AU2021225035A1 - Compositions and methods for treating non-age-associated hearing impairment in a human subject - Google Patents

Compositions and methods for treating non-age-associated hearing impairment in a human subject Download PDF

Info

Publication number
AU2021225035A1
AU2021225035A1 AU2021225035A AU2021225035A AU2021225035A1 AU 2021225035 A1 AU2021225035 A1 AU 2021225035A1 AU 2021225035 A AU2021225035 A AU 2021225035A AU 2021225035 A AU2021225035 A AU 2021225035A AU 2021225035 A1 AU2021225035 A1 AU 2021225035A1
Authority
AU
Australia
Prior art keywords
amino acids
acid substitutions
amino acid
amino
acids
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
AU2021225035A
Other languages
English (en)
Inventor
Robert NG
Emmanuel John Simons
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.)
Akouos Inc
Original Assignee
Akouos 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 Akouos Inc filed Critical Akouos Inc
Publication of AU2021225035A1 publication Critical patent/AU2021225035A1/en
Pending legal-status Critical Current

Links

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
    • 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
    • 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
    • C12N15/87—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/50—Vectors for producing vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2021225035A 2020-02-21 2021-02-19 Compositions and methods for treating non-age-associated hearing impairment in a human subject Pending AU2021225035A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062979792P 2020-02-21 2020-02-21
US62/979,792 2020-02-21
PCT/US2021/018919 WO2021168362A1 (en) 2020-02-21 2021-02-19 Compositions and methods for treating non-age-associated hearing impairment in a human subject

Publications (1)

Publication Number Publication Date
AU2021225035A1 true AU2021225035A1 (en) 2022-10-13

Family

ID=75302627

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021225035A Pending AU2021225035A1 (en) 2020-02-21 2021-02-19 Compositions and methods for treating non-age-associated hearing impairment in a human subject

Country Status (14)

Country Link
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
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
IL325122A (en) * 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
WO2020093018A1 (en) * 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A 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
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
MX2024003320A (es) * 2021-09-18 2024-06-28 Skyline Therapeutics Ltd Virus adenoasociado para terapia génica de degeneración macular húmeda asociada con la edad.
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

Family Cites Families (155)

