TWI886216B - 用於治療人類個體之非年齡相關性聽力障礙的組成物及方法 - Google Patents

用於治療人類個體之非年齡相關性聽力障礙的組成物及方法 Download PDF

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TWI886216B
TWI886216B TW110105997A TW110105997A TWI886216B TW I886216 B TWI886216 B TW I886216B TW 110105997 A TW110105997 A TW 110105997A TW 110105997 A TW110105997 A TW 110105997A TW I886216 B TWI886216 B TW I886216B
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amino acids
amino acid
acid substitutions
amino
seq
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TW110105997A
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Chinese (zh)
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TW202144578A (zh
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伊曼紐爾 約翰 西蒙斯
羅伯特 伍
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美商阿科奧斯公司
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • 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
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    • 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
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/103—Ovine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • 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
    • 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
    • C12N2750/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors

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  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
TW110105997A 2020-02-21 2021-02-20 用於治療人類個體之非年齡相關性聽力障礙的組成物及方法 TWI886216B (zh)

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US202062979792P 2020-02-21 2020-02-21
US62/979,792 2020-02-21

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TW202144578A TW202144578A (zh) 2021-12-01
TWI886216B true TWI886216B (zh) 2025-06-11

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US (4) US11807867B2 (https=)
EP (1) EP4114958A1 (https=)
JP (1) JP7807380B2 (https=)
KR (1) KR20220157944A (https=)
CN (1) CN116096905A (https=)
AR (1) AR121390A1 (https=)
AU (1) AU2021225035A1 (https=)
BR (1) BR112022016596A2 (https=)
CA (1) CA3168055A1 (https=)
CL (1) CL2022002276A1 (https=)
IL (1) IL295747A (https=)
MX (1) MX2022010050A (https=)
TW (1) TWI886216B (https=)
WO (1) WO2021168362A1 (https=)

Families Citing this family (11)

* 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
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
IL325122A (en) * 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
US12188041B2 (en) * 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
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
AU2021247172A1 (en) 2020-04-01 2022-09-15 University Of Florida Research Foundation, Incorporated Dual AAV-MY07A vectors with improved safety for the treatment of USH1B
EP4401836A1 (en) * 2021-09-18 2024-07-24 Skyline Therapeutics Limited Aav for the gene therapy of wet-amd
CN117305367A (zh) * 2023-08-21 2023-12-29 复旦大学附属眼耳鼻喉科医院 一种表达全长耳畸蛋白的双aav载体系统及其应用
WO2025201333A1 (zh) * 2024-03-28 2025-10-02 苏州星奥拓维生物技术有限公司 递送Otof基因的双载体系统和其用途

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019165292A1 (en) * 2018-02-22 2019-08-29 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject

