CL2022002276A1 - Composiciones y métodos para tratar discapacidad auditiva no asociada con la edad en un sujeto - Google Patents

Composiciones y métodos para tratar discapacidad auditiva no asociada con la edad en un sujeto

Info

Publication number
CL2022002276A1
CL2022002276A1 CL2022002276A CL2022002276A CL2022002276A1 CL 2022002276 A1 CL2022002276 A1 CL 2022002276A1 CL 2022002276 A CL2022002276 A CL 2022002276A CL 2022002276 A CL2022002276 A CL 2022002276A CL 2022002276 A1 CL2022002276 A1 CL 2022002276A1
Authority
CL
Chile
Prior art keywords
compositions
subject
methods
age related
hearing impairment
Prior art date
Application number
CL2022002276A
Other languages
English (en)
Inventor
John Simons Emmanuel
Ng Robert
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 CL2022002276A1 publication Critical patent/CL2022002276A1/es

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)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Se proporcionan composiciones que incluyen por lo menos dos vectores de ácido nucleico diferentes, en donde cada uno de los por lo menos dos vectores diferentes incluye una secuencia codificadora que codifica una porción diferente de una proteína otoferlina, y el uso de estas composiciones para tratar la pérdida auditiva en un sujeto.
CL2022002276A 2020-02-21 2022-08-19 Composiciones y métodos para tratar discapacidad auditiva no asociada con la edad en un sujeto CL2022002276A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202062979792P 2020-02-21 2020-02-21

Publications (1)

Publication Number Publication Date
CL2022002276A1 true CL2022002276A1 (es) 2023-04-21

Family

ID=75302627

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2022002276A CL2022002276A1 (es) 2020-02-21 2022-08-19 Composiciones y métodos para tratar discapacidad auditiva no asociada con la edad en un sujeto

Country Status (14)

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

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
JP7240675B2 (ja) 2017-05-05 2023-03-16 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
IL276764B2 (en) * 2018-02-22 2026-05-01 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
JP7665609B2 (ja) 2019-10-30 2025-04-21 デシベル セラピューティクス インコーポレイテッド オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
CA3174500A1 (en) 2020-04-01 2021-10-07 University Of Florida Research Foundation, Incorporated Dual aav-myo7a 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

