JP2025506419A - Ｇｊｂ２調節エレメント及びその使用 - Google Patents

Ｇｊｂ２調節エレメント及びその使用 Download PDF

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Publication number
JP2025506419A
JP2025506419A JP2024546193A JP2024546193A JP2025506419A JP 2025506419 A JP2025506419 A JP 2025506419A JP 2024546193 A JP2024546193 A JP 2024546193A JP 2024546193 A JP2024546193 A JP 2024546193A JP 2025506419 A JP2025506419 A JP 2025506419A
Authority
JP
Japan
Prior art keywords
seq
gjb2
polynucleotide
sequence identity
promoter
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
JP2024546193A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023150689A5 (https=
JP2025506419A5 (https=
Inventor
バーンズ、ジョセフ
エリス、キャサリン
ギブソン、タイラー
レボ、ケビン
プレーゲルニッヒ、ガブリエラ
サミュエルソン、メーガン ドラモンド
カンセラリッチ、サラ
サビン、レア
バッティスタ ミアーニ、ダニエラ ディ
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.)
Regeneron Pharmaceuticals Inc
Decibel Therapeutics Inc
Original Assignee
Regeneron Pharmaceuticals Inc
Decibel 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 Regeneron Pharmaceuticals Inc, Decibel Therapeutics Inc filed Critical Regeneron Pharmaceuticals Inc
Publication of JP2025506419A publication Critical patent/JP2025506419A/ja
Publication of JPWO2023150689A5 publication Critical patent/JPWO2023150689A5/ja
Publication of JP2025506419A5 publication Critical patent/JP2025506419A5/ja
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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0075—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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0046—Ear
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2024546193A 2022-02-04 2023-02-03 Ｇｊｂ２調節エレメント及びその使用 Pending JP2025506419A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263306928P 2022-02-04 2022-02-04
US202263306941P 2022-02-04 2022-02-04
US63/306,928 2022-02-04
US63/306,941 2022-02-04
PCT/US2023/061953 WO2023150689A2 (en) 2022-02-04 2023-02-03 Gjb2 regulatory elements and uses thereof

Publications (3)

Publication Number Publication Date
JP2025506419A true JP2025506419A (ja) 2025-03-11
JPWO2023150689A5 JPWO2023150689A5 (https=) 2026-02-04
JP2025506419A5 JP2025506419A5 (https=) 2026-02-04

Family

ID=87552983

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024546193A Pending JP2025506419A (ja) 2022-02-04 2023-02-03 Ｇｊｂ２調節エレメント及びその使用

Country Status (11)

Country Link
US (1) US20250127928A1 (https=)
EP (1) EP4473005A4 (https=)
JP (1) JP2025506419A (https=)
KR (1) KR20240156606A (https=)
AU (1) AU2023214481A1 (https=)
CA (1) CA3250345A1 (https=)
CL (1) CL2024002306A1 (https=)
CO (1) CO2024012057A2 (https=)
IL (1) IL314705A (https=)
MX (1) MX2024009520A (https=)
WO (1) WO2023150689A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2026025095A2 (en) 2024-07-26 2026-01-29 Regeneron Pharmaceuticals, Inc. Otic drug delivery system and methods

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3159549A1 (en) * 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP4164695A4 (en) * 2020-05-13 2024-06-05 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS

Also Published As

Publication number Publication date
CA3250345A1 (en) 2023-08-10
CL2024002306A1 (es) 2024-12-27
IL314705A (en) 2024-10-01
MX2024009520A (es) 2024-09-11
WO2023150689A2 (en) 2023-08-10
WO2023150689A8 (en) 2023-10-12
KR20240156606A (ko) 2024-10-30
CO2024012057A2 (es) 2024-09-30
EP4473005A4 (en) 2026-02-18
WO2023150689A3 (en) 2023-11-16
US20250127928A1 (en) 2025-04-24
AU2023214481A1 (en) 2024-08-08
EP4473005A2 (en) 2024-12-11

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Effective date: 20260127

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