* 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
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania 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
AU7739000A (en) 1999-10-01 2001-05-10 University Of Florida 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
AU2001240018A1 (en) 2000-03-03 2001-09-17 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral compositions and methods
AU2001245947A1 (en) 2000-03-23 2001-10-03 Collaborative Technologies, Inc Method for preparing high pressure/high shear dispersions containing physiologically active ingredients
AU4445701A (en) 2000-03-24 2001-10-03 Centre National De La Recherche Scientifique-Cnrs 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
JP2006512401A (ja) 2001-06-05 2006-04-13 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン ナノエマルジョンワクチン
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
AU2003223766A1 (en) 2002-04-30 2003-11-17 University Of North Carolina At Chapel Hill Secretion signal vectors
WO2003093479A1 (en) * 2002-05-01 2003-11-13 University Of Florida Research Foundation, Inc. Improved 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
US6864336B2 (en) 2002-09-12 2005-03-08 3M Innovative Properties Company Fluoroelastomers having low temperature characteristics and solvent resistance
US7329688B2 (en) 2002-12-17 2008-02-12 Soft Gel Technologies, Inc. Natural vitamin E compositions with superior antioxidant potency
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
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
WO2005009287A2 (en) 2003-07-21 2005-02-03 University Of Maryland, Baltimore Drug delivery to the inner ear and methods of using same
US20060151899A1 (en) 2003-08-06 2006-07-13 Akira Kato Process for producing drug ultramicroparticle and apparatus therefor
EP1510206A1 (en) 2003-08-29 2005-03-02 Novagali Pharma SA Self-nanoemulsifying oily formulation for the administration of poorly water-soluble drugs
WO2005042539A1 (en) 2003-10-29 2005-05-12 Sonus Pharmaceuticals, Inc. Tocopherol-modified therapeutic drug compounds
CA2553676A1 (en) 2004-01-28 2005-08-11 Research Development Foundation Human soluble neuropilin-1 primary polyadenylation signal and uses thereof
CA2557261A1 (en) 2004-02-23 2005-09-15 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
BRPI0511764B8 (pt) 2004-06-01 2021-05-25 Avigen Inc método de prevenção de agregação de vírions de vírus adeno-associado recombinante (raav) em uma preparação purificada de virions raav
EP1755400A2 (en) 2004-06-18 2007-02-28 The University Of Montana Aav mediated gene delivery to cochlear cells
WO2006012414A2 (en) 2004-07-20 2006-02-02 Critical Therapeutics, Inc. Novel polyadenylation signal for use in expression vectors
US8591457B2 (en) 2005-08-10 2013-11-26 Alza Corporation Method for making a needle-free jet injection drug delivery device
EP2311966A3 (en) 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) 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
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
US20080065002A1 (en) 2006-09-07 2008-03-13 Neurosystec Corporation Catheter for Localized Drug Delivery and/or Electrical Stimulation
DK2173888T3 (en) 2007-07-26 2016-11-28 Uniqure Ip Bv Baculovirus vectors comprising repeating coding sequences WITH differential preferred codons
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
CN102239261A (zh) 2008-11-28 2011-11-09 奥地利经济服务有限责任公司 人工染色体载体
KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 ａａｖ 벡터에 대한 개선된 정제 방법
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
WO2011094198A1 (en) 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
EP2545165B1 (en) 2010-03-11 2020-07-29 uniQure 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
CA2853482C (en) 2011-10-28 2020-05-19 Joshua Grieger 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
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.
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
AU2014255665B2 (en) 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
WO2015031686A1 (en) 2013-08-30 2015-03-05 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
CN106232618A (zh) 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
CA2942289C (en) 2014-03-10 2024-05-21 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
WO2016004319A1 (en) 2014-07-02 2016-01-07 University Of Florida Research Foundation, Inc. Compositions and methods for purifying recombinant adeno-associated virus
US9862936B2 (en) 2015-01-13 2018-01-09 Alfa Wassermann, Inc. Methods of purifying adeno-associated virus (AAV) and/or recombinant adeno-associated virus (rAAV) and gradients and flow-through buffers therefore
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
EP3258955A1 (en) 2015-02-20 2017-12-27 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
SI3265571T1 (sl) 2015-03-03 2022-10-28 Fondazione Telethon Več vektorski sistem in njegove uporabe
EP3748007B1 (en) 2015-03-06 2024-06-26 Massachusetts Eye & Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
CN108603235A (zh) 2015-07-30 2018-09-28 马萨诸塞眼科耳科诊所 祖先病毒序列及其用途
EP3384015A4 (en) 2015-12-01 2019-05-29 Spark Therapeutics, Inc. SCALABLE PROCESS FOR THE PREPARATION OF A RECOMBINANT ADENO ASSOCIATED VIRAL VECTOR IN A SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR THE CLINICAL APPLICATION
SG10201913266UA (en) 2015-12-11 2020-02-27 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
JP7007273B2 (ja) 2015-12-22 2022-01-24 アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） 遺伝子治療用の改良された複合型二重組換えａａｖベクターシステム
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
WO2017136764A1 (en) 2016-02-05 2017-08-10 The General Hospital Corporation Hybrid system for efficient gene delivery to cells of the inner ear
US10201455B2 (en) 2016-06-22 2019-02-12 The Charles Stark Draper Laboratory, Inc. System for inner ear drug delivery via trans-round window membrane injection
GB201612248D0 (en) 2016-07-14 2016-08-31 Puridify Ltd New process
IL301963A (en) 2016-07-21 2023-06-01 Spark Therapeutics Inc Scalable High Recovery Methods for High-Throughput Recombinant Adeno-Associated Viral Vector Production and Recombinant Adeno-Associated Viral Vectors Made So
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
WO2018080895A1 (en) * 2016-10-24 2018-05-03 Cummins Inc. Waste heat recovery vehicle cooling optimization
CA3040288A1 (en) 2016-11-04 2018-07-12 Baxalta Incorporated Adeno-associated virus purification methods
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
PT3568483T (pt) 2017-01-10 2025-11-18 Childrens Medical Res Institute Polinucleótidos e vetores para a expressão de transgenes
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
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
JP2020518258A (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． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
EP3619310A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. Modulatory polynucleotides
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
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20200377887A1 (en) 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of 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
AU2019209770B2 (en) 2018-01-16 2025-07-31 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
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
IL325122A (en) 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
EP3762040A4 (en) 2018-03-05 2022-01-05 Children's Medical Center Corporation COMPOSITIONS AND METHODS OF ADMINISTRATION OF NUCLEIC ACIDS TO COCHLEAR AND VESTIBULAR CELLS
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
KR20210032984A (ko) 2018-07-11 2021-03-25 박스알타 인코퍼레이티드 Aav 조성물
CN112770812A (zh) 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
EP3856227A4 (en) 2018-09-27 2022-07-06 Akouos, Inc. METHODS OF TREATMENT OF AUDITORY NEUROPATHY SPECTRUM DISEASE
WO2020077295A1 (en) 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications
WO2020093018A1 (en) 2018-11-01 2020-05-07 University Of Florida Research Foundation, Incorporated A codon optimized otoferlin aav dual vector gene therapy
EP4063510A1 (en) 2018-11-07 2022-09-28 Akouos, Inc. Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear
EP4295913A3 (en) 2019-01-18 2024-03-06 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
CA3129422A1 (en) 2019-02-08 2020-08-13 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
WO2020223933A1 (zh) 2019-05-08 2020-11-12 中国科学院脑科学与智能技术卓越创新中心 高效感染支持细胞的aav突变体
WO2021067448A1 (en) 2019-09-30 2021-04-08 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of genetic hearing loss
AU2020366531A1 (en) 2019-10-18 2022-05-26 Research Institute At Nationwide Children's Hospital Gene therapy targeting cochlear cells
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
IL292904A (en) 2019-11-11 2022-07-01 Salk Inst For Biological Studi 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
EP4100533A1 (en) 2020-02-07 2022-12-14 University of Rochester Ribozyme-mediated rna assembly and expression
CA3170709A1 (en) 2020-02-07 2021-08-12 The Children's Medical Center Corporation Large gene vectors and delivery 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
US20210284699A1 (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
EP4294460A4 (en) 2021-02-19 2025-06-25 Decibel Therapeutics, Inc. Methods for treating sensorineural hearing loss using otoferlin dual vector systems
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