Family Cites Families (155)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CH587021A5 (es) 1974-06-07 1977-04-29 Nestle Sa
DE3584341D1 (de) 1984-08-24 1991-11-14 Upjohn Co Rekombinante dna-verbindungen und expression von polypeptiden wie 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
JP2001500015A (ja) 1996-09-06 2001-01-09 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 Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
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
DE59914485D1 (de) 1998-03-30 2007-10-18 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
AU773834B2 (en) 1998-12-03 2004-06-10 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
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
WO2001049829A1 (en) 2000-01-05 2001-07-12 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
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
WO2002080864A1 (en) 2001-03-30 2002-10-17 Color Access, Inc. Novel nanoemulsions
KR100463167B1 (ko) 2001-04-13 2004-12-23 주식회사 태평양 고분자 나노입자를 이용한 경피흡수제 및 이를 함유한외용제 조성물
AU2002367976B2 (en) 2001-06-05 2007-06-21 The Regents Of The University Of Michigan Nanoemulsion vaccines
US7319002B2 (en) 2001-08-08 2008-01-15 The Trustees Of The University Of Pennsylvania Method for purification of viral vectors having proteins which bind sialic acid
US20040115159A1 (en) 2002-03-29 2004-06-17 Tadlock Charles C Novel nanoemulsions
US20060292117A1 (en) * 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
US7071172B2 (en) 2002-04-30 2006-07-04 The 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
ATE389675T1 (de) 2002-09-12 2008-04-15 3M Innovative Properties Co Fluorelastomere mit niedertemperatureigenschaften und lösungsmittelbeständigkeit
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
CA2528095C (en) 2003-06-04 2014-10-14 Nanobio Corporation Compositions for inactivating pathogenic microorganisms, methods of making the compositions, and methods of use thereof
KR20060052849A (ko) 2003-08-06 2006-05-19 에자이 가부시키가이샤 약물 초미립자의 제조 방법 및 제조 장치
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
WO2005042539A1 (en) 2003-10-29 2005-05-12 Sonus Pharmaceuticals, Inc. Tocopherol-modified therapeutic drug compounds
WO2005073384A2 (en) 2004-01-28 2005-08-11 Research Development Foundation Human soluble neuropilin-1 primary polyadenylation signal and uses thereof
MXPA06009529A (es) 2004-02-23 2007-04-10 Texas A & M Univ Sys Composiciones antioxidantes y metodos para el uso de las mismas.
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
AU2005287393A1 (en) 2004-06-18 2006-03-30 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
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
AU2006304997B2 (en) 2005-10-20 2012-03-01 Uniqure Ip B.V. Improved AAV vectors produced in insect cells
US9198984B2 (en) 2006-04-28 2015-12-01 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
ES2785223T3 (es) 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
US20080065002A1 (en) 2006-09-07 2008-03-13 Neurosystec Corporation Catheter for Localized Drug Delivery and/or Electrical Stimulation
SI2173888T1 (sl) 2007-07-26 2017-01-31 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
EP2356241B1 (en) 2008-11-28 2018-06-20 Anton Bauer Artificial chromosome vector
KR101812813B1 (ko) 2009-06-16 2017-12-27 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
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
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
WO2012022920A1 (fr) 2010-08-19 2012-02-23 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
US20140304847A1 (en) 2011-06-07 2014-10-09 Ralf Kühn 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
KR102057540B1 (ko) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
EP2986635B1 (en) 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
NZ718926A (en) 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
EP4450630A3 (en) 2014-03-10 2025-01-29 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
CN107428799A (zh) 2015-01-13 2017-12-01 阿尔法韦士曼公司 纯化腺相关病毒(AAV)及/或重组腺相关病毒(rAAV)之方法及其梯度和流通式缓冲液
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
WO2016131981A1 (en) 2015-02-20 2016-08-25 Institut Pasteur Prevention and/or treatment of hearing loss or impairment
US20180327779A1 (en) 2015-03-03 2018-11-15 Fondazione Telethon (IT/IT) Multiple vector system and uses thereof
PT3265568T (pt) 2015-03-06 2020-08-20 Massachusetts Eye & Ear Infirmary Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31
EP4134375A1 (en) 2015-07-30 2023-02-15 Massachusetts Eye & Ear Infirmary Ancestral adeno associated virus sequences and uses thereof
MX2018006682A (es) 2015-12-01 2018-09-26 Spark Therapeutics Inc Metodos escalables para producir vector viral adenoasociado (aav) recombinante en un sistema de cultivo celular en suspension libre de suero adecuado para su uso clinico.
CN116236591B (zh) 2015-12-11 2026-02-27 马萨诸塞眼科耳科诊所 用于将核酸递送至耳蜗和前庭细胞的材料和方法
EP3393522B1 (en) 2015-12-22 2021-07-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Improved hybrid dual recombinant aav vector systems for gene therapy
CA3013038A1 (en) 2016-01-29 2017-08-03 Massachusetts Eye And 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
KR20190045174A (ko) 2016-07-21 2019-05-02 스파크 테라퓨틱스, 인코포레이티드 재조합 아데노신 관련 바이러스(rAAV) 벡터를 고수율로 생산하기 위한 확장 가능한 고회수 방법 및 이에 의해 생산된 재조합 아데노-관련 바이러스(rAAV) 벡터
AU2017301600B2 (en) 2016-07-26 2022-08-04 Biomarin Pharmaceutical 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
US11125139B2 (en) * 2016-10-24 2021-09-21 Cummins Inc. Waste heat recovery vehicle cooling optimization
CN110168081B (zh) 2016-11-04 2024-08-16 武田药品工业株式会社 腺相关病毒纯化方法
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
AU2018215785B2 (en) 2017-02-06 2023-08-31 Children's Medical Center Corporation Materials and methods for delivering nucleic acids to cochlear and vestibular cells
JP7240675B2 (ja) 2017-05-05 2023-03-16 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． オトフェルリンを発現させるための組成物および方法
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating 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
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
EP3684388A4 (en) 2017-09-22 2021-09-22 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF HUNTINGTON'S DISEASE
US11439749B2 (en) 2017-10-25 2022-09-13 The Charles Stark Draper Laboratory, Inc. Apparatus and method for trans-round window membrane drug delivery
US11905522B2 (en) 2018-01-16 2024-02-20 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
KR102860981B1 (ko) 2018-02-09 2025-09-18 데시벨 테라퓨틱스, 인크. 항-백금 화학보호제를 함유하는 고장성 약제학적 조성물
DE102018103924A1 (de) 2018-02-21 2019-08-22 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Gentherapeutische Behandlung von Schwerhörigkeit
IL276764B2 (en) 2018-02-22 2026-05-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
CA3093490A1 (en) 2018-03-05 2019-09-12 Children's Medical Center Corporation Compositions and methods for delivering 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
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
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
AU2019299964A1 (en) 2018-07-11 2021-01-21 Takeda Pharmaceutical Company Limited AAV compositions
JP2021530548A (ja) 2018-07-24 2021-11-11 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. 遺伝子治療製剤を生産するための系および方法
US20200101122A1 (en) 2018-09-27 2020-04-02 Yen-Fu Cheng Method for treating an auditory neuropathy spectrum disorder
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
BR112021013996A2 (pt) 2019-01-18 2021-10-26 Institut Pasteur Terapia gênica mediada por aav que recupera o gene otoferlina
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
JP7562544B2 (ja) 2019-02-08 2024-10-07 デシベル セラピューティクス インコーポレイテッド ミオシン１５プロモーター及びその使用
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
WO2021077115A1 (en) 2019-10-18 2021-04-22 Research Institute At Nationwide Children's Hospital Gene therapy targeting cochlear cells
JP7665609B2 (ja) 2019-10-30 2025-04-21 デシベル セラピューティクス インコーポレイテッド オトフェリン二重ベクター系及びそれを含む感音難聴を治療するための組成物
WO2021096605A1 (en) 2019-11-11 2021-05-20 Salk Institute For Biological Studies Compositions and methods for high-efficiency recombination of rna molecules
CN113840915A (zh) 2020-01-24 2021-12-24 美利坚合众国, 由健康及人类服务部部长代表 腺相关病毒及其用于内耳疗法的用途
US20230090778A1 (en) 2020-02-07 2023-03-23 The Children's Medical Center Corporation Large gene vectors and delivery and uses thereof
CN115335526B (zh) 2020-02-07 2025-04-18 罗切斯特大学 核酶介导的rna组装和表达
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
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
WO2022056440A1 (en) 2020-09-14 2022-03-17 President And Fellows Of Harvard College Dual-aav vector delivery of pcdh15 and uses thereof
WO2022099007A1 (en) 2020-11-06 2022-05-12 Applied Genetic Technologies Corporation 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
WO2022178298A1 (en) 2021-02-19 2022-08-25 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
BR112022016596A2 (pt) 2022-11-16
AU2021225035A1 (en) 2022-10-13
JP7807380B2 (ja) 2026-01-27
US20240093237A1 (en) 2024-03-21
EP4114958A1 (en) 2023-01-11
IL295747A (en) 2022-10-01
US20250313856A1 (en) 2025-10-09
TW202600830A (zh) 2026-01-01
US11807867B2 (en) 2023-11-07
CA3168055A1 (en) 2021-08-26
TW202144578A (zh) 2021-12-01
KR20220157944A (ko) 2022-11-29
TWI886216B (zh) 2025-06-11
US12077773B2 (en) 2024-09-03
US20240425881A1 (en) 2024-12-26
MX2022010050A (es) 2022-09-05
AR121390A1 (es) 2022-06-01
JP2023515072A (ja) 2023-04-12
US20230108913A1 (en) 2023-04-06
WO2021168362A1 (en) 2021-08-26
US12305191B2 (en) 2025-05-20
CN116096905A (zh) 2023-05-09

