CN118497276A - 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途 - Google Patents

腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途 Download PDF

Info

Publication number
CN118497276A
CN118497276A CN202410531995.8A CN202410531995A CN118497276A CN 118497276 A CN118497276 A CN 118497276A CN 202410531995 A CN202410531995 A CN 202410531995A CN 118497276 A CN118497276 A CN 118497276A
Authority
CN
China
Prior art keywords
amino acid
acid substitutions
amino acids
amino
cell target
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
CN202410531995.8A
Other languages
English (en)
Chinese (zh)
Inventor
E·J·西蒙斯
R·恩格
D·伦兹
M·瓦莱罗
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.)
Akus Co ltd
Original Assignee
Akus Co ltd
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 Akus Co ltd filed Critical Akus Co ltd
Publication of CN118497276A publication Critical patent/CN118497276A/zh
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/16Otologicals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/65Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression using markers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/03Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03001Alkaline phosphatase (3.1.3.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/40Systems of functionally co-operating vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202410531995.8A 2018-11-07 2019-11-07 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途 Pending CN118497276A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201862756953P 2018-11-07 2018-11-07
US62/756,953 2018-11-07
US201962888093P 2019-08-16 2019-08-16
US62/888,093 2019-08-16
PCT/US2019/060328 WO2020097372A1 (en) 2018-11-07 2019-11-07 Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear
CN201980082439.4A CN113853437A (zh) 2018-11-07 2019-11-07 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201980082439.4A Division CN113853437A (zh) 2018-11-07 2019-11-07 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途

Publications (1)

Publication Number Publication Date
CN118497276A true CN118497276A (zh) 2024-08-16

Family

ID=70611216

Family Applications (2)

Application Number Title Priority Date Filing Date
CN202410531995.8A Pending CN118497276A (zh) 2018-11-07 2019-11-07 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途
CN201980082439.4A Pending CN113853437A (zh) 2018-11-07 2019-11-07 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN201980082439.4A Pending CN113853437A (zh) 2018-11-07 2019-11-07 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途

Country Status (7)

Country Link
US (2) US20230056182A1 (https=)
EP (2) EP4063510A1 (https=)
JP (2) JP2022506613A (https=)
CN (2) CN118497276A (https=)
AU (1) AU2019377115A1 (https=)
CA (1) CA3119065A1 (https=)
WO (1) WO2020097372A1 (https=)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019530737A (ja) 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
MX2020006435A (es) 2017-12-19 2021-02-09 Akouos Inc Administracion de anticuerpos terapeuticos mediada por vector de virus adenoasociado (aav) en el oido interno.
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
AU2020218547A1 (en) 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
JP7807380B2 (ja) 2020-02-21 2026-01-27 アコーオス インコーポレイテッド ヒト対象において非加齢関連聴力障害を処置するための組成物および方法
EP4164695A4 (en) * 2020-05-13 2024-06-05 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS
US20240016955A1 (en) * 2020-09-14 2024-01-18 President And Fellows Of Harvard College Dual-aav vector delivery of pcdh15 and uses thereof
JP2023541443A (ja) * 2020-09-14 2023-10-02 プレジデント アンド フェローズ オブ ハーバード カレッジ GJB2をコードする組換えアデノ随伴ウイルス(rAAV)およびその使用。
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
AU2021376225A1 (en) * 2020-11-06 2023-06-22 Eli Lilly And Company Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss
IL303317A (en) * 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
MX2023007800A (es) 2020-12-29 2023-07-11 Akouos Inc Composiciones y metodos para tratar la perdida de audicion y/o la perdida de vision asociada a clrn1.
US20220267795A1 (en) * 2021-02-24 2022-08-25 Kinase Pharma Inc. Compositions and methods for regulating production of an angiogensis inhibitor
EP4473011A1 (en) * 2022-02-02 2024-12-11 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
KR20250080886A (ko) * 2022-09-30 2025-06-05 데시벨 테라퓨틱스, 인크. Slc26a4 조절 요소 및 이의 용도
WO2025179016A1 (en) * 2024-02-20 2025-08-28 Skylark Bio, Inc. Recombinant adeno associated virus and uses thereof

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0928202A4 (en) * 1996-09-25 2001-09-05 Cell Genesys Inc NON-INVASIVE ADMINISTRATION OF ADENO-ASSOCIATED VIRAL VECTORS
EP1082445A2 (en) * 1998-05-27 2001-03-14 Cell Genesys, Inc. Adeno-associated viral vector-mediated expression of factor viii activity
WO2011075838A1 (en) * 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
CA2890061A1 (en) * 2012-12-04 2014-06-12 Merck Sharp & Dohme Corp. Conditional replicating viral vectors
CA2909733C (en) * 2013-04-18 2023-10-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
TWI813532B (zh) * 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的crispr酶突變
SG11201804814YA (en) * 2015-12-11 2018-07-30 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2017136764A1 (en) * 2016-02-05 2017-08-10 The General Hospital Corporation Hybrid system for efficient gene delivery to cells of the inner ear
WO2017181021A1 (en) * 2016-04-15 2017-10-19 Regenxbio Inc. Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab
JP2019530737A (ja) * 2016-08-23 2019-10-24 アコーオス インコーポレイテッド ヒト対象において非加齢性聴力障害を治療するための組成物および方法
KR102746901B1 (ko) * 2017-03-03 2024-12-26 옵시디안 테라퓨틱스, 인크. Cd19 조성물 및 면역요법을 위한 방법

Also Published As

Publication number Publication date
WO2020097372A8 (en) 2021-04-29
US20230056182A1 (en) 2023-02-23
US20250041457A1 (en) 2025-02-06
EP4063510A1 (en) 2022-09-28
JP2024086735A (ja) 2024-06-28
CN113853437A (zh) 2021-12-28
JP2022506613A (ja) 2022-01-17
EP3880826A1 (en) 2021-09-22
AU2019377115A1 (en) 2021-05-20
EP3880826A4 (en) 2022-08-24
CA3119065A1 (en) 2020-05-14
WO2020097372A1 (en) 2020-05-14

Similar Documents

Publication Publication Date Title
CN118497276A (zh) 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途
US12264317B2 (en) Compositions and methods for treating non-age-associated hearing impairment in a human subject
US20220040327A1 (en) Compositions and methods for treating non-age-associated hearing impairment in a human subject
JP7807380B2 (ja) ヒト対象において非加齢関連聴力障害を処置するための組成物および方法
US12275960B2 (en) AAV-mediated delivery of therapeutic antibodies to the inner ear
JP2023153320A (ja) Clrn1に関連する聴力喪失及び/または視力喪失を治療する方法
CN118679182A (zh) 用于治疗前庭神经鞘瘤相关症状的抗vegf抗体构建体和相关方法
KR20230117179A (ko) 청신경초종 연관 증상을 치료하기 위한 항-vegf 항체작제물 및 관련된 방법
HK40115203A (zh) 腺相关病毒载体用於在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途
JP2024500786A (ja) Clrn1関連難聴及び/または視力低下を治療するための組成物及び方法
EA051334B1 (ru) Композиции и способы лечения невозрастных нарушений слуха у человека
EA052823B1 (ru) Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40115203

Country of ref document: HK