Family Cites Families (154)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH587021A5 (https=) 1974-06-07 1977-04-29 Nestle Sa
EP0173552B1 (en) 1984-08-24 1991-10-09 The Upjohn Company Recombinant dna compounds and the expression of polypeptides such as tpa
US5168062A (en) 1985-01-30 1992-12-01 University Of Iowa Research Foundation Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence
US5651991A (en) 1987-10-28 1997-07-29 Nippon Shinyaku Co. Ltd. Drug carriers
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
CA2091152C (en) 1993-03-05 2005-05-03 Kirsten Westesen Solid lipid particles, particles of bioactive agents and methods for the manfuacture and use thereof
JPH07285863A (ja) 1994-02-25 1995-10-31 Takeda Chem Ind Ltd 注射剤組成物およびその製造方法
ES2078190B1 (es) 1994-05-20 1996-08-01 Cusi Lab Procedimiento para el recubrimiento de goticulas o particulas de tamaño nanometrico.
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
US5510118A (en) 1995-02-14 1996-04-23 Nanosystems Llc Process for preparing therapeutic compositions containing nanoparticles
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6007856A (en) 1997-08-08 1999-12-28 The Procter & Gamble Company Oil-in-water dispersions of β-carotene and other carotenoids stable against oxidation prepared from water-dispersible beadlets having high concentrations of carotenoid
ATE372377T1 (de) 1998-03-30 2007-09-15 Mibelle Ag Cosmetics Verwendung von nanoemulsionen zur bestimmung der biokompatibilität von lipophilen stoffen im zellkultur-test und dafür geeignete nanoemulsionen
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
US8026096B1 (en) 1998-10-08 2011-09-27 Protein Sciences Corporation In vivo active erythropoietin produced in insect cells
ATE226449T1 (de) 1998-12-03 2002-11-15 Avigen Inc Hilfsstoffe zur verwendung in adeno-assoziiertes virus enthaltenden pharmazeutischen zubereitungen und damit hergestellte pharmazeutische zubereitungen
FR2787728B1 (fr) 1998-12-23 2001-01-26 Oreal Nanoemulsion a base d'esters gras d'acide phosphorique, et ses utilisations dans les domaines cosmetique, dermatologique, pharmaceutique et/ou ophtalmologique
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6627617B1 (en) 1999-10-01 2003-09-30 University Of North Carolina At Chapel Hill Temperature-sensitive regulation of viral vector production
US7208315B2 (en) 2000-01-05 2007-04-24 Fred Hutchinson Cancer Research Center Compositions and methods for efficient AAV vector production
WO2001066782A1 (en) 2000-03-03 2001-09-13 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral compositions and methods
MXPA02009275A (es) 2000-03-23 2004-09-06 Collaborative Technologies Inc Metodo para preparar dispersiones de alta presion/alto esfuerzo cortante que contienen ingredientes fisiologicamente activos.
WO2001070972A2 (en) 2000-03-24 2001-09-27 Yasunaga, Shin'ichiro Multiple human and mouse otoferlin isoforms
RS49646B (sr) 2000-07-04 2007-09-21 Slobodan Arsenijević Instrument za ubrizgavanje fluida i dilataciona sonda za uvođenje u telesne šupljine
FR2811561B1 (fr) 2000-07-13 2003-03-21 Oreal Composition, notamment cosmetique, renfermant la dhea et/ou un precurseur ou derive chimique ou biologique de celle-ci, et un inhibiteur de metalloproteinase
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US20040048836A1 (en) 2001-03-23 2004-03-11 Wilmott James M Method for preparing high pressure/ high shear dispersions containing physiologically active ingredients
EP1377255A4 (en) 2001-03-30 2008-11-19 Color Access Inc NEW NANOEMULSIONS
KR100463167B1 (ko) 2001-04-13 2004-12-23 주식회사 태평양 고분자 나노입자를 이용한 경피흡수제 및 이를 함유한외용제 조성물
WO2004030608A2 (en) 2001-06-05 2004-04-15 The Regents Of The University Of Michigan Nanoemulsion vaccines
JP2004538005A (ja) 2001-08-08 2004-12-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア シアル酸に結合するタンパク質を有するウイルスベクターの精製法
US20040115159A1 (en) 2002-03-29 2004-06-17 Tadlock Charles C Novel nanoemulsions
AU2003221733A1 (en) * 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
US7071172B2 (en) 2002-04-30 2006-07-04 The University Of North Carolina At Chapel Hill Secretion signal vectors
US20060127358A1 (en) * 2002-05-01 2006-06-15 Nicholas Muzyczka Raav expression systems and methods for enhancing transduction of mammalian neural cells
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
JP4824311B2 (ja) 2002-09-12 2011-11-30 スリーエム イノベイティブ プロパティズ カンパニー 低温特性および耐溶剤性を有するフルオロエラストマー
US7329688B2 (en) 2002-12-17 2008-02-12 Soft Gel Technologies, Inc. Natural vitamin E compositions with superior antioxidant potency
DE602004030327D1 (de) 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
AU2004273779B2 (en) 2003-06-04 2009-10-22 Nanobio Corporation Compositions for inactivating pathogenic microorganisms, methods of making the compositions, and methods of use thereof
WO2005013938A1 (ja) 2003-08-06 2005-02-17 Eisai Co., Ltd. 薬物超微粒子の製造法及び製造装置
US7387614B2 (en) 2003-08-26 2008-06-17 University Of Maryland, Baltimore Drug delivery to the inner ear and methods of using same