Similar Documents

Publication Publication Date Title
AR121390A1 (es) Composiciones y métodos para tratar la deficiencia auditiva no relacionada con la edad en un sujeto humano
CL2020002161A1 (es) Composiciones y métodos para tratar el deterioro auditivo no asociado con la edad en un sujeto humano.
EP4219724A3 (en) Compositions and methods for treating non-age-associated hearing impairment in a human subject
CL2021002212A1 (es) Tratamiento de lesión renal
CO2021005884A2 (es) Moduladores de la alfa-1 antitripsina
MX2020008494A (es) Nanomateriales de silicio-carbono, metodo de su elaboracion y usos.
MX2020003915A (es) Proteinas trispecificas y metodos de uso.
BR112018008326A2 (pt) composições e métodos para o tratamento de câncer
MX384205B (es) Metodos para usar agonistas de fxr.
ECSP21026021A (es) Moduladores de la expresión de pnpla3
MX2019012352A (es) Composiciones, sistemas, kits y metodos para ablacion neural.
CL2021001903A1 (es) Tratamiento de la hepatotoxicidad
CL2020003010A1 (es) Moduladores de la expresión de apol1
MX2021008216A (es) Anticuerpos anti-tigit.
MX2021011609A (es) Moduladores de interacciones de proteinas de superficie celular y metodos y composiciones relacionados con estos.
CO2020006907A2 (es) Péptidos antimicrobianos y métodos para usarlos
BR112019003816A2 (pt) composição de tratamento de enxágue dos cabelos, método para tratamento de cabelos quimicamente danificados e uso de uma composição
CO2022009691A2 (es) Formulaciones líquidas de tasimelteón y métodos para su uso
WO2019241730A3 (en) Increasing immune activity through modulation of postcellular signaling factors
MX2021000322A (es) Enzimas de variante fosfopentomutasa modificada geneticamente.
PY1829651A (es) Combinaciones fungicidas
BR112019007365A2 (pt) métodos e composições para imunoterapia com tusc2
BR112018014762A2 (pt) método de tratamento da doença de alzheimer (da) precoce
CL2020003346A1 (es) Métodos para tratar la pérdida de la audición y/o pérdida de la visión asociadas con clrn1
MX2025007132A (es) Metodos para tratar la enfermedad de farber