Also Published As

Publication number Publication date
US20250313856A1 (en) 2025-10-09
KR20220157944A (ko) 2022-11-29
TW202600830A (zh) 2026-01-01
US20240425881A1 (en) 2024-12-26
US20230108913A1 (en) 2023-04-06
CL2022002276A1 (es) 2023-04-21
CN116096905A (zh) 2023-05-09
BR112022016596A2 (pt) 2022-11-16
JP2023515072A (ja) 2023-04-12
EP4114958A1 (en) 2023-01-11
IL295747A (en) 2022-10-01
US12305191B2 (en) 2025-05-20
MX2022010050A (es) 2022-09-05
WO2021168362A1 (en) 2021-08-26
TW202144578A (zh) 2021-12-01
US20240093237A1 (en) 2024-03-21
US11807867B2 (en) 2023-11-07
AR121390A1 (es) 2022-06-01
TWI886216B (zh) 2025-06-11
CA3168055A1 (en) 2021-08-26
JP7807380B2 (ja) 2026-01-27
US12077773B2 (en) 2024-09-03

Similar Documents

Publication Publication Date Title
US12264317B2 (en) Compositions and methods for treating non-age-associated hearing impairment in a human subject
AU2019224121B2 (en) Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12077773B2 (en) Compositions and methods for treating non-age-associated hearing impairment in a human subject
HK40098362A (en) Compositions and methods for treating non-age-associated hearing impairment in a human subject
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