EP1510206A1 (en) 2003-08-29 2005-03-02 Novagali Pharma SA Self-nanoemulsifying oily formulation for the administration of poorly water-soluble drugs
BRPI0415858A (pt) 2003-10-29 2007-01-09 Sonus Pharma Inc compostos de medicamentos terapêuticos modificados por tocoferol
CA2553676A1 (en) 2004-01-28 2005-08-11 Research Development Foundation Human soluble neuropilin-1 primary polyadenylation signal and uses thereof
EP1718163A1 (en) 2004-02-23 2006-11-08 The Texas A&M University System Antioxidant compositions and methods of use thereof
US7419497B2 (en) 2004-04-21 2008-09-02 Acclarent, Inc. Methods for treating ethmoid disease
EP1751275B1 (en) 2004-06-01 2017-08-16 Avigen, Inc. Compositions and methods to prevent aav vector aggregation
US20050281786A1 (en) 2004-06-18 2005-12-22 David Poulsen AAV mediated gene delivery to cochlear cells
US20060040354A1 (en) 2004-07-20 2006-02-23 O'keefe Theresa L Novel polyadenylation signal for use in expression vectors
US20070055199A1 (en) 2005-08-10 2007-03-08 Gilbert Scott J Drug delivery device for buccal and aural applications and other areas of the body difficult to access
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US7851196B2 (en) 2006-05-01 2010-12-14 The Regents Of The University Of California Methods for purifying adeno-associated virus particles
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
US20080065002A1 (en) 2006-09-07 2008-03-13 Neurosystec Corporation Catheter for Localized Drug Delivery and/or Electrical Stimulation
SI3093345T1 (sl) 2007-07-26 2019-08-30 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami
US20090042825A1 (en) 2007-08-06 2009-02-12 Majed Matar Composition, method of preparation & application of concentrated formulations of condensed nucleic acids with a cationic lipopolymer
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
PL2356241T3 (pl) 2008-11-28 2018-12-31 Anton Bauer Wektor sztucznego chromosomu
HUE028341T2 (en) 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purification of recombinant aav vectors
JP5697123B2 (ja) 2009-07-27 2015-04-08 国立大学法人 千葉大学 酸性化ポリエチレンイミンを用いる細胞への核酸導入方法
WO2011075838A1 (en) 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
WO2011112089A2 (en) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Mutated rep encoding sequences for use in aav production
EP2392208B1 (en) 2010-06-07 2016-05-04 Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Fusion proteins comprising a DNA-binding domain of a Tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use
EP2605761A1 (fr) 2010-08-19 2013-06-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Dispositif implantable dans l'os temporal pour administrer un produit et prothèse auditive pourvue d'un tel dispositif.
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
WO2012168307A2 (en) 2011-06-07 2012-12-13 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Improved recombination efficiency by inhibition of nhej dna repair
US8852165B2 (en) 2011-06-16 2014-10-07 II Edward G. Mackay Endoluminal drug delivery devices and methods
US9441206B2 (en) 2011-10-28 2016-09-13 The University Of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
AU2013221212B2 (en) 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
CA2909733C (en) 2013-04-18 2023-10-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors
EP3039129B1 (en) 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
HUE052676T2 (hu) 2013-10-11 2021-05-28 Massachusetts Eye & Ear Infirmary Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik
WO2015137802A1 (en) 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
US10526583B2 (en) 2014-07-02 2020-01-07 University Of Florida Research Foundation, Incorporated Compositions and methods for purifying recombinant adeno-associated virus
SG11201704919SA (en) 2015-01-13 2017-07-28 Alfa Wassermann Inc Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav)
EP3054006A1 (en) 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography
US10751385B2 (en) 2015-02-20 2020-08-25 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
RS63416B1 (sr) 2015-03-03 2022-08-31 Fond Telethon Sistem višestrukih vektora i njegove primene
PL3265568T3 (pl) 2015-03-06 2020-11-30 Massachusetts Eye & Ear Infirmary Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31
CA2994160C (en) 2015-07-30 2021-08-10 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
AU2016362317B2 (en) 2015-12-01 2023-03-16 Spark Therapeutics, Inc. Scalable methods for producing recombinant Adeno-Associated Viral (AAV) vector in serum-free suspension cell culture system suitable for clinical use
SG11201804814YA (en) 2015-12-11 2018-07-30 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2017108931A1 (en) 2015-12-22 2017-06-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Improved hybrid dual recombinant aav vector systems for gene therapy
CN109219439B (zh) 2016-01-29 2022-09-20 马萨诸塞眼科耳科诊所 内耳支持细胞的扩增和分化及其使用方法
