JP7807393B2 - 調節核酸配列 - Google Patents

調節核酸配列

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Publication number
JP7807393B2
JP7807393B2 JP2022564157A JP2022564157A JP7807393B2 JP 7807393 B2 JP7807393 B2 JP 7807393B2 JP 2022564157 A JP2022564157 A JP 2022564157A JP 2022564157 A JP2022564157 A JP 2022564157A JP 7807393 B2 JP7807393 B2 JP 7807393B2
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Prior art keywords
cns
promoter
seq
expression
synthetic
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JP2022564157A
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English (en)
Japanese (ja)
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JP2023525476A (ja
JP2023525476A5 (enExample
Inventor
トニー・オーステルフェーン
アン・ブラーエ
シンクレア・クーパー
フアン・マヌエル・イグレシアス
マイケル・エル・ロバーツ
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アスクバイオ インコーポレイテッド
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Publication of JP2023525476A publication Critical patent/JP2023525476A/ja
Publication of JP2023525476A5 publication Critical patent/JP2023525476A5/ja
Priority to JP2026005291A priority Critical patent/JP2026082853A/ja
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Publication of JP7807393B2 publication Critical patent/JP7807393B2/ja
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Classifications

    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • 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
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2750/14111—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
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022564157A 2020-04-20 2021-04-19 調節核酸配列 Active JP7807393B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2026005291A JP2026082853A (ja) 2020-04-20 2026-01-15 調節核酸配列

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB2005732.9 2020-04-20
GBGB2005732.9A GB202005732D0 (en) 2020-04-20 2020-04-20 Regulatory nucleic acid sequences
PCT/GB2021/050939 WO2021214443A1 (en) 2020-04-20 2021-04-19 Regulatory nucleic acid sequences

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2026005291A Division JP2026082853A (ja) 2020-04-20 2026-01-15 調節核酸配列

Publications (3)

Publication Number Publication Date
JP2023525476A JP2023525476A (ja) 2023-06-16
JP2023525476A5 JP2023525476A5 (enExample) 2024-05-01
JP7807393B2 true JP7807393B2 (ja) 2026-01-27

Family

ID=70860129

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022564157A Active JP7807393B2 (ja) 2020-04-20 2021-04-19 調節核酸配列
JP2026005291A Pending JP2026082853A (ja) 2020-04-20 2026-01-15 調節核酸配列

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2026005291A Pending JP2026082853A (ja) 2020-04-20 2026-01-15 調節核酸配列

Country Status (8)

Country Link
US (1) US20230143758A1 (enExample)
EP (1) EP4139466A1 (enExample)
JP (2) JP7807393B2 (enExample)
CN (1) CN115702246B (enExample)
AU (1) AU2021260914A1 (enExample)
CA (1) CA3176038A1 (enExample)
GB (1) GB202005732D0 (enExample)
WO (1) WO2021214443A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023211606A1 (en) * 2022-01-27 2024-07-11 AskBio Inc. Compositions for treating neurological disease
AU2023226080A1 (en) * 2022-02-24 2024-10-03 Allen Institute Artificial expression constructs for modulating gene expression in dopaminergic neurons

Citations (3)

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Publication number Priority date Publication date Assignee Title
US20100129903A1 (en) 2008-08-11 2010-05-27 Simpson Elizabeth M Pitx3 expression promoters
JP2013531471A (ja) 2010-04-23 2013-08-08 ユニバーシティ オブ マサチューセッツ Ｃｎｓターゲティングａａｖベクターおよびその使用方法
US20140364336A1 (en) 2009-09-16 2014-12-11 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods of use

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FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
EP0968284B1 (en) 1996-11-20 2006-12-13 Introgen Therapeutics, Inc. An improved method for the production and purification of adenoviral vectors
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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100129903A1 (en) 2008-08-11 2010-05-27 Simpson Elizabeth M Pitx3 expression promoters
US20140364336A1 (en) 2009-09-16 2014-12-11 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods of use
JP2013531471A (ja) 2010-04-23 2013-08-08 ユニバーシティ オブ マサチューセッツ Ｃｎｓターゲティングａａｖベクターおよびその使用方法

Also Published As

Publication number Publication date
CN115702246B (zh) 2026-06-05
GB202005732D0 (en) 2020-06-03
JP2023525476A (ja) 2023-06-16
US20230143758A1 (en) 2023-05-11
EP4139466A1 (en) 2023-03-01
AU2021260914A1 (en) 2022-12-08
JP2026082853A (ja) 2026-05-19
WO2021214443A1 (en) 2021-10-28
CA3176038A1 (en) 2021-10-28
CN115702246A (zh) 2023-02-14

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