JP2022553824A - 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

Info

Publication number
JP2022553824A
JP2022553824A JP2022525803A JP2022525803A JP2022553824A JP 2022553824 A JP2022553824 A JP 2022553824A JP 2022525803 A JP2022525803 A JP 2022525803A JP 2022525803 A JP2022525803 A JP 2022525803A JP 2022553824 A JP2022553824 A JP 2022553824A
Authority
JP
Japan
Prior art keywords
seq
nucleic acid
vestibular
cells
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
JP2022525803A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022553824A5 (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
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 JP2022553824A publication Critical patent/JP2022553824A/ja
Publication of JP2022553824A5 publication Critical patent/JP2022553824A5/ja
Pending legal-status Critical Current

Links

Images

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
    • 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
    • A61K48/005—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • 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
    • 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
    • C12N15/861—Adenoviral 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
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022525803A 2019-11-04 2020-11-04 前庭支持細胞プロモーター及びその使用 Pending JP2022553824A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962930520P 2019-11-04 2019-11-04
US62/930,520 2019-11-04
US202063024959P 2020-05-14 2020-05-14
US63/024,959 2020-05-14
PCT/US2020/058795 WO2021091950A1 (en) 2019-11-04 2020-11-04 Vestibular supporting cell promoters and uses thereof

Publications (2)

Publication Number Publication Date
JP2022553824A true JP2022553824A (ja) 2022-12-26
JP2022553824A5 JP2022553824A5 (https=) 2023-11-10

Family

ID=75848663

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022525803A Pending JP2022553824A (ja) 2019-11-04 2020-11-04 前庭支持細胞プロモーター及びその使用

Country Status (6)

Country Link
US (1) US20220331449A1 (https=)
EP (1) EP4076467A4 (https=)
JP (1) JP2022553824A (https=)
CN (2) CN119391775A (https=)
TW (1) TWI902721B (https=)
WO (1) WO2021091950A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220054640A1 (en) * 2018-12-11 2022-02-24 Decibel Therapeutics, Inc. Compositions and methods for the delivery of therapeutic agents across the round window membrane
CN118043468A (zh) * 2021-06-11 2024-05-14 分贝治疗公司 用于内耳中的细胞类型特异性基因表达的组合物和方法

Citations (4)

* 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
WO2018145111A1 (en) * 2017-02-06 2018-08-09 Children's Medical Center Corporation Materials and methods for delivering nucleic acids to cochlear and vestibular cells
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002082904A2 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
US20070161031A1 (en) * 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
WO2008076556A2 (en) * 2006-11-15 2008-06-26 Massachusetts Eye & Ear Infirmary Generation of inner ear cells
WO2009108274A2 (en) * 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
WO2012149038A1 (en) * 2011-04-25 2012-11-01 Advanced Bioscience Laboratories, Inc. Truncated hiv envelope proteins (env), methods and compositions related thereto
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
US20150209406A1 (en) * 2012-09-07 2015-07-30 Massachusetts Eye And Ear Infirmary Methods and compositions for regenerating hair cells and/or supporting cells
EP3407901B1 (en) * 2016-01-29 2021-07-21 Massachusetts Eye & Ear Infirmary Expansion and differentiation of inner ear supporting cells and methods of use thereof
US11191847B2 (en) * 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
US20190345516A1 (en) * 2018-03-12 2019-11-14 Musc Foundation For Research Development Methods for treating eye disease

Patent Citations (4)

* 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
WO2018145111A1 (en) * 2017-02-06 2018-08-09 Children's Medical Center Corporation Materials and methods for delivering nucleic acids to cochlear and vestibular cells

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
"Definition: Homo sapiens solute carrier family 6 member 14 (SLC6A14), RefSeqGene on chromosome X", DATABASE：NCBI[ONLINE], ACCESSION NO. NG_021305.2, JPN7025003439, 24 August 2019 (2019-08-24), ISSN: 0005774603 *
ACTA OTO-LARYNGOLOGICA, vol. 130 (2), JPN7024004582, 2010, pages 215 - 222, ISSN: 0005656484 *
ADVANCES IN OTO-RHINO-LARYNGOLOGY, vol. 66, JPN7024004581, 2009, pages 52 - 63, ISSN: 0005656483 *
GENETICS, vol. 177, JPN7024004580, 2007, pages 631 - 653, ISSN: 0005656486 *
HEARING RESEARCH, vol. 276 (1-2), JPN7024004579, 2011, pages 44 - 51, ISSN: 0005656485 *

Also Published As

Publication number Publication date
TWI902721B (zh) 2025-11-01
CN115052634B (zh) 2024-11-12
CN119391775A (zh) 2025-02-07
EP4076467A4 (en) 2023-12-27
WO2021091950A1 (en) 2021-05-14
TW202124719A (zh) 2021-07-01
US20220331449A1 (en) 2022-10-20
EP4076467A1 (en) 2022-10-26
CN115052634A (zh) 2022-09-13

Similar Documents

Publication Publication Date Title
JP7686709B2 (ja) ミオシン１５プロモーター及びその使用
US20250304637A1 (en) Myosin 15 promoters and uses thereof
US20220288236A1 (en) Cochlear outer hair cell promoters and uses thereof
US12545930B2 (en) Cochlear inner hair cell promoters and uses thereof
JP2024538076A (ja) ステレオシリンプロモーター及びその使用
US20220331449A1 (en) Vestibular supporting cell promoters and uses thereof
US20230181767A1 (en) Compositions and methods for promoting hair cell regeneration
US20240218391A1 (en) Vestibular supporting cell promoters and uses thereof
EP4093410A1 (en) Methods and compositions for generating type i vestibular hair cells
KR20260026049A (ko) 청력 장애에 대한 유전자 치료

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20231101

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20231101

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20241021

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241029

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250116

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250428

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250805

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251031

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20260120