WO2017136764A1 (en) 2016-02-05 2017-08-10 The General Hospital Corporation Hybrid system for efficient gene delivery to cells of the inner ear
JP2019522539A (ja) 2016-06-22 2019-08-15 ザ チャールズ スターク ドレイパー ラボラトリー， インク．Ｔｈｅ Ｃｈａｒｌｅｓ Ｓｔａｒｋ Ｄｒａｐｅｒ Ｌａｂｏｒａｔｏｒｙ， Ｉｎｃ． 経正円窓膜注入による内耳薬物送達用のシステム
GB201612248D0 (en) 2016-07-14 2016-08-31 Puridify Ltd New process
KR102695908B1 (ko) 2016-07-21 2024-08-14 스파크 테라퓨틱스, 인코포레이티드 재조합 아데노-관련 바이러스(rAAV) 벡터를 고수율로 생산하기 위한 확장 가능한 고회수 방법 및 이에 의해 생산된 재조합 아데노-관련 바이러스(rAAV) 벡터
PE20190401A1 (es) 2016-07-26 2019-03-13 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado
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
WO2018080895A1 (en) * 2016-10-24 2018-05-03 Cummins Inc. Waste heat recovery vehicle cooling optimization
KR102482744B1 (ko) 2016-11-04 2022-12-30 다케다 야쿠힌 고교 가부시키가이샤 아데노-연관 바이러스 정제 방법
US10626376B2 (en) 2016-11-14 2020-04-21 St. Jude Children's Research Hospital Method for isolating and purifying adeno-associated virus particles using salt
FI3568483T3 (fi) 2017-01-10 2025-11-21 Childrens Medical Res Institute Polynukleotideja ja vektoreita siirtogeenien ilmentämiseen
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
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP2020518266A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 調節性ポリヌクレオチド
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
WO2019060649A1 (en) 2017-09-22 2019-03-28 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
WO2019084145A1 (en) 2017-10-25 2019-05-02 The Charles Stark Draper Laboratory Inc. APPARATUS AND METHOD FOR DRUG DELIVERY ACROSS THE ROUND WINDOW MEMBRANE
KR20200122320A (ko) 2018-01-16 2020-10-27 씨엘에스 테라퓨틱스 리미티드 데옥시리보뉴클레아제 (dnase) 활성을 갖는 효소의 간 발현에 의한 질환의 치료
CN112020359A (zh) 2018-02-09 2020-12-01 分贝治疗公司 含有抗铂化学保护剂的高渗药物组合物
DE102018103924A1 (de) 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
JP7430652B2 (ja) 2018-03-05 2024-02-13 ザ チルドレンズ メディカル センター コーポレーション 蝸牛および前庭細胞に核酸を送達するための組成物および方法
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
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒（ａａｖ）之組成物和方法
AU2019299964A1 (en) 2018-07-11 2021-01-21 Takeda Pharmaceutical Company Limited AAV compositions
AU2019310459A1 (en) 2018-07-24 2021-02-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020069320A1 (en) 2018-09-27 2020-04-02 Akouos, Inc. A method for treating an auditory neuropathy spectrum disorder
CA3115810A1 (en) 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications
US12188041B2 (en) 2018-11-01 2025-01-07 University Of Florida Research Foundation, Incorporated Codon optimized otoferlin AAV dual vector gene therapy
CN118497276A (zh) 2018-11-07 2024-08-16 阿库斯股份有限公司 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
WO2020148458A1 (en) 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CN110437317B (zh) 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
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
AU2020218547A1 (en) 2019-02-08 2021-08-26 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3967706A4 (en) 2019-05-08 2022-12-28 Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences AAV MUTANT FOR EFFICIENT INFECTION OF SUPPORT CELLS
CA3153133A1 (en) 2019-09-30 2021-04-08 Chris Bartolome Adeno-associated virus (aav) systems for treatment of genetic hearing loss
EP4045092A1 (en) 2019-10-18 2022-08-24 Research Institute at Nationwide Children's Hospital Gene therapy targeting cochlear cells
CA3159549A1 (en) 2019-10-30 2021-05-06 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP4058571A1 (en) 2019-11-11 2022-09-21 Salk Institute for Biological Studies Compositions and methods for high-efficiency recombination of rna molecules
WO2021150850A1 (en) 2020-01-24 2021-07-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Svcs. Adeno-associated viruses and their uses for inner ear therapy
CA3168903A1 (en) 2020-02-07 2021-08-12 University Of Rochester Ribozyme-mediated rna assembly and expression
JP7846626B2 (ja) 2020-02-07 2026-04-15 ザ チルドレンズ メディカル センター コーポレーション 大型遺伝子ベクターならびにその送達および使用の方法
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
WO2021183825A1 (en) 2020-03-12 2021-09-16 California Institute Of Technology Adeno-associated viral capsids with expanded sizes
CN111349148A (zh) 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途
EP3885440A1 (en) 2020-03-26 2021-09-29 Splicebio, S.L. Split inteins and their uses
US20240016955A1 (en) 2020-09-14 2024-01-18 President And Fellows Of Harvard College Dual-aav vector delivery of pcdh15 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
EP4008788A1 (en) 2020-12-03 2022-06-08 Medizinische Hochschule Hannover Lentiviral vector technology for inner ear gene therapy
CN117295529A (zh) 2021-02-19 2023-12-26 分贝治疗公司 使用耳畸蛋白双载体系统治疗感觉神经性听力损失的方法
CN113337507B (zh) 2021-08-05 2021-11-12 首都医科大学附属北京友谊医院 Otof 1273 (C>T)基因定点突变的耳聋小鼠模型的构建方法及其应用

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019165292A1 (en) * 2018-02-22 2019-08-29 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject

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