JP2024517250A - 前庭支持細胞プロモーター及びその使用 - Google Patents

前庭支持細胞プロモーター及びその使用 Download PDF

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Publication number
JP2024517250A
JP2024517250A JP2023568110A JP2023568110A JP2024517250A JP 2024517250 A JP2024517250 A JP 2024517250A JP 2023568110 A JP2023568110 A JP 2023568110A JP 2023568110 A JP2023568110 A JP 2023568110A JP 2024517250 A JP2024517250 A JP 2024517250A
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JP
Japan
Prior art keywords
nucleic acid
vestibular
seq
acid vector
protein
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
JP2023568110A
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English (en)
Japanese (ja)
Other versions
JP2024517250A5 (https=
Inventor
バーンズ、ジョセフ
ギブソン、タイラー
プレーゲルニッヒ、ガブリエラ
ソー、キャシー
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.)
Decibel Therapeutics Inc
Original Assignee
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 Decibel Therapeutics Inc filed Critical Decibel Therapeutics Inc
Publication of JP2024517250A publication Critical patent/JP2024517250A/ja
Publication of JP2024517250A5 publication Critical patent/JP2024517250A5/ja
Pending legal-status Critical Current

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Classifications

    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Display Devices Of Pinball Game Machines (AREA)
  • Absorbent Articles And Supports Therefor (AREA)
  • Orthopedics, Nursing, And Contraception (AREA)
JP2023568110A 2021-05-04 2022-05-04 前庭支持細胞プロモーター及びその使用 Pending JP2024517250A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163184015P 2021-05-04 2021-05-04
US63/184,015 2021-05-04
PCT/US2022/027679 WO2022235805A1 (en) 2021-05-04 2022-05-04 Vestibular supporting cell promoters and uses thereof

Publications (2)

Publication Number Publication Date
JP2024517250A true JP2024517250A (ja) 2024-04-19
JP2024517250A5 JP2024517250A5 (https=) 2025-05-09

Family

ID=83932529

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023568110A Pending JP2024517250A (ja) 2021-05-04 2022-05-04 前庭支持細胞プロモーター及びその使用

Country Status (13)

Country Link
US (1) US20240218391A1 (https=)
EP (1) EP4334460A4 (https=)
JP (1) JP2024517250A (https=)
KR (1) KR20240031224A (https=)
CN (1) CN117616127A (https=)
AU (1) AU2022271238A1 (https=)
BR (1) BR112023023092A2 (https=)
CA (1) CA3219057A1 (https=)
CL (2) CL2023003257A1 (https=)
CO (1) CO2023016746A2 (https=)
IL (1) IL308143A (https=)
MX (1) MX2023013058A (https=)
WO (1) WO2022235805A1 (https=)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140024663A1 (en) * 2012-07-17 2014-01-23 Georgia Health Sciences University Research Institute, Inc. Atb(0,+) amino acid transporter as a drug target for treatment of estrogen receptor-positive breast cancer
US20160298080A1 (en) * 2013-12-03 2016-10-13 The Johns Hopkins University Method for highly efficient conversion of human stem cells to lineage-specific neurons
JP2018536420A (ja) * 2015-12-11 2018-12-13 マサチューセッツ アイ アンド イヤー インファーマリー 蝸牛および前庭細胞に核酸を送達するための材料および方法

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140024663A1 (en) * 2012-07-17 2014-01-23 Georgia Health Sciences University Research Institute, Inc. Atb(0,+) amino acid transporter as a drug target for treatment of estrogen receptor-positive breast cancer
US20160298080A1 (en) * 2013-12-03 2016-10-13 The Johns Hopkins University Method for highly efficient conversion of human stem cells to lineage-specific neurons
JP2018536420A (ja) * 2015-12-11 2018-12-13 マサチューセッツ アイ アンド イヤー インファーマリー 蝸牛および前庭細胞に核酸を送達するための材料および方法

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
"Mouse DNA sequence from clone RP23-99K18 on chromosome X, complete sequence", GENBANK [ONLINE], JPN6026008979, 13 December 2012 (2012-12-13), ISSN: 0005813226 *
"Mus musculus targeted KO-first, conditional ready, lacZ-tagged mutant allele Slc6a14:tm1a(KOMP)Wtsi;", GENBANK [ONLINE], JPN6026008977, 5 November 2011 (2011-11-05), ISSN: 0005813224 *
"Mus musculus targeted non-conditional, lacZ-tagged mutant allele Slc6a14:tm1e(KOMP)Wtsi; transgenic", GENBANK [ONLINE], JPN6026008976, 5 November 2011 (2011-11-05), ISSN: 0005813223 *
GENETICS, vol. 177, JPN7024004580, 2007, pages 631 - 653, ISSN: 0005813229 *
HEARING RESEARCH, vol. Vol. 276, No. 1-2, JPN7024004579, 2011, pages 44 - 51, ISSN: 0005813228 *
MUS MUSCULUS SOLUTE CARRIER FAMILY 6 (NEUROTRANSMITTER TRANSPORTER), MEMBER 14 (SLC6A14), MRNA, JPN6026008980, 10 April 2021 (2021-04-10), ISSN: 0005813227 *
NATURE, vol. 474, no. 7351, JPN6026008978, 2011, pages 337 - 342, ISSN: 0005813225 *

Also Published As

Publication number Publication date
AU2022271238A9 (en) 2024-01-04
CN117616127A (zh) 2024-02-27
BR112023023092A2 (pt) 2024-01-30
WO2022235805A1 (en) 2022-11-10
IL308143A (en) 2023-12-01
CO2023016746A2 (es) 2024-02-26
MX2023013058A (es) 2023-12-14
CL2024002594A1 (es) 2025-01-03
EP4334460A1 (en) 2024-03-13
AU2022271238A1 (en) 2023-12-14
KR20240031224A (ko) 2024-03-07
EP4334460A4 (en) 2025-11-05
US20240218391A1 (en) 2024-07-04
CA3219057A1 (en) 2022-11-10
CL2023003257A1 (es) 2024-05-31

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