IL291789A - Adeno-associated virus (aav) systems for the treatment of genetic hearing loss - Google Patents

Adeno-associated virus (aav) systems for the treatment of genetic hearing loss

Info

Publication number
IL291789A
IL291789A IL291789A IL29178922A IL291789A IL 291789 A IL291789 A IL 291789A IL 291789 A IL291789 A IL 291789A IL 29178922 A IL29178922 A IL 29178922A IL 291789 A IL291789 A IL 291789A
Authority
IL
Israel
Prior art keywords
gjb2
aav
nucleic acid
promoter
polynucleotide
Prior art date
Application number
IL291789A
Other languages
English (en)
Hebrew (he)
Original Assignee
Applied Genetic Tech Corporation
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 Applied Genetic Tech Corporation filed Critical Applied Genetic Tech Corporation
Publication of IL291789A publication Critical patent/IL291789A/en

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    • 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
    • A61K48/005—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
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL291789A 2019-09-30 2020-09-30 Adeno-associated virus (aav) systems for the treatment of genetic hearing loss IL291789A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962907834P 2019-09-30 2019-09-30
PCT/US2020/053561 WO2021067448A1 (en) 2019-09-30 2020-09-30 Adeno-associated virus (aav) systems for treatment of genetic hearing loss

Publications (1)

Publication Number Publication Date
IL291789A true IL291789A (en) 2022-06-01

Family

ID=75161339

Family Applications (1)

Application Number Title Priority Date Filing Date
IL291789A IL291789A (en) 2019-09-30 2020-09-30 Adeno-associated virus (aav) systems for the treatment of genetic hearing loss

Country Status (9)

Country Link
US (2) US12241077B2 (https=)
EP (1) EP4037771A4 (https=)
JP (2) JP7713130B2 (https=)
KR (1) KR20220133854A (https=)
CN (1) CN115103710A (https=)
AU (1) AU2020357740A1 (https=)
CA (1) CA3153133A1 (https=)
IL (1) IL291789A (https=)
WO (1) WO2021067448A1 (https=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
WO2021231808A2 (en) * 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating gjb2-associated hearing loss
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
TW202227476A (zh) * 2020-09-14 2022-07-16 哈佛大學校長及研究員協會 編碼GJB2之重組腺相關病毒(rAAV)及其用途
US20230340038A1 (en) * 2020-09-14 2023-10-26 President And Fellows Of Harvard College Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof
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
WO2022155588A2 (en) * 2021-01-18 2022-07-21 Life Technologies Corporation Compositions, kits and methods for direct amplification from crude biological samples
EP4330413A4 (en) * 2021-04-27 2026-01-28 Childrens Hospital Philadelphia Adeno-associated viral vectors for cochlear transduction
MX2023013394A (es) * 2021-05-13 2023-12-14 Akouos Inc Composiciones de suministro de terapia genica y metodos para tratar perdida auditiva.
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
WO2023106256A1 (ja) * 2021-12-06 2023-06-15 学校法人順天堂 改変型アデノ随伴ウイルスベクター
US20240252685A1 (en) * 2022-05-09 2024-08-01 Akouos, Inc. Cell specific gene therapy delivery compositions and methods for treating hearing loss
EP4536862A1 (en) * 2022-06-09 2025-04-16 Wisconsin Alumni Research Foundation Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation
WO2024081687A2 (en) * 2022-10-12 2024-04-18 The Johns Hopkins University Method of treating lbsl by enhancing dars2 expression
CN115717166B (zh) * 2022-12-15 2026-04-14 东南大学 遗传性耳聋检测系统、标志物或探针、药物及应用
US20240263143A1 (en) * 2023-02-02 2024-08-08 Agathos Biologics Baby Hamster Kidney (BHK) Cells Transformed with the Adenoviral E1 Gene for Production of Recombinant Adeno-Associated Virus
EP4713463A1 (en) * 2023-05-18 2026-03-25 Decibel Therapeutics, Inc. Compositions and methods for treating gjb2-related hearing loss
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
CN119510774A (zh) * 2024-11-26 2025-02-25 华中科技大学同济医学院附属协和医院 Sptbn1作为靶点在治疗gjb2相关感音神经性听力损失中的应用

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1755400A2 (en) * 2004-06-18 2007-02-28 The University Of Montana Aav mediated gene delivery to cochlear cells
US9085777B2 (en) * 2006-05-11 2015-07-21 Virttu Biologics Limited Mutant HSV, materials and methods for generation of mutant HSV
JP2010505442A (ja) * 2006-10-10 2010-02-25 ビロメッド カンパニー， リミテッド 安全性の改善された発現ベクター
WO2015130840A2 (en) * 2014-02-25 2015-09-03 Coda Therapeutics, Inc. Treatment of resistant lesions
GB201513176D0 (en) * 2015-07-27 2015-09-09 Glaxosmithkline Biolog Sa Novel methods for inducing an immune response
GB2547179A (en) 2015-10-26 2017-08-16 Quethera Ltd Genetic construct
SG10201913266UA (en) * 2015-12-11 2020-02-27 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
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
EP3585883A4 (en) * 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
CN109055499B (zh) * 2018-08-30 2021-01-19 杭州杰毅生物技术有限公司 基于CRISPR-Cas的等温核酸检测方法及试剂盒
TW202227476A (zh) * 2020-09-14 2022-07-16 哈佛大學校長及研究員協會 編碼GJB2之重組腺相關病毒(rAAV)及其用途

Also Published As

Publication number Publication date
JP7713130B2 (ja) 2025-07-25
EP4037771A4 (en) 2023-10-25
EP4037771A1 (en) 2022-08-10
CN115103710A (zh) 2022-09-23
JP2025110904A (ja) 2025-07-29
KR20220133854A (ko) 2022-10-05
US20250236887A1 (en) 2025-07-24
JP2022549380A (ja) 2022-11-24
AU2020357740A1 (en) 2022-04-28
US20210095313A1 (en) 2021-04-01
CA3153133A1 (en) 2021-04-08
US12241077B2 (en) 2025-03-04
WO2021067448A1 (en) 2021-04-08

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