregulation of alpha-synuclein expression as a novel parkinson's disease therapeutic

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
JP4012243B2 (ja) 1994-01-18 2007-11-21 ザ スクリップス リサーチ インスティチュート 亜鉛フィンガータンパク質誘導体およびそのための方法
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
CA2196419C (en) 1994-08-20 2007-08-21 Yen Choo Improvements in or relating to binding proteins for recognition of dna
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (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
US7030215B2 (en) * 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
AU2001263155A1 (en) 2000-05-16 2001-11-26 Massachusetts Institute Of Technology 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
US20050235369A1 (en) * 2001-03-28 2005-10-20 Yen Choo Gene regulation II
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
CA2442670A1 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
US20040224385A1 (en) 2001-08-20 2004-11-11 Barbas Carlos F Zinc finger binding domains for cnn
US7595306B2 (en) * 2003-06-09 2009-09-29 Alnylam Pharmaceuticals Inc Method of treating neurodegenerative disease
WO2005004794A2 (en) * 2003-06-09 2005-01-20 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease
AU2005233583B2 (en) * 2004-04-08 2011-02-03 Sangamo Therapeutics, Inc. Methods and compositions for modulating cardiac contractility
DK3246403T3 (da) 2005-10-18 2020-10-26 Prec Biosciences Rationelt designede meganucleaser med ændret sekvens-specificitet og dna-bindende affinitet
AU2009212247A1 (en) * 2008-02-08 2009-08-13 Sangamo Therapeutics, Inc. Treatment of chronic pain with zinc finger proteins
EP2566972B1 (en) 2010-05-03 2020-01-15 Sangamo Therapeutics, Inc. Compositions for linking zinc finger modules
WO2012094132A1 (en) * 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
BR112014020325A2 (pt) * 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
IL264792B2 (en) 2016-08-24 2023-10-01 Sangamo Therapeutics Inc Engineered target-specific zinc-finger 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
US20190345516A1 (en) * 2018-03-12 2019-11-14 Musc Foundation For Research Development Methods for treating eye disease
TW202134288A (zh) * 2020-01-22 2021-09-16 美商聖加莫治療股份有限公司 用於抑制tau蛋白表現之鋅指蛋白轉錄因子

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019152433A1 (en) * 2018-01-30 2019-08-08 Parkinson's Institute Crispr-based downregulation of alpha-synuclein expression as a novel parkinson's disease therapeutic

Also Published As

Publication number Publication date
WO2021067871A1 (en) 2021-04-08
AU2020356962A1 (en) 2022-04-14
AU2020356962B2 (en) 2026-08-06
US20220324928A1 (en) 2022-10-13
EP4041754A1 (en) 2022-08-17
CA3156258A1 (en) 2021-04-08
WO2021067871A9 (en) 2021-05-06
KR20220071248A (ko) 2022-05-31
TW202126681A (zh) 2021-07-16
TW202539725A (zh) 2025-10-16
IL291827A (en) 2022-06-01
CN114466867A (zh) 2022-05-10
AU2020356962A9 (en) 2026-07-30
JP2022551286A (ja) 2022-12-08

Similar Documents

Publication Publication Date Title
JP7824873B2 (ja) プリオン病の治療のためのジンクフィンガータンパク質転写因子
US20230242602A1 (en) Zinc finger protein transcription factors for repressing tau expression
CN113766921B (zh) 染色体9开放阅读框72基因表达的调节物及其用途
US20220324928A1 (en) Zinc Finger Protein Transcription Factors for Repressing Alpha-Synuclein Expression
US20240018203A1 (en) Novel zinc finger protein transcription factors for repressing alpha-synuclein expression
RU2828216C2 (ru) Модуляторы экспрессии гена открытой рамки считывания 72 хромосомы 9 и их применения
TW202547548A (zh) 使用血腦屏障穿透性蛋白殼之ｔａｕ抑制子遞送
CN116761812A (zh) Neurod1和dlx2载体